Download PRIMARY IMMUNE DEFICIENCY DISEASES IN AMERICA:

TREATMENT EXPERIENCES AND
PREFERENCES AMONG PATIENTS WITH
PRIMARY IMMUNODEFICIENCY DISEASES
NATIONAL SURVEY OF PATIENTS: 2008
The National Patient Organization Dedicated to Advocacy, Education and Research
for
Primary Immunodeficiency Diseases
Prepared by:
Abt SRBI, Inc.
May 6, 2009
Table of Contents
Overview ............................................................................................................... 1
Background: Immune Deficiency Diseases .......................................................... 1
Treatment Experiences Survey Of IVIG Users: 2002 .......................................... 2
Treatment Experiences Survey Of IVIG Users: 2008 .......................................... 3
Characteristics Of Patients with PIDD In The Treatment Survey ........................ 3
Health Before Diagnosis ....................................................................................... 9
Current Sources Of Care And Health Care Coverage ........................................ 13
Treatment With IgG ............................................................................................ 18
Product Effectiveness, Safety And Satisfaction ................................................. 24
Efficacy Of Treatment ........................................................................................ 30
Availability ......................................................................................................... 38
This survey was funded in part by an unrestricted educational grant provided by
Talecris Biotherapeutics
TREATMENT EXPERIENCES AND PREFERENCES
Overview
The Immune Deficiency Foundation (IDF) conducted its Second National Survey
of the Treatment Experiences and Preferences of Patients with Primary
immunodeficiency Diseases in 2008.
A total of 2,500 patients with primary
immunodeficiency diseases (PIDD), who had reported either intravenous
immunoglobulin (IVIG) therapy, or subcutaneous immunoglobulin (SCIG) therapy, were
randomly selected from the IDF database for this survey. These individuals were mailed
an eight-page, self-administered questionnaire, which they were asked to complete and
return to IDF. A second mailing was made to those who did not respond to the first
mailing. A total of 1,088 respondents returned a completed questionnaire for a 43.5%
response rate. After removing cases of respondents with non-primary immunodeficiency
disease diagnoses, or those not currently using immunoglobulin therapies, the survey
yielded a total of 1,030 patients with primary immunodeficiency diseases who were
currently being treated with either IVIG or SCIG. These cases provide a relatively large
national sample of persons with primary immunodeficiency diseases, who have been
treated with immunoglobulin therapy. Although this is not a probability sample from
which we can make population estimates within known limits of sampling error, it
provides the most representative sample currently available of patients with a rare disease
from which we can examine treatment experiences.
Background: Immunodeficiency Diseases
Primary immunodeficiency diseases represent a class of disorders in which there
is an intrinsic defect in the human immune systems (rather than immune disorders that
are secondary to infection, chemotherapy, or some other external agent). In some cases,
the body fails to produce any or enough antibodies to fight infection. In other cases, the
cellular defenses against infection fail to work properly. There are more than 150
different primary immunodeficiency diseases currently recognized by the World Health
Organization.
Medical recognition of primary immunodeficiency disease is a little more than
fifty years old. Although these disorders may have existed in antiquity, it was not until
the development of antibiotics that infections could be controlled long enough to
recognize there was an underlying defect in the immune system. Also, the parallel
development of gamma globulin in World War II provided a replacement therapy for the
antibody deficiency forms of immune deficiency. Today, immunoglobulin replacement
therapy (IG therapy) is available in intravenous (IVIG) and subcutaneous (SCIG) modes
of administration.
The major health surveys conducted by the government in the United States, the
National Health Interview Survey and the National Health and Nutrition Examination
Survey, do not collect information on primary immunodeficiency diseases. No
comprehensive population survey has ever been undertaken by the federal government to
estimate the prevalence or the population characteristics of these diseases in the United
States. The 2005 prevalence survey conducted by the Immune Deficiency Foundation
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TREATMENT EXPERIENCES AND PREFERENCES
provided the first reliable estimate of the prevalence of diagnosed primary
immunodeficiency diseases in the United States. However, a probability sample of
10,000 households yielded only 23 cases of primary immunodeficiency diseases, which is
too small to yield reliable estimates of the characteristics of patients and their treatment
experiences. Hence, although these diseases are clinically described in the medical
literature, there is no current, comprehensive portrait available of the patient with primary
immunodeficiency disease, their medical condition and their treatment.
Treatment Experiences Survey of IVIG Users: 2002
In 1997 IDF conducted its first treatment survey based on respondents who stated
that they were IVIG users in IDF’s first patient survey. In 2002, the Immune Deficiency
Foundation issued its first report on the Treatment Experiences and Preferences among
Patients with Primary immunodeficiency Diseases. Two national surveys of patients with
primary immunodeficiency diseases conducted by IDF in 1996/97 and 2002 provided the
sampling frame for a follow-up survey of 1,000 persons identified as currently using
intravenous immunoglobulin (IVIG).
The treatment survey was first mailed to the
2,589 unique cases with a name and address in a database that could be linked with the
1996 patient survey information. However, since the 1996 survey information was more
than five years old, a very substantial portion of the sample was expected to be bad
addresses. The original addresses were matched with the National Change of Address
database to update these addresses where possible.
An eight-page questionnaire with a cover letter was mailed by SRBI to these
persons on August 29, 2002. The cover letter included toll-free numbers for both IDF
and SRBI. The package included a postage-paid business reply envelope for respondents
to return the completed questionnaire. A postcard reminder was sent to all potential
respondents one week later. The questionnaire for the “old patients” from the 1996
survey was sent to all, regardless of their reported IVIG use in 1996/97. Consequently,
the questionnaire for this population was structured so that patients who were no longer
using IVIG ended the interview.
A second sample of new patients that did not participate in the 1996 survey was
drawn from the Second National Patient Survey. Only patients with primary
immunodeficiency diseases who were currently using IVIG to treat their condition were
included in this sample. Assuming a response rate of approximately fifty percent to the
second survey, nearly 1,000 eligible participants from the 2002 survey were sent
questionnaire for the treatment survey. The first mailing to the new patients was sent on
November 9, 2002. A second mailing to non-respondents was sent on January 2, 2003. A
total of 651 out 957 (68.0%) cases in the “new patient” sample completed and returned
the treatment questionnaires by the end of the field period.
The 2002 Treatment Experiences report was based on the first 1,186 completed
questionnaires from the “old patient” and “new patient” samples. This included 535
patients who currently use IVIG, who were first identified in the baseline 1996 survey. It
also includes 651 patients currently using IVIG who were first identified in the 2002
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TREATMENT EXPERIENCES AND PREFERENCES
survey. The findings from the 2002 survey have been widely used by patients, health
providers, third party payers and manufacturers to understand treatment patterns and
outcomes in this population.
Treatment Experiences Survey of IG Therapy Users: 2008
There have been a number of developments since 2002 that are likely to have
affected the pattern of treatment for patients with primary immunodeficiency diseases.
First, since the earlier treatment survey, subcutaneous immunoglobulin has emerged as a
treatment option with a licensed product in the United States. Second, changes to
Medicare have affected reimbursement for IVIG, which has had an impact on site of
service and access to treatment for Medicare patients. Third, there is anecdotal evidence
that reimbursement for IVIG has become more difficult from private third-party payers as
well. Fourth, standards of care guidelines for the treatment of primary immunodeficiency
disease have been developed and disseminated within the specialist community. Finally,
changes in the size and demographics of the PIDD population may have affected the
patterns of treatment in that community. Consequently, IDF conducted a Third Treatment
Experiences Survey among patients with PIDD in 2008.
