Download Learn How to Manage Your PNH

You have done well to manage
your PNH with Soliris®. Living
with a rare disease, like PNH,
can be challenging, but by
sticking with your Soliris therapy,
you are doing a lot to help
yourself. Soliris works to reduce
the signs, symptoms, and health
risks associated with PNH.
This brochure provides helpful
tips for continued, long-term
management of your PNH with
Soliris therapy.
You have done well to manage
your PNH with Soliris®. Living
with a rare disease, like PNH,
can be challenging, but by
sticking with your Soliris therapy,
you are doing a lot to help
yourself. Soliris works to reduce
the signs, symptoms, and health
risks associated with PNH.
This brochure provides helpful
tips for continued, long-term
management of your PNH with
Soliris therapy.
PNH
Refresh your memory about PNH
Symptoms you can see or feel if your PNH is left untreated
Fatigue:
Our commitment
•
•
Alexion’s objective is that
every patient with PNH who
can benefit from Soliris®
will have access to Soliris.
•
•
•
Tiredness
Difficulty performing
daily activities
Trouble concentrating
Dizziness
Weakness
Other signs and symptoms:
•
•
•
•
•
Dark-colored urine
Shortness of breath
Difficulty swallowing
Yellowing of the skin
and/or eyes
Erectile dysfunction (ED)
PNH is just
like an iceberg — what you can’t see
or feel can hurt
you the most.
Pain:
•
•
•
• PNH is a lifelong disease. Once it occurs, it remains for life
for most patients
•
Stomach pain
Leg pain or swelling
Chest pain
Back pain
• PNH is serious. It starts with your blood, but it can affect
your entire body
• PNH is dangerous. If you don’t take care of PNH, it can
lead to serious health problems, including some that can
be life-threatening
• PNH is constant. Even though you can’t see it, destruction
of blood cells, or hemolysis, is always happening. Ongoing
hemolysis is the leading cause of the signs, symptoms, and
serious health problems of PNH
Lowering hemolysis is
the key to managing
your PNH.
• PNH is manageable. In fact, management is vital
Blood clots
Kidney disease
Damage to your
organs
Stroke
Heart attack
R emember, you and your doctor should consider all of your signs,
symptoms, and lab results for a complete picture of your PNH.
Signs you may not always see or feel
2
Please see accompanying full prescribing information and Medication Guide for Soliris, including
boxed WARNING regarding serious meningococcal infection.
Please see Important Safety Information on pages 16-19.
If you would like more
in-depth information
about PNH, for yourself
or to share with others,
call OneSource™ at
1.888.SOLIRIS
(1.888.765.4747).
An Alexion Nurse Case
Manager can assist you
with additional education
and helpful resources.
3
What are the risks with PNH?
You may have heard your
doctor use the term “clone
size.” This refers to the
percentage of blood cells in
your body that are affected
by PNH. A larger clone size
means you have more PNH
cells, but even patients
who have a small clone size
can have symptoms that may
greatly impact their lives,
such as fatigue, stomach
pain, chest pain, shortness of
breath, and dark-colored urine.
Almost 1/2 of PNH patients
have signs of lung problems
Almost 2/3 of people with
PNH have chronic kidney
disease
Lung problems:
PNH can lead to lung problems caused by pulmonary hypertension.
Symptoms can include shortness of breath and other serious
health problems.
Kidney damage:
PNH can cause chronic kidney disease, a life-threatening condition.
In fact, kidney failure can cause death in patients with PNH.
Left untreated, PNH can lead to
blood clots in 40% of patients
Fatigue occurs in 96% of
people with PNH
Blood clots:
If left untreated, PNH can lead to blood clots. They can block veins
and arteries and lead to heart attack, stroke, and organ damage,
as well as other problems; and are the main cause of death in
patients with PNH.
Fatigue:
Hemolysis affects the way oxygen gets to parts of your body.
This can make you feel weak and tired to the point where
once-normal everyday activities are a struggle.
Why is PNH serious?
