Download Fall 2016 Newsletter

ITP Awareness Special Edition
The
Platelet News
VOL. 18; NO. 3 FALL 2016
In This Issue
Message From the
Executive Director
2
Communicating
with your Doctor
3
Making a Difference
4
Walk/Run News
8
PDSA Launches First ITP Natural History
Study Registry
Teen Session at
ITP Conference 2016
10
By Alexandra Kruse
ITP in Adults: Q&A
11
Starting this winter, you can join thousands of
patients living with ITP from across the globe and
register with the Platelet Disorder Support Association’s (PDSA) “ITP Natural History
Study Registry” to advance research and improve the quality of life of ITP patients.
Purple Power Spreads
New ITP Awareness!
Special Photo Spread
12
The ITP Natural History Study Registry is an international, patient-consented registry of
individuals with ITP. The registry aims to collect data on the natural progression of ITP,
enabling PDSA to gather data on diagnosis and treatment, management of care, quality
of life, and clinician reporting. The registry is administered by PDSA and overseen by the
(CONTINUED ON PAGE 7)
National Organization for Rare Disorders (NORD), the US Food
It’s Official!
ITP Advocacy
and Support
is a Worldwide
Initiative
In Appreciation
14
Fundraising News
15
PDSA’s First
Research Coordinator
Alexandra Kruse
16
In Memory / In Honor
17
New Patient-Centered
Research Guidelines for
ITP Research Proposals
17
Local Support Groups
18
Support Group News
19
Q&A with ICON
Pediatrician Dr. Klaassen 20
GLOBAL PARTNERS MEET
AS THE INTERNATIONAL
ITP ALLIANCE
By Nancy Potthast
PDSA’s ITP Poke-R Club
Abstract One of the Top
Posters at NORD Summit 14
International delegates from 8 countries attended the ITP
Global Support Group Meeting on Friday, September 16, 2016
at The Shaw Country House Hotel in the U.K. Representatives
from International ITP Alliance member organizations included
Denmark, Finland, India, Italy, Netherlands, Sweden, United
Kingdom, and United States of America.
Ahhhh, September. It’s that
magical time of year when
purple fills the hearts of a rare
community with strength,
pride and a sense of belonging — when warriors and advocates come together to raise
awareness for immune thrombocytopenia (ITP). Just six short years ago, PDSA requested
and received the designation of September as National ITP Awareness Month with the
last Friday of the month named as Sport Purple for Platelets Day and for the first time
this year, the last week of September was established as Global ITP Awareness
Week. In all its purple splendor, it seems quite natural that September would
also be the month in which (after many years of collaboration) patient support
and advocacy groups from around the world should come together as the
International ITP Alliance.
(CONTINUED ON PAGE 7)
IMPORTANT: The information contained in this newsletter is for educational purposes only.
For advice on your unique medical condition please consult a health care professional.
What Our Friends Are
Saying on Facebook
21
Are You Looking for
Great Holiday Gifts?
21
Advocacy Alert
22
ITP in Kids Q&A
23
Living with ITP – Patients
Share Their Stories
26
Kids Korner
27
2
The Platelet News
www.pdsa.org
From the Executive Director
Ten years ago when I was
serving as a PDSA board
member and working in the
media industry, there was a
rumor that actress Julia
Roberts had ITP. Not that I
would ever wish ITP on
anyone, but we were excited
with the possibility that one
of the biggest stars on the planet would
bring much needed awareness for ITP,
and help raise funding for research. I
sent a media kit with information and
resources about ITP and PDSA to Julia’s
publicist, but I never heard back. I can’t
say whether Julia Roberts had ITP. I
have heard rumors that there are
several well-known people living with
ITP, but keep their diagnosis a secret
because of fear that having a chronic
illness might impact their career.
At the recent NORD (National
Organization for Rare Disorders) Summit,
parent advocate Kristen Gray shared
with 600 conference attendees her
family's personal and inspiring story to
try and cure Batten disease. Kristen and
her husband Gordon founded The
Charlotte & Gwenyth Gray Foundation
after learning that both their daughters
had this aggressive, degenerative and
fatal brain disease. Less than 10
children in the world currently live
with Batten disease. Almost one year to
the day after learning that their older
daughter had been diagnosed with
Batten disease, the Grays raised $3.2
million dollars and were funding a gene
therapy clinical trial.
The Gray’s story is very compelling and
I was astonished that they could raise so
much money in such a short period of
time for an extremely rare disease. I know
a mother’s (and father’s) love can move
mountains, but how were they able to
accomplish so much in such a short
period of time? Then Kristen shared
with the audience that her
husband was a big Hollywood
producer. He enlisted his
celebrity friends like Jennifer
Garner, Dwayne Johnson,
Rihanna and Jon Hamm to
host fundraisers. People were
motivated to donate to their
cause because their daughters
had been handed a death sentence and
time was not on their side.
PDSA might not have any big celebrities
to promote our cause, but what we do
have are thousands of “stars” who
quietly support our organization with
their generous donations and our
Awareness Champions who raise funds
and awareness about ITP.
Over the past 18 years, PDSA has done
an incredible job of supporting and
educating ITP patients and their
families. But we need to do more.
ITP is an invisible illness — most of us
with the disease “look fine” and it’s not
considered a fatal disease. However,
while dying from ITP is rare, it still
happens. I know patients and caregivers
don’t want to hear that, but it is the
cruel reality of this disease. Just in the
past couple of months PDSA received
donations in memory of three ITP
patients — one was a ten-month old
boy, Talen Chao, who passed away two
days after being diagnosed with ITP
from catastrophic bleeding in his brain.
His mom, Anna Symuong-Chao,
contacted PDSA looking for answers
and wanted to share her family’s story.
And what about the ITP teen who is
hospitalized on a monthly basis with
excessive bleeding or the young adult
who cannot work, participate in
“normal” activities, and barely leaves
the house because of his ITP?
(CONTINUED ON THE NEXT PAGE)
PDSA Board Members
Peter Pruitt, Chairman, Coral Gables, FL
Jay Charness, Vice Chair, Arvada, CO
Kim Everett, Treasurer, Mishicot, WI
Beth Siegelbaum, Secretary, Norwalk, CT
Karen Avrick, Woodbury, NY
Brian Bamesberger, Loch Lloyd, MO
Jamie Harwell, Helena, MT
Diane Joseph, McLean, VA
Linda McGuirl, Basking Ridge, NJ
Dale Paynter, Cambridge, ON, Canada
Join…
Your valuable donation will enable PDSA
to enhance the programs and services
that help those suffering with ITP
and other platelet disorders.
For more information about making
a gift of $1,000 or more, contact
Caroline Kruse, Executive Director,
at (877) 528-3538 toll free
or by e-mail, [email protected].
Thank you to sponsor
for supporting
the PDSA Website:
www.pdsa.org
Platelet Disorder Support Association
The Platelet News
Carol Hoxie, Managing Editor
Platelet Disorder
Support Association
Caroline Kruse, Executive Director
Brenda Foster, Administrative Manager
Carol Hoxie, Communication Specialist
Alexandra Kruse, Research Coordinator
Nancy Potthast, Director of Marketing
Jody Shy, Programs & Events Manager
Sue Zemon, Chief Financial Officer
Jeff Cooper, Web site and
Social Media Manager
phone 1-87-PLATELET
3
ITP Conference 2016: Part 2
In this fall issue PDSA continues its coverage of highlights from presentations at
the 2016 ITP Conference in Orlando, FL on July 8-10. Part 1 of the highlights
appeared in the summer issue of The Platelet News. To hear full details of the 2016
conference presentations be sure to order your copy of the Conference USB Flash
Drive now available: https://www.pdsa.org/products-a-publications/the-plateletstore.html#!/2016-ITP-Conference-Video/p/69192534/category=4605129
Communicating with your Doctor:
What Every ITP Patient Wants Their
Doctor to Know
ITP CONFERENCE 2016 • DRS. DONALD ARNOLD, JAMES BUSSEL, TERRY GERNSHEIMER,
CRAIG KESSLER, DAVID KUTER, HOWARD LIEBMAN, AND JOHN SEMPLE
By Alexandra Kruse
Joan Young, Founder
If any of the PDSA medical advisors wanted to quit their day job (and we hope that
they wouldn’t!), we now know they have a promising career in acting thanks to the
ITP Conference Saturday session, “Communicating with Your Doctor.” Each physician
team role-played common scenarios that ITP patients experience with their hematologist.
The Platelet News
is published quarterly by
A nonprofit organization dedicated to
enhancing the lives of people with ITP
and other platelet disorders through
education, advocacy, research, and support.
The first ITP patient, Donald
(Dr. Arnold), has been bruising
for the past month. He was
admitted to the ER with a
platelet count of 2,000 and feels
afraid. He was referred to Dr.
Craig Kessler, who inquired
about Donald’s family history
of low platelets. Dr. Kessler
examined Donald’s blood smear
(CONTINUED ON PAGE 6)
PDSA medical advisors Dr. John Semple (L) and Dr. David
Kuter (R) in this entertaining conference session team
role-played common scenarios that ITP patients
experience with their hematologist
Thank you to our 2016 National ITP Conference Sponsors
Platinum Sponsor
Gold Sponsor
Gold Sponsor
Silver Sponsor
For more information contact:
PDSA
8751 Brecksville Road, Suite 150
Cleveland, OH 44141
Phone: 1-87-PLATELET or (440) 746-9003
Fax: (844) 270-1277
www.pdsa.org • [email protected]
PDSA is a 501(c)3 organization.
All contributions are tax deductible.
From the Executive Director
CONTINUED FROM PAGE 2
Thanks to the financial contributions of
so many, we are excited to announce
that we are now at a place where we can
start funding and conducting several
research studies on an annual basis, and
have even added a research coordinator
to our staff. There are still so many
unanswered questions when it comes to
ITP, and we hope this new research will
yield some much needed answers.
Caroline Kruse, Executive Director
Platelet Disorder Support Association
4
The Platelet News
www.pdsa.org
Making a Difference
George R. Buchanan, MD
By Caroline Kruse
Dr. George Buchanan, pediatric hematologist and ITP specialist, has
served as President of the American Society of Pediatric HematologyOncology (ASPHO) and twice on the Executive Committee of the
American Society of Hematology (ASH). His national honors include
the Distinguished Career Award from ASPHO, the Mentoring Award in
Clinical Research from ASH, and the Arnold Gold Foundation Award
for Humanism in Medicine from the American Association of Medical
Colleges. Dr. Buchanan has retired from the University of Texas at
Southwestern Medical Center in Dallas, TX.
Bleeding severity and quality of life are
key factors when an ITP expert
physician and patient/caregiver consider
a treatment plan. Unfortunately, it
wasn’t always that way. Less than 10
years ago most hematologists relied
solely on the platelet count (and some
still do) when deciding whether to treat
or not to treat. But not Dr. George
Buchanan. Calling himself a ‘radical
nihilistic “non-treater”,’ Dr. Buchanan
has worked tirelessly for 40 years to
improve the lives of children with ITP.
Dr. Buchanan, a pediatric hematologist,
recently retired from the University of
Texas Southwestern Medical Center in
Dallas TX. In honor of Dr. Buchanan’s
distinguished career and numerous
contributions to the field of ITP
research and advancement of patient
care, I asked him to answer a few
questions about his lifework.
Q: Why did you decide to specialize in
ITP and what has been your approach
to treating children with ITP?
