ArticlePDF Available

How do patients and physicians perceive immune thrombocytopenia (ITP) as a disease? Results from Indian analysis of ITP World Impact Survey (I-WISh)

Authors:
  • Vivekananda Institute of Medical Sciences Kolkata

Abstract and Figures

Purpose Immune thrombocytopenia (ITP) is primarily considered a bleeding disorder; its impact on patients’ health-related quality of life (HRQoL) is under-recognized. We aimed to assess how aligned patient and physician perceptions are regarding ITP-associated symptoms, HRQoL, and disease management in India. Methods Patients and physicians (hematologists/hemato-oncologists) from India who participated in the global ITP World Impact Survey (I-WISh) were included in this subgroup analysis (survey). Physicians were recruited via a local, third party recruiter in India. In addition to completing a survey themselves, physicians were asked to invite consulting patients on a consecutive basis to complete a survey. All surveys were completely independently by the respondents online in English. The respondents took 30 min to complete the questionnaire. Patients also completed the newly developed ITP Life Quality Index (ILQI) that included 10 questions on the impact of ITP on the following: work or studies, time taken off work or education, ability to concentrate, social life, sex life, energy levels, ability to undertake daily tasks, ability to provide support, hobbies, and capacity to exercise. Results A total of 65 patients and 21 physicians were included in this study. Average disease duration from diagnosis-to-survey-completion was 5.3 years. The most severe symptoms reported by patients at diagnosis were menorrhagia (15 of 19 patients [79%]), anxiety surrounding unstable platelet counts (17 of 28 patients [61%]), and fatigue (27 of 46 patients [59%]); these were also the key symptoms they wanted to be resolved. In contrast, physicians perceived petechiae (19 of 21 patients [90%]), bleeding-from-gums (8 of 21 patients [86%]), and purpura (16 of 21 patients [76%]) as the most common symptoms. While the important treatment goals for patients were healthy blood counts (42 of 65 patients [65%]), improved QoL (35 of 65 patients [54%]), and prevention of worsening of ITP (33 of 65 patients [51%]), physicians’ goals were reduction in spontaneous bleeding (17 of 21 physicians [81%]), better QoL (14 of 21 physicians [67%]), and symptom improvement (9 of 21 physicians [43%]). More than half the patients reported that ITP affected their work life/studies, social life, and energy levels, thereby negatively impacting their QoL. Patients were almost entirely dependent on family and friends for support. Conclusions This survey highlights the substantial discrepancy in patients’ and physicians’ perceptions regarding ITP-associated symptoms and treatment goals in India. Based on the identified gaps, educating physicians on aspects of ITP beyond bleeding, and highlighting patients’ under-recognized symptoms/needs through support-systems should be prioritized in the future.
Content may be subject to copyright.
Chakrabartietal.
Journal of Patient-Reported Outcomes (2022) 6:24
https://doi.org/10.1186/s41687-022-00429-y
RESEARCH
How dopatients andphysicians perceive
immune thrombocytopenia (ITP) asadisease?
Results fromIndian analysis ofITP World Impact
Survey (I-WISh)
Prantar Chakrabarti1, Biju George2, Chandrakala Shanmukhaiah3, Lalit Mohan Sharma4, Shashank Udupi5 and
Waleed Ghanima6*
Abstract
Purpose: Immune thrombocytopenia (ITP) is primarily considered a bleeding disorder; its impact on patients’ health-
related quality of life (HRQoL) is under-recognized. We aimed to assess how aligned patient and physician perceptions
are regarding ITP-associated symptoms, HRQoL, and disease management in India.
Methods: Patients and physicians (hematologists/hemato-oncologists) from India who participated in the global ITP
World Impact Survey (I-WISh) were included in this subgroup analysis (survey). Physicians were recruited via a local,
third party recruiter in India. In addition to completing a survey themselves, physicians were asked to invite consulting
patients on a consecutive basis to complete a survey. All surveys were completely independently by the respondents
online in English. The respondents took 30 min to complete the questionnaire. Patients also completed the newly
developed ITP Life Quality Index (ILQI) that included 10 questions on the impact of ITP on the following: work or stud-
ies, time taken off work or education, ability to concentrate, social life, sex life, energy levels, ability to undertake daily
tasks, ability to provide support, hobbies, and capacity to exercise.
Results: A total of 65 patients and 21 physicians were included in this study. Average disease duration from diagno-
sis-to-survey-completion was 5.3 years. The most severe symptoms reported by patients at diagnosis were menor-
rhagia (15 of 19 patients [79%]), anxiety surrounding unstable platelet counts (17 of 28 patients [61%]), and fatigue
(27 of 46 patients [59%]); these were also the key symptoms they wanted to be resolved. In contrast, physicians
perceived petechiae (19 of 21 patients [90%]), bleeding-from-gums (8 of 21 patients [86%]), and purpura (16 of 21
patients [76%]) as the most common symptoms. While the important treatment goals for patients were healthy blood
counts (42 of 65 patients [65%]), improved QoL (35 of 65 patients [54%]), and prevention of worsening of ITP (33 of 65
patients [51%]), physicians’ goals were reduction in spontaneous bleeding (17 of 21 physicians [81%]), better QoL (14
of 21 physicians [67%]), and symptom improvement (9 of 21 physicians [43%]). More than half the patients reported
that ITP affected their work life/studies, social life, and energy levels, thereby negatively impacting their QoL. Patients
were almost entirely dependent on family and friends for support.
Conclusions: This survey highlights the substantial discrepancy in patients’ and physicians’ perceptions regarding
ITP-associated symptoms and treatment goals in India. Based on the identified gaps, educating physicians on aspects
© The Author(s) 2022. Open Access This article is licensed under a Creative Commons Attribution 4.0 International License, which
permits use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit to the
original author(s) and the source, provide a link to the Creative Commons licence, and indicate if changes were made. The images or
other third party material in this article are included in the article’s Creative Commons licence, unless indicated otherwise in a credit line
to the material. If material is not included in the article’s Creative Commons licence and your intended use is not permitted by statutory
regulation or exceeds the permitted use, you will need to obtain permission directly from the copyright holder. To view a copy of this
licence, visit http:// creat iveco mmons. org/ licen ses/ by/4. 0/.
Open Access
Journal of Patient-
Reported Outcomes
*Correspondence: wghanima@gmail.com
6 Departments of Research and Hemato-Oncology, Østfold Hospital,
Østfold Hospital, PB 300, 1714 Grålum, Norway
Full list of author information is available at the end of the article
Page 2 of 14
Chakrabartietal. Journal of Patient-Reported Outcomes (2022) 6:24
Background
Immune thrombocytopenia (ITP) is an acquired autoan-
tibody-mediated bleeding disorder characterized by
both accelerated platelet destruction and impaired plate-
let production, which an estimated incidence in adults
between 1.6 and 3.9 per 100,000 person-years based
on the platelet count threshold used [1]. ITP requires
lifelong treatment in a substantial proportion of adult
patients, thereby negatively impacting the patient quality
of life (QoL) [1, 2]. Improvement in health-related QoL
(HRQoL) parameters has been identified as an important
treatment objective in the updated ITP guidelines (ASH,
ICR 2019) [3, 4]. However, in resource-limited countries,
such as India, where physicians have a higher patient
burden and can afford only limited in-clinic time [57],
assessment and treatment of HRQoL parameters is chal-
lenging. Physicians often tend to underestimate or ignore
HRQoL parameters in routine clinical practice, as the
major treatment goal for ITP is to treat or prevent bleed-
ing [2].
Recently, the ITP World Impact Survey (I-WISh) was
conducted to discern how ITP and associated treatments
affect patient lives and to evaluate how aligned patient
and physician perceptions are regarding symptoms,
HRQoL, and disease management [8, 9], and we have
conducted an analysis of data from the Indian patient
subgroup included in the I-WISh study. With ITP being
one of the most common non-infectious causes of throm-
bocytopenia in India [10, 11], the major objectives of this
study were to understand the challenges in the diagnostic
journey of patients with ITP in India; patient and physi-
cian perceptions of disease and symptoms; impact of ITP
on patient QoL, daily activities, and work; and existing
support systems for ITP and its management.
Methods
Survey participants andstudy conduct
e I-WISh India-specific analysis is based on data col-
lected as part of I-WISh 1.0, a cross-sectional survey of
adult patients (age 18years) with ITP and hematolo-
gists or hemato-oncologists who treat patients with ITP.
e global I-WISh study was conducted in 13 countries
(Canada, China, Colombia, Egypt, France, Germany,
India, Italy, Japan, Spain, Turkey, the United Kingdom,
and the United States).
Patient surveys were sent via mass email to patient sup-
port networks and physicians who were requested to dis-
seminate the surveys to patients. A steering committee
of disease experts and patient advocates led the design
of the survey materials and endorsed them prior to ini-
tiation of data collection. Physician surveys were emailed
by local fieldwork agencies. Physicians were recruited via
a local, third party recruiter in India. Physicians invited
patients to complete the survey following a routine con-
sultation for their ITP. In addition to completing a survey
themselves, physicians were asked to invite consulting
patients on a consecutive basis to complete a survey. All
surveys were completely independently by the respond-
ents online in English. As the surveys were online, it was
not possible for any respondent to omit to answer any
question. However, where deemed appropriate, respond-
ents were allowed to select ‘Not applicable’ or ‘Don’t
know’ for certain questions. In these cases, the ‘Don’t
know’ or ‘Not applicable’ responses were removed from
the individual analyses, but these patients were otherwise
eligible for inclusion in all other analyses. Overall, the
respondents took 30min to complete the questionnaire.