Defining
clone size
The biggest risk with PNH is that you cannot always see or feel
its effects. Or, you can get so used to the symptoms that you start
to feel as though they are “normal.” This means serious health risks
can take you by surprise. Fortunately, you’ve decided to take an
active role with Soliris®. Soliris reduces hemolysis and the serious
health risks associated with PNH. Remember, if left untreated,
PNH can lead to:
Other bone
marrow disorders
Like PNH, aplastic anemia
(AA) and myelodysplastic
syndrome (MDS) are
also bone marrow failure
disorders. But unlike PNH,
which is a disease of red
blood cell destruction,
AA and MDS are diseases
that affect the production
of blood cells in the bone
marrow. If you have AA or
MDS in addition to PNH, ask
your doctor about different
treatment options for these
conditions. In fact, the
presence of PNH clones
may actually increase your
chances of responding well
to some treatment options
for AA and MDS.
MYT
H:
TRU
TH:
MYT
Other common symptoms of PNH include stomach pain, difficulty
swallowing, and shortness of breath, which may indicate more
serious, underlying health risks.
4
Please see accompanying full prescribing information and Medication Guide for Soliris, including
boxed WARNING regarding serious meningococcal infection.
Please see Important Safety Information on pages 16-19.
5
What are the benefits of taking Soliris® regularly?
Soliris® is a
complement
inhibitor indicated
for the treatment
of patients with
paroxysmal nocturnal
hemoglobinuria (PNH)
to reduce hemolysis.
Helpful Tip
Give yourself credit for
taking control of your PNH
with Soliris. By sticking
with a regular infusion
schedule, you’re taking
the right steps to protect
against the possible health
risks associated with PNH.
Taking care of your health
is something to be proud of.
Keep up the great work!
For Soliris to inhibit hemolysis, the drug needs to stay above a
certain level in your blood. However, like all drugs, Soliris is broken
down and removed from your body over time. The time that it takes
your body to remove half of the drug is called the “half-life” of that
drug. The half-life of Soliris is about 11 days. A regular therapy
schedule keeps Soliris in your body at a level where it works best.
• Soliris is the only drug approved by the FDA to treat
patients with PNH. Soliris is the first and only drug approved
by the FDA to treat hemolysis, the underlying cause of all the
signs and symptoms of PNH
• Soliris should be taken every 2 weeks to reduce
hemolysis. A well-managed therapy schedule keeps Soliris in
your body at a level where it works best
You must receive a meningococcal vaccine at least 2 weeks before
your first dose of Soliris unless you have already had this vaccine.
6
Please see accompanying full prescribing information and Medication Guide for Soliris, including
boxed WARNING regarding serious meningococcal infection.
Please see Important Safety Information on pages 16-19.
If you forget or miss a
Soliris infusion, call your
doctor right away.
Helpful Tip
• Soliris inhibits hemolysis. Soliris works by inhibiting a part of
your body’s defense system from attacking red blood cells that
are missing an important protective protein
• Soliris provides clinical benefits. In clinical trials, people
saw significant improvements in hemolysis, blood clots, and
health-related quality of life
Soliris ® therapy
TH:
Refresh your memory about Soliris®
?
MYTH:
TRUTH:
MYTH:
TRUTH:
What would happen if I missed a dose?
If you forget or miss a Soliris infusion, call your doctor right away.
Soliris needs to be infused according to the recommended dosing
schedule for you to get the most out of your treatment. If the level
of Soliris in your body gets too low, hemolysis can occur. Hemolysis
is the underlying cause of the major health problems in PNH.
Missing doses can cause hemolysis to happen. Keep taking Soliris
and work closely with your doctor to best manage your PNH.
To get the most from your
Soliris therapy, stick with a
well-planned and ongoing
treatment schedule.
Remember, your Alexion
Nurse Case Manager
can help when you plan
to be away from home.
She'll do what she can
to make sure your Soliris
infusions continue without
interruption. Regular
infusions are important
to get the most from your
Soliris therapy. Just call
1.888.SOLIRIS about a
month before you go.
7
Refresh your memory about the benefits of Soliris ® therapy
• Improvement in energy level
Between 2 and 3 weeks
• Improved health-related quality of life
• Improvement in breathing
In clinical trials, people with PNH taking Soliris®
experienced the following:
Between 2 and 6 months
• 86% less hemolysis as measured by LDH
• Stabilized hemoglobin levels
• 92% fewer blood clots—one of the serious health
problems with PNH
After 6 months
• 73% reduction in the average number of
blood transfusions
• Improvement in energy level; improvement in healthrelated quality of life
Common side effects in people with PNH treated with Soliris include:
headaches, runny nose and colds, sore throat, back pain, and nausea.