A sampling frame for the Third Treatment Survey was constructed from the IDF
patient database. The Foundation constructed a listing of persons identified as users of
IG therapy in previous IDF surveys or new patients that had been identified as IgG users
in the database. Next, all non-patients, deceased patients and non-US addresses were
removed from this listing, which was then matched with the National Change of Address
database to update addresses where possible. Finally, 2,500 potential respondents were
selected from this sampling frame through random case selection. The sample size for
the survey was selected to ensure a minimum sample size of 1,000 completed interviews,
equivalent to the achieved sample for the 2002 Treatment Survey.
An eight-page questionnaire with a cover letter was initially mailed to the sample
of 2,500 persons between July 9th and August 20, 2008. The package included a postagepaid business reply envelope for respondents to return the completed questionnaire. A
second mailing to non-respondents began on a rolling basis on August 26, 2008. A total
of 1,088 questionnaires were returned (43.5% response rate). From that total, 13 cases
were eliminated for being neither a patient nor parent/caretaker of a person with PIDD, 7
cases were eliminated due to a diagnosis other than PIDD, and 38 cases were eliminated
for having never used IG therapy. This resulted in a total of 1,030 cases in the sample,
which form the basis for this report.
Characteristics of Patients with PIDD in the 2008 Treatment Survey
The population for the Treatment Survey was restricted to those patients with
primary immunodeficiency diseases who have been treated with immunoglobulin
therapy. Immunoglobulin is the medically recommended treatment for some, but not all
primary immunodeficiency diseases. In the 1996 national survey of nearly 3,000 patients
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TREATMENT EXPERIENCES AND PREFERENCES
with primary immunodeficiency diseases, 70% reported that they had been treated with
IVIG. In the Third National Patient Survey in 2007, 83% reported that they had ever
been treated regularly with IVIG or SCIG.
Nearly three-quarters (73%) of respondents in the 2008 Treatment Survey were
Patients with PIDD. Only 24% were either patients or caregivers of a patient with PIDD,
and just 2% were both a patient and the caregiver/parent of a patient (Figure 1).
Patient Or Parent: 2008
Patient
73%
Parent/caregiver
24%
Both
2%
Blank
1%
Q1. Are you a patient with a primary immunodeficiency disease (PIDD) or parent/caregiver of a child in the
household with PIDD? N=1030
Figure 1
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TREATMENT EXPERIENCES AND PREFERENCES
Similar to the findings of the 2002 report, primary immunodeficiency diseases are
no longer a pediatric condition. Only one-in-ten patients (10%) were aged 12 or younger
in 2008, compared to 14% in 2002. Eight percent were adolescents, aged thirteen to
seventeen in 2008, compared to 9% in 2002. In total, only 18% of patients in the 2008
Treatment Survey were under the age of 18. Six years ago, in the 2002 study, 23% of
patients were under 18 (Figure 2).
Twelve percent are young adults, aged 18 to 29, and 16% of the patient
population is 30 to 44 years old. Nearly forty percent of patients (39%) are middle aged,
45 to 64 years old, and, sixteen percent (16%) of Patients with PIDD that have been
treated with immunoglobulin therapy in 2008 are aged 65 or older. In 2002, only 8% of
PIDD current users of IVIG were aged 65 and older (Figure 2).
Comparison Of Patient Age:
2008 And 2002
50%
39%
40%
2008
34%
2002
30%
23%
20%
16%
14%
10%
10%
16%
12% 12%
8% 9%
8%
0%
0-12
13-17
18-29
30-44
45-64
Q2. What is the date of birth of the (adult patient/oldest child) in the household with a primary
immunodeficiency disease? N=1030 (2008)
Q2. What is the date of birth of the PIDD patient being treated with IGIV? N=1186 (2002)
65+
Figure 2
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TREATMENT EXPERIENCES AND PREFERENCES
In the 2008 treatment survey, 39% of respondents were male and 61% were
female. There was a slightly higher percentage of males in the 2002 survey (43%) and a
slightly lower percentage (57%) of females (Figure 3). There are, however, dramatic
differences across age groups. Among respondents under 18, 71% were male, and just
29% were female. Among respondents over 18, the ratio of males to females drops to
33% male, 67% female.
Comparison Of Patient Gender:
2008 And 2002
80%
70%
61%
60%
2008
57%
2002
50%
40%
43%
39%
30%
20%
10%
0%
Male
Female
Q3. What is the gender of that person (adult patient/oldest child with PIDD)? N=1030 (2008)
Q7. What is the gender of that person? N=1,165 (2002)
Figure 3
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TREATMENT EXPERIENCES AND PREFERENCES
One-third of respondents in the Treatment Survey (32%) had been diagnosed with
a primary immunodeficiency disease before the age of 18. Four percent of respondents
had been diagnosed before the age of 1, 15% were diagnosed between ages 1 and 5, 9%
between ages 6 and 12, and 4% between ages 13 and 17. Another 10% were diagnosed
as young adults, between the ages of 18 and 29. Just over one quarter (26%) were
diagnosed between ages 30 and 44, and another 24% were diagnosed between ages 45
and 64. Only 2% of respondents in the Treatment Survey were diagnosed as having a
primary immunodeficiency disease at age 65 or older (Figure 4).
Patient Age At Diagnosis: 2008
30-44
26%
45-64
24%
65+
2%
18-29
10%
1-5
15%
13-17
4%
6-12
9%
Blank
5%
0
4%
Q4. At what age (in years) was that person first diagnosed with a primary immunodeficiency
disease? N=1030
Figure 4
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TREATMENT EXPERIENCES AND PREFERENCES
Most respondents (79%) had no family history of primary immunodeficiency
disease (Figure 5). However, about one in five of these Patients with PIDD did have
other family members with the disease, including 5% having a diagnosed brother, 4%
having a diagnosed daughter, son or sister, 2% with a diagnosed mother and 1% with a
diagnosed father. Another 6% had some other family member that had been diagnosed
with a primary immunodeficiency disease.
Other Diagnosed Family Members:
2008
90%
79%
80%
70%
60%
50%
40%
30%
20%
10%
5%
4%
4%
4%
Brother
Daughter
Son
Sister
6%
2%
1%
Mother
Father
0%
Q6. Which other family member(s) have been diagnosed as having a primary
immunodeficiency disease? (net of cases) N=1030
Other
No other
member
diagnosed
Figure 5
8
TREATMENT EXPERIENCES AND PREFERENCES
Health Before Diagnosis
Although the vast majority of patients with PIDD have no family history of
immune deficiency diseases, almost all experienced repeated, serious or unusual
infections prior to diagnosis. More than nine in ten Patients with PIDD (93%) that have
been treated with immunoglobulin therapy reported experiencing repeated, serious, or
unusual infections prior to diagnosis (Figure 6). Only 6% reported no serious or repeated
infections prior to diagnosis.
Infections Prior To Initial
Diagnosis: 2008
No
6%
Yes
93%
Blank
1%
Q7a. Did the patient experience repeated, serious, or unusual infections prior to initial diagnosis as
immune deficient? N=1030
Figure 6
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TREATMENT EXPERIENCES AND PREFERENCES
Only 10% of patients missed no days of work or school in the 12 months prior to
diagnosis. Another 10% missed 1 week or less, 12% missed between 1 and 2 weeks,
18% missed between two weeks and 1 month, 11% missed between 32 and 100 days, and
another 7% missed over 100 days (Figure 7). Excluding those who were too young to go
to work or school (16%) and those who did not answer the question (16%), the survey
found that Patients with PIDD were too sick to attend work or school or perform their
usual activities an average of 36.8 days during the 12 months prior to diagnosis.