Fully deidentified respondent information was collated
and aggregated by local fieldwork partners such that the
surveys were unlinked and anonymized. Surveys and
details of the survey methods, including how patients
and physicians were identified, have been outlined in the
supplementary material and published previously [8, 9].
To understand the level of agreement that the respond-
ents had with a statement in the survey, a Likert scale of
1–7 was used; for assessment of symptoms, a score 5
on the Likert scale implied that the symptom in question
was considered to be “severe” by the patient. Patients also
completed the newly developed ITP Life Quality Index
(ILQI) that included 10 questions on the impact of ITP
on the following: work or studies, time taken off work
or education, ability to concentrate, social life, sex life,
energy levels, ability to undertake daily tasks, ability to
provide support, hobbies, and capacity to exercise [12];
additional details can be accessed from the global I-WISh
study [10].
All methods were carried out in compliance with
EphMRA guidelines and in full accordance with the US
HIPAA 1996. Separate protocols were submitted for
the patient and physician surveys (patient survey refer-
ence number: 02018/1056; physician survey reference
of ITP beyond bleeding, and highlighting patients’ under-recognized symptoms/needs through support-systems
should be prioritized in the future.
Keywords: Disease management, Health-related quality of life (HRQoL), Immune thrombocytopenia (ITP), India, ITP
World Impact Survey (I-WISh), ITP symptoms
Page 3 of 14
Chakrabartietal. Journal of Patient-Reported Outcomes (2022) 6:24
number: 02018/1049). Survey materials and protocol
were reviewed and approved by the Western Institutional
Review Board. Patients and physicians were given an
overview of the study and ethical approval details; those
who wished to participate had to provide consent via a
tick/check box before initiating.
Statistical analyses
It being a descriptive, exploratory study, it did not include
sample size calculations. e local recruiter conducted a
feasibility assessment prior to launching data collection
to confirm the achievable sample size for this study.
Patient and physician survey data were analyzed sepa-
rately using descriptive statistics. Analyses were descrip-
tive, and no formal hypothesis was tested. Missing data
were not imputed. Analyses were conducted using
STATA statistical software version 15.1 (StataCorp, Col-
lege Station, TX).
Results
Demographic characteristics andthediagnostic journey
ofpatients withITP
Overall, 21 physicians and 65 patients completed the
survey questionnaire from March 09, 2018 and May
02, 2018. Patients were recruited by either experienced
physicians treating ITP (64 of 65 patients [98%]) or
patient association groups (1 of 65 patients [2%]). Accu-
rate estimates on the number of individuals who were
approached for participation in the survey could not be
obtained. All respondents who participated in the survey
questionnaire provided their demographic information,
along with details of their diagnostic processes (Table1).
Patients
e mean (standard deviation [SD]) age of the patients
was 33 (12.62) years, with 39 of 65 patients (60%) being
male. e symptom burden was moderate to high in 24 of
57 patients (42.1%), of whom 17 patients (71%) reported
a poor health score ( 4 on the Likert scale). Patients met
an average of 5 healthcare professionals (HCPs; including
primary care physicians, nurses, emergency care doctors,
dentists, and others) before an accurate diagnosis of ITP,
which was confirmed by physicians specialized in the
management of ITP in 56 of 65 patients (86%). Overall,
21 of 65 patients (32%) expressed a delay in ITP diagno-
sis, thereby leading to severe anxiety ( 5 on the Likert
scale) in 8 of 21 patients (38%) (Table1).
Physicians
All physicians included in the survey were either hema-
tologists (n = 13) or hemato-oncologists (n = 8). More
than half of the physicians who participated in the sur-
vey (12 of 21 physicians [57%]) practiced in a private
setting. Of the average caseload, about 87 of 625 (13.9%)
were patients with ITP. ITP was rated as a “somewhat
less important” condition by 14 of 21 physicians (67%).
Nearly one-fourth (5 of 21 physicians [24%]) of the physi-
cians perceived that 26%-50% of patients were misdiag-
nosed (Table1).
Patient andphysician perception ofITP symptoms
andseverity
Patient perspective
e most commonly reported symptoms at diagno-
sis were heavy menstrual bleeding (19 of 26 patients
[73%]), fatigue (46 of 65 patients [71%]), petechiae (39 of
65 patients [60%]), hematoma (30 of 65 patients [46%]),
and anxiety surrounding unstable platelet counts (28 of
65 patients [43%]). e mean (SD) duration of the disease
from diagnosis to survey completion was 5.3 (6.77) years.
At survey completion, the most commonly reported
symptoms were fatigue (35 of 65 patients [54%]), heavy
menstrual bleeding (10 of 26 patients [38%]), anxiety
surrounding unstable platelet counts (23 of 65 patients
[35%]), petechiae (19 of 65 patients [29%]), and hema-
toma (11 of 65 patients [17%]) (Fig. 1A). Menorrhagia
(15 of 19 patients [79%]), anxiety surrounding unstable
platelet counts (17 of 28 patients [61%]), and fatigue (27
of 46 patients [59%]) were the most commonly reported
severe symptoms at diagnosis (considering symptoms
reported by at least 15 patients) (Fig.1A). e key symp-
toms that patients wanted to be resolved included fatigue
(27 of 65 patients [42%]), heavy menstrual bleeding (10
of 26 patients [38%]), and anxiety surrounding unstable
platelet counts (21 of 65 patients [32%]) (Additional file1:
Figure S1).
Physician perspective
e most common signs and symptoms reported by phy-
sicians, based on the inputs received from their patients,
were similar at diagnosis vs survey completion; these
included petechiae (19 of 21 physicians [90%] vs 19 of
21 physicians [90%]), bleeding from gums (18 of 21 phy-
sicians [86%] vs 18 of 21 physicians [86%]), purpura (16
of 21 physicians [76%] vs 18 of 21 physicians [86%]),
epistaxis (14 of 21 physicians [67%] vs 13 of 21 physicians
[62%]), and heavy menstrual bleed (11 of 21 physicians
[52%] vs 12 of 21 physicians [57%]) (Fig.1B). According
to physicians, hematuria, melena, or rectal bleed (17 of
21 physicians [81%]); profuse bleeding during surgery (16
of 21 physicians [76%]); menorrhagia (14 of 21 physicians
[67%]); anxiety surrounding unstable platelet counts (12
of 21 physicians [57%]); and hematoma (12 of 21 phy-
sicians [57%]) could have a major negative impact on
patient HRQoL (scored 5 on the Likert scale). Accord-
ing to physicians, about 37% patients experienced fatigue,
Page 4 of 14
Chakrabartietal. Journal of Patient-Reported Outcomes (2022) 6:24
Table 1 Patient/physician demographic characteristics and patient diagnostic pathways
Patients
N = 65
Mean age, years 33
Male, n (%) 39 (60%)
Female, n (%) 26 (40%)
Current health state (Score: 1, very poor health; 7, excellent health)
4 26 (40%)
Splenectomized, n (%) 6/64 (9.3%)
Diagnosis
Median (IQR) time from symptom presentation to diagnosis, months 1.5 (0.5–5.7)
Symptom presentation to first consultation, months 0.7 (0.1–3.0)
First consultation to diagnosis 0.5 (0.2–1.0)
Patients with a median time from initial presentation to ITP diagnosis > 6 months, n (%) 9/55 (16%)
Patients in whom diagnosis of ITP confirmed as a result of another health condition, n (%) 2 (3%)
Delay in diagnosis, n (%) 21 (32%)
Awaiting additional test results 8 (38%)
Specialist reference 7 (33%)
Patient support following diagnosis, n (%)
Family/friends 59 (91%)
Physicians 50 (77%)
Nurses 24 (37%)
Patients who needed more support during the diagnosis process, n (%) 27 (42%)
Physicians 20 (74%)
Family/friends 13 (48%)
Patient support groups 11 (41%)
Physicians
N = 21
Average total patient caseload 625
Number of ITP patients seen in the last 12 months 81
Practice setting
Private care 12 (57%)
Specialty cancer center 5 (24%)
University teaching hospital and community teaching hospital 4 (19%)
Year of qualification
Before 1981 1 (5%)
1981–1993 3 (14%)
1994–2003 5 (24%)
2004–2014 10 (48%)
After 2014 2 (10%)
Diagnosis
Median (IQR) time from symptom presentation to diagnosis, months 0.25 (0.25–0.62)
Primary ITP 70%
Secondary ITP 30%
Reasons for delay in diagnosis
Specialist reference 13 (62%)
Exclusion of other potential causes 12 (57%)
Causes of secondary ITP
Systemic lupus erythematosus 11 (52%)
Drug-induced thrombocytopenia 11 (52%)
Hepatitis C virus 10 (48%)
Page 5 of 14
Chakrabartietal. Journal of Patient-Reported Outcomes (2022) 6:24
and the severity was considered as low ( 4 on the Lik-
ert scale) in most patients (17 of 21 physicians [81%]).
Fatigue was considered to be very severe by 12 of 21
physicians (57%), 10 of 21 physicians (48%), and 8 of 21
physicians (38%) when platelet counts were <10 × 109/L,
10-29 × 109/L, and 30-39 × 109/L, respectively (Addi-
tional file2: Figure S2). Overall, fatigue was considered as
a major concern by only 7 of 21 physicians (33%).