In Soliris clinical trials, most people also received blood-thinning
medicine. The effect of stopping blood-thinning medicine during
treatment with Soliris has not been studied.
A measure of success
• Less hemolysis (measured by doctor)
• Fewer blood transfusions (if you need them)
• Continued improvement in health-related
quality of life
• Near normal levels of hemolysis
One way to check your
progress with Soliris is
through a simple LDH blood
test ordered by your doctor.
LDH measures hemolysis,
so when there’s less of it in
your blood, it means Soliris
is working. Soliris can lower
LDH in people with PNH
right away and can maintain
that level over time.
• Continued improvement in energy levels
• Even fewer blood transfusions (if needed)
• Continued stabilization in hemoglobin levels
At 3 years and longer
• Continued reduction in hemolysis
• Sustained reduction in blood clots
*Fictitious patient and account. Adapted from real patient story.
8
Please see accompanying full prescribing information and Medication Guide for Soliris, including
boxed WARNING regarding serious meningococcal infection.
Please see Important Safety Information on pages 16-19.
9
Soliris ® benefits
—Abby *
In 1 week
In clinical trials, blood clots were reduced.
“For me, managing my
PNH means having the
energy to spend quality
time with my husband.
Remembering my life
with PNH before I started
taking Soliris reminds
me never to take
everyday things like that
for granted again.”
Individual results for patients with PNH on Soliris may vary, so
the improvements you see in your health and your experiences
with your therapy may differ from others. But one thing that
every patient in global clinical trials experienced on Soliris was a
lowering of hemolysis. Even if you can’t see or feel it, hemolysis,
if left untreated, is the main cause of your PNH symptoms,
including those that can be life-threatening.
Helpful Tip
You, your doctor, and your
Alexion Nurse Case Manager
are a team. By working
together, you can get the
most out of your Soliris
therapy over the long term.
TH:
Stay on track to get the most from Soliris®
Soliris and hemolysis reduction
In clinical studies, patients on Soliris experienced continued
improvement in their symptoms through 4.5 years. However,
everyone is different, and everyone experiences PNH differently.
Even if it has been a while and you aren’t feeling 100%, it’s
important to remember that in global clinical trials Soliris reduced
hemolysis in everyone who received it. Continued improvement in
fatigue and health-related quality of life, reduction in the average
number of blood transfusions (if needed), and stabilization of
hemoglobin occurred over time. Remember, there’s no one sign,
symptom, or lab result that defines PNH, so looking at your full
clinical picture is important. Talk openly with your doctor and
your Alexion Nurse Case Manager about possible next steps.
—Dan*
*Fictitious patient and account. Adapted from real patient story.
10
Please see accompanying full prescribing information and Medication Guide for Soliris, including
boxed WARNING regarding serious meningococcal infection.
Please see Important Safety Information on pages 16-19.
11
Staying on track
Soliris reduces the main cause of serious PNH health problems:
ongoing hemolysis. However, in some patients, the body breaks
down drugs faster. In rare cases, hemolysis can start up again
even if you are taking Soliris on your regular schedule. This is
called “breakthrough hemolysis.” If you are not experiencing the
full benefits of your Soliris therapy, your doctor may test you for
breakthrough hemolysis by measuring the amount of LDH in your
blood just before a scheduled infusion. If you are experiencing
breakthrough hemolysis, your doctor may decide to reduce the
amount of time between doses. Talk with your doctor if you
experience any changes in the way Soliris has been working
for you.
What if my results on Soliris® are different from
other patients with PNH?
“Dealing with a rare
disease can be lonely.
But OneSource™ put me
in touch with other
people managing their
PNH, just like me. It’s
been great to be able
to share my experiences
with my new friends—
they understand what
it’s like to live with PNH.”
Get the most from your Soliris® infusion therapy
Why is Soliris given as an infusion?