Days Too Sick To Attend Work/School In
The 12 Months Prior To Diagnosis: 2008
Mean number of
days 36.8
15 days - 1 month
18%
32-100 days
11%
8 days - 2 weeks
12%
101-200 days
3%
201 days+
4%
1 week or less
10%
Infant/NA
16%
None
10%
Blank
16%
Q18. Not counting hospitalizations, how many days was he/she too sick to work, go to school or perform
usual activities in the 12 months prior to diagnosis? N=1030
Figure 7
10
TREATMENT EXPERIENCES AND PREFERENCES
Only a little less than one-third of patients with PIDD (32%) reported that they
were never hospitalized before diagnosis as immune deficient. Another 11% were
hospitalized only once prior to diagnosis, while 19% were hospitalized 2-3 times. By
contrast, 11% of patients with PIDD were hospitalized 4-5 times before diagnosis, and
10% were hospitalized 6-10 times. Another 5% reported 11-20 hospitalizations, and 3%
reported over 20 hospitalizations before being diagnosed as immune deficient (Figure 8).
The cost of undiagnosed primary immunodeficiency to patients, the health care system
and society as a whole is staggering, just in terms of hospitalizations.
Times Hospitalized Prior To
Diagnosis: 2008
2-3
19%
1
11%
4-5
11%
6-10
10%
0
32%
11-20
5%
Other
9%
21+
3%
Q8. How many times had he/she been hospitalized before diagnosis as immune deficient? N=1030
Figure 8
11
TREATMENT EXPERIENCES AND PREFERENCES
As a result of late diagnosis, and repeated and severe infections prior to diagnosis,
over one half of patients with PIDD reported at least some permanent impairment before
their diagnosis as immune deficient. Nearly two out of five patients with PIDD reported
permanent impairment of lung function (37%) prior to diagnosis. Additionally, 17% had
permanent impairment to digestive function, 13% hearing loss or impairment, 8%
neurological impairment, 7% loss of mobility, and 5% had either vision loss or
impairment. Another 11% had some other permanent impairment or loss prior to
diagnosis as immune deficient (Figure 9).
Permanent Impairments Or Losses
Prior To Initial Diagnosis: 2008
60%
46%
40%
37%
17%
20%
13%
11%
8%
7%
5%
on
e
N
th
er
O
isi
on
V
ob
ili
ty
M
eu
ro
lo
gi
ca
l
N
ea
rin
g
H
Fu
nc
tio
n
ig
es
tiv
e
D
Lu
ng
Fu
nc
tio
n
0%
Q9. By the time of initial diagnosis as immune deficient, had he/she suffered any permanent impairment
or loss of________? (net of cases) N=1030
Figure 9
12
TREATMENT EXPERIENCES AND PREFERENCES
Current Sources of Care and Health Care Coverage
The majority of patients with primary immunodeficiency diseases who have been
treated with immunoglobulin therapy see a primary care doctor most often for their health
care, not a specialist. More than half of patients reported that the doctor they see most
often for their health care is in family practice (26%), internal medicine (21%) or
pediatrics (8%). However, one-in-five (21%) reported that they see an immunologist
most often for their primary health care.
Only a minority reported seeing other
specialists most often for their health care, including 3% who see a pulmonologist as their
primary doctor, 2% use an allergist, 1% each say they see an ear, nose and throat
specialist (ENT) or hematologist, and 3% reported seeing an other type of doctor most
often for their health care (Figure 10).
Type of Primary Doctor: 2008
26%
General/Family practice
Immunology
21%
Internal Medicine
21%
8%
Pediatrics
3%
Pulmonologist
2%
Allergist
2%
Infectious Disease
1%
Ear, Nose & Throat
1%
Hematology
Gastroenterologist
1%
No regular doctor
0.2%
Other
3%
12%
Blank
0%
5%
10%
15%
20%
25%
Q19. What kind of doctor does the patient see most often for his/her primary health care? N=1030
30%
Figure 10
13
TREATMENT EXPERIENCES AND PREFERENCES
However, only a quarter of patients with PIDD (24%) said that they did not see
another physician for the treatment and management of their immune deficiency
condition. Three-quarters of these patients with PIDD (74%) reported that there was
another physician responsible for the treatment and management of their PIDD (Figure
11). Among patients who had another doctor who was responsible for treatment and
management of their primary immunodeficiency disease, over three-quarters (78%) said
they saw an immunologist to treat their PIDD. In addition, 11% saw an allergist, 10% a
hematologist, 5% a pulmonologist, 4% an infectious disease doctor, and 3% saw an ENT
specialist. Another 8% had another type of doctor to treat their PIDD (Figure 12).
Hence, while most patients with PIDD that have used immunoglobulin therapy rely on a
primary care doctor as their main source of health care, the vast majority report also
having a specialist responsible for the management and treatment of their PIDD
condition.
Another Physician Responsible For
PIDD Treatment And Management: 2008
Yes
74%
No
24%
Blank
2%
Q22a. Is another physician responsible for the treatment and management of the patient’s PIDD? N=1030
Figure 11
14
TREATMENT EXPERIENCES AND PREFERENCES
Type Of Doctor Responsible For PIDD
Treatment And Management: 2008
100%
90%
78%
80%
70%
60%
50%
40%
30%
20%
11%
10%
5%
10%
4%
8%
3%
th
er
Ea
r,
no
se
an
d
O
th
ro
at
ise
as
e
D
In
fe
ct
io
us
on
ol
og
ist
em
Pu
lm
at
ol
og
ist
lle
rg
ist
A
H
Im
m
un
ol
og
ist
0%
Q22b. Which type of physician is responsible for the treatment and management of the patient’s PIDD?
BASE: Those who have a doctor other than the primary doctor to treat PIDD (net of cases) N=761
Figure 12
15
TREATMENT EXPERIENCES AND PREFERENCES
Moreover, in addition to their primary doctor and the doctor most responsible for
the treatment of their immune deficiency disease, more than four out of five patients with
PIDD (82%) saw at least one other specialist during the past year. Between one-quarter
and one third of patients with PIDD saw a hematologist (35%), dermatologist (30%),
ENT specialist (28%) pulmonologist (27%), or an immunologist (26%) other than their
primary care doctor and the one responsible for the management of their condition.
Twenty-two percent saw another allergist or cardiologist. One in seven patients with
PIDD (16%) have seen an endocrinologist in the past year, while others report seeing an
infectious disease specialist (13%), gastroenterologist (12%), rheumatologist (5%) or
neurologist (3%) in the past year. Only 18% of patients saw none of these specialists
unless they were their primary care doctor or the one responsible for the treatment of
their immune deficiency disease (Figure 13).