A: My introduction to ITP was during
my pediatrics residency in Chicago in
the early 1970’s. After encountering
several affected children with ITP I came
across and was influenced by a 1966
publication in Journal of Pediatrics by
Drs. Jeanne Lusher, then a fellow in
pediatric hematology in Detroit, and
her mentor Wolf Zuelzer, a legendary
figure who was one of the “fathers” of
pediatric hematology as a distinct
discipline. They described the extensive
use of steroids to treat ITP, indicating that
it could temporarily raise the platelet
count but was associated with many
undesirable side effects. Although they
did not perform a rigorous randomized
study (virtually no one did in those
days!), their conclusion was that steroids
should be avoided in most children with
ITP given that the quite substantial side
effects did not usually justify their use
in such a self-limited condition.
This experience influenced me greatly
to become a hematologist and paved
the way to my approach to managing
ITP (which continues to the present) as
a “minimalist” or “non-interventionist”
who treats the patient, not the platelet
count, but instead uses bleeding
severity and quality of life as the
primary determinants of management.
Q: What research have you been
personally involved in and what do you
hope your contributions or legacy will
be in regards to ITP?
A: After completing my hematologyoncology training in 1977 I accepted a
faculty position at UT Southwestern and
Children’s Medical Center Dallas, where
I have since remained. For years I
(CONTINUED ON THE NEXT PAGE)
Medical Advisors
Donald M. Arnold, M.D.
McMaster University
Hamilton, Ontario, Canada
James Bussel, M.D.
Weill Medical College, Cornell University
New York, NY
Douglas Cines, M.D.
University of Pennsylvania Hospital
Philadelphia, PA
Amy Geddis, M.D., Ph.D.
Seattle Children’s Hospital
Seattle, WA
Terry Gernsheimer, M.D.
Puget Sound Blood Center • Seattle, WA
Andra H. James, M.D.
Duke University • Durham, NC
Craig Kessler, M.D.
Georgetown University Hospital
Washington, DC
David Kuter, M.D., D.Phil.
Massachusetts General Hospital
Boston, MA
Michele P. Lambert, M.D.
Children’s Hospital of Philadelphia (CHOP)
Philadelphia, PA
Howard A. Liebman, M.D.
Norris Cancer Center Hospital
Los Angeles, CA
Diane J. Nugent, M.D.
Children’s Hospital of North Orange
County (CHOC) • Orange, CA
Drew Provan, M.D.
Barts & The London Queen Mary’s
School of Medicine & Dentistry
London, UK
John Semple, Ph.D.
St. Michael’s Hospital • Toronto, Canada
Michael Tarantino, M.D.
Bleeding & Clotting Disorders Institute
Peoria, IL
Emeritus
Robert McMillan, M.D.
The Scripps Research Institute • La Jolla,CA
Platelet Disorder Support Association
“For more than 40 years,
Dr. Buchanan has made countless,
meaningful contributions to our
understanding of pediatric benign
and malignant hematology, especially
on the optimal management of ITP
in children. Like many pediatric
hematologists in the US and around
the world, I am fortunate to have
learned from Dr. Buchanan over
the years. He is truly a legend in
the field.” – PDSA Medical Advisor
Michael Tarantino, MD,
Medical Director and President,
The Bleeding and Clotting
Disorders Institute
“Dr. Buchanan has been a leader in
the field of pediatric hematology.
He has conducted numerous research
studies that have improved the care
of children. He especially focused on
understanding the disease from the
perspective of the patient and
learning how to capture clinically
relevant information. In ITP for
example, he led the way in capturing
important outcomes like bleeding
symptoms and changed practice by
focusing treatment decisions on
symptoms rather than the platelet
count. As a clinician he engaged
families in shared decision making,
taking the time to discuss the disease
and treatment options, and
understand their goals and concerns.
He has trained over 80 fellows and
has made an impact on the career of
many individuals. As his mentee, he
was always supportive and you
worked hard because he believed in
your success.” – Cindy Neunert, MD
MSCS, Columbia University Medical
Center, Chair of the Pediatric ITP
Consortium of North America (ICON)
phone 1-87-PLATELET
5
Making a Difference
CONTINUED FROM PAGE 4
practiced oncology as well as hematology
and was Division Director between
1980 and 2011. Our program was a
large one, so I had the opportunity to
see and care for many children with ITP
and noted, like Dr. Lusher and many
others, that most of them did quite well
without steroid treatment. By this time
the debate (steroids vs no steroids) was
intense in the childhood ITP world, as
were other controversial issues such as
the need for a bone marrow exam to
“rule out” leukemia and whether
hospitalization was necessary when the
platelet count was extremely low. Being
a bit frustrated by the lack of formal
prospective studies in children with
newly diagnosed ITP, I designed and
conducted a clinical trial randomizing
such patients to steroids or a placebo/
sugar pill and measured over the next
four weeks their platelet counts,
bleeding times, and clinical “bleeding
scores.” Upon its publication in 1984
it was the first (or one of the first) to
randomize ITP patients to steroids
versus no drug treatment and to
quantitatively assess bleeding severity.
The 1980’s were when IVIg was
introduced and shown to be beneficial
(and in most ways superior to steroids)
as a means to promptly raise the platelet
count. Although I endorsed its use to
treat significant bleeding or prevent it
upon injury or need for surgery, I was
cautious in view of its great expense,
adverse effects, requirement for an
intravenous infusion, and widespread
acceptance by many ITP treaters. In
editorials, commentaries, and face to face
“debates” with colleagues I stuck by a
“non-interventionist” philosophy, which
was shortly thereafter challenged by the
introduction of anti-D immunoglobulin
as a seemingly more “user friendly” means
of quickly raising the platelet count
(albeit introducing intravascular hemolysis
as a potentially serious adverse effect).
My reputation as radical nihilistic
“non-treater” probably continued to
grow, yet during the 1990’s it was
gratifying to me that many others
shared similar views, as manifested by
their willingness to “protest” in a letterto-the-editor in Blood accompanying its
publication in 1996 of guidelines
recommending routine steroid or IVIg
treatment of children with newly
diagnosed ITP. Although the debates
have continued regarding ITP treatment
philosophy, I and many others
subsequently “moderated” our views
such that an “era of good feeling” began
to prevail in the pediatric ITP arena and
— much to everyone’s satisfaction —
has continued to the present.
Interaction among ITP “treaters” and
investigators is now rarely heated and
often very productive. Organizations
such as the Platelet Disorder Support
Association (PDSA), the ITP Support
(CONTINUED ON THE NEXT PAGE)
PDSA’s Executive Director Caroline Kruse with
Dr. George Buchanan at the American Society
of Pediatric Hematology/Oncology’s (ASPHO)
29th Annual Meeting in Minneapolis, May 2016
6
The Platelet News
www.pdsa.org
Communicating with your Doctor
CONTINUED FROM PAGE 3
and didn’t notice abnormalities on his
red or white cells — were there other
autoimmune symptoms that could
contribute? Donald thought his
diagnosis was a mistake and mentioned
his physician wanted to perform a bone
marrow examination and started him
on prednisone, which made him feel
hyperactive.
Dr. Kessler informed Donald that most
hematologists don’t perform bone
marrow biopsies on ITP patients anymore.
He explained that controversy on how
to treat ITP patients exists because of
the variety of treatment options. Dr.
Kessler recommended the next step is
raising Donald’s platelet count to
minimize bleeding and that Donald needs
to give up his hobby of playing hockey.
ITP patient, Terry (Dr. Gernsheimer), has
taken prednisone for six months and is
seeing Dr. Howard Liebman to seek new
treatment. She’s worried about having
cancer because she’s being treated at a
cancer center. Dr. Liebman explained the
close association between hematology
and oncology medical training,
meaning many hematology patients
(without cancer) just receive treatment
at cancer centers. Terry disclosed that
prednisone has made her gain weight
and feel sick, restless, and moody.
Dr. Liebman reassured Terry of alternative
approaches: some treatments have a high
frequency of response and eliminate the
need for steroids; others have lower
responses but could result in remission.
Dr. Liebman recommended Rituxan,
explaining that although Rituxan was
originally developed to treat immune
system tumors, it’s now used in immune
disorders to deplete B-cells. Splenectomy
was also suggested but has potential long
term risks and could be managed by a
patient observant about immunizations,
infections, and blood clots. Dr. Liebman
recommended TPO-agents, which elevate
platelet production so that Terry won’t
have to take prednisone. Dr. Liebman
clarified that although ITP therapies
cannot cure ITP, treatment could improve
Terry’s symptoms and quality of life.
The last patient of the day, “Ms. Semple,”
(Dr. John Semple) has had ITP with a
count of 10,000 for 30 years and has
tried steroids, IVIg, Rituxan®, and TPO.
She didn’t want to remove her spleen
because of her fear that the treatment
won’t work. She saw Dr. David Kuter
who reassured her that most patients
initially see high rates of response and
tried to calm her anxiety by telling Ms.
Semple that her spleen could be taken
out safely, either by getting her blood
counts up with platelet transfusions or
maybe having an older surgeon perform
the operation, as older surgeons are
more used to performing splenectomies
with low platelet counts. After inquiring
about Ms. Semple’s family history and
bone marrow biopsy results, Dr. Kuter
wanted to check for anti-platelet
antibodies and thrombopoietin levels to
validate her ITP diagnosis. Dr. Kuter also
suggested combining therapies such as
steroids and Nplate®/Promacta®, or
enrolling in new experimental research
studies. Although Ms. Semple felt that
she had tried most of the common ITP
therapies without any luck, there were
still a variety of other treatments that
remained and could allow her to live a
normal life with ITP or cause remission.
Making a Difference
CONTINUED FROM PAGE 5
Association in the U.K., the
Intercontinental ITP Cooperative Study
Group in Switzerland (ICIS), and ITP
Consortium of North America (ICON)
have provided highly effective
networking opportunities between
investigators, clinicians, and patients
with ITP. I have had the great fortune of
being involved with each of these
marvelous groups.
ITP management guidelines that are
evidence-based are now consulted and
frequently employed as well as being
subject to periodic review. New agents
to effectively manage persistent and
chronic ITP are available and undergoing
study in children as well as adults.
Bone marrow exam and hospitalization
are no longer features of “routine” care
in most centers. We’ve come a long way
since my “initiation” to ITP 30+ years ago!
Q: What does the future hold for you
and will you still be involved in the
world of ITP?
A: An important obligation we have as
academic leaders in the ITP arena is
identifying, nurturing, and supporting
the success of young investigators. I
take special pride in the many past and
current physician trainees in our Dallas
program who have focused on ITP.
As my career as an ITP doctor is winding
down, I have not regretted for a moment
my longstanding involvement with ITP
patients, their families, and their primary
physicians. I will no longer be doing
clinical work seeing patients, but I hope
to remain connected with PDSA
(including their wonderful teleconferences
with young ITP patients and their
parents), ICON, ICIS, and the ASH
Guidelines panel. I will also watch from
the “sidelines” what I expect to be major
advances during the coming years in
better understanding the pathophysiology,
treatment, and cure of ITP. It has been a
wonderful privilege being a member of
the international “ITP Family!”
Platelet Disorder Support Association
phone 1-87-PLATELET
7
September is ITP Awareness Month
CONTINUED FROM PAGE 1
On Friday, September 16th, delegates
from Denmark, Finland, India, Italy,
Netherlands, Sweden, United Kingdom
and the United States met at The Shaw
Country House Hotel in Melksham,
Wiltshire, United Kingdom for a
meeting organized by the ITP Support
Association. For many years, these
patient groups, as well as others from
around the world, have been
collaborating to educate, support and
advocate for the ITP community and
International ITP Alliance members (pictured
left to right): Barbara Lovrencic (Italy), PDSA
Executive Director, Caroline Kruse, Monika
Westerberg (Sweden), Marjo Holmala (Finland)
and PDSA Marketing Director, Nancy Potthast
have “officially” joined forces as the
International ITP Alliance — the
intercontinental partnership of ITP
patient support organizations
committed to education, awareness and
establishing a global voice for immune
thrombocytopenia patients.