Impact ofITP onQoL
Patient
Based on the ILQI scores, the parameters that signifi-
cantly had a negative impact on patient QoL very often
(more than half of the time) were work life/studies (19
of 50 patients [38%]), absence of work/education (16 of
48 patients [33%]), and energy levels (19 of 65 patients
[29%]) (Fig.2A).
e overall impact on emotional well-being was
scored 5 on the Likert scale by 25 of 65 patients (38%),
and the top 4 reported reasons with a severe impact were
anxiety surrounding unstable platelet counts (38 of 65
patients [58%]), importance of stable platelet counts (38
of 65 patients [58%]), fluctuation in platelet counts for no
apparent reason (34 of 65 patients [52%]), and frustra-
tion with ITP symptoms (31 of 64 patients [48%]) (Fig.3).
Overall, 60 of 65 patients (92%) did not receive any pro-
fessional support, of whom 20 patients (33%) expressed a
desire for additional support (data not shown).
ITP adversely affected the work and financial situation
of patients, with 10 of 38 patients (26%) reducing their
work hours and 9 of 36 patients (25%) seriously consid-
ering a reduction in their work hours. Patients reported
an average of 11.1h of missed work per week due to the
impact of ITP (Fig.4). e total monthly out-of-pocket
expense for a patient with ITP was $211 (~ 16,000 INR),
with medicines accounting for more than 60% of this
expense ($132 [~ 9600 INR]). Patients also spent an aver-
age of 6.1h/month traveling for their appointments.
Overall, 39 of 65 patients (60%) expressed the need
for support (either ‘rarely’, ‘sometimes’, or ‘often’) for an
average of 33.7h/week; homemaking (27 of 39 patients
[69%]), transportation (26 of 39 patients [67%]), health-
care (25 of 39 patients [64%]), and management of
finances (20 of 39 patients [51%]), were the primary rea-
sons for which support was requested. e key support
providers were parents (19 of 39 patients [49%]) and
spouses (9 of 39 patients [23%]) (data not shown).
Physician
Physicians felt that anxiety about platelet counts and
frustrations around having a long-term, rare disease had
a severe adverse impact on most of the patients (~ 90%).
Daily activities were severely impacted in 4 of 20 patients
(21%) and 5 of 20 physicians (25%) felt that ITP had nega-
tively impacted patients’ relationship with their spouses.
Overall, interference of ITP in the level of patients’ physi-
cal activity was reported as severe by 6 of 20 physicians
(30%), and 18 of 20 physicians (90%) felt that ITP greatly
impacted patients’ ability to play contact sports or sports
with a chance of bleeding injury. A negative impact of
ITP on patients’ sex lives (8 of 18 physicians [44%]) and
concerns around increased risk of bleeding impacting
travel plans (9 of 20 physicians [45%]) was reported by
45% physicians (data not shown).
Table 1 (continued)
Physicians
N = 21
Chronic lymphocytic leukemia 8 (38%)
Human immunodeficiency virus 7 (33%)
Investigation rates (asymptomatic vs high symptom burden)
Spleen evaluation 12 (57%) vs 16 (76%)
Coomb’s test 5 (24%) vs 13 (76%)
H. pylori 3 (14%) vs 8 (28%)
Computed tomography scan 1 (5%) vs 7 (33%)
Platelet specific assay 0% vs 4 (19%)
Misdiagnosis rates
Upto 25% patients are misdiagnosed 14 (67%)
26–50% patients are misdiagnosed 5 (24%)
Most commonly misdiagnosed conditions
Drug induced thrombocytopenia 12 (63%)
Leukemia 11 (58%)
Aplastic anemia 10 (53%)
Page 6 of 14
Chakrabartietal. Journal of Patient-Reported Outcomes (2022) 6:24
Fig. 1 Frequency and/or severity of symptoms of ITP at diagnosis and survey completion – Patient and physician perspective
Page 7 of 14
Chakrabartietal. Journal of Patient-Reported Outcomes (2022) 6:24
Almost all physicians (20 of 21 physicians [95%]) did
not use any QoL tool, but expressed their desire to use a
patient self-assessment questionnaire (12 of 20 physicians
[60%] would use it during every consultation, and 7 of
20 physicians [35%] would use it every 6months). Most
physicians (16 of 21 physicians [76%]) expressed that use
of a mobile-based app would help in recording patient
QoL, while 10 of 21 physicians (48%) expressed that com-
bining paper- and mobile app-based approach would be
the preferred method to use. No major differences were
Fig. 2 Impact of ITP on QoL – Patient and physician perspective
Page 8 of 14
Chakrabartietal. Journal of Patient-Reported Outcomes (2022) 6:24
Fig. 3 Impact of ITP on emotional well-being – Patient perspective
Fig. 4 Impact of ITP on work
Page 9 of 14
Chakrabartietal. Journal of Patient-Reported Outcomes (2022) 6:24
observed in the response assessments of physicians based
on their workload (data not shown).
Management ofgoals andtreatment options inITP
Patient
ITP diagnosis to treatment required an average of
0.9 months, with over half of the patients (34 of 65
patients [52%]) undergoing a period of “wait and watch.
e important treatment goals for patients were healthy
blood counts (42 of 65 patients [65%]), improvement in
QoL (35 of 65 patients [54%]), prevention of episodes
on worsening of ITP (33 of 65 patients [51%]), reduction
in spontaneous bleeding (18 of 65 patients [28%]), and
an overall improvement in symptoms (17 of 65 patients
[26%]) (Fig.5A). A majority of patients (41 of 65 patients
[63%]) strongly agreed that their current treatment was
helping them reach their treatment goals.
A once-daily oral pill was preferred by 49 of 65 (75%)
patients. At the time of survey completion, the most fre-
quently administered treatments were corticosteroids (38
of 65 patients [58%]), androgens (9 of 65 patients [14%]),
anti-CD20 (9 of 65 patients [14%]), thrombopoietin
receptor agonists (TPO-RAs; 8 of 65 patients [12%]), and
other immunosuppressants (7 of 65 patients [11%]); the
average duration of these medications was 4.7months.
When the symptom burden was low, most patients
reported undergoing treatment with corticosteroids
(27 of 33 patients [82%]), and as the burden increased
to moderate and above, corticosteroid use decreased
slightly (17 of 24 patients [71%]). e use of androgens
(8 of 33 patients [24%] to 11 of 24 patients [46%]), anti-
CD20 (9 of 33 patients [27%] to 11 of 24 patients [46%]),
and TPO-RAs (3 of 33 patients [9%] to 7 of 24 patients
[29%]) increased with increasing symptom burden. Data
on treatment satisfaction were based on a low patient
number (data not presented here) (data not shown).
Physician
Approximately 39% of the newly diagnosed patients were
given a trial of observation only. Even among patients
who had been previously treated for > 12 months since
diagnosis, 30% were put on observation instead of being
treated. Splenectomy was considered in 23% of the
patients with chronic and recurrent course. Platelet count
Fig. 5 Treatment goals
Page 10 of 14
Chakrabartietal. Journal of Patient-Reported Outcomes (2022) 6:24
monitoring was done more routinely in newly diag-
nosed patients (every 15 days) compared with patients
with chronic ITP (every 1.7 months). e major treat-
ment goals for physicians were reduction in spontaneous
bleeding (17 of 21 physicians [81%]), better QoL (14 of 21
physicians [67%]), symptom improvement (9 of 21 phy-
sicians [43%]), healthy blood counts (6 of 21 physicians
[29%]), and reduction in fatigue symptom (5 of 21 phy-
sicians [24%]). Nearly 90% of physicians (18 of 21 physi-
cians [86%]) believed that they discussed and agreed on
treatment goals with their patients, and 14 of 21 physi-
cians (67%) aimed to limit the immunosuppressive effect
of the treatment (Fig.5B).
e most important attributes while making treatment
decisions for patients with ITP were offering cure or sus-
tained remission (83%), the ability to reduce bleeding risk
(80%), and keeping side effects to a minimum (79%). For
both newly diagnosed and chronic ITP, ~ 80% of physi-
cians preferred oral treatment options as the first line of
treatment. Corticosteroids (19 of 21 physicians [90%])
and intravenous immunoglobulins (IVIgs; 16 of 21 phy-
sicians [76%]) were the preferred treatments in newly
diagnosed patients with ITP. TPO-RAs (19 of 21 physi-
cians [90%]) and anti-CD20 (17 of 21 physicians [81%]),
followed by androgens (16 of 21 physicians [76%]), were
the preferred treatment options in patients with persis-
tent and chronic ITP (Fig.6; Additional file3: Figure S3).
For patients relapsing for the first time, corticoster-
oids (14 of 21 physicians [67%]), followed by androgens
(8 of 21 physicians [38%]) and IVIgs/anti-CD20 (7 of
21 physicians [33%] each), were preferred, while dur-
ing second relapse other immunosuppressants (9 of 21
physicians [43%]) followed by corticosteroids (8 of 21
physicians [38%]) were preferred; by the third relapse,
TPO-RAs (11 of 21 physicians [52%]), followed by anti-
CD20 therapy (8 of 21 physicians [38%]), were the pre-
ferred treatment option (data not shown).
Based on physician perspective, patients treated with
TPO-RAs had the least incidence of side effects. A total
of 13 of 21 physicians (62%) agreed that they were sat-
isfied with the current treatment options available. Lack
of efficacy (21 of 21 physicians [100%]), followed by side
effects (19 of 21 physicians [90%]), and cost/coverage (17
of 21 physicians [81%]), was the most important reason
for a change in therapy (data not shown).
Patient andphysician relationship
When compared with patients, a lower proportion of
physicians were completely satisfied with the various
aspects of ITP disease–related care and management
(data not shown). While responding to questions on
access to information on ITP for their patients, 9 of 21
physicians (43%) expressed that patients faced at least
some level of difficulty in accessing information. About
half of the physicians indicated that they provided dis-
ease management–related information in a leaflet format
explaining the contents of the leaflet (9 of 21 physicians
[43%]). From the patient perspective, 40 of 65 patients
(62%) had not received any information from their HCP.