Other Specialists Seen In Past Year: 2008
35%
Hematologist
30%
Dermatology/skin specialist
28%
ENT/Otolaryngology
27%
Pulmonary/lung specialist
26%
Immunologist
Allergy/asthma specialist
22%
Cardiology/heart Specialist
22%
16%
Endocrinologist
13%
Infectious Disease
12%
Gastroenterologist
5%
Rheumatologist
Neurologist
3%
27%
Other
18%
None of these
0%
5%
10%
15%
20%
25%
30%
Q23. Aside from the patient’s primary care physician and/or the physician most responsible for
the treatment of the patient’s PIDD, hast he patient seen any of the following specialists in the
past year? (net of cases) N=1030
35%
40%
Figure 13
16
TREATMENT EXPERIENCES AND PREFERENCES
Almost all persons with primary immunodeficiency diseases being treated with
IgG had some form of health insurance coverage. The majority (66%) had insurance
through an employer group policy. Ten percent reported health care coverage through a
non-employer group policy, while 7% had an individual policy. More than a quarter of
the Patients with PIDD in this sample (28%) had Medicare coverage, while 10% had
Medicaid coverage, and 2% were covered by a state or county health program. Three
percent had health coverage through COBRA. Nine percent reported other forms of
coverage, such as military or veterans. Less than one percent reported no health care
coverage through any of these sources (Figure 14).
Health Care Coverage: 2008
Insurance through employe r
/work
66%
28%
Medicare
10%
Other group insurance
10%
Medicaid
7%
Individual policy
COBRA
State/county health agency
3%
2%
9%
Other
No health insurance coverage
0%
0.5%
10%
20%
30%
40%
50%
Q73. What is the coverage of the patient’s health care coverage? (net of cases) N=1030
60%
70%
80%
Figure 14
17
TREATMENT EXPERIENCES AND PREFERENCES
Treatment with IG Therapy
The Treatment Survey is restricted to patients with primary immunodeficiency
diseases who have ever been treated with either IVIG (intravenous immunoglobulin) or
SCIG (subcutaneous immunoglobulin). Almost all of these patients (93%) have been
treated with IVIG, including those who have only been treated with IVIG (75%) and
another 18% who have been treated with both IVIG and SCIG. A quarter of this sample
has been treated with SCIG, including 7% who have only been treated with SCIG and
another 18% who have been treated with both IVIG and SCIG (Figure 15). It should be
noted that the proportion of SCIG users in the sample should not be used as a market
estimate since the patients with PIDD in the IDF database are more likely to be exposed
to SCIG as a treatment option either through the IDF or through the major medical
centers from which many of the IDF connected patients are drawn.
Ever IVIG Or SCIG Therapy: 2008
Yes, SCIG
7%
Yes, IVIG
75%
Yes, both
18%
Yes, unsure
which
0.10%
Q24a. Has the patient ever been treated with intravenous immunoglobulin (IVIG) or
subcutaneous immunoglobulin (SCIG) therapy on a regular basis? N=1030
Figure 15
18
TREATMENT EXPERIENCES AND PREFERENCES
Nearly seven in ten patients with PIDD (69%) in the treatment sample are
currently using IVIG. Another 23% of the patients in the sample are currently using
SCIG. Seven percent of respondents who have been treated in the past with either IVIG
or SCIG are no longer using IG therapy (Figure 16). Once again, these proportions are
more likely to reflect the nature of the patients with PIDD known to IDF, rather than true
market share. However, the treatment survey provides very useful subsample sizes of
IVIG users, SCIG users, and former IG therapy users for our analysis.
Current IVIG Or SCIG Therapy: 2008
Yes, SCIG
23%
Yes, IVIG
69%
No
7%
Blank
1%
Q26a. Is he/she currently being treated with IVIG or SCIG for his/her immune deficiency
disease? N=1030
Figure 16
19
TREATMENT EXPERIENCES AND PREFERENCES
Among patients that are no longer being treated with IG therapy, nearly two in
five (39%) report that IG therapy is no longer prescribed for them. Of greater concern,
29% of patients with PIDD who have discontinued immunoglobulin therapy had to stop
due to a lack of insurance or inadequate insurance, and 18% claimed it was too expensive
despite good insurance. For 8% of those who have stopped, safety issues or side effects
were a concern, and for 6% availability was a problem. Forty-two percent of patients no
longer using IG therapy reported other or additional issues as the reason why they have
stopped (Figure 17).
Reasons For Past Users Not Currently
Receiving IG Therapy: 2008
50%
42%
45%
40%
39%
35%
29%
30%
25%
18%
20%
15%
8%
10%
6%
5%
0%
No longer
prescribed
Lack of or
inadequate
insurance
Too expensive
(despite good
insurance)
Safety/side
effects
IVIG not
available or
hard to get
Q26b. Why is the patient no longer being treated with immunogloblin? BASE: Those who are no longer
receiving treatment (net of cases) N=68
Other
Figure 17
The appropriateness for IG therapy depends on the specific diagnosis of primary
immunodeficiency disease. The current IDF Clinical Care Guidelines for Patients with
Primary Immunodeficiency Diseases recommend treatment with immunoglobulin for
Agammaglobulinemia, Common variable immunodeficiency (CVID), Hyper IgM,
Wiskott-Aldrich, and Severe combined immunodeficiency (SCID). Specific antibody
deficiency, which may be a part of IgG subclass deficiency, is also indicated, although
IgG subclass deficiency alone is not.
20
TREATMENT EXPERIENCES AND PREFERENCES
Among this national sample of patients with primary immunodeficiency diseases
who were currently using either as IVIG or SCIG, over three-quarters (77%) reported that
their current diagnosis is CVID.
Another 9% reported a diagnosis of
Agammaglobulinemia. There were also small proportions of patients with SCID (2%),
IgA deficiency (2%), Hyper IgM syndrome (1%) or Specific antibody disorder (1%).
Only 9% of the current IG therapy users had a diagnosis of IgG subclass deficiency. The
remaining IG therapy users (3%) had another diagnosis (Figure 18). Hence, about nine
out of ten current IG therapy users among patients with primary immunodeficiency
diseases in this sample had a diagnosis for which IG therapy is the current standard for
care.
Immunodeficiency Disease Diagnosis
Among Current IG Users: 2008
90%
77%
80%
70%
60%
50%
40%
30%
20%
9%
10%
9%
2%
2%
1%
1%
0.2%
2%
0.1%
0.6%
Bl
an
k
th
er
O
m
ag
lo
bu
lin
em
Se
ia
ve
re
Co
m
bi
ne
d
Ig
A
D
ef
H
ic
yp
ien
er
cy
Ig
M
Sp
ec
D
ef
ifi
ici
cA
en
nt
cy
ib
od
y
D
iso
rd
W
er
is
ko
ttA
Al
ta
dr
xi
ich
a
Te
la
ng
iec
ta
sis
D
Su
ga
m
A
bc
la
ss
on
m
om
Ig
G
C
ef
ic
ie
nc
y
Va
ri
ab
le
0%
Q5. What is the specific diagnosis of that person’s immunodeficiency disease? BASE: Those who are
currently using SCIG or IVIG therapy N=955 (net of cases)
Figure 18
21
TREATMENT EXPERIENCES AND PREFERENCES
Despite the appropriateness of this treatment, over half of the respondents that had
discontinued immunoglobulin therapy because it was no longer prescribed for them had a
diagnosis of CVID (52%), while others were diagnosed with SCID,
Agammaglobulinemia, IgA Deficiency, Specific antibody disorder, and Wiskott-Aldrich
(Figure 19).