As each representative shared details
relating to their organization’s size,
communication vehicles and means of
support, the gateways and barriers
existing within geographic borders
became clearly evident — there are
substantial differences in patient needs
based on their country of origin.
Amidst the vast contrasts in healthcare
funding and patient support, however,
there emerged critical common ground
— patients overwhelmingly agree that
the patient voice is intensely deficient
regardless of geographic location.
There are four short years left as we
round out a decade of ITP awareness.
The time has come to share resources,
learn from each other’s trials and
Pictured L to R at the ITP Support Association
Charity Event in Putney, London are Caroline
Kruse (Executive Director, PDSA), Barbara
Taylor (Novartis), Nancy Potthast (Marketing
Director, PDSA), Rosemary Elston, John
Flemming, MD (Novartis), and Derek Elston
(Board Member ITP Support Association)
successes, advance research, focus on
patient-centered care and treatments,
and create a global voice for immune
thrombocytopenia patients. For more
information about the International ITP
Alliance, photos, global news, and plans
for ITP Awareness in 2017 visit
www.GlobalITP.org.
PDSA Launches First ITP Natural History Study Registry
CONTINUED FROM PAGE 1
and Drug Administration (FDA), and a
committee of leading hematologists,
ITP patients, and caregivers.
Registries can provide health care
professionals and researchers with firsthand information about people with
certain conditions like ITP and other
platelet disorders, individually and as a
disease population, to increase our
understanding of that condition over time.
Rare diseases like ITP have posed unique
challenges to researchers and drug
developers because of small patient
populations, lack of data, clinical
endpoints that are often unclear, and
enrollment and retention challenges for
clinical trials. NORD’s Natural History
Studies project empowers patients and
families to help eliminate some of the
uncertainty in rare disease research,
making way for progress.
Natural history studies are longitudinal
studies that aim to fill research gaps,
which help medical researchers better
understand how diseases progress over
time. They yield vital information essential
to clinical trial design, such as biomarkers,
demographics, important clinical
symptoms, genetic and environmental
variables, and patient perspectives.
Your participation in our secure,
confidential HIPAA-compliant registry is
likely to increase what we know about
ITP and other platelet disorders. It will
help healthcare professionals improve
treatment, and allow researchers to
design better studies on a particular
condition, including development and
testing of new treatments. The more
patients who are registered the more
data researchers will have to further
their work, which can accelerate
research into new therapies for ITP and
potentially a cure one day. More
information on the patient registry
will be coming soon.
8
The Platelet News
www.pdsa.org
Walk / Run News
VISIT WWW.ITPWALK.ORG TO FIND A LOCATION NEAR YOU!
Cranberry Township, PA Pump It Up For Platelets!
Event Date: August 7, 2016 • Total Raised: $5,627.65
Read Cayden’s ITP Journey:
http://bit.ly/2fRGiGI
Superheroes unite in the fight in support of ITP Survivor,
Cayden Tomczak (pictured front center with his sister and event
organizer, Lanni Mozzoni)
Team Cayden strikes a pose in support of their little
superhero, Cayden Tomczak. Cayden’s Mom Kristen (back row, second
from left) knows first-hand that real superheroes often live in the
hearts of small children fighting big battles. Her little ITP Warrior beat
ITP.
Cranberry Township, PA event organizer, Lanni
Mozzoni takes a break from event set up to pose with her brother
and ITP Survivor, Cayden Tomczak.
Rancho Cucamonga, CA Pump It Up For Platelets!
Event Date: September 10, 2016 • Total Raised: $3,340
Read Barbara’s ITP Journey: http://bit.ly/2eXTTLv
ITP Warrior, Freddy Tinajero, Jr. (center) takes a break
from the festivities for a picture with his parents, Maria and Freddy,
Sr.;
ITP Warrior Freddy (left) walks with one of his
fans, younger brother Jose (right).
Team Freddy with their impressive showing in support of their
young ITP Warrior, Freddy Tinajero, Jr.
Thank you to our National
2016 Walk / Run Sponsors
Silver Sponsor
Silver Sponsor
Bronze Sponsor
Platelet Disorder Support Association
phone 1-87-PLATELET
9
Chicago, IL Pump It Up For Platelets!
Event Date: September 17, 2016
Total Raised: $15,039.31
Read Trish’s ITP Journey: http://bit.ly/2froG3V
It’s a wrap! Chicago event organizers and volunteers
gather in celebration of another successful event.
ITP Warrior Caden Slad
(center with purple hair) lets loose
with team members after a job
well-done. Team Caden raised over
$1,600 in support of PDSA programs
and research;
And they’re
off! Chicago participants hit the
trail for the third annual Chicago,
IL Pump It Up For Platelets! event
at Half Day Forest Preserve in
Vernon Hills, IL.
Orange, CA Pump It Up For Platelets!
Event Date: September 18, 2016
Total Raised: $7,446
Read the Journey of Four ITP Warriors,
a Mom and their fight against ITP:
http://bit.ly/2eTBaNT
Orange, CA event
organizers (left to right) ITP
Warrior Cathy Aldama, ITP Mom
Leilani DeCastro, ITP Warrior
Melissa Hilsabeck and ITP Warrior
Kelly Torres with little ITP Warrior,
Joey DeCastro (front center)
Bridging the geographic gap.
Because they are spread all across the
country, the Ugly Potatoes set up a virtual
team in support of ITP Warrior Aandrea
Hays (center) and raised over $1,100
The Cal State Fullerton Titans
baseball team shines their support in honor
of little ITP Warrior Joey DeCastro as part
of Team Ikaika
Sandgap, KY
Pump It Up For Platelets!
Event Date: September 30, 2016
Total Raised: $1,555
Read Anthony’s ITP Journey:
http://bit.ly/2fRLZ7s
The Sandgap, KY group of
participants was small and mighty (like
little ITP Warrior Anthony) raising
$1,555 for PDSA programs and research!
Event Organizer and ITP
Mom, Amanda Johnson with her little
ITP Warrior Anthony.
(CONTINUED ON THE NEXT PAGE)
10
The Platelet News
www.pdsa.org
Outstanding Teen Session
at ITP Conference 2016
By Sam Kalina
For many teenagers with ITP, finding
people of similar age who understand
the adversity of a chronic blood
disorder is a challenge. At the annual
ITP conferences, the teen session is a
chance for teenagers and young adults
to meet, connect, and form lasting
friendships.
This year’s 2016 conference offered not
just a single teen session, but an entire
separate track with multiple special
sessions dedicated to the teen/young
adult age group. Leading the sessions
were Dr. Michael Tarantino and Dr.
Michele Lambert. On the first night of
the conference, all the teens gathered
for a casual meet and greet, and each
participant was given a fun, blank
“heroes of science” T-shirt to personalize
and color with fabric markers.
During this time, the teens
were able to discuss a topic
given to them by Dr.
Tarantino that would be the
The teen group session at ITP Conference 2016
segue into the next day’s
session: what is the biggest
controlled, and issues that could affect
problem in ITP? For some, their biggest
the study results. With the combined
problem was bruising. For others, it was
brainpower of all the teens, as well as
frequent hospital visits. But as a group,
Dr. Tarantino, and Dr. Lambert, a very
the teens decided to progress further
realistic study was created.
with the issue of fatigue.
In the next session, Dr. Tarantino and
Dr. Lambert assisted the teens in creating
a pseudo-study in order to solve the
issue of fatigue in ITP patients’ lives.
Some of the discussion included ideas on
how to measure fatigue, how frequently
it should be measured, variables to be
Overall, the teen sessions were
wonderful environments for teens/young
adults to learn about the scientific
process, meet and befriend others with
ITP, and have fun. The ITP Conference
2016 teen sessions were great successes,
and next year’s will only be better!
Walk/Run News
CONTINUED FROM PAGE 9
Cleveland, OH
Pump It Up For Platelets!
Event Date: October 22, 2016
Total Raised: $6,263.00
Read Caroline’s ITP Journey:
http://bit.ly/2eyi5VW
The 42 degree rainy weather didn’t put a damper on the first Cleveland, OH Pump It Up for
Platelets! event. PDSA Executive Director Caroline Kruse with her family (L to R), sister Suzanne Kerner, parents
Gil and Pat George, ITP Warrior Caroline and husband Ken, and niece Lexi Kerner (front). The Kerner
Purple Platelets team raised $1,160!
Healing Hailey was the top fundraising team, raising
$1,310 for PDSA programs and research. 29 family members and friends rallied to support seven-year old
ITP Warrior Hailey Leffel!
The Johnson Family from
Warren, Michigan drove about three
hours to attend the event and support
little 3-year-old ITP Warrior Ava
The Brecksville/Broadview Heights
High School National Honor Society
members who volunteered as part of
their community service enjoyed sporting
purple to show support for PDSA and the
six ITP Warriors of this year’s event!
Platelet Disorder Support Association
phone 1-87-PLATELET
11
ITP in Adults: Q&A
ITP CONFERENCE 2016 • PANEL WITH DRS. DONALD ARNOLD, CRAIG KESSLER, JAMES BUSSEL, HOWARD LIEBMAN,
TERRY GERNSHEIMER, JOHN SEMPLE, AND DAVID KUTER
By Carol Hoxie
During this Saturday afternoon session
patients had an opportunity to ask
questions of the doctors’ panel on a
wide range of topics.
Q: How often should bone marrow
biopsies be done in ITP patients?
A: Dr. Kuter: Never. I haven’t done one
in a bonafide ITP patient in about 30
years. It is not part of the guidelines for
evaluating any ITP patient. There is no
need for it if you get a rise in platelets
from steroids.
Dr. Kessler: If someone has regular runof-the-mill ITP, then it’s not needed, but
if you’ve been treated for ITP a while,
are not responding to treatment, and
have physical exam findings not in
keeping with ITP (enlarged spleen or
anemia, for example), it might be
justified to do bone marrow testing.
Dr. Arnold: Some studies included bone
marrow tests on patients who were on
the TPO treatments Nplate®
(romiplostim) or Promacta®/Revolade®
(eltrombopag). The studies found no
significant differences in bone marrow
that was of concern. They concluded
there was no need for surveillance bone
marrows in patients on TPO agents.
Q: I understand CD4+ Treg cells can
reduce the tolerance to infection in their
absence. When there is reduced number
of Tregs can it result in a loss of tolerance
to the bacteria in food? My hematologist
plans to test me for celiac disease.
A: Dr. Semple: I don’t think so.
If anything, you’d be more hyperresponsive to the bacteria. I don’t think
the low Tregs that you see in active
autoimmunity will set you up for
increased bacterial infections.”
Q: Is there any likelihood ITP could
evolve into leukemia or lymphoma?
A: Dr. Kessler: There’s no direct link
between ITP and development of
underlying malignancy.
Lymphoproliferative disorders can be
affected by autoimmune problems. If you
have a patient who presents with ITP and
has some other physical abnormalities
then it’s fair game to look for some
other secondary stimulus. However, ITP
itself does not lead to malignancy.
My hematologist thinks my ITP is
triggered by multiple viruses in my
body. I was tested and found positive
for Epstein-Barr virus (EBV), CMV,
herpes, and a couple others. Can I
eliminate those viruses from my body?
I had black toxic mold syndrome where
this all started. Doctors are telling me
those viruses are triggering my ITP
episodes. I’ve been on steroids a long
time but off them the past four weeks.
A: Dr. Kuter: To verify someone is
virally infected two documented tests of
antibody are needed: 1) Evidence you had
the virus and have now cleared it, and
2) the actual viral load. If you have an
active virus in circulation that could
possibly be a trigger for ITP, a high viral
load could affect a person’s immune status.