Helpful Tip
Now that you are managing
your PNH with Soliris, you
may be feeling better and
doing things you haven’t
been able to do for a while,
like travel. Your Alexion Nurse
Case Manager can review
your insurance coverage with
you and—if your insurance
allows—help find a Soliris
trained doctor at an alternate
infusion site while you're
away from home.
UTH:
?
TRUTH:
MYTH:
TRUTH:
Soliris is a drug called a “humanized monoclonal antibody.” Soliris
works by blocking part of your immune system from attached red
blood cells missing an important protective protein (PNH cells).
For Soliris to work properly, the way that it is given to you is very
important. To make sure Soliris gets to where it works—your
blood—Soliris is given as an infusion into a vein. The actual
infusion generally takes about 35 minutes, and you only need to get
it every 2 weeks (after the first 5 weekly treatments). Common side
effects in people receiving Soliris include headaches, runny nose
and colds, sore throat, back pain, and nausea.
Helpful infusion tips
Some people don’t like infusions, but here are ways that you can
make it a better experience:
• Drink plenty of water. This will help your doctor find your
veins more easily
• Wear comfortable, layered clothing that you can adjust in case
you become overly warm or cool
Quick facts about long-term Soliris®
infusion therapy
• Hemolysis is the cause of serious health problems in PNH
• Ongoing hemolysis needs ongoing management
• Soliris infusion therapy offers results—both right away and
over time
• To get the most from your treatment, stick with a regular
Soliris therapy schedule
• It is possible to successfully manage your PNH
The same mechanism
that Soliris uses to
stop hemolysis can
increase your risk
of getting serious
and life-threatening
meningococcal
infections. Call your
doctor or get emergency
medical care right away
if you get any of these
signs or symptoms
of meningococcal
infection:
• Headache with
nausea or vomiting
• Headache and a fever
• Headache with a stiff
neck or stiff back
• Fever of 103˚F
(39.4˚C) or higher
• Fever and a rash
• Confusion
• Muscle aches with
flu-like symptoms
• Eyes sensitive to light
Infusion therapy
• Keep busy during your infusion by reading, doing a crossword
puzzle, knitting, or watching TV
If your insurance allows, your healthcare provider may approve
Soliris infusions in the comfort of your own home. Contact
OneSource™ for information about whether your insurance covers
home infusions.
12
Please see accompanying full prescribing information and Medication Guide for Soliris, including
boxed WARNING regarding serious meningococcal infection.
Please see Important Safety Information on pages 16-19.
13
How can I help successfully manage my PNH?
Maximize the benefits of Soliris®
You have been working hard to manage your PNH. To continue
to get the most from your Soliris therapy, keeping up with your
regular infusion schedule is important. If you aren’t already doing
so, you may want to use a planner or an electronic app to help
remind you of your infusion appointments. In most cases, your
doctor will schedule an infusion every 2 weeks.
The PNH Registry
Would you like to help
increase medical
understanding and
knowledge about PNH?
The PNH Registry is a
confidential program
designed to collect safety,
effectiveness, and quality
of life data on patients with
PNH. Its ultimate goal is to
help improve the diagnosis
and treatment of patients
with PNH. To take part in the
advancement of treatment
for PNH, contact OneSource
for help with finding a
doctor to enroll you.
?
MYTH:
Tell your doctor how you are feeling
Soliris® lowers LDH, the measure of hemolysis, in people with
PNH right away and over time. A high level of LDH doesn’t
necessarily mean Soliris isn’t working. It’s important to remember
that your LDH goes up and down for reasons other than PNH.
Stress, colds, or the flu can cause your level to temporarily
increase above normal.
Remember, no one sign, symptom, or lab result defines PNH. It’s
important to consider the full clinical picture. To get the most
from your Soliris therapy, it’s important to stick with your regular
infusions, as prescribed by your doctor. Speak with your doctor
whenever you experience a change in your health.
TRUTH:
T R E A T M E N T
MYTH:
TRUTH:
S U P P O R T
Remember, your Nurse Case Manager is here for you, to provide
continued support. Call 1.888.SOLIRIS (1.888.765.4747) for
answers to any questions you may have or for information about:
• Assistance with access to Soliris
®
• Ongoing treatment support
OneSource can help you if you are traveling, changing your
insurance, or experiencing a major life event that could affect
your regular Soliris schedule, such as a change in family, job,
or insurance status
Helpful Tip
Carry your Patient Safety
Card with you at all times.