Immunodeficiency Disease Diagnosis Among
Those No Longer Prescribed IG Therapy: 2008
90%
80%
70%
60%
52%
50%
40%
30%
18%
20%
11%
11%
7%
10%
4%
4%
4%
th
er
O
ko
ttAl
dr
ich
W
is
D
ef
ic
ien
cy
ia
ga
m
A
Ig
A
D
y
nt
ib
od
A
m
ag
lo
bu
lin
em
iso
rd
er
bi
ne
d
Co
m
Sp
ec
ifi
c
Su
Ig
G
Se
ve
re
D
bc
la
ss
on
m
om
C
ef
ic
ie
nc
y
Va
ri
ab
le
0%
Q5. What is the specific diagnosis of that person’s immunodeficiency disease? BASE: Those who are no
longer receiving treatment because it is no longer prescribed for them (net of cases) N=27
Q26b. Why is the patient no longer being treated with immunogloblin?
Figure 19
22
TREATMENT EXPERIENCES AND PREFERENCES
Two out of five (42%) IVIG users reported that they usually received their
infusion at home. Of those, 7% were able to self-infuse, while the other 35% had a nurse
perform the infusion. Twenty-six percent of IVIG users usually got their infusion at a
hospital outpatient department (21%), or at a hospital clinic (5%). Most of the remainder
said that they usually got their infusion in a doctor’s private office (9%) or an infusion
suite (16%). (Figure 20)
Nearly all SCIG users (93%) received their treatment at home, and were able to
self-infuse. Of the remaining SCIG users, 3% also received their treatment at home, with
a nurse performing the infusion, or at a doctor’s private office (1%). (Figure 20)
Therapy Location By Type of
IG Therapy: 2008
100%
93%
80%
IVIG
SCIG
60%
35%
40%
21%
16%
20%
7%
0%
9%
3%
1%
5%
0%
0%
4% 2%
0.4% 2% 0.4%
At home,
self-infused
At home,
Doctor's
Hospital
Hospital Infusion site
Other
nurseprivate
outpatient
clinic
infused
office
Q36. Where does the patient usually receive his/her IgG therapy? BASE: Those who are currently using
IVIG (N=716) or SCIG Therapy (N=239)
Q26a. Is he/she currently being treated with IVIG or SCIG for his/her immune deficiency disease?
Blank
Figure 20
23
TREATMENT EXPERIENCES AND PREFERENCES
Product Effectiveness, Safety and Satisfaction
The vast majority of patients with PIDD on IG therapy are satisfied with their
product’s effectiveness in controlling their immune deficiency. Nearly three out of five
patients say that their immunodeficiency is either completely controlled (13%) or well
controlled (46%) by their immunoglobulin. Another third of patients with PIDD (32%)
feel their immunodeficiency is adequately controlled by IG therapy. Only 7% of patients
with PIDD who are currently on IG therapy say that the therapy provides less than
adequate control of their immunodeficiency, while less than 1% say it is poorly
controlled (Figure 21).
Immunoglobulin Control Over
Immunodeficiency: 2008
Adequately
controlled
32%
Less than
adequately
controlled
7%
Poorly
controlled
0.4%
Well controlled
46%
Completely
controlled
13%
Blank
2%
Q47. How well does immunoglobulin control the patients immunodeficiency? BASE: Those who are
currently using IVIG or SCIG Therapy N=955
Figure 21
24
TREATMENT EXPERIENCES AND PREFERENCES
There is no significant difference between users of IVIG and SCIG in perceived
effectiveness of the product in controlling their immune deficiency. The survey finds that
13% of IVIG users and 12% of SCIG users say their treatment completely controls their
PIDD; while another 45% of IVIG users and 49% of SCIG users say their
immunodeficiency is well controlled by the treatment; and 32% of IVIG users and 29%
of SCIG users feel their condition is adequately controlled by their treatment. By
contrast, only seven percent of IVIG users and 8% of SCIG users feel that their immune
deficiency is less than adequately controlled by their treatment, while almost none
(0.04%), regardless of treatment type, think that their immunodeficiency is poorly
controlled by their treatment (Figure 22).
IgG Control Over Immunodeficiency By
Type Of Treatment: 2008
70%
60%
49%
50%
45%
40%
32%
30%
IVIG
29%
SCIG
20%
13%12%
7% 8%
10%
0.4% 0.4%
2% 1%
Poorly
controlled
Blank
0%
Completely
controlled
Well controlled
Adequately
controlled
Less than
adequately
controlled
Q47. How well does immunoglobulin control the patients immunodeficiency? BASE: Those who are currently
using IVIG (N=716) or SCIG Therapy (N=239)
Figure 22
Q26a. Is he/she currently being treated with IVIG or SCIG for his/her immune deficiency disease?
25
TREATMENT EXPERIENCES AND PREFERENCES
Many patients with PIDD have experienced side effects either during or after their
IVIG or SCIG treatment. Among IVIG users, more than three quarters (77%) reported
headaches during or after IG therapy. At least two out of five IVIG users reported
experiencing muscle aches (45%) or fever/chills (40%) during or after IG therapy in the
past 12 months. Less commonly, IVIG users also reported increased blood pressure
(16%), anxiety (15%), redness at infusion site (13%), abdominal pain (13%), swelling at
the infusion site (13%), blood pressure drops (12%) or wheezing (10%) during or after
treatment in the past 12 months. A smaller proportion of IVIG users experienced
vomiting (9%), hives (6%), aseptic meningitis (4%), hepatitis (3%) or kidney problems
(2%) during or after their IVIG in the past 12 months (Figure 23).
Side Effects From IVIG Therapy
In Past 12 months: 2008
77%
Headaches
45%
Muscle aches
40%
Fever/chills
16%
Increased blood pressure
15%
Anxiety
# of times
(mean)
7.0
12.9
6.1
9.8
7.6
Redness at infusion site
13%
7.0
Abdominal pain
13%
7.0
Swelling at infusion site
13%
6.4
12%
Blood pressure drop
10%
Wheezing
9%
Vomiting
Hives
Aseptic Meningitis
Hepatitis
Kidney problems
Other
6%
4%
7.5
5.1
7.9
5.9
3.2
3%
7.9
2%
4%
4.4
0%
20%
40%
60%
80%
Q41. During the past 12 months, has he/she experienced any of the following during or after IgG therapy?
BASE: Those who are currently using IVIG Therapy (net of cases) N=716
100%
Figure 23
Of these side effects, muscle aches were felt the most often in the past 12 months
(12.9 times on average), followed by increased blood pressure (9.8 times on average).
26
TREATMENT EXPERIENCES AND PREFERENCES
For SCIG users, the most commonly experienced symptoms during or after IG
therapy in the past 12 months was redness (78%) or swelling (70%) at the infusion site.
They were also the symptoms felt most often (37.2 and 38.2 times on average) during the
past 12 months. In addition, 54% experienced headaches, 25% experienced muscle
aches, and 21% experienced fever/chills during or after treatment in the past 12 months.
SCIG users also reported abdominal pains (15%), anxiety (12%), increased blood
pressure (8%), hives (7%), blood pressure drops (6%), vomiting (5%), wheezing (5%)
and aseptic meningitis (2%) during or after treatment. (Figure 24)
Side Effects From SCIG Therapy
In The Past 12 Months: 2008
78%
Redness at infusion site
70%
Swelling at infusion site
54%
Headaches
25%
Muscle aches
21%
Fever/chills
15%
Abdominal pain
12%
Anxiety
8%
Increased blood pressure
Hives
Blood pressure drop
7%
6%
# of times
(mean)
37.2
38.2
14.2
39.1
5.6
18.0
16.2
10.3
3.1
4.1
Vomiting
5%
8.5
Wheezing
5%
48.9
Aseptic Meningitis
Other
2%
1.7
5%
0%
20%
40%
60%
80%
Q41. During the past 12 months, has he/she experienced any of the following during or after IgG therapy?