Dr. Liebman: In Chronic Fatigue
Syndrome there is a subset of people
who can’t handle Herpes viruses,
including EBV and CMV. However, I’ve
not heard of a case of ITP associated
with this syndrome. You should see a
good immunologist for a full work up.
Dr. Kuter: Getting off steroids may be
key here. It’s important to look at using
other agents to raise your platelets
instead of chronic steroids.
some people who are doing research on
rapid screening on a Smartphone for
hematocrit (hemoglobin) but I’m not
aware of anything for platelet testing.
Dr. Kuter: I have a patent on a device
that never got picked up by industry. It
could be done by measuring serotonin
in platelets using spectra photography
the way they measure blood sugar.
Testing is affected by people taking
SSRIs and a lot of other stuff, which is
why it didn’t get picked up by industry.
Most patients don’t need platelet
counts more than about once per
month (twice per month in some
exceptions). The idea that you need a
platelet count every time you get an
Nplate shot is nonsense. As to testing
frequency, unless you live in a remote
area, there’s usually a blood center
nearby to get your test done readily.
You need frequent counts only if you
are changing dosage of your treatment.
Dr. Liebman: As to platelet count
results, be aware that sometimes blood
is drawn one day but platelets are not
tested until a day later, which can lead
to lower counts and false readings. On
your lab results check the time of the
draw and the time tested.
Dr. Kuter: There are some problems
with differences in how labs count the
platelets. Some labs, for instance, don’t
measure the large platelets, which leads
to a lower count. Please be nice to your
hematologist — get your labs done
early in the morning. When you get
your counts done at 4 p.m., then (by
law) your hematologist gets notified by
labs running the tests at 4 a.m. letting
me know someone has a low count.
Q: Is there any fast blood-screening
Q: How well does Rituximab work? Is it
platelet test that could be done at home
or any ongoing research on home
platelet tests?
A: Dr. Gernsheimer: I’m working with
safe to use it every 2 – 3 years? Could it
cause CVID (common variable immune
disease)? What are the long-term side
Q: Do viruses in the body affect ITP?
(CONTINUED ON PAGE 24)
12
www.pdsa.org
The Platelet News
Purple Power Spreads New ITP Awareness!
In fall 2016 Sport Purple for Platelets Day,
held September 30th, once again raised
public awareness of ITP and platelets
across the US and around the world.
Many of this year’s participants sent
PDSA their great ‘sport purple’ photos
(shown here). Adults, teens, and kids
wore bracelets, tee shirts, and sported
purple attire, fingernails, and hair. Even
pets wearing purple joined in the fun.
Schools, offices, and sports teams wore
their purple shirts. Some ate purple
cookies and cupcakes! Whatever activities
or events you took part in to help PDSA
raise ITP awareness this fall, we heartily
thank you for joining in this year’s effort.
Platelet Disorder Support Association
phone 1-87-PLATELET
13
Dear Caroline,
I wanted to share with you that we had
a great deal of fun at Rigel Sporting
Purple for Platelets!! Our employees are
very pumped up to be part of the effort
and I wanted to compliment you and
your team for doing this event. It binds
the entire ITP community together. I am
glad we were able to be part of it.
Best regards, Raul Rodriguez,
President & CEO, Rigel Pharmaceuticals, Inc.
14
The Platelet News
www.pdsa.org
PDSA’s ITP Poke-R Club Abstract Chosen
as One of the Top Posters at NORD Summit
By Alexandra Kruse
At this year’s NORD Rare
Diseases and Orphan
Products Breakthrough
Summit, participants
were invited to submit a
scientific poster abstract
on “Life-Transforming
Treatments: Innovative
Research, Medical
Education Advancement,
and Patient Community
Building.” We felt that
PDSA’s ITP Poke-R Club
(L – R) PDSA’s Alexandra Kruse and Nancy Potthast pose with
for Kids with ITP fit the
their winning ITP Poke-R Club poster at the October 17-18 NORD
criteria and we knew
Rare Diseases and Orphan Products Breakthrough Summit in
that the Poke-R Club
Arlington, VA
was a unique and
Sixty-seven percent of participants
uplifting program for children battling
enjoyed everything about the program,
ITP, but we needed more information to
including BUZZY, the distraction cards,
assess the real impact it has on our
the prizes, and how it eased their
pediatric community.
anxiety and helped them to feel less
alone in their ITP journey. Overall, the
We created a survey and sent it to
Poke-R Club program received an
participants in the Poke-R-Club. We
average rating of 9/10 by participants.
then analyzed their responses to
questions regarding demographic
Needle phobia significantly decreased
information, needle phobia and anxiety
after enrollment in the Poke-R Club,
before and after introduction to the
demonstrating that PDSA has
Poke-R Club, their favorite and least
accomplished its goal in minimizing
favorite parts about the program, and
the anxiety correlated with treatments,
how they felt overall about the
draws, and pokes, although we also
program. A majority of respondents
found that Buzzy doesn’t completely
claimed the best part of the Poke-R
eliminate pain from venipuncture. The
Club program was the BUZZY device or
program has a significant positive
that it made the child feel special.
impact on pediatric patients with ITP,
and is most effective for children and
preteens who have chronic ITP.
Maddie F., who has ITP, happily displays the
award and prize she won in the Poke-R Club
Not only was PDSA selected to present
“ITP Poke-R Club: A Clinical Support
Program Bringing Comfort and Support
to Kids Coping with Immune
Thrombocytopenia” at the Summit, but
our poster abstract was chosen as one of
the top three abstracts out of 40 posters
(CONTINUED ON THE NEXT PAGE)
In Appreciation
The following individuals were active
members of the CIRCLE OF HOPE
between July 1 and September 30, 2016.
Circle of Hope members donate or raise
$1,000 or more within a calendar year and
serve as PDSA’s philanthropic leaders.
April – June, 2016
Cathy and Raul Aldama • Emily Auterson
• Andrew Avrick • Karen and Adam Avrick
• Stuart Avrick • Sydney Avrick • Cindy
and Philip Ayliff • Kenda and Brian
Bamesberger • Kim and Robert Barber
• Amita and Ranjive Bhalla • James
Bussel, MD • Brendan Cameron • Leilani
de Castro • Barry Deutsch • Rachel and
Patrick English • Robert Feiner • Allison
and Keith Flowers • Susan Frank • Jan and
Steve Gardner • Terri and Michael Goldberg
• Wanda Gregory • Jen and Benjamin
Grimes • Kim and Rodney Hall • Kristin
Henrikson and Jon Brandt • Noelle and
Mat Heyman • Melissa Hilsabeck • Madeline
and John Hromyak • Kristin Hunt • Karen
and David Imig • Amanda Johnson • Anne
and Pitkin Johnson • Joan and Richard
Jordan • Diane and Robert Joseph • Sharon
Kaye • Emily and Kris Kile • Louise Kittel
• Alexandra Kruse • Caroline and Ken
Kruse • Irene and David Kuter, MD, DPhil
• Paula Lagree • David Lihani • Jenn and
Andy Lindal/For the Love of Gracie
• Barbara Link • Mary Lou Lyons • Kristie
Lyons • Eleonore and Russell McCabe
• The McGuirl Family • Irma and Daniel
Miles • Stephanie and Mike Moran • Lani
Mozzoni • Destiny Owen • Jeanne and
Dan Pinnell • Nancy and Michael Potthast
• Jacqueline and Bruce Prescott • Meredith
Prescott • Barbara and Peter Pruitt, Jr.
• Shirley and Peter Pruitt, Sr. • Patricia Pulley
• Angela Racoosin • Virginia Rennie
• Martha Roberts • Steven Rodgers
• Karen Rosenbaum • Maria Soledad Ruiz
(CONTINUED ON PAGE 17)
Platelet Disorder Support Association
15
phone 1-87-PLATELET
PDSA Fundraising News
2016 Awareness Champion Team
Intensifies in Support of ITP Warriors
By Nancy Potthast
ITP Warrior, John Camera (center,
holding medal) and his Student Council
celebrate Sport Purple Day.
The starting line-up of awareness champions continues to grow as they inspire,
share and make the world #ITPAWARE! Do you or someone you know run, cycle or
participate in community fitness events? There’s still time to join our team of
Awareness Champions! Contact PDSA’s Programs and Events Manager, Jody Shy at
[email protected] for more information and to set up your personal fundraising page
and get started today!
EVENT
ORGANIZER(S)
LOCATION
AMOUNT
Key West Half Marathon
Cheri Zimmerman
Key West, FL
$4,080.19
Maple City Savings Bank
Raffle prizes and literature at Sport
Purple Day organized by ITP Warrior
John Camera and his Student Council.
The group raised over $270 for
PDSA programs and research.
“A tribute to all patients with
platelet disorders — our office
collected ‘Pennies for Platelets’
during September ITP Awareness
Month — we collected $250.00
for ITP awareness!!”
– Bleeding and Clotting Disorders
Institute, Peoria, IL
Hornell, NY
$232.00
#FASTRUBY
The Tillinghast Family
Lincoln, NE
$100.00 to-date
Lagree Fundraisers
Paula Lagree
McCormick, SC
$100.00 to-date
Cleveland Half Marathon
Alex Kruse
Cleveland, OH
$3,165.50
Tri-Beta Bio Honor Society
Jennifer Roberts, PhD
Romeoville, IL
$825.00
Linda’s Angels
Nicole Novello
Bellmore, NY
$245.28 to-date
Lugo Fundraiser
John Camera
Brooklyn, NY
$722.00
August Challenge
Tammy Fassett
Bothell, WA
$169.27 to-date
Virtual Walk
Kristie Lyons
Oak Ridge, NJ
$655.00 to-date
Bravelets
Linda Hanson
Decatur, GA
$40.00 to-date
Pennies for Platelets
Cade Kleisner
Neenah, WI
$200.00
Parents Partner for PDSA
Kim Barber
St. Petersburg, FL
$345.00
Clinch’s Hope for a Cure
Yvonne Molino
Longs, SC
$361.88
Pennies for Platelets
BCDI
Peoria, IL
$250.00
For a complete list of fundraising events, visit:
http://pdsa.org/contribute/fundraising/fundraising-events.html
PDSA is a 501 (c)3 nonprofit corporation. All contributions are tax deductible to the
extent of the law. PDSA receives NO federal funding.
Poke-R Club Abstract One of the Top Posters at NORD Summit
CONTINUED FROM PAGE 14
Staff at the Bleeding Clotting Disorders
Institute (BCDI), in Peoria, IL decked out in
purple for September ITP Awareness Month
(of which 80% were submitted by
hospitals, prestigious research
institutions, and pharmaceutical
companies). We were asked to provide a
one page-flyer about the Poke-R Club
that was printed in the NORD Summit
program and distributed to over 600
rare disease stakeholders.
You can find both the ITP Poke-R Club
flyer and the poster abstract on the
PDSA Web site here:
http://pdsa.org/resources/researchprogram.html. To enroll your child in
the Poke-R Club contact PDSA Programs
and Events Manager Jody Shy at
1-87-PLATELET or [email protected].
16
The Platelet News
www.pdsa.org
PDSA Welcomes Alexandra Kruse —
Its First Research Coordinator
By Carol Hoxie
PDSA is pleased to welcome Alexandra
Kruse as PDSA’s first Research Coordinator.