Show it to any doctor or
nurse who treats you.
PATIENT SAfETy INfORMATION CARd
Important Safety Information for
Patients Taking Soliris®
Soliris can lower the ability of your immune system to
fight infections, especially meningococcal infection,
which requires immediate medical attention. If
you experience any of the following symptoms, you
should immediately call your doctor or seek emergency
medical care, preferably in a major emergency medical
care center:
• headache with nausea or vomiting
• headache and a fever
• headache with a stiff neck or stiff back
• fever of 103ºF (39.4ºC) or higher
• fever and a rash
• confusion
• muscle aches with flu-like symptoms
• eyes sensitive to light
!
Get emergency medical care right
away if you have any of these signs
or symptoms and show this card.
Even if you stop using Soliris, keep this card with
you after your last Soliris dose, since side effects
may occur up to 3 months after your last dose.
• Shared connections
If you would like to speak with someone who knows what you’re
going through, your Alexion Nurse Case Manager can put you in
touch with another person also living with PNH.
You can manage your PNH by staying committed to Soliris therapy.
Continue working with your doctor and your Alexion Nurse Case
Manager, and stay on track with your Soliris infusions.
15
How can I help?
14
Please see accompanying full prescribing information and Medication Guide for Soliris, including
boxed WARNING regarding serious meningococcal infection.
Please see Important Safety Information on pages 16-19.
Indications and Important Safety Information
WARNING: SERIOUS MENINGOCOCCAL INFECTIONS
See full prescribing information for complete boxed warning
Life-threatening and fatal meningococcal infections have
occurred in patients treated with Soliris and may become rapidly
life-threatening or fatal if not recognized and treated early.
• Comply with the most current Advisory Committee on
Immunization Practices (ACIP) recommendations for
meningococcal vaccination in patients with complement
deficiencies.
• Immunize patients with a meningococcal vaccine at least
2 weeks prior to administering the first dose of Soliris,
unless the risks of delaying Soliris therapy outweigh the
risks of developing a meningococcal infection. (See Serious
Meningococcal Infections for additional guidance on the
management of the risk of meningococcal infection.)
• Monitor patients for early signs of meningococcal infections,
and evaluate immediately if infection is suspected.
Soliris is available only through a restricted program under
a Risk Evaluation and Mitigation Strategy (REMS). Under the
Soliris REMS, prescribers must enroll in the program.
Indications and Usage
Soliris is indicated for the treatment of patients with paroxysmal
nocturnal hemoglobinuria (PNH) to reduce hemolysis.
Soliris is indicated for the treatment of patients with atypical hemolytic
uremic syndrome (aHUS) to inhibit complement-mediated thrombotic
microangiopathy.
Limitation of Use
Soliris is not indicated for the treatment of patients with Shiga toxin
E. coli related hemolytic uremic syndrome (STEC-HUS).
16
Please see accompanying full prescribing information and Medication Guide for Soliris, including
boxed WARNING regarding serious meningococcal infection.
Important Safety Information
Contraindications
Soliris should not be used in:
• Patients who have meningococcal infection
• P atients who have not been vaccinated against meningitis infection
unless the doctor decides that urgent treatment with Soliris is needed
Warnings and precautions
Soliris® increases your risk of getting serious and life-threatening
meningococcal infections.
Meningococcal infections may quickly become life-threatening and
cause death if not recognized and treated early.
1. You must receive a meningococcal vaccine at least 2 weeks before
your first dose of Soliris unless you have already had this vaccine.
If your doctor decides that urgent treatment with Soliris is needed,
you should receive a meningococcal vaccine as soon as possible.
2. I f you had a meningococcal vaccine in the past, you might need a
booster dose before starting Soliris. Your doctor will decide if you
need another dose of a meningococcal vaccine.