For patients who recieved information from their HCP, it
Fig. 6 Management of ITP – Prescribed treatments
Page 11 of 14
Chakrabartietal. Journal of Patient-Reported Outcomes (2022) 6:24
was either through a leaflet (10 of 25 patients [40%]) or
through HCPs showing the website content during con-
sultation (8 of 25 patients [32%]) or by being directed to
the website for accessing information about the disease (7
of 25 patients [28%]). A large proportion of patients did
not have any contact with patient support groups (61 of
65 patients [94%]) (data not shown).
Among 64 of 65 patients (98%) who visited a specialist
doctor, an average of 6.5 visits were recorded in the last
12months, and of these patients, 49 (77%) perceived the
frequency of visits to be adequate. None of the patients
reported consultation with a psychologist (data not
shown).
Discussion
To the best of our knowledge, this questionnaire-based
survey is the first of its kind among patients with ITP
and treating physicians in the Indian subcontinent, and
provides an insight into the perceptions of both patients
and physicians regarding disease diagnosis, signs and
symptoms, impact of patient HRQoL, and the approach
toward disease management.
A marked difference was observed in the num-
ber of patients with ITP seen by physicians in the last
12months before survey completion between the Indian
and global survey data (India: 81, global: 43) [9]. In
India, the overall doctor-to-population ratio is 1:1800,
which is lower than that the ratio of 1:1000 suggested
by ‘High Level Expert Group (HLEG) for Universal
Health Coverage’ constituted by the Planning Commis-
sion, and endorsed by WHO [7, 13]. Moreover, in India,
the population-to-specialist ratio is high [7], which fur-
ther increases the patient burden of hematologists and
hemato-oncologists. It is now widely accepted that ITP
is a diagnosis of exclusion and an estimated 15% patients
with primary ITP are misdiagnosed (McMaster ITP Reg-
istry) [14]. It is also pertinent that the initial diagnosis of
ITP is accurate, while avoiding delays based on the chal-
lenges associated with patient navigation. Encouragingly,
in this survey, the time from symptom presentation to
first consultation, and first consultation to diagnosis were
both < 1 month based on patients’ response, although
from the physicians’ perspective the accuracy of the diag-
nosis was low, with ~ 25% of physicians estimating that up
to 50% patients were misdiagnosed. Even if an accurate
diagnosis is made, the high patient burden and the asso-
ciated low average primary care physician consultation
time of ~ 2 min in India [5], physicians tend to primar-
ily treat for bleeding episodes and often underestimate
the impact of ITP on QoL. It is therefore imperative that
auxiliary healthcare service providers, especially nurses,
are trained to assess HRQoL parameters, and along with
physicians, adopt app-based or other validated QoL tools
for better disease management.
Heavy menstrual bleeding, fatigue, and anxiety sur-
rounding unstable platelet counts were predominantly
reported as severe by patients at both diagnosis and
survey completion. Physician perspectives on the fre-
quency and/or severity of the most common symptoms
and their impact on QoL were not always similar to
those reported by patients. While fatigue was reported
as severe by ~ 60% of patients at diagnosis, about 33%
of physicians perceived it as a symptom that severely
affects patient QoL. is trend in underestimation of
fatigue by physicians was observed in both the Indian
and global data [9]. However, fatigue adversely impacts
patient work productivity and social life, and physicians
should consider patient-reported fatigue as an important
symptom that affects HRQoL [15]. A high frequency of
menorrhagia, iron-deficiency anemia, and other nutri-
tional anemias found among Indian patients could be an
important contributing factor for fatigue [1618]. Similar
to fatigue events, menorrhagia also impacts a number of
HRQoL measures [19, 20] and was reported by a major-
ity of women (> 70%) in this analysis. e fear concerning
heavy menstruation could be a major cause of anxiety in
most women at the time of ITP diagnosis (based on low-
grade evidence) [21, 22]. Of note, anxiety was reported
by 43% patients at diagnosis and 35% patients at survey
completion. Given that anxiety could be associated with
repeated blood count testing, more healthcare visits than
required, and changing the consulting physician fre-
quently (doctor shopping), it could result in an overall
increase in healthcare cost. erefore, counselling and
participatory medicine is important to ensure a common
treatment goal for physicians and patients to address
anxiety in ITP. Interestingly, the proportion of patients
reporting anxiety as a severe symptom reduced from 61
to 16% from diagnosis to survey completion. is could
be partially attributed to the fact that the average disease
duration from the time of diagnosis to survey completion
was 5.3years, implying that most patients evaluated in
this analysis had chronic ITP. It is often speculated that
patients with newly diagnosed ITP have higher anxiety
levels due to the uncertainty associated with their disease
course [23].
e assessment and improvement of HRQoL param-
eters generally require a multidimensional approach
and should be tailored for the patient, while taking into
account the healthcare system, cultural, and economic
backgrounds of individual countries [24]. In this sub-
group analysis among patients from India, the ILQI
questionnaire scores showed that daily life was severely
impacted by ITP, with more than half the patients
reporting that their work life, education, concentration
Page 12 of 14
Chakrabartietal. Journal of Patient-Reported Outcomes (2022) 6:24
levels, social lives, and energy levels were negatively
affected. In general, the QoL parameters that were
highlighted as being a concern include anxiety about
platelet counts and frustrations around having a long-
term rare disease, high out-of-pocket expenses, inabil-
ity to perform intense physical exercise or play sports
with chances of bleeding injuries, and impact on travel
plans due to concerns about increased risk of bleed-
ing and taking medications abroad. e out-of-pocket
expenses account for nearly 63% of the total healthcare
expenditure in India—one of the highest in the world—
reiterating the importance of a country’s healthcare
infrastructure in supporting improvement of patients’
HRQoL [2426]. Although a few public health insur-
ance programs in India cover nonmedical expenditure,
such as transportation, lodging, and food costs, for
patients and caregivers, there is no provision for incur-
ring the loss of pay suffered by patients or their spouses
[26], thereby increasing the socioeconomic burden
of the disease. e lack of patient support groups and
other professional support for patient counseling add
to the emotional burden of ITP in India, as patients
almost entirely depend on family, friends, and the treat-
ing doctor for support. Patient support groups could
not only provide a platform for patients to share their
disease experience and provide emotional and moral
support but also help educate patients/families, raise
public awareness, and aid in raising funds [27]. How-
ever, in India, engagement in patient support groups is
low. e major constraints in ensuring higher engage-
ment rates could be the lack of awareness, lack of time,
or anxiety around discussing the negative aspects of
the disease publicly [27]. ere is a need to consider a
holistic approach toward assessment of symptom bur-
den and impact of ITP on QoL in routine clinical prac-
tice in India.
Physician ability to effectively and compassionately
communicate the nature of disease and management
options is important to build trust in a patient–physi-
cian relationship, and shared decision-making is a key
element in improving patient–physician communica-
tion [28]. Although nearly 90% of physicians included
in this survey mentioned that they had included their
patients’ perspective during decision-making, the
implementation of a participatory decision-making
model in ITP, which has been in place for cancer man-
agement for a considerable period of time [29], may not
be feasible in the Indian context. is could be due to
the existing gaps in patient knowledge of the disease
and effectiveness of available treatment options [30].
Implementation of a shared-decision model in India
needs greater patient education, along with physician
awareness and willingness; patient support groups can
play a major role in bringing about this change.
A shared-decision model could also help in ensur-
ing that the treatment goals of patients and physicians
are completely aligned. Our survey results showed that
achievement of healthy blood counts was the most
important goal for patients, while for physicians, it
was reduction in spontaneous bleeds. Interestingly,
improvement in QoL was one of the most impor-
tant treatment goals for both patients and physicians,
underlining the importance of assessing HRQoL among
patients with ITP. is was consistent with the global
I-WISh data, wherein improvement in QoL was one of
the top 3 goals among 38% of patients and 64% of phy-
sicians [8, 9].
Overall, the survey data outcomes and driven conclu-
sions must be interpreted with caution, given the small
sample size of the respondents, specifically the patient
group. Also, this survey did not capture the number of
treatment lines received by patients or the remission sta-
tus. Recall bias and the use of a non-validated HRQoL
questionnaire (ILQI) are some of the other limitations of
the study. However, the study results need to be consid-
ered in the light of the fact that ITP is a rare disease, and
currently, in India, there is limited education/awareness
among patients regarding the disease.
Conclusion
Based on the overall respondent assessment, the study high-
lights the need for education/training of physicians and
other healthcare workers on all aspects of ITP disease man-
agement—especially fatigue, anxiety, and menorrhagia—and
general awareness among physicians and patients on disease
management, including treatment goals, and the impact of
ITP on QoL. Additionally, it also emphasizes some of the
neglected aspects of ITP and provides a good starting point
for large-scale future studies in this therapy area.
Abbreviations
HCP: Healthcare professional; HRQoL: Health-related quality of life; ILQI: ITP Life
Quality Index; IRB: Institutional Review Board; ITP: Immune thrombocytopenia;
IVIgs: Intravenous immunoglobulins; I-WISh: ITP World Impact Survey; QoL:
Quality of life; TPO-RAs: Thrombopoietin receptor agonists.
Supplementary Information
The online version contains supplementary material available at https:// doi.
org/ 10. 1186/ s41687- 022- 00429-y.
Additional le1. Figure S1: Symptoms that patients want to be resolved.
Additional le2. Figure S2: Physician perspective on fatigue.
Additional le3. Figure S3: Prescribed treatments based on platelet
counts.