She is responsible for overseeing and
executing all research studies affiliated
with PDSA. She designed PDSA’s
research framework as part of an award
from the Patient-Centered Outcomes
Research Institute (PCORI), creating new
grant guidelines for clinical, laboratory,
and observational research. Alexandra
plans to work with patients and clinicians
to pilot patient-centered research
studies conducted by PDSA. She is also
PCOR-science trained as a PCORI
ambassador. Her other main responsibility
is creating and overseeing PDSA’s new
ITP Natural History Study/ Patient
Registry. She will construct a clinical
variables database for ITP patients in
conjunction with the National
Organization of Rare Disorders (NORD)
and the US Food and Drug Administration
(FDA). This new database will help
advance research in ITP and further
understand the individual and
community profiles of ITP patients.
Alexandra runs PDSA’s Instagram account
(follow us at @pdsa_itp!), live tweeted
the conference this past July, and writes
several of the scientifically-based
articles for the quarterly newsletter The
Platelet News and the monthly E-news.
Although originally from Cleveland,
OH she currently lives in Washington,
DC, which she said is truly the
epicenter of healthcare policy and
research in the United States. Because
of her DC location, Alexandra has been
able to attend several meetings and
conferences as a PDSA representative,
such as the Rally for Medical Research
Day on Capitol Hill to advocate for NIH
funding, the National Organization for
Rare Disorders (NORD) Summit, the
Patient-Centered Outcomes Research
Annual Conference, various research
training programs, and several meetings
at the National Institutes of Health (NIH).
She has her B.A. in Modern Languages
and Literatures (Spanish and Arabic)
with a Biology minor and Pre-Medicine
concentration from Kenyon College,
and completed a Post-Baccalaureate
Program at Cleveland State University
in Pre-Medical Studies. Alexandra also
attended a Public Health program with
the School for International Training in
Buenos Aires, Argentina, where she
studied public health policy, urban
epidemiology, and health care equity,
and researched barriers to healthcare for
socioeconomically disadvantaged
pregnant women in Argentina.
Before joining PDSA, Alexandra worked
as a Clinical Research Intern at Weill
Cornell Medical Center with PDSA
Medical Advisor, Dr. James Bussel. She
was responsible for reviewing electronic
medical records, interviewing and
consenting patients, analyzing data,
and providing administrative assistance
to research coordinators. Alexandra has
authored and collaborated on a number
of abstracts through Weill Cornell and
presented research at annual
conferences for the European
Hematology Association (EHA), the
American Society of Hematology (ASH),
and even at PDSA conferences!
When she decided to train for the
Cleveland Rite Aid Half Marathon this
past spring, Alexandra wanted to use
the event to raise awareness for a
disease that has impacted every aspect
of her life. Most people who know her
know that she would never have labeled
herself “a runner.” However, when she
was training Alexandra thought about
all of the people who have touched her
life as a result of her involvement with
PDSA. She knew the run would
Alexandra Kruse, PDSA’s new Research
Coordinator, with Dr. James Bussel at the
2016 ITP Conference in Florida
definitely be worth it. She finished her
half marathon in less than 2 ½ hours (in
rain, hail, and 30 degree temperatures!)
in the top quartile of half-marathon
runners. To keep herself motivated while
she ran she carried a list of ITP warriors
in her pocket! Now she is training to
run half-marathon #2 and to Sport
Purple for Platelets in Washington!
Alexandra described her relationship
to ITP and PDSA this way. She was
only seven years old when her mom,
PDSA’s Executive Director Caroline
Kruse, was diagnosed with Idiopathic
Thrombocytopenic Purpura. She
couldn’t even pronounce those words,
let alone understand them. With her
being the oldest sibling, her mom
insisted Alexandra needed to be able to
explain her mom’s condition in case they
ever got into a car accident or she cut
herself. As a result, Alexandra’s exposure
to medicine began at a young age, often
waiting at the hematologist for the results
of a CBC or sitting with her mom during
lengthy IVIg treatments. Instead of being
fearful, she was empowered to learn as
much as she could about autoimmune
disease and bleeding disorders. Alexandra
volunteered at ITP support group meetings
and several PDSA conferences running
mics during sessions, preparing conference
materials, and helping with the Kids
(CONTINUED ON THE NEXT PAGE)
Platelet Disorder Support Association
In Memory
We received contributions from
July 1, 2016 to September 30, 2016
in memory of:
Talen Chao
Gregory Chin
Barbara Crawford
Pamela Yukihiro
In Honor
We received contributions from July 1,
2016 to September 30, 2016 in honor of:
Jessica Cohen
Guests of Rebecca Antas’ Wedding
Susan Frank
Mark Jacobson
David L. Johnson
Barb Link
Easton Parson
Hayley Shimanek
Cade Slad
If you are considering an honorarium
donation, please contact PDSA, 8751
Brecksville Road, Ste. 150, Cleveland, OH
44141 or [email protected] or call
1-87-platelet (1-877-528-3538) toll free.
In Appreciation
CONTINUED FROM PAGE 14
• Trish and Paul Santaromana • John
Semple, PhD • Kimberly and Ronald
Sewald • Hayley and Nathan Shimanek
• Jody and Jon Shy • Jennifer and Aaron
Slad • Michael Tarantino, MD • The
Tillinghast Family • Kristen and Steve
Tomczak • Kelly Torres • Lois Umhoefer
• Stephanie Zane, Esq. • Cheri and Derek
Zimmerman • Joyce and Dale Zimmerman
If you have any questions about the
CIRCLE OF HOPE, please contact
PDSA at (877) 528-3538 or visit
www.pdsa.org/hope to learn more.
phone 1-87-PLATELET
17
PDSA Develops New Patient-Centered
Research Guidelines for ITP Research
Proposals
By Alexandra Kruse
Part of PDSA’s mission statement is
“enhancing the lives of people with
immune thrombocytopenia (ITP) and
other platelet disorders through research.”
When we received an opportunity to
update our research protocol with the
help of an award from the PatientCentered Outcomes Research Institute
(PCORI), we knew our goal of working to
engage the needs of ITP patients would
remain our first priority when developing
new investigative guidelines.
At the 2016 ITP conference, the newlyestablished “PDSA Patient Research
Panel” comprised of PDSA medical
advisors Dr. James Bussel and Dr.
Michael Tarantino, and five ITP patients
and caregivers convened to discuss how
research supported by PDSA should be
conducted with the patient voice
specifically in mind. The panel decided
that research must be focused on
clinically-oriented, observational, and
laboratory research proposals related to
primary ITP. Research methods must
include evidence of consultation and/or
collaboration with ITP patients.
Funding should be allocated to
hypothesis-driven research projects
showing patient benefit, according to
PCORI. Benefits could include “disease
incidence, prevalence, burden, causes,
prevention and management; gaps in
evidence; potential for new evidence to
improve health, well-being, and quality
of care [such as patient qualitative
studies to clarify symptoms or other
quality of life issues of living with ITP];
patient needs, outcomes, and
preferences; [and] relevance to patients
and clinicians in making informed
health decisions [about treating ITP].”
Applications from investigators will be
evaluated by PDSA’s Patient Research
Panel. The PDSA Research Coordinator
will screen proposals for proper
adherence to our newly updated
guidelines, and PDSA Medical Advisors
will review for feasibility and relevance to
ITP. After receiving updated proposals,
grants will be awarded based on
selection by the patient panel. Proposals
will be evaluated based on importance,
relevance, investigation plan, and
interest to the ITP patient community.
PDSA is especially grateful to our
Patient Research Panel for their opinions
in the creation of this new program.
With their cooperation, we will be
accepting grant applications starting
this month to fund new and exciting
ITP research, selected with the ITP
community’s needs in mind. You can
find the full text of PDSA’s Research
Guidelines and donate to the research
fund on PDSA Web site’s Research
Program page https://www.pdsa.org/
resources/research-program.html
PDSA Welcomes Alexandra Kruse — Its First Research Coordinator
CONTINUED FROM PAGE 16
Camp. She gained insight into the lives
of patients with rare and chronic diseases.
From listening to heart-wrenching ITP
patient stories, she learned the
importance of patient advocacy, a correct
diagnosis, the opportunity to see an
expert in the field, and ensuring proper
treatment. Although Alexandra loves
research, she is drawn to the personal
connection that comes with listening to
the patient story, and she plans to
apply to medical school this spring.
18
The Platelet News
www.pdsa.org
PDSA Local Support Groups
PDSA has support groups across the United States and Canada and new groups continue to form. Listed below are existing groups
with meeting dates. No group listed in your area? Consider forming a group and becoming a facilitator. We’ll provide you with a
New Facilitator Kit to help you get a group started. Just contact Jody Shy at [email protected] or call toll free at 1-877-528-3538.
For information on meeting locations, dates, and times please contact the group facilitator.
ITP PARENTS TELECONFERENCE GROUP
ATLANTA, GEORGIA
RALEIGH, NORTH CAROLINA
Facilitator: Jay Charness, [email protected] or (303) 731-7731
2016 Meetings: December 7
2017 Meetings: February 1, April 4, June 7,
August 1, October 4, December 12
Facilitators: Joan Coppolino,
[email protected] or (770) 924-2258
Facilitator: Amy Prokopowicz,
[email protected] or (919) 280-0872
SOUTHEAST, GEORGIA
CINCINNATI, OHIO
Facilitator: Patricia Johnson,
[email protected] or (229) 247-0699
Facilitators: Mary and Mark Griffith,
[email protected] or (513) 922-4255
Currently looking for a Facilitator
CHICAGO, ILLINOIS
CLEVELAND, OHIO
OTTAWA, ONTARIO - CANADA
Facilitator: Trish Santaromana,
[email protected] or (630) 292-1485,
http://chicagoitpsupport.org
Facilitators: Barbara Hise, fourstrings@
ameritech.net or (216) 310-3926 and Caroline
Kruse, [email protected] or (440) 526-0629
PEORIA, ILLINOIS
DAYTON, OHIO
CANADIAN TELECONFERENCE GROUP
Facilitator: Livia Chyurlia and Vanessa Clermont,
[email protected] or (613) 612-9689
2017 Meetings: January 17, March 21
WATERLOO-WELLINGTON, ONTARIO CANADA
Facilitator: Dale Paynter,
[email protected] or (519) 651-2352
PHOENIX, ARIZONA
Facilitator: Bunnie Stevenson,
[email protected] or (480) 451-7661
ROGERS, ARKANSAS
Facilitator: James Martin,
[email protected] or (479) 270-7760
LOS ANGELES (SIMI VALLEY), CALIFORNIA
Facilitators: Nina and Stephanie Schussman,
[email protected] or (818) 370-8877
ORANGE COUNTY, CALIFORNIA
Facilitators: Melissa Hilsabeck &
Leilani de Castro, [email protected]
or (714) 598-7102
PATTERSON, CALIFORNIA
Facilitator: Carla Nelson,
[email protected] or (209) 892-8813
SACRAMENTO, CALIFORNIA
Facilitators: Dawn and John Phillips,
[email protected] or (916) 607-1699
2016 Meetings: December 29
2017 Meetings: February 18
SAN DIEGO, CALIFORNIA
Facilitator: Shanna Beard, [email protected] or
(390) 692-5337
Facilitator: Tina Garland,
[email protected] or (937) 643-1705
BOSTON, MASSACHUSETTS
POWHATAN POINT, OHIO –
SOUTHEASTERN OH/WV
Facilitator: Michael Westfort, michael@platelet
health.org or (508) 366-1073 or (508) 930-0309
DETROIT, MICHIGAN
Facilitators: Sharon Cisco and Linda Galka,
[email protected] or (586) 783-8014
ST. PAUL, MINNESOTA
Facilitator: Joe Winter,
[email protected] or (651) 792-5277
OMAHA, NEBRASKA
Facilitator: Heidi Green, [email protected]
or (402) 498-3826
CENTRAL/NORTH NEW JERSEY
Facilitator: Linda McGuirl, [email protected]
or (908) 764-1819
NORTH NEW JERSEY
Facilitators: Dianne Danielle, [email protected]
or (201) 265-8875 and Lanie Gastman,
[email protected] or (201) 592-1181
Facilitator: Camie Sims,
[email protected] or (304) 816-2608
POCONOS, PENNSYLVANIA
Facilitator: John Catalano,
[email protected] or (917) 892-4264
RHODE ISLAND
Facilitators: Lisa & Steve Sack,
[email protected] or (401) 884-5711
CHATTANOOGA, TENNESSEE
Facilitator: Sharon Putnam,
[email protected] or (423) 991-6450
NASHVILLE, TENNESSEE
Facilitator: Charity Hasty Backs,
[email protected] or (615) 473-3372
AMARILLO, TEXAS (PANHANDLE)
Facilitator: Robin Abshire, [email protected]
or (337) 296-7052
DALLAS FORT WORTH, TEXAS
SOUTH NEW JERSEY
Facilitator: Michael Vitale, [email protected]
CAPITAL DISTRICT (ALBANY AREA),
NEW YORK
Facilitators: Linda and Kris Dorasami,
[email protected] or Linda (817) 7272351 or Kris (817) 727-2361 and Marsha
Inman (817) 249-5391, [email protected]
Facilitator: Susan Pounder, [email protected]
or (858) 217-6587
Currently looking for a new facilitator
SALT LAKE CITY, UTAH
LONG ISLAND, NEW YORK
WASHINGTON, D.C.