BASE: Those who are currently using SCIG Therapy (net of cases) N=239
100%
Figure 24
Wheezing and muscle aches, though experienced by only a small percentage of
SCIG users, were experienced frequently by those who had them. On average, muscle
aches were experienced 39.1 times, and wheezing was experienced 48.9 times.
27
TREATMENT EXPERIENCES AND PREFERENCES
Just one-third of IVIG users (33%) and 28% of SCIG users report that they
tolerate all immunoglobulin products similarly. Nearly half of SCIG users (48%) and
37% of IVIG users reported that they tolerate some immunoglobulin products better than
others. Another 27% of IVIG users and 23% of SCIG users have only used one product
so they could not evaluate their tolerance of different products (Figure 25).
Tolerance Of Immunoglobulin Products
By Type Of Treatment: 2008
70%
60%
48%
50%
IVIG
SCIG
37%
40%
33%
30%
28%
27%
23%
20%
10%
3%
2%
0%
About the same
Some better than others
Only used one product
Blank
Q49. Does he/she tolerate any immunoglobulin products better than others, or are they all about the
same? BASE: Those who are currently using IVIG (N=716) or SCIG therapy (N=239)
Q26a. Is he/she currently being treated with IVIG or SCIG for his/her immune deficiency disease?
Figure 25
28
TREATMENT EXPERIENCES AND PREFERENCES
Overall, nearly all patients with PIDD being treated with IG therapy are at least
somewhat satisfied with their IG therapy product. Seventy-eight percent of respondents,
regardless of treatment type, are very satisfied with their current IG therapy product.
Another 15% of IVIG users and 14% of SCIG users are somewhat satisfied. Three
percent of patients say that they are neither satisfied nor dissatisfied with the product that
they are currently using. By comparison, only two percent of IVIG and SCIG users are
somewhat dissatisfied with their current product, while just 1% of IVIG users and 2% of
SCIG users are very dissatisfied with their current IG therapy product (Figure 26).
Satisfaction With Current IgG Product By
Type Of Treatment: 2008
90%
80%
78%78%
70%
60%
50%
IVIG
SCIG
40%
30%
20%
15% 14%
10%
3% 3%
2% 2%
1% 2%
2% 2%
Neither
Somewhat
dissatisfied
Very dissatisfied
Blank
0%
Very satisfied
Somewhat
satisfied
Q59. How satisfied is the patient with the IgG product currently being used? BASE: Those who are currently
using IVIG (N=716) or SCIG therapy (N=239)
Q26a. Is he/she currently being treated with IVIG or SCIG for his/her immune deficiency disease?
Figure 26
29
TREATMENT EXPERIENCES AND PREFERENCES
Efficacy of Treatment
To best measure the efficacy of IG therapy among patients, a series of questions
were asked to gauge the health of the patient in the twelve months prior to diagnosis and
in the most recent twelve months. The comparison in health status provides the most
compelling evidence of the efficacy of IG therapy to control PIDD, and demonstrates the
improvement in the health and well-being of the patient since treatment has begun.
Prior to diagnosis, only one-in-seven patients with PIDD who currently use IVIG
or SCIG (16%) described their health status as excellent (2%), very good (4%) or good
(10%). Indeed, one-quarter (24%) would describe their health status prior to diagnosis as
just fair. More than half of patients with PIDD described their health status as either poor
(36%) or very poor (22%) in the 12 months prior to diagnosis (Figure 27).
Comparison Of Health Status –
Prior to Diagnosis and Current
50%
40%
36%
Prior to diagnosis
Past 12 months
32%
30%
26%
24%23%
22%
20%
10%
8%
10%
2%
7%
4%
2%
2% 2%
0%
Excellent
Very good
Good
Fair
Poor
Very Poor
Q10. Would you describe his/her health in the 12 months prior to diagnosis…..? BASE: Those who are
currently using IVIG or SCIG therapy N=955
Q61. Would you describe his/her health in the past 12 months as…..? BASE: Those who are currently
using IVIG or SCIG therapy N=955
Blank
Figure 27
However, in the most recent twelve months, the same patients with PIDD are now
describing their health status in a much more positive way. Two-thirds of patients with
PIDD currently using IG therapy describe their health in the past 12 months as excellent
(8%) very good (26%) or good (32%). Just 23% would describe their recent health status
as fair. By contrast, less than one-in-ten current IgG users say their health status over the
past twelve months has been poor (7%) or very poor (2%).
30
TREATMENT EXPERIENCES AND PREFERENCES
One reason that most patients with PIDD describe their health as good or better in
the past 12 months, compared to the 12 months prior to treatment, are a lower rate of
chronic diseases. These patients reported a decrease in many chronic conditions,
including a decrease in asthma (39% prior to diagnosis, 35% recently), malabsorption
(12% prior, 9% recent), Lymphoepenia (11% prior, 8% recent), Neutropenia (5% prior,
4% recent), and Hepatitis (2% prior, 1% recent). There is also a decrease in the rate of
other chronic diseases not described in the survey (57% prior, 40% recently). By
contrast, there has been no change in the rate of digestive diseases (25%) or
Cancer/Leukemia (2%), while there has been a slight increase in some age-related
conditions including arthritis (22% prior to diagnosis, 31% recently), COPD (10% prior,
12% recently), and neurological disease (6% prior, 8% recently).
Overall, the
percentage of patients with PIDD that reported no chronic conditions nearly doubled,
from 14% in the 12 months before diagnosis to 26% in the past 12 months (Figure 28).
Comparison Of Chronic Conditions:
Prior to Diagnosis and Current
70%
60%
Prior to diagnosis
57%
Past 12 months
50%
40%
39%
35%
40%
31%
25%25%
30%
26%
22%
20%
12% 11%
12%
9%
8% 10%
10%
14%
8%
6%
5% 4% 2%2% 2%
1%
on
e
N
th
er
O
eu
C
O
ro
PD
lo
gi
ca
lD
ise
as
e
N
eu
tr
op
C
an
en
ce
ia
r/L
eu
ke
m
ia
H
ep
at
iti
s
al
ab
so
rp
tio
Ly
n
m
ph
op
en
ia
rt
hr
iti
s
M
A
N
D
ig
es
tiv
e
A
sth
m
a
Di
se
as
es
0%
Q11. During the 12 months prior to diagnosis, did he/she have__________? BASE: Those who are currently
using IVIG or SCIG therapy (net of cases) N=955
Q62. During the past 12 months has he/she have______ ____? BASE: Those who are currently using IVIG or Figure 28
SCIG therapy (net of cases) N=955
31
TREATMENT EXPERIENCES AND PREFERENCES
There is also a dramatic decrease in the rate of acute conditions before diagnosis
compared to the past 12 months among patients with PIDD currently on IG therapy. IG
therapy users are much less likely to suffer from pneumonia (57% prior to diagnosis,
15% recently), ear infections (46% prior, 18% recently), and bronchitis (69% prior, 37%
recently). There is also a significant decrease in sinusitis (80% experienced prior to
diagnosis, 70% in past 12 months). The proportion of patients who have experienced
diarrhea, Candida, skin infections, eye infections, abscess and sepsis have also decreased,
but not quite as dramatically. However, the average number of incidents of these
infections has dropped even more dramatically. The proportion of patients with PIDD
being treated with IG therapy that report no acute conditions increased from 2% prior to
diagnosis to 10% in the past 12 months (Figure 29).