Page 13 of 14
Chakrabartietal. Journal of Patient-Reported Outcomes (2022) 6:24
Acknowledgements
We extend enormous thanks to all the patients and physicians who took the
time to complete this survey. Funding was provided by Novartis to Adelphi
Real World for the survey design, data collection, and data analysis. Tom Bailey
coordinated data collection and statistical analysis. We thank Anupama Singh
of Novartis Healthcare Private Limited for providing medical writing assistance.
Authors’ contributions
All authors contributed in the interpretation of data, writing and critically
reviewing the manuscript, and approval of the final draft. All authors read and
approved the final manuscript.
Funding
This study was sponsored by Novartis Pharmaceuticals Corporation.
Availability of data and materials
Not applicable.
Declarations
Ethics approval and consent to participate
Survey materials and the study protocol were reviewed and approved by a
centralized Institutional Review Board (IRB). Patients and physicians were given
an overview of the study and the ethical approval details; those who wished
to participate were required to provide consent via a tick/check box before
initiation.
Consent for publication
Not applicable.
Competing interests
Dr. Shashank Udupi is a full-time employee of Novartis Healthcare Private
Limited. None of the other authors had any conflict of interest to declare.
Author details
1 Department of Hematology, Vivekananda Institute of Medical Sciences,
Machan, L 16 Panchasayar, Kolkata, West Bengal 700094, India. 2 Department
of Hematology, CMC Vellore, Vellore, Tamil Nadu 632004, India. 3 Department
of Clinical Hematology, KEM Hospital, 1902, 19th floor UG PG hostel, KEM
Hospital Campus, Parel, Mumbai, Maharashtra 400012, India. 4 Department
of Medical Oncology, MG Medical College, 67/166, Sector 6, Pratap Nagar,
Jaipur, Rajasthan, India. 5 Medical Affairs, Oncology (Hematology), Novartis
Healthcare Private Limited, Inspire BKC, Part of 601 & 701, 7th Floor, Bandra
Kurla Complex, Bandra (East), Mumbai, Maharashtra 400051, India. 6 Depart-
ments of Research and Hemato-Oncology, Østfold Hospital, Østfold Hospital,
PB 300, 1714 Grålum, Norway.
Received: 9 September 2021 Accepted: 28 February 2022
References
1. Michel M, Suzan F, Adoue D et al (2015) Management of immune throm-
bocytopenia in adults: a population-based analysis of the French hospital
discharge database from 2009 to 2012. Br J Haematol 170(2):218–222
2. Sestøl HG, Trangbæk SM, Bussel JB, Frederiksen H (2018) Health-related
quality of life in adult primary immune thrombocytopenia. Expert Rev
Hematol 11(12):975–985
3. Neunert C, Terrell DR, Arnold DM et al (2019) American Society of
Hematology 2019 guidelines for immune thrombocytopenia. Blood Adv
3(23):3829–3866
4. Provan D, Arnold DM, Bussel JB et al (2019) Updated international con-
sensus report on the investigation and management of primary immune
thrombocytopenia. Blood Adv 3(22):3780–3817
5. Irving G, Neves AL, Dambha-Miller H et al (2017) International variations
in primary care physician consultation time: a systematic review of 67
countries. BMJ Open 7(10):e017902
6. Density of Physicians (Total Number per 1000 Population, Latest Available
Year), Global Health Observatory (GHO) Data. Situation and Trends. http://
www. who. int/ gho/ health_ workf orce/ physi cians_ densi ty/ en/. Accessed
15 Feb 2021.
7. Deo MG (2013) Doctor population ratio for India—the reality. Indian J
Med Res 137(4):632–635
8. Cooper N, Kruse A, Kruse C et al (2020) Immune thrombocytopenia (ITP)
World Impact Survey (iWISh): patient and physician perceptions of diag-
nosis, signs and symptoms, and treatment. Am J Hematol 96(2):188–198
9. Cooper N, Kruse A, Kruse C et al (2020) Immune thrombocytopenia (ITP)
World Impact Survey (I-WISh): impact of ITP on health-related quality of
life. Am J Hematol 96(2):199–207
10. Nampoothiri RV, Singh C, Lad D et al (2019) Immune thrombocytopenia
is still the commonest diagnosis on consultative hematology. Indian J
Hematol Blood Transfus 35(2):352–356
11. Mishra K, Pramanik S, Jandial A et al (2020) Real-world experience
of eltrombopag in immune thrombocytopenia. Am J Blood Res
10(5):240–251
12. Griffiths P, Grant L, Bonner N et al (2019) The psychometric proper-
ties of the ITP Life Quality Index assessed in a large multinational
“real-world” cohort of immune thrombocytopaenia patients. Blood.
134(Supplement_1):386–386
13. High Level Expert Group Report on Universal Health Coverage for India.
Planning Commission of India. New Delhi, November, 2011. http:// nhm.
gov. in/ images/ pdf/ publi cation/ Plann ing_ Commi ssion/ rep_ uhc08 12. pdf.
Accessed 19 Mar 2021.
14. Arnold DM, Nazy I, Clare R et al (2017) Misdiagnosis of primary immune
thrombocytopenia and frequency of bleeding: lessons from the McMas-
ter ITP Registry. Blood Adv 1(25):2414–2420
15. Hill QA, Newland AC (2015) Fatigue in immune thrombocytopenia. Br J
Haematol 170(2):141–149
16. Didzun O, De Neve JW, Awasthi A et al (2019) Anaemia among men
in India: a nationally representative cross-sectional study. Lancet Glob
Health 7(12):e1685–e1694
17. Kapil U, Bhadoria AS (2014) National Iron-plus initiative guidelines
for control of iron deficiency anaemia in India, 2013. Natl Med J India
27(1):27–29
18. Shankar B, Agrawal S, Beaudreault AR et al (2017) Dietary and nutritional
change in India: implications for strategies, policies, and interventions.
Ann N Y Acad Sci 1395(1):49–59
19. Shankar M, Chi C, Kadir RA (2008) Review of quality of life: menorrhagia
in women with or without inherited bleeding disorders. Haemophilia
14(1):15–20
20. Rajpurkar M, O’Brien SH, Haamid FW, Cooper DL, Gunawardena S, Chitlur
M (2016) Heavy menstrual bleeding as a common presenting symptom
of rare platelet disorders: illustrative case examples. J Pediatr Adolesc
Gynecol 29(6):537–541
21. Rodeghiero F, Marranconi E (2020) Management of immune thrombocy-
topenia in women: current standards and special considerations. Expert
Rev Hematol 13(2):175–185
22. Kruse C, Kruse A, Watson S et al (2018) Patients with immune thrombo-
cytopenia (ITP) frequently experience severe fatigue but is it under-rec-
ognized by physicians: results from the ITP World Impact Survey (I-WISh).
Blood 132(Supplement 1):2273–2273
23. Kruse A, Kruse C, Potthast N, Milligan K, Bussel JB (2019) Mental health
and treatment in patients with immune thrombocytopenia (ITP); Data
from the Platelet Disorder Support Association (PDSA) Patient Registry.
Blood 134(Supplement_1):2362–2362
24. Hurst JR, Agarwal G, van Boven JFM et al (2020) Critical review of
multimorbidity outcome measures suitable for low-income and middle-
income country settings: perspectives from the Global Alliance for
Chronic Diseases (GACD) researchers. BMJ Open 10(9):e037079–e037079
25. Sriram S, Khan MM (2020) Effect of health insurance program for the poor
on out-of-pocket inpatient care cost in India: evidence from a nationally
representative cross-sectional survey. BMC Health Serv Res 20(1):839
26. Patel V, Parikh R, Nandraj S et al (2015) Assuring health coverage for all in
India. Lancet 386(10011):2422–2435
27. Hu A (2017) Reflections: the value of patient support groups. Otolaryngol
Head Neck Surg 156(4):587–588
28. ACOG Committee Opinion No. 587 (2014) Effective patient-physician
communication. Obstet Gynecol 123(2 Pt 1):389–393
Page 14 of 14
Chakrabartietal. Journal of Patient-Reported Outcomes (2022) 6:24
29. Butow P, Harrison JD, Choy ET, Young JM, Spillane A, Evans A (2007)
Health professional and consumer views on involving breast cancer
patients in the multidisciplinary discussion of their disease and treatment
plan. Cancer 110(9):1937–1944
30. Wang KKW, Charles C, Heddle NM, Arnold E, Molnar L, Arnold DM (2014)
Understanding why patients with immune thrombocytopenia are deeply
divided on splenectomy. Health Expect 17(6):809–817
Publisher’s Note
Springer Nature remains neutral with regard to jurisdictional claims in pub-
lished maps and institutional affiliations.
... -primary immune thrombocytopenia HRQoL -Health-related quality of life TPO-RAs -agonists of thrombopoietin receptors PC -Platelet counts TFR -Treatment-free response TGF-beta -Transforming growth factor beta I-WISh -ITP World Impact Survey as the principal treatment goal for ITP is to treat or prevent bleeding [3,24]. ...
... The impact of ITP and its treatments on patientsꞌ HRQoL may affect the entire spectrum of patients' lives, encompassing daily activities, emotional health, energy level, fatigue, and work productivity. Compared with age-matched and sex-matched controls, patients with ITP have lower work productivity, more physician visits, and are more likely to take sick leave [3,5,24]. Interestingly, with the current standard of care, HRQoL appears to deteriorate in the first year after diagnosis and improve later in the disease course [5]. ...