Facilitator: Claudia Montuori,
[email protected] or (718) 725-2645
Facilitator: Cory Bushman,
[email protected] or (801) 404-4580
Facilitator: Cindy Ayliff, [email protected] or
(703) 849-0054
TRIANGLE, NORTH CAROLINA
Facilitator: Barbara Pruitt,
[email protected]; (305) 409-6887
Facilitators: Donna Goldstein, ds.goldstein@
mindspring.com and Emily Goldstein,
[email protected] or (919) 942-4082
NORTH FLORIDA
CHARLOTTE, NORTH CAROLINA
Facilitator: Irene Xynides-Rincon, xynidesi@
bellsouth.net; (904)710-9055 or (904) 823-9055
Facilitator: Carol Mullis, carolhmullis@
windstream.net or (704) 843-2734
MIAMI, FLORIDA
SEATTLE, WASHINGTON
Facilitators: Tammy Fassett,
[email protected] or (206) 465-3451 and
Taylor White, [email protected] or
(253) 250-5873
MILWAUKEE, WISCONSIN
Facilitators: Kim Everett, [email protected]
or (920) 755-8430 and Trudy Sensat,
[email protected] or (414) 202-6291
Platelet Disorder Support Association
phone 1-87-PLATELET
19
News from Our Support Groups
Our support groups are the grassroots that help us BUILD STRENGTH THROUGH SUPPORT and ADVANCE ADVOCACY
THROUGH EDUCATION. When you share your personal stories with others, you give hope to those who may have felt all
alone in dealing with their ITP. We hope you will find comfort in knowing that whether you are part of a meeting to listen,
talk or both that you are not alone. We encourage you to make your medical providers, family and friends aware of our
numerous resources that help make the World of ITP a more manageable place to be. It’s this strong network that will continue
to create awareness, educate, and advocate. Here are some inspirational stories and news we’ve received from our support
groups recently. To share your group’s news, please contact Nancy Potthast at [email protected]
Atlanta, GA Support Group
Meeting
Facilitator: Joan Coppolino
[email protected] or 707-924-2258
The group warmly welcomed a new
attendee to their August 27th meeting
and discussed updates from ITP
Conference 2016 in Orlando, FL.
Central/North NJ Support Group
Meeting
Facilitator: Linda McGuirl,
[email protected] or 908-764-1819
The group welcomed four new members
to their September 20th meeting with
13 in attendance. The engaged group
reviewed ITP Conference 2016.
Attendees shared their experiences with
ITP, discussed the need to be one’s own
advocate, and reviewed personal
experiences with treatments (side
effects, successes and failures). They
celebrated what lifts their spirits —
meditation, energy, healing, daily
exercise, good nutrition — and agreed
to reflect on five things to be grateful
for upon awakening each day.
welcomed 15 attendees (familiar and
new) who shared their journeys in
living life with ITP! It was a wonderful
afternoon with the first PDSA ITP
Network Meeting at the historic
Runnymede Library in Bloor West
Village. The meeting offered great
discussions about treatments and what
is working, not working and wishing
their doctors had the resources they
have through groups like PDSA. The
group also wants all its American friends
to come up and see them sometime!
Sacramento, CA Support Group
Meeting
Facilitators: Dawn and John Phillips
[email protected] or 916-607-1699
Facilitators Dawn and John welcomed
two new families to the group at the
October 18th meeting in Sacramento.
The newcomers (one newly diagnosed,
Waterloo-Wellington/Greater
Toronto Area, Ontario – Canada
Support Group Meeting
Facilitator: Dale Paynter
[email protected] or 519-651-2352
The Waterloo-Wellington group
expanded their area, held their October
16th meeting in Toronto, and
Members of the Sacramento, CA Support
Group sport purple at a game with ITP
Warrior Josh Phegley (catcher for the Oakland
A’s) in July. ITP Warrior and Sacramento group
co-facilitator John Phillips is pictured (center,
left) with Josh Phegley (center, right).
the other an ITP veteran) found
comfort in hearing the journeys of
others, were relieved to uncover
resolution to unanswered questions,
and are grateful to have new sources of
support through the group and PDSA.
Chicago, IL Support Group
Facilitator: Trish Santaromana
[email protected] or
630-292-1485
Happy Anniversary to all who have
shared their ride on the ITP Rollercoaster
in Chicagoland for the past nine years!
Parents ITP Teleconference
Support Group
Facilitator: Jay Charness
[email protected] or
303-731-7731
The ITP parents had a great
teleconference call on Aug. 3rd with
several families joining in on the call.
The first half hour was dedicated to the
kids giving them time to share their
ITP and life experiences. Parents on the
call had an outstanding question and
answer session with ICON Pediatric
Hematologist Robert Klaassen, MD.
Questions and answers from the call
are printed in this issue of The Platelet
News. Following the Q&A session,
parents discussed many other topics of
concern for their children with ITP.
The next teleconference call is
scheduled for Dec. 7th, 7 pm EST.
20
The Platelet News
www.pdsa.org
Parents Get Answers During Q&A with ICON
Pediatrician Dr. Robert Klaassen
By Carol Hoxie
During PDSA’s Aug. 3rd ITP Parents’ Teleconference Support Group call parents asked their questions of
ICON pediatric hematologist, Dr. Robert Klaassen. He is Professor of Pediatrics in the Division of Pediatric
Hematology/ Oncology at the University of Ottawa in Ontario, Canada. Dr. Klaassen has been on staff at
the Children’s Hospital of Eastern Ontario (CHEO) since 1998. His current area of research focuses on
quality of life of patients with hematologic disorders. His clinical expertise is in benign hematology
including bleeding disorders.
Dr. Klaassen is a Site Investigator for ICON (ITP Consortium of North America), a collaborative research
effort dedicated to improving the understanding, treatment, and quality of life of pediatric ITP patients.
ICON is pleased to partner with and work in collaboration with PDSA.
Dr. Robert Klaassen,
Pediatric
Hematologist from
the University of
Ottawa in Ontario,
Canada answered
questions during the
August 3rd Parents’
Teleconference Call
Here are the parents’ questions and Dr. Klaassen’s answers.
Q: Our child’s migraines are triggered
Q: Are there long-term effects of the
when her platelets are low. We have a
30 year-old house and recently got an
ionizer that kills mold. Is mold
something that triggers ITP?
A: Infection is a trigger for ITP. It’s
important to do anything you can to
avoid infection, as it will drop platelet
counts and might exacerbate ITP.
platelet growth drugs?
A: These treatments have been used for
ITP about 7-8 years in addition to the
years of clinical trials. Romiplostim
(Nplate®) is injected, eltrombopag
(Promacta®) is a pill. These work equally
well and seem quite safe. We are getting
more data as time goes on. Promacta
was approved for use in children in 2015.
Q: Is there a chance for remission from
ITP after being on Nplate?
A: About 5 – 10% of patients on Nplate
or Promacta have gone into remission
for a while after treatment was ended.
The question is: Was it the drug that led
to the remission or was it from the
natural history of the disease? In
children a majority of ITP patients go
into remission.
Q: What other ITP treatments are you
and ICON working on?
Q: If one of these two drugs doesn’t
Q: On IVIg our child’s platelet counts
A: The ICON study has 26 centers.
work well or has bad side effects, is it
worth switching to the other one?
A: Just because someone gets side
effects with Promacta doesn’t mean
they will have the same effect with
Nplate. It’s worth a try.
were up and down and then was treated
with Dapsone. First, the count was
48,000, then fell to 30,000 and recently
to 20,000. Our hematologist is thinking
of trying Promacta.
A: Dapsone works in some ITP patients.
Some patients respond, some don’t.
Again, was it the Dapsone doing
something, or was it the natural history
of the child’s ITP?
As to treatments, Rituxan® (the most
common one used) is similar to IVIg
but is directed at one type of cells in
the immune system (B cells that make
antibodies). Rituxan works in about 2/3
of patients with initial response. About
1/3 get a longer response time. As to
side effects we see some infusion
reactions, blood pressure changes, and
hives. The downside is higher risk of
infection after treatment. Risk is lower
if we give Rituxan and no other drugs
at the same time.
There is also research on drugs that
stimulate platelet-making cells
(megakaryocytes), whereby pieces bud
off to make platelets. These growth
drugs are very effective, working in
about 80% who get them. However,
they only work as long as you take
them and are very expensive.
Q: Our child bleeds at lot at an 8,000
count. We try to treat when the count
is down to 10,000.
A: I have some patients who at 5,000
platelet counts don’t bleed. However, if
a child bleeds at a particular level, they
should be treated.
Q: What about possible head injuries at
low counts?
A: Studies have shown about a 1-2% rate
of bleeds in the head. There’s about a 99%
chance of not having a head bleed. In a
study in Switzerland with 3,000 kids,
they only had three bleeds (a toddler, an
elementary school age child, and a teen).
Q: What is your general consultation
about ITP children playing standard
school sports and what is a safe
platelet level?
A: We did a survey of US doctors
published in Pediatric Blood and Cancer.
We got lots of different responses. On
any given day you can’t know exactly
what your child’s count is. It could be
or down. Try to get a picture of what
your child’s trend is. Do avoid contact
(CONTINUED ON THE NEXT PAGE)
Platelet Disorder Support Association
What Our Friends Are Saying
on Facebook
PDSA has a Facebook page that provides
a direct online forum for comments and
sharing news and information about ITP,
treatments, and the ups and downs of
having this platelet disorder.
If you LOVE PDSA, then be sure to:
www.facebook.com/plateletdisorder
Note: The views expressed are personal
experiences and are not endorsed by PDSA.
Here are some recent comments we’ve
received on our Facebook page:
Comments on fatigue:
“The fatigue is like nothing I’ve ever
experienced in my life. I have had weeks
where I could only muster the strength to
roll over in bed. This disease is a dirty
rotten scoundrel! It’s 100% a symptom.
All autoimmune diseases have fatigue as a
symptom; your body is hell-bent on
destroying itself. It is exhausting!” – Tara C.
“I was diagnosed 15 years ago…fatigue is
real. Sadly, for years I was told it was all in
my head.” – Shannon K.