Comparison Of Acute Conditions:
Prior to Diagnosis and Current
Sinusitis
Bronchitis
70%
69%
37%
Pneumonia
57%
15%
Ear infections
80%
46%
18%
38%
33%
Diarrhea
Candida
16%
Skin infection
Eye infection
Abscess
5%
25%
24%
18%
23%
13%
10%
4% 9%
Sepsis
2%
None
Other
0%
Prior to Diagnosis
Last 12 months
10%
22%
18%
20%
40%
60%
80%
Prior to
diagnosis*
Past 12
months*
4.46
3.05
2.85
0.80
1.26
0.26
1.76
0.55
5.01
4.37
0.75
0.49
0.55
0.39
0.47
0.28
0.20
0.09
0.26
0.07
* Mean number of mentions
100%
Q12. Did the patient experience any of the following infections during the 12 months prior to diagnosis? BASE: Those who are
currently using IVIG or SCIG therapy (net of cases) N=955
Q63. Did he/she experience any of the following infections during the past 12 months? BASE: Those who are currently using Figure 29
IVIG or SCIG therapy (net of cases) N=955
32
TREATMENT EXPERIENCES AND PREFERENCES
As a result of reduced chronic and acute conditions, current IgG patients have
seen a significant decrease in their physical limitations between the time before they were
diagnosed and the past twelve months. In the year prior to diagnosis, a majority of IgG
users reported moderate (36%) or severe (22%) limitations in their work, play or normal
physical activity as a result of their health. One-quarter (25%) reported only slight
limitations, and only 13% reported no limitations. In the past year, however, IgG users
are far less limited in their activities. Nearly two-thirds of current users reported no
limitation (30%) or slight limitations (34%) in their lives. Just 23% reported moderate
limitations, and only 12% reported severe limitations (Figure 30).
Comparison Of Limitations:
Prior to Diagnosis and Current
50%
40%
34%
Prior to diagnosis
36%
Past 12 months
30%
30%
25%
23%
22%
20%
13%
12%
10%
3% 2%
0%
No limitation
Slight limitation
Moderate limitation
Severe limitation
Blank
Q13. During the 12 months prior to diagnosis, how much was he/she limited in work, play or normal physical
activity as a result of his/her health? BASE: Those who are currently using IVIG or SCIG therapy N=955
Q64. During the past 12 months, how much has he/she been limited in work, play, or normal physical
Figure 30
activity as a result of his/her health? BASE: Those who are currently using IVIG or SCIG therapy N=955
33
TREATMENT EXPERIENCES AND PREFERENCES
As a result of better health, there was a decline in the number of days the patient
missed from school or work. The average number of days missed by a current IgG user
in the twelve months prior to diagnosis was 36.3 days. In the past twelve months, that
number dropped to 30.4 days. More dramatically, the median number of missed days
from work or school as been cut in half from the 12 months prior to diagnosis (10.0) and
the past 12 months (5.0) (Figure 31).
Comparison Of Missed Days:
Prior to Diagnosis and Current
40%
30%
25%
Mean
Median
Prior to diagnosis
36.3
10.0
Past 12 months
30.4
5.0
25%
Prior to diagnosis
20%
Past 12 months
18%
10%
10%
11%
12%11%
12%
16%
15%
11%
9%
7%
3%3%
4%4%
3%
0%
None
1 week or 8 days - 2 15 days - 1 32-100
101-200 201 days+ Infant/NA
Blank
less
weeks
month
days
days
Q18. Not counting hospitalizations, how many days was he/she too sick to work, go to school or perform usual
activities in the BASE: Those who are currently using IVIG or SCIG therapy N=955
Q69. Not counting hospitalizations, how many days was he/she too sick to work, go to school or perform usual
Figure 31
activities in the past 12 months? BASE: Those who are currently using IVIG or SCIG therapy N=955
One half of respondents missed either no school or work (25%) or less than one
week (25%) in the past 12 months. In the 12 months prior to diagnosis, only 10% did not
miss any work or school, and 11% missed less than one week.
34
TREATMENT EXPERIENCES AND PREFERENCES
Current IG therapy users have also spent less time in the hospital in the past
twelve months. A majority of IG users (53%) were hospitalized at least once in the
twelve months prior to diagnosis. Sixteen percent were hospitalized once, 13% were
hospitalized twice, 16% were hospitalized three or more times and an additional 8%
couldn’t recall the number of times they were hospitalized. Only 47% reported no
hospitalizations (Figure 32). In the most recent twelve months, over three-quarters of IG
users (76%) were not hospitalized at all.
Comparison Of Times Hospitalized:
Prior to Diagnosis and Current
100%
76%
80%
Prior to diagnosis
Past 12 months
60%
47%
40%
16%
20%
12%
16%
13%
8% 6%
6%
0.2%
0%
None
Once
Twice
Three or more
Don’t know
times
number of times
Q14b. How many times was he/she hospitalized in the 12 months prior to diagnosis? BASE: Those who are
currently using IVIG or SCIG therapy N=955
Q65b. How many times was he/she hospitalized in the past 12 months? BASE: Those who are currently
Figure 32
using IVIG or SCIG therapy N=955
35
TREATMENT EXPERIENCES AND PREFERENCES
On average, patients on IG therapy were hospitalized 1.22 times in the twelve
months prior to diagnosis. During the most recent year, these same patients were
hospitalized an average of 0.50 times. In the 12 months prior to diagnosis, they spent an
average of 5.32 nights in the hospital, compared to just 2.23 nights in the hospital in the
past 12 months. In the year before diagnosis, these patients spent an average of 0.61
nights in the ICU compared to 0.33 nights in the ICU in the past 12 months (Figure 33).
Hence, the improved health found in patients with PIDD after treatment with IG therapy
has a significant impact on health care costs associated with hospitalization.
Comparison Of Hospitalization:
Prior to Diagnosis and Current
10
8
Mean number of
times/nights
6
Prior to diagnosis
Past 12 months
5.32
4
2.23
2
1.22
0.61
0.50
0.33
0
Number of times hospitalized
Number of nights hospitalized
Number of nights in ICU
Q14b/c/d. How many times/nights was he/she hospitalized (in an ICU) during the 12 months prior to diagnosis? BASE:
Those who are currently using IVIG or SCIG therapy N=955
Q65b/c/d. How many times/nights was he/she hospitalized (in an ICU) in the past 12 months? BASE: Those who are
Figure 33
currently using IVIG or SCIG therapy N=955
There was also a decrease in the number of doctor’s visits and surgical procedures
for IgG users. Prior to diagnosis, IG users had an average of 8.26 visits to their primary
care physician and 5.20 visits to specialists. In the past 12 months, IG users made only
4.82 visits to their primary care physician and 6.79 visits to specialists (Figure 34).
Furthermore, the number of operations that IG users had to have also decreased with IG
therapy use. Prior to diagnosis, IG users underwent an average of 0.44 in-patient and
0.59 out-patient procedures. In the past 12 months, those averages decreased to 0.14 inpatient procedures and 0.29 out-patient procedures (Figure 35).