... However, I-WISh survey reflected the increased awareness of reduced HRQoL and low energy in patients with ITP by physicians who were experienced in the care of ITP [5]. On the other hand, data from India showed that fatigue was reported as severe by 60% of patients at diagnosis, and only 33% of physicians perceived it as a severe problem [24]. Even more, 58% of physicians felt their patients experienced anxiety about PC, whereas this was identified as an issue in 53% of patients. ...
Article
Full-text available
Introduction. Primary immune thrombocytopenia is a chronic acquired autoimmune disorder that is characterized by isolated thrombocytopenia (<100 x109/L) and the absence of any underlying cause. Treatment of primary immune thrombocytopenia. While splenectomy has a curable potential, it carries long-term risk of infection and thromboembolic complications. Therefore, the use of splenectomy has declined with the advent of rituximab and agonists of thrombopoietin receptors. The efficacy of rituximab is good for the short-term outcome, and the majority of patients will relapse. On the other hand, agonists of thrombopoietin receptors induce remission in only 10-30% of patients after treatment discontinuation, and long-term treatment is often required. Health - related quality of life. Immune thrombocytopenia and its treatments may affect the entire spectrum of patients? lives, encompassing daily activities, emotional health, energy level, fatigue, and work productivity. Primary immune thrombocytopenia World Impact Survey was conducted to discern how immune thrombocytopenia and associated treatments affect patient lives. Concerns about unstable platelet count, low energy levels, inability to exercise, and reduced participation in hobbies and work had the greatest negative impact. While most patients reported ?good health?, nonetheless half of patients reported a negative impact on their emotional well-being that worsened with increasing burden of disease and was often substantial. Conclusion. Although several important improvements have been made in immune thrombocytopenia treatment algorithms, there is still room for improvement. One of the possible options could be early, intensive treatment of immune thrombocytopenia, which might reduce the risk of disease progression and consequently improve patients? quality of life.
... During the disease, the proportion of self-reported HMB ranged from −17% to 79%. 4,42,43 A higher proportion was found in the Indian subset of the I-WISh study than in the complete study population (73% vs. 51% at diagnosis and 38% vs. 17% during the disease). Again, none of these studies used a clear definition of HMB. ...
... Of note, the prevalence of HMB throughout the course of disease was found to vary largely in different studies be much lower (17%-79%). 4,42,43 These studies all relied on self-rapportage, which is known to be unreliable. 22 For example, in one study, only 27% of patients with a PBAC score of ≥100 reported having HMB through self-assessment. ...
Article
Full-text available
Immune thrombocytopenia (ITP) may cause menstrual problems. This cross‐sectional study assessed menstrual problems in premenopausal chronic ITP women by several questionnaires, including the pictorial bleeding assessment calendar (PBAC; score ≥100 indicates heavy menstrual bleeding [HMB]), and the menorrhagia multiattribute scale (MMAS). Spearman was used for assessing correlations. A literature review was performed in Pubmed. The cohort comprised 37 women (mean age 31 ± 9). A total of 29/37 (78%) had experienced clinical menstrual problems in the present or past. Of the 33 patients who returned the PBAC, 13 (39%) had a score of ≥100. The median MMAS score was 79 (IQR 60–95). The PBAC scores correlated with the MMAS. Both questionnaires were unrelated to the platelet count. Patients with a levonorgestrel intrauterine device (LNG‐IUD) had lower PBAC scores than patients with other or no hormonal therapy. MMAS scores were correlated with fatigue. The review identified 14 papers. HMB occurred in 6%–55% at ITP diagnosis and 17%–79% during disease. Menstrual symptoms influenced the quality of life, particularly in patients with a low platelet count. This explorative study suggested that HMB is frequent in women with chronic ITP despite management and platelet counts >50 *109/l. An LNG‐IUD seemed to reduce blood loss significantly.
... severity, suffering, and prognosis) are interpreted according to the observer's socio-economic status, race, previous experience, and level of education. Patients, caregivers, and health care providers may have differing perceptions of a specific disease even though they all observe the same progression [56,57]. Specifically, family and friends tend to overestimate the suffering experienced by ET patients as compared to the patient's own ratings [57]. ...
Article
Full-text available
Brain donation is a challenging process, comprising four sequential stages: (1) the brain donation decision, (2) pre-mortem arrangements and follow up, (3) specimen collection and (4) tissue processing. It is important to understand the factors that are pertinent to each stage. Currently, there is extensive information on factors that involve donor's personal and cultural backgrounds and how these could affect the process. However, little is known about disease-specific factors that influence the process. The Essential Tremor Centralized Brain Repository was established in 2003, and after nearly 20 years of collecting essential tremor (ET) brain tissue, we are well-positioned to discuss the brain donation process from a disease-specific standpoint. In the current manuscript, we discuss ET disease-specific factors that influence the first two stages of the brain donation process. We center our discussion around three points: (1) factors that influence the patient's decision to donate, (2) the involvement of next of kin in the donation, and (3) the rationale for enrolling patients prospectively and evaluating them longitudinally before the anatomical gift takes place. This discussion shares our understanding of the background from which our repository operates and may be of value for other brain banks that study similar neurodegenerative diseases.
Article
Full-text available
Immune thrombocytopenia (ITP) is now well‐known to reduce patients' health‐related‐quality‐of‐life. However, data describing which signs and symptoms patients and physicians perceive as having the greatest impact are limited, as is understanding the full effects of ITP treatments. I‐WISh (ITP World Impact Survey) was an exploratory, cross‐sectional survey designed to establish the multifaceted impact of ITP, and its treatments, on patients' lives. It focused on perceptions of 1507 patients and 472 physicians from 13 countries regarding diagnostic pathway, frequency and severity of signs and symptoms, and treatment use. Twenty‐two percent of patients experienced delayed diagnosis (caused by several factors), 73% of whom felt anxious as a result. Patients rated fatigue among the most frequent, severe symptom associated with ITP at diagnosis (58% most frequent; 73% most severe), although physicians assigned it lower priority (30%). Fatigue was one of the few symptoms persisting at survey completion (50% and 65%, respectively) and was the top symptom patients wanted resolved (46%). Participating physicians were experienced at treating ITP, thereby recognizing the need to limit corticosteroid use to newly‐diagnosed or first‐relapse patients and espoused increased use of thrombopoietin receptor agonists and anti‐CD20 after relapse in patients with persistent/chronic disease. Patient and physicians were largely aligned on diagnosis, symptoms, and treatment use. I‐WISh demonstrated that patients and physicians largely align on overall ITP symptom burden, with certain differences eg fatigue. Understanding the emotional and clinical toll of ITP on the patient will facilitate shared decision‐management, setting and establishment of treatment goals and disease stage‐appropriate treatment selection. This article is protected by copyright. All rights reserved.
Article
Full-text available
Immune thrombocytopenia (ITP) has a substantial, multifaceted impact on patients’ health‐related quality of life (HRQoL). Data describing which aspects of ITP physicians and patients perceive as having the greatest impact are limited. ITP World Impact Survey (I‐WISh) was a cross‐sectional survey, including 1507 patients and 472 physicians, to establish the impact of ITP on HRQoL and productivity from patient and physician perspectives. Patients reported that ITP reduced their energy levels (85% of patients), capacity to exercise (77%), and limited their ability to perform daily tasks (75%). Eighty percent of physicians reported that ITP symptoms reduced patient HRQoL, with 66% reporting ITP‐related fatigue substantially reduced patient HRQoL. Patients believed ITP had a substantial impact on emotional well‐being (49%) and 63% worried their condition would worsen. Because of ITP, 49% of patients had already reduced, or seriously considered reducing their working hours, and 29% had considered terminating their employment. Thirty‐six percent of patients employed at the time of the survey felt ITP decreased their work productivity, while 51% of patients with high/very high symptom burden reported that ITP affected their productivity. I‐WISh demonstrated substantive impact of ITP on patients’ HRQoL both directly for patients and from the viewpoint of their physicians. Patients reported reduced energy levels, expressed fears their condition might worsen, and those who worked experienced reduced productivity. Physicians should be aware not only of platelet counts and bleeding but also the multi‐dimensional impact of ITP on patients’ lives as an integral component of disease management. This article is protected by copyright. All rights reserved.
Article
Full-text available
Immune thrombocytopenia (ITP) is characterized by decreased platelet count in the peripheral circulation. The first-line therapy is corticosteroids with 53-80% overall response rate. Eltrombopag has been used as second-line therapy in ITP for over a decade now. The long-term efficacy and safety profile have been widely reported in the western world. However, the data from the resource-constraint settings of the developing world is scarce. We aim to present the real-life experience of efficacy and safety of eltrombopag from the resource-constraint settings. This was a retrospective, single-center study conducted at a tertiary care hospital in Northern India from 2012-2019. On audit of medical records, patients of ITP receiving eltrombopag were screened for inclusion. Patients whose treatment outcomes were not available were excluded. Finally, 53 patients were analyzed using statistical packages of Python v3.7. The patients' median age was 35 years (range 17-78), with 23 (43.4%) being female. The median time to response was 35 days (range 28-50 days) and the cumulative overall response rates (ORR) at day 30, day 60 and day 90 were 41.5%, 69.8%, and 81.1% respectively. A total of 10 patients on eltrombopag relapsed during follow up. The cumulative rate of relapse at one year, three years, and five years were 6.6%, 25.3%, and 47.7%, respectively. There was no significant difference in outcome (response rate or relapse) in any subgroups depending on age, sex, duration of disease, number of prior lines of treatment, splenectomy, or baseline platelet count. Six patients stopped eltrombopag after having a median sustained response for 796 days (range 658-1185), and after a median follow up of 624 days (range 92-1339), they continued to be in remission. Seventeen patients (17/53, 32%) reported one or more adverse events while on eltrombopag therapy. A total of 49 adverse events (n=4, grade ≥3 CTCAEv4) were noted. Anemia was the most frequent adverse event followed by hepatobiliary dysfunction as reflected by deranged AST/ALT or raised bilirubin. The use of eltrombopag among adult ITP patients in resource-constraint settings was well-tolerated and yielded excellent overall response. The benefit was found to be sustained on long-term follow up. However, events like anemia, hepatobiliary, and thrombotic complications merit closer follow up.