3. A meningococcal vaccine does not prevent all meningococcal
infections. Call your doctor or get emergency medical care
right away if you get any of these signs and symptoms of a
meningococcal infection:
• Headache with nausea or vomiting
• Headache and a fever
• Headache with a stiff neck or stiff back
• Fever of 103°F (39.4°C) or higher
• Fever and a rash
• Confusion
• Muscle aches with flu-like symptoms
• Eyes sensitive to light
17
Indications and Important Safety Information (continued)
Other Infections
Adverse Reactions
Soliris may also increase the risk of other types of serious infections
If your child is treated with Soliris, make sure that your child receives
the following vaccinations:
• Streptococcus pneumoniae
• Haemophilus influenza type b (Hib)
Common side effects in people with PNH treated with Soliris include:
• Headaches
• Runny nose and colds
• Sore throat
• Back pain
• Nausea
Monitoring Disease Manifestations After Soliris Discontinuation
If you have PNH, your doctor will need to monitor you closely for at
least 8 weeks after stopping Soliris. Stopping treatment with Soliris
may cause breakdown of your red blood cells due to PNH.
Symptoms or problems that can happen due to red blood cell breakdown
include:
• Drop in the number of your red blood cell count
• Drop in your platelet count
• Confusion
• Chest pain
• Kidney problems
• Blood clots
• Difficulty breathing
Treatment with Soliris should not stop you from taking your
anticoagulant medicine (medicine that keeps your blood from clotting).
Common side effects in people with aHUS treated with Soliris include:
• Headache
• Low red blood cell count
• Diarrhea
• Cough
• High blood pressure
• Swelling of the arms
and legs
• Common cold (upper
respiratory infection)
• Nausea
• Abdominal pain
• Urinary tract infections
• Vomiting
• Fever
• Nasopharyngitis
Serious Allergic Reactions
Serious allergic reactions can happen during your Soliris infusion. Tell
your doctor or nurse right away if you get any of these symptoms during
your Soliris infusion:
• Chest pain
• Trouble breathing or shortness of breath
• Swelling of your face, tongue, or throat
• Feel faint or pass out
If you have an allergic reaction to Soliris, your doctor may need to infuse
Soliris more slowly, or stop Soliris.
18
Please see accompanying full prescribing information and Medication Guide for Soliris, including
boxed WARNING regarding serious meningococcal infection.
19
Want to learn more about
PNH or Soliris®?
• Visit www.Soliris.net
• Call 1.888.SOLIRIS (1.888.765.4747)
Soliris offers clinical benefits:
• 86% less hemolysis as measured by LDH
• 92% fewer blood clots—one of the serious health problems with PNH
• 73% reduction in the average number of blood transfusions
• Improvement in energy level; improvement in health-related quality of life
Indications and Usage
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Soliris (eculizumab) is indicated for the treatment of patients with paroxysmal nocturnal
hemoglobinuria (PNH) to reduce hemolysis.
WARNING: SERIOUS MENINGOCOCCAL INFECTIONS
See full prescribing information for complete boxed warning
Life-threatening and fatal meningococcal infections have occurred in patients treated with
Soliris and may become rapidly life-threatening or fatal if not recognized and treated early.
• Comply with the most current Advisory Committee on Immunization Practices (ACIP)
recommendations for meningococcal vaccination in patients with complement deficiencies
• Immunize patients with a meningococcal vaccine at least 2 weeks prior to administering
the first dose of Soliris, unless the risks of delaying Soliris therapy outweigh the risks of
developing a meningococcal infection. (See Serious Meningococcal Infections for additional
guidance on the management of the risk of meningococcal infection.)
• Monitor patients for early signs of meningococcal infections, and evaluate immediately if
infection is suspected
Soliris is available only through a restricted program under a Risk Evaluation and Mitigation
Strategy (REMS). Under the Soliris REMS, prescribers must enroll in the program.
Common side effects in people with PNH treated with Soliris include headaches, runny nose
and colds, sore throat, back pain, and nausea.
In Soliris clinical trials, most people also received blood-thinning medicine. The effect of
stopping blood-thinning medicine during treatment with Soliris has not been studied.
Please see accompanying full prescribing information and Medication Guide for Soliris, including
boxed WARNING regarding serious meningococcal infection.
Please see Important Safety Information on pages 16-19.
Soliris® is a registered trademark of Alexion Pharmaceuticals, Inc.
© 2014, Alexion Pharmaceuticals, Inc.
All rights reserved.
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