36
TREATMENT EXPERIENCES AND PREFERENCES
Comparison Of Doctor Visits:
Prior to Diagnosis and Current
15
Mean number of doctor visits
Prior to diagnosis
Past 12 months
10
8.26
6.79
5.20
4.82
5
0
Primary care visits
Specialist visits
Q17. Not counting hospitalizations, about how many doctor visits did the patient make during the 12
months prior to diagnosis? BASE: Those who are currently using IVIG or SCIG therapy N=955
Q68. Not counting hospitalizations, about how many doctor visits did the patient make during the past 12
months? BASE: Those who are currently using IVIG or SCIG therapy N=955
Figure 34
Comparison Of Surgical Procedures:
Prior to Diagnosis and Current
0.9
Mean number of Procedures
0.8
0.7
0.6
0.5
Prior to diagnosis
Past 12 months
0.59
0.44
0.4
0.29
0.3
0.2
0.14
0.1
0.0
In-Patient
Out-Patient
Q15. How many operations, if any, did he/she have in the 12 months prior to diagnosis? BASE: Those
who are currently using IVIG or SCIG therapy N=955
Q66. How many operations, if any, did he/she have in the past 12 months? BASE: Those who are currently
using IVIG or SCIG therapy N=955
Figure 35
37
TREATMENT EXPERIENCES AND PREFERENCES
Unfortunately, many IG users still require additional medical treatments, and
some of these treatments are at a higher rate than prior to diagnosis. The use of
prescription drugs among IG users has increased from an average of 90 days per year in
the year prior to diagnosis to 171 days in the past 12 months. Antibiotic use increased
from an average of 75 days to 78 days per year, respiratory therapy use increased from an
average of 28 days to 35 days per year and oxygen use increased from an average of 4
days to 14 days in the recent twelve months. There is also an increase in the average use
of a physical therapist (2 days prior, 3 days recently) and visiting nurses (2 days prior, 4
days recently). (Figure 36)
Comparison Of Medical Treatments:
Prior to Diagnosis and Current
200
Mean number of days
171
150
100
Prior to diagnosis
90
Last 12 months
75 78
50
28
35
4
14
2
3
2
4
0
Other
Antibiotics
Respiratory
Oxygen
Physical
prescription
therapy
therapist
drugs
Q16. In the 12 months prior to diagnosis, approximately how many days did he/she use__________?
BASE: Those who are currently using IVIG or SCIG therapy N=955
Visiting Nurse
Q67. Approximately how many days in In the past 12 months did he/she use__________? BASE: Those
who are currently using IVIG or SCIG therapy N=955
Figure 36
It should be noted that many of these treatments (e.g., respiratory therapy,
physical therapists, and oxygen) represent treatments for functional disabilities incurred
as a result of infections prior to treatment.
38
TREATMENT EXPERIENCES AND PREFERENCES
Availability
Few current users reported that they had difficulties in getting their regular IG
therapy in the past few years. Two-thirds (66% of IVIG users and 69% of SCIG users)
have not had a problem getting their regular therapy in the past three years. Among the
remaining users, only 2% of IVIG and SCIG users reported a problem in the past month,
7% of IVIG users and 3% of SCIG users in the past 6 months, 8% of IVIG users and 7%
of SCIG users in the past year. However, 17% of IVIG and 18% of SCIG patients
reported having difficulty in getting IG therapy 2-3 years ago (Figure 37).
Most Recent Problem Getting Regular
Infusion By Type Of Treatment: 2008
100%
80%
66%
60%
IVIG
69%
SCIG
40%
17% 18%
20%
7%
2% 2%
8%
3%
7%
0%
Past month
Past six months
Past year
2-3 years ago
No problem in past
3 years
Q54. When was the most recent time the patient had a problem getting his/her regular infusion? BASE: Those
who are currently using IVIG (N=716) or SCIG (N=293)
Figure 37
Q26a. Is he/she currently being treated with IVIG or SCIG for his/her immune deficiency disease?
39
TREATMENT EXPERIENCES AND PREFERENCES
Among respondents that had experienced a problem getting their regular IG
therapy in the past three years, nearly half (49%) reported delayed infusions as the
problem. Another 31% had problems with reimbursements, 28% had no product
available, 20% had to deal with increased intervals between IG therapies, or cancelled
therapies. In addition, there were problems of change in infusion site (17%), a switch to
a less preferred product (15%) or less tolerated product (9%), or a reduced dosage of
infusions (Figure 38).
Recent Problems Experienced
Getting Infusion
Delayed infusions
49%
Reimbursement problems
31%
No product available
28%
Increased interval between infusions
20%
Cancelled infusions
20%
Change in site of infusion
17%
Switched to less preferred product
15%
Switched to less tolerated product
9%
Reduced dosage of infusions
8%
Other
23%
3%
None
0%
10%
20%
30%
40%
50%
Q55. In the past 12 months, which of the following problems, if any, has the patient experienced? BASE:
Those who are currently using IVIG or SCIG and have had a problem in getting regular infusion in the
past three years (Net of cases) N=143
60%
Figure 38
40
TREATMENT EXPERIENCES AND PREFERENCES
Although relatively few patients experienced current difficulties with the supply
of IG therapy, the majority of IG therapy users remain concerned about the reliability of
the IG supply. Indeed, 59% of immune deficient patients using IVIG and 55% using
SCIG said reliability of IG supply is a major concern for them. Another 21% of IVIG
and 24% of SCIG users said that reliability of supply is a moderate concern. Only 10%
of IVIG and 13% of SCIG patients said that reliability of the IG therapy supply is only a
minor concern, while 8% said that it is not a concern for them (Figure 39).
Concern Over Reliability Of Supply By
Type Of Treatment: 2008
70%
60%
59%
55%
50%
40%
IVIG
30%
21%
SCIG
24%
20%
10%
13%
10%
8% 8%
1% 0%
0%
Major concern
Moderate concern
Minor concern
Not really a
concern
Blank
Q52. How much of a concern to you is the reliability of the immunoglobulin supply? BASE: Those who are
currently using IVIG (N=716) or SCIG therapy (N=239)
Figure 39
Q26a. Is he/she currently being treated with IVIG or SCIG for his/her immune deficiency disease?
Since the patients with primary immunodeficiency diseases have experienced
dramatic improvements in their health since treatment with IG therapy, there is
widespread satisfaction with the treatment. Not surprisingly, patients are concerned with
the continued availability of these products and their access to them.
41
TREATMENT EXPERIENCES AND PREFERENCES
Conclusions
The results of this survey continue to support the tremendous efficacy of
immunoglobulin replacement therapy for patients with primary immunodeficiency
diseases who are antibody deficient. Once these patients begin receiving IG therapy on a
regular basis their health status improves markedly, their activity limitations drop
significantly and their quality of life improves dramatically. The differences in health,
activity limitation, and infections before diagnosis and after the initiation of replacement
therapy are considerable. Immunoglobulin replacement therapy is life saving.
Regrettably, there continues to be a long lag time between symptom onset of
PIDD and the diagnosis and initiation of IG therapy in this patient population. As a
result, many patients suffer debilitating, permanent functional impairments. Despite the
effectiveness of IG therapy in dramatically improving patients’ ability to avoid repeated,
unusual and severe infections, IG therapy cannot reverse the permanent organ damage
done by such infections prior to diagnosis and the initiation of IG therapy.
Given the effectiveness of IG therapy, it’s imperative that patients receive proper
diagnosis and have unimpeded access to this life-sustaining therapy. The early diagnosis
of PIDD, and subsequent treatment with IG therapy is critical if antibody deficient
patients with PIDD are to avoid many of the long-term debilitating conditions that place
an increased burden not only on the patients, but on the U.S. health care system as well.
42