Article
Full-text available
Background: In India, Out-of-pocket expenses accounts for about 62.6% of total health expenditure - one of the highest in the world. Lack of health insurance coverage and inadequate coverage are important reasons for high out-of-pocket health expenditures. There are many Public Health Insurance Programs offered by the Government that cover the cost of hospitalization for the people below poverty line (BPL), but their coverage is still not complete. The objective of this research is to examine the effect of Public Health Insurance Programs for the Poor on hospitalizations and inpatient Out-of-Pocket costs. Methods: Data from the recent national survey by the National Sample Survey Organization, Social Consumption in Health 2014 are used. Propensity score matching was used to identify comparable non-enrolled individuals for individuals enrolled in health insurance programs. Binary logistic regression model, Tobit model, and a Two-part model were used to study the effects of enrolment under Public Health Insurance Programs for the Poor on the incidence of hospitalizations, length of hospitalization, and Out-of- Pocket payments for inpatient care. Results: There were 64,270 BPL people in the sample. Individuals enrolled in health insurance for the poor have 1.21 higher odds of incidence of hospitalization compared to matched poor individuals without the health insurance coverage. Enrollment under the poor people health insurance program did not have any effect on length of hospitalization and inpatient Out-of-Pocket health expenditures. Logistic regression model showed that chronic illness, household size, and age of the individual had significant effects on hospitalization incidence. Tobit model results showed that individuals who had chronic illnesses and belonging to other backward social group had significant effects on hospital length of stay. Tobit model showed that days of hospital stay, education and age of patient, using a private hospital for treatment, admission in a paying ward, and having some specific comorbidities had significant positive effect on out-of-pocket costs. Conclusions: Enrolment in the public health insurance programs for the poor increased the utilization of inpatient health care. Health insurance coverage should be expanded to cover outpatient services to discourage overutilization of inpatient services. To reduce out-of-pocket costs, insurance needs to cover all family members rather than restricting coverage to a specific maximum defined.
Article
Full-text available
Objectives: There is growing recognition around the importance of multimorbidity in low-income and middle-income country (LMIC) settings, and specifically the need for pragmatic intervention studies to reduce the risk of developing multimorbidity, and of mitigating the complications and progression of multimorbidity in LMICs. One of many challenges in completing such research has been the selection of appropriate outcomes measures. A 2018 Delphi exercise to develop a core-outcome set for multimorbidity research did not specifically address the challenges of multimorbidity in LMICs where the global burden is greatest, patterns of disease often differ and health systems are frequently fragmented. We, therefore, aimed to summarise and critically review outcome measures suitable for studies investigating mitigation of multimorbidity in LMIC settings. Setting: LMIC. Participants: People with multimorbidity. Outcome measures: Identification of all outcome measures. Results: We present a critical review of outcome measures across eight domains: mortality, quality of life, function, health economics, healthcare access and utilisation, treatment burden, measures of 'Healthy Living' and self-efficacy and social functioning. Conclusions: Studies in multimorbidity are necessarily diverse and thus different outcome measures will be appropriate for different study designs. Presenting the diversity of outcome measures across domains should provide a useful summary for researchers, encourage the use of multiple domains in multimorbidity research, and provoke debate and progress in the field.
Article
Full-text available
Background: Population-based studies on anaemia in India have mostly focused on women and children, with men with anaemia receiving much less attention despite anaemia's adverse effect on health, wellbeing, and economic productivity. This study aimed to determine the national prevalence of anaemia among men in India; how the prevalence of anaemia in men varies across India among states and districts and by sociodemographic characteristics; and whether the geographical and sociodemographic variation in the prevalence of anaemia among men is similar to that among women to inform whether anaemia reduction efforts for men should be coupled with existing efforts for women. Methods: In this cross-sectional study, we analysed data from a nationally representative household survey carried out from January, 2015, to December, 2016, among men aged 15-54 years and women aged 15-49 years in all 29 states and seven Union Territories of India. Haemoglobin concentration was measured using the portable HemoCue Hb 201+ (HemoCue AB, Ängelholm, Sweden) and a capillary blood sample. In addition to disaggregating anaemia prevalence (separately in men and women) by state and age group, we used mixed-effects Poisson regression to determine individual-level and district-level predictors of anaemia. Findings: 106 298 men and 633 305 women were included in our analysis. In men, the prevalence of any anaemia was 23·2% (95% CI 22·7-23·7), moderate or severe anaemia was 5·1% (4·9-5·4), and severe anaemia was 0·5% (0·5-0·6). An estimated 21·7% (20·9-22·5) of men with any degree of anaemia had moderate or severe anaemia compared with 53·2% (52·9-53·5) of women with any anaemia. Men aged 20-34 years had the lowest probability of having anaemia whereas anaemia prevalence among women was similar across age groups. State-level prevalence of any anaemia in men varied from 9·2% (7·7-10·9) in Manipur to 32·9% (31·0-34·7) in Bihar. The individual-level predictors of less household wealth, lower education, living in a rural area, smoking, consuming smokeless tobacco, and being underweight and the district-level predictors of living in a district with a lower rate of primary school completion, level of urbanisation, and household wealth were all associated with a higher probability of anaemia in men. Although some important exceptions were noted, district-level and state-level prevalence of anaemia among men correlated strongly with that among women. Interpretation: Anaemia among men in India is an important public health problem. Because of the similarities in the patterns of geographical and sociodemographic variation of anaemia between men and women, future efforts to reduce anaemia among men could target similar population groups as those targeted in existing efforts to reduce anaemia among women. Funding: Alexander von Humboldt Foundation.
Article
Background: ITP has a multifaceted impact on patients' (pts) lives but there are limited data on which signs and symptoms treating physicians (MDs) and pts perceive as having the greatest impact, especially on pt quality of life (QoL). As in many chronic autoimmune diseases, fatigue can significantly affect QoL in ITP pts. Aims: I-WISh studied the burden of ITP and its impact on QoL, especially fatigue, using a global pt and MD sampling frame. This analysis reports pt vs MD perception of frequency and severity of signs and symptoms. Methods : I-WISh is a cross-sectional survey of ITP pts, recruited via MDs and pt support groups, and also of MDs, recruited via local fieldwork agencies. Participants (pts and MDs) completed a 30-minute online survey that included demographics, signs and symptoms, impact of symptoms, and pt-MD relationships. A steering committee of expert MDs and pt advocacy ITP specialists designed and endorsed survey materials. Results: 1491 pts from 12 countries completed the survey; 65% female, with a mean (SD) age of 47 (16) yrs. 472 MDs from 13 countries completed the survey, with a mean (SD) ITP pt caseload of 34 (50) and a mean (SD) of 18 (36) newly-diagnosed pts in the past yr. The most frequent pt-reported signs and symptoms at diagnosis and at survey completion (Figure A,B) were petechiae (65%; 31%), bruising (64%; 30%), fatigue (58%; 50%), and anxiety around a stable platelet count (34%; 32%). The most common signs and symptoms MDs reported at diagnosis and overall (ie at any stage) were similar to those reported by pts, with petechiae (82% overall; 83% at diagnosis), purpura (74%; 73%), bleeding of the gums (69%; 70%), and epistaxis (69%; 70%) being the most common; there were few differences between diagnosis and overall. By contrast, fatigue was under-reported by MDs (31% overall; 30% at diagnosis) compared with pt-reported data (58% diagnosis; 50% survey completion). Pts rated the severity of their current symptoms. Fatigue was one of the most severe pt-reported symptoms, ie scored ≥5 on a 7-point Likert scale (7=worst imaginable severity), both at diagnosis (74%) and survey completion (65%), and remained consistently frequent and severe over time (Figure A,B). Of pts experiencing heavy menstrual bleeding (84%; 63%) and anxiety around a stable platelet count (78%; 65%) these were also reported as severe, although less so at survey completion. Thrombosis, while not common, was considered severe (62%; 74%; Figure A,B). When asked to consider their current symptoms, the three symptoms pts would most like to resolve were heavy menstrual bleeding (75%, n=118/158), thrombosis (74%, n=25/34), and fatigue (73%, n=544/743). MDs perceived several signs and symptoms as having a high impact on pt QoL (scored ≥5 on a 7-point Likert scale; 7=a great deal), with blood in urine/stool (81%), profuse bleeding during surgery (79%), and menorrhagia (78%) considered the most impactful; 59% believed fatigue has a high impact. Overall, 80% of MDs felt ITP symptoms reduce QoL (scored ≥5 on a 7-point Likert scale; 7=a great deal), and 66% believed that ITP-related fatigue reduces QoL. 46% of MDs believe fatigue is severe (scored ≥5 on a 7-point Likert scale; 7=completely fatigued) and believe fatigue increases as platelet levels decrease (Figure C). Pts are generally satisfied (79% satisfaction) with their MD's management of their disease, and overall satisfaction between pts and MDs was high regarding communication (80% vs 88%), management (79% vs 86%), and understanding of treatment goals (77% vs 90%). Summary/conclusions: The pt-reported symptom with highest frequency and greatest severity both at diagnosis and survey completion was fatigue. MDs who participated were experienced with treating ITP and believed fatigue would greatly affect pts, nonetheless MDs did not consider fatigue to be as substantial a problem as pts did. Pts were also concerned about platelet count stability, heavy menstrual bleeding, and thrombosis. Likelihood of fatigue increased as platelet count reduced suggesting fatigue may be intrinsically related to disease activity and could be alleviated by increasing the platelet count. These results indicate that pts and MDs align on overall symptom burden in ITP but highlight that improved understanding and awareness of the relationships between fatigue, platelet count, and QoL is needed. Disclosures Kruse: Novartis/ITP: Consultancy; Rigel/ITP: Honoraria; Amgen/ITP: Consultancy. Watson:Novartis: Membership on an entity's Board of Directors or advisory committees. Cooper:Amgen, Novartis: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees. Ghanima:Amgen, Novartis: Honoraria, Membership on an entity's Board of Directors or advisory committees; Bayer, BMS, Novartis: Research Funding. Provan:Amgen, Novartis: Honoraria, Research Funding. Arnold:Amgen: Consultancy, Research Funding; UCB: Consultancy; Bristol Myers Squibb: Research Funding; UCB: Consultancy; Amgen: Consultancy, Research Funding; Novartis: Consultancy, Research Funding; Bristol Myers Squibb: Research Funding; Novartis: Consultancy, Research Funding. Santoro:Bayer, CSL, Novo Nordisk, Pfizer, Shire, Sobi: Other: advisory boards, Speakers Bureau; Grifols, Gilead: Other: advisory boards; Amgen, Glaxo: Speakers Bureau. Tomiyama:Sysmex Corporation: Consultancy; Chugai Pharmaceutical Co., Ltd.: Honoraria; Novartis Pharma Co., Ltd.: Honoraria, Membership on an entity's Board of Directors or advisory committees; Kyowa Hakko Kirin Co., Ltd.: Honoraria. Waller:Novartis: Consultancy. Taylor-Stokes:Novartis: Consultancy. Bailey:Novartis: Consultancy. Stankovic:Novartis: Employment. Bussel:Novartis: Consultancy, Research Funding; Uptodate: Honoraria; Rigel: Consultancy, Research Funding; Momenta: Consultancy; Protalex: Consultancy; Amgen Inc.: Consultancy, Research Funding; Prophylix: Consultancy, Research Funding.