“Fatigue is really horrible because my brain
is telling me to get up and go but my body
just won’t oblige. I was told by one of my
consultants that ITP doesn’t cause fatigue.
What does he know — obviously not a lot
— he should try having it!” – Stephanie S.
“Every single time I tell the nurse that my
husband is tired/exhausted she’ll say, ‘His
hemoglobin is fine.’ Ugh!” – Jill F.
phone 1-87-PLATELET
21
Parents Get Answers During Q&A with ICON Pediatrician
CONTINUED FROM PAGE 20
sports, so no rugby, football or ice
hockey. These all have head injury risk.
Stay away from sports that involve
slamming into others as part of the
game. With basketball there is some
banging into others and bruising.
Soccer could be okay as long as there’s
no ‘heading’ and leg injuries are not
usually life-threatening. In general, let
your child have as normal a life as
possible. Don’t put them in a bubble.
Have them wear helmets for sports and
use common sense.
Q: What about fatigue in kids with ITP?
I notice a difference in my daughter.
She’s more active at 70,000 but her
endurance is off at 20,000. It’s like she
hits a wall.
A: In our study, we looked at fatigue.
We found that the group got fatigued
when counts were low. It might seem
counterintuitive but the best antidote
for fatigue is some activity — keep
moving. Don’t just let the kids sit on
the couch and watch TV or play video
games. It’s important that kids get
outside and get some exercise.
Q: In terms of splenectomy, what are
your personal parameters for your
patients?
A: For ITP patients, the younger you are
the greater the risk as removing the
spleen increases risk for infections. If
kids are over age 5, the risk is lower. As
kids get older they have fewer fevers
and infection concerns. We wait until
kids have had ITP at least a year, as
many kids go into remission by then.
For kids who have Evans syndrome (red
cell breakdown) and ITP, we avoid
splenectomy. For kids who are age 6 or
older and have ITP more than a year, it
is a possible solution. We found from a
quality of life (QOL) viewpoint, it can
be a dramatic change for the ITP child
and their family. If splenectomy works
and you see them much later, life is
much better for them.
22
The Platelet News
www.pdsa.org
Advocacy Alert
NEWS OF PDSA ADVOCACY ON BEHALF OF PATIENTS WITH ITP AND OTHER PLATELET DISORDERS
NORD 2016 Rare Diseases & Orphan
Products Breakthrough Summit
By Caroline Kruse
PDSA Executive Director Caroline Kruse,
Marketing Director Nancy Potthast, and
Research Coordinator Alexandra Kruse
were three of more than 600 members of
the rare disease community who came
together for the National Organization
of Rare Diseases (NORD) annual
conference, October 17 and 18 in
Arlington, Virginia. The “Rare Diseases &
Orphan Products Breakthrough Summit”
was a two-day event focused on fueling
collaboration to advance progress for
patients. It featured expert speakers from
multiple stakeholder groups, including
patients and patient groups, FDA, NIH,
industry, researchers, academia, and
venture capitalists, among others.
This year, two themes emerged as
necessary for continued progress:
collaboration and involving the patient
in every step along the way. Speakers
discussed national election implications,
federal and state policy issues,
collaboration nationally and
internationally, barriers to access, and
new drug development. There were also
networking opportunities on how to
drive progress for people with rare disease.
Keynote speakers included:
FDA Commissioner Dr. Robert Califf
spoke of the obligation of public health
agencies to foster collaboration with
patient advocates. Calling this an era of
remarkable progress, he announced more
than 20 new grants, totaling $23 million,
to fund rare disease research in the coming
years. Dr. Califf praised NORD and its
Summit for marshaling the strengths of
the rare disease community, saying,
“This organization brings together so
many different experts across the
different spectrums and most importantly
the patients and caregivers who provide
the best resources in providing expertise.”
Dr. Janet Woodcock, Director, Center
for Drug Evaluation and Research
(CDER), FDA, stated there is no better
time for patient involvement in the rare
disease drug development process. She
discussed the importance of natural
history studies and called for greater
collaboration between researchers
through consortia.
Dr. Peter Marks, Director, Center for
Biologics Evaluation and Research
(CBER), FDA, said we are on the threshold
of the first gene therapy approval. He
discussed CBER’s goal to accelerate
progress and get important therapies for
rare and orphan diseases to patients. His
reviewers get tremendous satisfaction
and help by meeting with patients and
advocacy groups to understand what
they face with their diseases.
Here are highlights from the NORD
Breakthrough Summit:
Focus on Pediatric Diseases – Noting
that the process of achieving pediatric
labeling needs to move more quickly,
Lynne Yao, MD, FDA, said, “It takes a
long time, currently 9 to 10 years on
average, to get from adult approval to
pediatric labeling,” adding there are
now 637 products labeled with
pediatric-specific information.
Potential Advances through Genetic
Innovation – There is an explosion of
gene studies with approximately 530 new
Investigational New Drug applications.
Collaborations Across Borders – The
panel endorsed patient-driven registries
to collect natural history data and
NORD President and CEO Peter L. Saltonstall
with PDSA Executive Director Caroline Kruse
at the NORD Members Membership Meeting
suggested a global depository system,
similar to those used for HIV and
vaccine treatments, to expand access
and care across the globe.
The Crucial Role of Data – All members
of the panel, representing medical
professionals, academics, drug developers
and FDA, agreed this is an exciting time
in drug development. “We are sort of
entering the era of patient-empowered
medicine,” said Matt Might.
Driving Progress through Policy –
Paul Melmeyer and Tim Boyd of
NORD’s Policy team said upcoming
legislation will affect the rare disease
community, including the important
vote for 21st Century Cures during the
3-week lame duck session, and looked
ahead to 2017 issues.
Strategies to Address Patient
Challenges – Before a packed room,
speakers tackled a series of practical issues
that patients face. During an emotional
session, undiagnosed individuals spoke of
the stress and pain of not getting answers.
(CONTINUED ON THE NEXT PAGE)
Platelet Disorder Support Association
phone 1-87-PLATELET
23
ITP in Kids: Q&A
ITP CONFERENCE 2016 • DR. MICHELE LAMBERT AND DR. MICHAEL TARANTINO
By Alexandra Kruse
Q: My son has tried every treatment,
and is now keeping his platelet count
up on Promacta® and dapsone. Can he
expect remission?
A: Dr. Tarantino: It’s very common to
see a real remission with both TPO agents
(Nplate® and Promacta®). The Bleeding
and Clotting Disorders Institute was part
of the original Promacta trial (REPEAT),
and there was at least one patient who
had a sustained elevation in their platelet
count without Promacta after the study
ended. At the time we thought it was a
fluke, but every year we hear about more
cases of patients whose counts remain
elevated. Both Amgen and Novartis,
the pharmaceutical companies who
make the TPO agents, are interested in
the mechanism of why TPO induces
remission and identifying those patients
who are likely to achieve remission.
Q: Our daughter’s platelet count has
been at 100,000 for a year on Promacta.
Last week her count was 25,000. Have
you ever seen that after a year on a drug
the patient’s platelet count plummets?
A: Dr. Lambert: Yes, we would first look
to determine whether there is something
else occurring immunologically based on
your CBC, or whether there isn’t something
other than the ITP causing issues, but
sometimes there is just a loose response.
Dr. Tarantino: Because medications are
biomolecules, there is a small chance
that they will be recognized by the
immune system after time and the
patient will develop antigens against
the medicines. However, a study done
in Europe found that switching a
patient from Promacta to Nplate has an
80% success rate in re-elevating platelet
count, and switching from Nplate to
Promacta has a 50% success rate.
Q: Can you explain dapsone?
A: Dr. Tarantino: Dapsone is a less
frequently used immunomodulating
drug that historically was used as a thirdline agent. The success rate in children
with chronic ITP is mostly anecdotal
with a 50% response rate, and usually
takes a while from beginning of
treatment to platelet elevation.
Q: For those who have a good response
to TPO, how high do you let their
platelet counts go and where do you
start to get concerned? Where do you
get nervous and where do you draw the
line for levels that are too high and that
it could cause a clot?
A: Dr. Tarantino: Initial assessment has
shown that there isn’t a correlation
between platelet count and an event of
thrombosis in Promacta. In those who
experienced clotting in an adult clinical
trial, 80% had another prior risk factor
for clotting that was unrelated, such as a
fractured ankle and the use of estrogenbased oral contraceptives. Thrombotic
events did not occur in the children’s
Promacta trial. The goal of TPO agents
is to achieve a hemostatic platelet
count, above 50,000. Your hematologist
should wean the dose after a platelet
count of 200,000 and stop
administering treatment if the platelet
count is consistently above 400,000.
NORD 2016 Rare Diseases & Orphan Products Breakthrough Summit
Predicting the Pipeline in 2017 – NIH,
FDA and industry representatives spoke
about rising trends in orphan drug
designations and the positive outlook
for continued high activity. Orphan
drugs tend to be innovative (based on
data showing what percentage are first-
Q: We have read some reports on
low-carb diets leading to remission.
What’s your opinion?
A: Dr. Lambert: It’s important to
maintain a healthy and balanced diet as
well as consistently exercising. Many
ITP patients have fatigue and are prone
to depression, so it is important to
maintain as much of a healthy lifestyle
as possible in order to be the healthiest
person they can be. Diet and exercise
definitely play into ITP as well as stress,
which can affect your platelet counts.
Q: How often do you recommend
treatment with IVIg and how often
should we take our son in for a CBC
when he is playing a sport?
A: Dr. Tarantino: Use altered doses of
IVIg at altered frequencies to keep the
platelet count above requisite level so that
your son can participate in his activity.
IVIg treatment is done in a secondary
mode, during the sport season, but then
we will wean them off of the medication
once the season is over as long as their
counts are in a safe range. You should
monitor counts fairly frequently, every
other week, but this is dependent on
bleeding level, activity and severity.
Above 100,000 is the threshold for
unrestricted physical activity.
Dr. Lambert: However, we do
sympathize in telling a patient that they
are not allowed to participate in an activity
that they love and is a huge part of
their life so we as pediatricians integrate
that into our discussion as well.
CONTINUED FROM PAGE 22
in-class) and there seems to be growing
willingness among developers to
expand into new conditions.
24
The Platelet News
www.pdsa.org
ITP in Adults: Q&A
CONTINUED FROM PAGE 11
effects of getting rituximab?
A: Dr. Kessler: We know from lymphoma
and lymphocytic leukemia patients using
rituximab that knocking out B cells can
downregulate (decreases the immune
response of) certain T cells. In some
patients we do see where long-term
rituximab can lead to immune deficiency
syndrome in adults as well as children.
Q: If you use rituximab once and it
doesn’t work, is it something you’d
recommend trying again a second time?
A: Dr. Kessler: All of us, I think, have
different philosophies. I think it’s
important to know what doses of
rituximab are given. I’ve seen a series of
patients that had received rituximab at
100 mg/m², which had been popularized
by an article in the UK. They had
indicated that that dose was adequate
for the treatment of ITP. Most of us on
this panel don’t think that is an
adequate dosing regimen for ITP. Most
of us use the traditional dose that is
recommended for lymphoproliferative
malignancies. As far as failure on one
series and redosing, my own theory is I
would go on to something else. But I
don’t think there’s anything wrong
with redosing but it has to be justified.
Dr. Kuter: Who do you declare a
‘rituximab failure’? One of the problems
is the typical oncologist will treat the
ITP patient with three doses at the
typical oncology dose level and if it
hasn’t worked yet, declare it a failure.