Article
Background Immune Thrombocytopenia (ITP) is an autoimmune disorder caused by immunologic destruction of otherwise normal platelets, most commonly occurring in response to an unknown stimulus. ITP is diagnosed after excluding other possible causes of disease, and symptoms can present across varying severities and treatments. The poor understanding of the symptoms and cause can result in both misdiagnosis and complex treatment patterns, which may significantly affect health related quality of life (HRQoL) in this patient population. There is currently no disease specific prospective tool in routine clinical practice to capture HRQoL in the adult ITP population. To help assess the impact of this condition on HRQOL, the ITP Life Quality Index (ILQI), a 10-item patient-reported outcome (PRO) measure was developed as a tool for clinical practice to aid discussions between patients and physicians about disease experience so to inform patient-centric treatment decisions. The ILQI was originally developed by clinical experts in the field of ITP and content validity was confirmed by conducting qualitative interviews with 15 adult patients with ITP. The ILQI was then cognitively debriefed patients with ITP and items refined following qualitative analysis and additional clinical input. The ILQI was included in the ITP World Impact Survey (I-WISh), a global observational study which collected data on the impact of ITP on patients' HRQoL. This large study provided an opportunity to assess the psychometric properties of the ILQI and confirm the scoring cut-offs. Methods The I-WISh survey resulted in data from 1,507 patients with ITP across 12 countries worldwide and was used to assess the structure, reliability and validity of the ILQI. The structure of the ILQI, how the items fit into total scores and subscales, was assessed by splitting the data into two datasets. One dataset was used for identifying the structure using exploratory factor analysis and one was used for checking the structure using confirmatory factor analysis. Validity, the ability of the ILQI to measure the correct construct, was assessed through known groups and convergent validity methods. Reliability, the consistency of the ILQI items and their ability to create reproducible scores, was assessed via internal consistency methods. To understand whether each ILQI item measured ITP in a similar way across countries, differential item functioning (DIF) was assessed using Cochran-Mantel-Haenszel test and Logistic regression. Finally, existing score cut-offs (20-"significantly impaired QoL"; 30 - "severely impaired QoL") were assessed using receiver operating characteristic (ROC) curves and a simulation study was conducted to develop rules for missing data. Results Results indicated that the ILQI has an essentially unidimensional structure, supporting the creation of a total score including all 10 items. The ILQI items work together to create a reproducible total score, usable for making judgements on an individual patient basis (Omega total and Cronbach's alpha coefficients ≥ 0.90). Known groups methods showed that ILQI monotonically increased with ITP severity (linear trends p's <0.001). Convergent validity methods confirmed hypothesized relationships between ILQI total/item scores and items which measure aspects of HRQOL, suggesting that the ILQI total score and its items measure the same concept of interest (i.e. HRQOL). DIF analyses showed that ILQI item responses were similar between the USA and Western countries. Some uniform DIF was discovered between the USA and other countries, and some non-uniform DIF was found between the USA and China for culturally relevant items as expected. The previous clinical cut-off of 20 for "significantly impaired QoL" was supported, but a cut-off 30 may be too conservative for assessing "severely impaired QoL". Missing data simulation suggests that a total score can be created even when some items are missing. Conclusion The ILQI is a valid and reliable, unidimensional measure to assess HRQoL of patients with ITP. Despite some variations in ILQI item responses between USA and China, adoption of the ILQI in routine care will improve consistency of patient-centric decision making and may lead to better outcomes for those patients whose HRQoL has been affected. The revised cut-off scores for the ILQI developed will also aid patient-centric decision making between patients and physician. Disclosures Griffiths: Adelphi Values Ltd: Employment; Novartis: Consultancy. Grant:Novartis: Consultancy; Adelphi Values Ltd: Employment. Bonner:Adelphi Values Ltd: Employment; Novartis: Consultancy. D'Alessio:Novartis: Employment, Equity Ownership. Hill:Apellis: Honoraria; Bioverativ, a Sanofi company: Honoraria; Novartis: Speakers Bureau; Alexion: Research Funding. Provan:Rigel ONO: Consultancy, Research Funding; Novartis: Consultancy, Honoraria, Research Funding; Amgen: Consultancy, Honoraria, Research Funding; UCB: Consultancy; MedImunne: Consultancy; ONO Pharmaceutical: Consultancy. Ghanima:Bayer: Honoraria, Research Funding; Amgen: Consultancy, Honoraria; Pfizer/BMS: Research Funding; Novartis: Consultancy, Honoraria, Research Funding. Cooper:Amgen: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees; Novartis: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees; Rigel: Consultancy, Membership on an entity's Board of Directors or advisory committees; Principia: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees. Viana:Novartis: Employment, Equity Ownership.
Article
Introduction: Immune thrombocytopenia (ITP) is an acquired autoimmune disorder, with an incidence rate of 20-40/million adults/year and an estimated prevalence in women of childbearing age of 24.5/million. Areas covered: Authors discuss management of ITP in pregnancy, treatment-related toxicity, delivery, neonatal thrombocytopenia and breastfeeding, and other women’s specific issues. Search of papers published between January 1990 and December 2019 was done on PubMed using combinations of the keywords below. Distinction between ITP and other thrombocytopenias in pregnancy is of paramount importance. The current belief (at variance with the past) that ITP is a relatively benign disease pregnancy, is emphasized. Expert opinion: The lack of randomized, prospective, controlled studies hampers evidence-based statements. Remarkably, ITP diagnosis is still one of exclusion, there are no clinical or laboratory criteria for prognosis and we still need more solid data on the risks related to neonatal thrombocytopenia. Corticosteroids and IVIG remain the mainstay of treatment, since rituximab, thrombopoietin-receptor agonists, fostamatinib may be toxic in pregnancy. Safety and efficacy of recombinant-human-thrombopoietin, available in China, requires confirmation studies. Quality of life and women-related toxicity of treatments in young girls, adults and elders are still an orphan area of investigation.
Article
Background Despite an increase in the number of therapies available to treat patients with immune thrombocytopenia (ITP), there are minimal data from randomized trials to assist physicians with the management of patients. Objective These evidence-based guidelines of the American Society of Hematology (ASH) are intended to support patients, clinicians, and other health care professionals in their decisions about the management of ITP. Methods In 2015, ASH formed a multidisciplinary guideline panel that included 8 adult clinical experts, 5 pediatric clinical experts, 2 methodologists with expertise in ITP, and 2 patient representatives. The panel was balanced to minimize potential bias from conflicts of interest. The panel reviewed the ASH 2011 guideline recommendations and prioritized questions. The panel used the Grading of Recommendations Assessment, Development and Evaluation (GRADE) approach, including evidence-to-decision frameworks, to appraise evidence (up to May 2017) and formulate recommendations. Results The panel agreed on 21 recommendations covering management of ITP in adults and children with newly diagnosed, persistent, and chronic disease refractory to first-line therapy who have non–life-threatening bleeding. Management approaches included: observation, corticosteroids, IV immunoglobulin, anti-D immunoglobulin, rituximab, splenectomy, and thrombopoietin receptor agonists. Conclusions There was a lack of evidence to support strong recommendations for various management approaches. In general, strategies that avoided medication side effects were favored. A large focus was placed on shared decision-making, especially with regard to second-line therapy. Future research should apply standard corticosteroid-dosing regimens, report patient-reported outcomes, and include cost-analysis evaluations.