As Dr. Kessler has shown usually you
see the true immune response and the
rise in platelet counts after weeks three
and four through maybe 10. I would
probably make sure a whole course (4
weeks) is given and wait maybe a
month or two before deciding if it
hasn’t worked. Then if it is a failure, I
would probably not go back and use it
again. The doctors on the panel
discussed dosages of rituximab they give
to treat ITP. Drs. Kuter, Gernsheimer, and
Liebman all give 375 mg/m². Dr.
Liebman combines that dose with
dexamethasone. Dr. Kessler has seen
when some patients who also had
another autoimmune disorder (such as
lupus) were given the higher lupus dose
(400 mg/m²) they responded well.
Q: Treatments like rituximab or
splenectomy depend on reducing
antiplatelet antibodies. Has anyone
noticed if they are less effective in ITP
patients who are antibody negative?
A: Dr. Arnold: A number of years ago it
was thought antiplatelet antibodies
could be a biomarker for ITP and then
that turned out to be a not-so-great test.
There were some refinements to the test
but it still hasn’t turned out to be a
great biomarker. In small studies we had
of patients who had gotten rituximab
we looked at platelet antibodies before
and after their treatments. Only about
40% of patients had a detectable
antibody by very specific, sensitive
techniques. When they got rituximab,
their antibody levels dropped more
than the placebo group but levels didn’t
respond with how well they would do.
Rituximab can reduce antibody levels.
However, reduction in antibody levels
did not seem to correlate with
improvement in outcomes.
Dr. Semple: The data Dr. Arnold
mentioned is from mouse studies. With
mice you can make ITP with one set of
antibodies or another and can see the
difference in those antibodies. Most ITP
patients have multiple types of
antibodies. You’re not going to be able
to get patients with just one type of
antibody. We know that certain
antiplatelet antibodies are good at
getting platelets destroyed and others
are good at shutting them off
(inhibiting platelet function). So which
is worse? Decreasing the count or
causing patients to have more bleeds?
Q: What is the long-term outlook for
ITP patients? Is remission likely?
A: Dr. Arnold: There’s an interesting
concept about treating ITP of ‘hit hard,
hit early’ to bring patients to remission.
That’s worth studying some more. The
argument can be made the other way —
ITP antibodies can accumulate over time
but can also disappear over time. It doesn’t
mean the ITP is getting worse or better,
it’s just evolving. And some patients
just get better on their own, so there’s a
burnout rate of the disease as well.
Dr. Bussel: I agree it kind of depends on
the patient and we don’t always know
which patient is which. Studies have
shown treating with rituximab very
early or with high dose dexamethasone
are curative. Our own studies showed if
you treat within a year you can cure
some people with ITP. I think maybe
certain treatments look worse over time
because some patients get better over
time and are no longer being treated.
Dr. Kuter: A doctor in Europe who
followed 187 ITP patients over five
years found 82 percent of the patients
who didn’t have splenectomy had a
remission and came off therapy after
five years. Her findings show that ITP is
a highly variable disease.
Dr. Bussel: Right, though I wouldn’t
want the audience to think that 82% of
adults are likely to get better in five
years based on the best of our
experience here in the U.S.
Dr. Kuter: On the contrary, I would like
the audience to hear that. It gives them
optimism that there’s a light at the end
of the tunnel.
Q: What about switching between the
two TPO agents?
A: Dr. Kuter: We looked at patients on
romiplostim and eltrombopag, not doing
well on their respective treatment. When
they were switched to the other agent,
83% of switched patients responded
well. Switching is worth a try.
26
The Platelet News
www.pdsa.org
Living with ITP –
Patients Share
Their Stories
ITP CONFERENCE 2016
PANEL: DALE PAYNTER, KATIE MELOY,
YVONNE MOLINO, AND KEITH ORI
By Carol Hoxie
Each ITP conference features a “Patient
Stories” session, where several members
share their ITP journey with the audience.
Hearing their fellow ITP patients’
experiences helps audience members
with ITP realize they are not alone. They
learn which treatments worked, which
didn’t, and coping strategies. This year’s
international panel included speakers
from the US, Canada, and the UK. First
was Katie Meloy, a 21-year-old from
York, UK, diagnosed with ITP in January
2015. After diagnosis she felt her hopes
and dreams were being taken away. Life
would never be normal. With counts as
low as 2,000 and as high as 160,000,
she experienced the “emotional roller
coaster” that is ITP. When put on
steroids Katie suffered sleep deprivation,
weight gain, and hallucinations. She
found Sandi Zancha on PDSA’s
Discussion Board and the PDSA Web
site, which made her feel less alone.
IVIg caused her serious side effects.
After treatment with Rituxan® she is in
remission, with stable 200.000+ counts.
Katie said, “I have ITP — ITP does not
have me.” Contacting people across the
world through social media she put
together an ITP awareness video that
can be viewed on YouTube (search for
‘ITP awareness video’). Katie said,
“Without ITP I definitely wouldn’t be
the person I am today.”
Dale Paynter, from Toronto, Ontario,
Canada has ITP since he was diagnosed
in 2010 after a company physical found
an 80,000 platelet count. When it fell to
30,000 he was sent to a hematologist.
Bone marrow testing showed healthy
platelet production. He tried prednisone
This year’s engaging ITP patient panel included: (L to R) Keith Ori, Yvonne Molino, Katie Meloy,
and Dale Paynter, moderated by PDSA Executive Director Caroline Kruse (C)
but it didn’t help. In 2011 Dale was
planning a golf trip to South Carolina;
his hematologist recommended IVIg
treatment. It raised his count to 350,000.
He’s been on IVIg treatments twice per
month ever since. After several years Dale
was tired of constant IVIg treatments so
during fall 2014 he had a splenectomy.
Unfortunately it didn’t work. He tried a
six-month fostamatinib clinical trial but
that didn’t raise his counts. He is back
to twice-monthly IVIg.
Dale’s PDSA connection began when he
attended the 2011 ITP conference in
Boston, MA. He has attended every
conference since. He runs the WaterlooWellington, Ontario support group.
Three years ago he joined PDSA’s Board
of Directors. ITP has changed his life.
He is more careful around home and he
no longer plays ice hockey as he did for
many years. Dale considers himself one
of the lucky ones, with few petechiae
and little bruising. He said, “You don’t
have to let ITP take over your life.”
Yvonne Molino, of Longs, SC is the
parent of a 10-year-old son with ITP and
shared his story. Clinch was diagnosed with
ITP at age 2. He was hospitalized nine days
and received IVIg and WinRho®, but there
was no real change in his platelets. When
they rose from 1,000 to 3,000 Yvonne was
excited until the doctor said, “Well, it’s
kind of like whether you have a thousand
pennies or three thousand pennies, you’re
still kind of broke.” Clinch was covered
in bruises and had bleeding mouth
blisters. His bone marrow test was
normal. They started him on steroids.
Doctors said it was probably ‘acute’ ITP
and the low counts wouldn’t happen
again. Four months later he was in the
hospital again. This routine of platelet
counts bottoming out and getting
treatments lasted until age 5. Between
ages 5-7 Clinch had two hospital visits.
Yvonne thought he was growing out of
it. Around age 7 -8 he was sick again, his
counts ranging from 2,000 to 40,000.
Then Yvonne found PDSA and got
involved. She said, “We realized then
this was probably ‘chronic’ ITP.” We
tried rituximab for Clinch. It lasted seven
months with a normal platelet count.
He is doing well on IVIg, rituximab,
and immune suppressant. Yvonne
shared her insights as an ITP parent:
1) Remember every person with ITP is
different; what works for one may not
work for another.
2) Try to not limit your child’s activities.
Don’t put them in a bubble. ‘No’ all the
time creates negativity and may result
in them being depressed and angry.
3) Don’t ‘compensate’ for your child’s
illness by buying things and spoiling him/
her. Our doctor said it’s not good to spoil
as that child has to face real-life in the
world. Help them be prepared for that.
4) Get involved for ITP and PDSA. We
take part in Sport Purple for Platelets
Day and are doing a fundraiser this year.
(CONTINUED ON THE NEXT PAGE)
Platelet Disorder Support Association
phone 1-87-PLATELET
27
Kids Korner
By Carol Hoxie
Teen’s School Fundraiser Increases ITP
Awareness and Raises $200 for PDSA
Dear PDSA, My name is Cade Kleisner
and I have ITP. I discovered I had ITP
when I started to spot bruises and
petechiae on my chest and face in
January of my 6th grade year. Thanks to
my homeroom and ELA teacher Mrs.
Schlaak, who had a son with ITP and
recognized the signs, I saw my doctor
and got the right diagnosis and help I
needed. It started out rather rough and
after reaching a low 9,000 platelet count,
I had my first treatment with IVIg. It
worked. After that my platelets declined
again and I had to get another treatment.
This process continued for quite a
while. I also switched to prednisone
and dexamethasone treatments.
Currently I’m an 8th grader and attend
Shattuck Middle School in Neenah, WI.
My platelet levels now rest at about
70,000. Thanks to Ben Jones, a close
friend of mine, as well as guidance
counselor, Mrs. Jamie Lasee, we were
able to include the PDSA in the yearly
Shattuck Penny Drive where we donate
to multiple support groups of different
disorders. I also wanted to thank you for
supplying me and my family with tons
of helpful information. It has definitely
made my experience with this blood
disorder a heck of a lot easier.
PDSA Note: Cade’s fundraiser raised ITP
awareness and a $200 donation to PDSA.
Thank you!
ITP Warrior Donates $150 in
Allowance Money to PDSA
Hi, My name is Kai and I am 8 years old.
I have had ITP since I was 2. I have tried
many different treatments, including
Promacta®, WinRho®, Nplate®, Cellcept,
prednisone, and my typical treatment is
Kai, an 8-year-old ITP Warrior saved his
allowance for 18 months and donated $150
to PDSA
IVIg. I have saved my allowance for 18
months, and I have donated $150.00.
Thank you PDSA for all of the hard
work that you have done trying to find
cures for ITP.
PDSA Note: Thank you to Kai for saving
up his allowance to give to PDSA to help
others with ITP!
If you have an interesting story or news
about a child/teen with ITP, please send to
PDSA at [email protected] or mail to:
PDSA, 8751 Brecksville Road, Suite 150,
Cleveland, OH 44141.
Living with ITP - Patients Share Their Stories
CONTINUED FROM PAGE 26
Keith Ori, from Orlando, FL was the last
speaker. He buys and flips houses on a
reality TV show, Zombie House Flipping.
He was fine until 2010 when he
experienced fatigue, brain fog and
collapsed while playing with his kids. It
was one of those “something is really
wrong” moments. At the doctor he
learned he had a 20,000 platelet count.
He received pulse dexamethasone and
had a bone marrow test. His hematologist
suggested splenectomy, telling him 2/3
of ITP patients get better, although 1/3
don’t and get worse. Keith promptly saw
a different hematologist. His count was
around 16,000. His doctor said since Keith
was Rh+ he could get WinRho®. So he
got the infusion. Unfortunately, later at
home, he suffered a rare severe hemolytic
reaction to WinRho®, a reaction that has
caused death in some ITP patients.
On prednisone Keith wasn’t much fun
to be around. He tried IVIg and
Rituxan® in 2011 but it didn’t help. He
got the Indium platelet screening test
done outside the U.S., which showed
his platelets were destroyed mainly in
his spleen. He graphed his platelet
counts. He noted they rose in the
winter and fell in the summer. Keith
has seasonal allergies and said in
Orlando they use air conditioning a lot
in the summer. After adding onto their
house in 2007 they experienced trouble
with the AC system, including
condensation and lots of mold. When
he put a UV light in the unit, it killed
the mold. After that his platelet counts
rose by 2013 to the 100,000 – 150,000
range. Keith asked, “So, after six years
of ITP am I cured?” He encouraged the
audience to put everything into finding
out what might be causing their ITP.
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