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How Do Patients and Physicians Perceive Immune Thrombocytopenia (ITP) As a Disease? Results From Indian Analysis of ITP World Impact Survey (I-WISh)

  • Vivekananda Institute of Medical Sciences Kolkata

Abstract and Figures

Background: 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). 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 (15/19;79%), anxiety surrounding unstable platelet counts (17/28;61%), and fatigue (27/46;59%); these were also the key symptoms they wanted to be resolved. In contrast, physicians perceived petechiae (19/21;90%), bleeding-from-gums [(8/21;86%), and purpura (16/21;76%) as the most common symptoms. While the important treatment goals for patients were healthy blood counts (42/65;65%), improved QoL (35/65;54%), and prevention of worsening of ITP (33/65;51%), physicians’ goals were reduction in spontaneous bleeding (17/21;81%]), better QoL (14/21;67%]), and symptom improvement (9/21;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. Educating physicians on aspects of ITP beyond bleeding, and highlighting patients’ under-recognized symptoms/needs through support-systems may bring about a meaningful change.
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How Do Patients and Physicians Perceive Immune
Thrombocytopenia (ITP) As a Disease? Results
From Indian Analysis of ITP World Impact Survey (I-
Prantar Chakrabarti
Vivekananda Institute of Medical Sciences
Biju George
CMC Vellore: Christian Medical College Vellore
Chandrakala Shanmukhaiah
KEM Hospital and Seth G S Medical College: King Edward Memorial Hospital and Seth Gordhandas
Sunderdas Medical College
Lalit Mohan Sharma
MG Medical College
Shashank Udupi
Novartis Healthcare Pvt Ltd
Waleed Ghanima ( )
Ostfold Hospital: Sykehuset Ostfold HF
Keywords: Disease management, health-related quality of life (HRQoL), immune thrombocytopenia (ITP),
India, ITP World Impact Survey (I-WISh), ITP symptoms
License: This work is licensed under a Creative Commons Attribution 4.0 International License. 
Read Full License
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Background: 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).
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 (15/19;79%), anxiety surrounding unstable platelet counts (17/28;61%), and
fatigue (27/46;59%); these were also the key symptoms they wanted to be resolved. In contrast,
physicians perceived petechiae (19/21;90%), bleeding-from-gums [(8/21;86%), and purpura (16/21;76%)
as the most common symptoms. While the important treatment goals for patients were healthy blood
counts (42/65;65%), improved QoL (35/65;54%), and prevention of worsening of ITP (33/65;51%),
physicians’ goals were reduction in spontaneous bleeding (17/21;81%]), better QoL (14/21;67%]), and
symptom improvement (9/21;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. Educating physicians on aspects of
ITP beyond bleeding, and highlighting patients’ under-recognized symptoms/needs through support-
systems may bring about a meaningful change.
Immune thrombocytopenia (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 identied 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,[5–7] assessment and treatment of
HRQoL parameters is challenging. 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 bleeding.[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
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non-infectious causes of thrombocytopenia 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 physician
perceptions of disease and symptoms; impact of ITP on patient QoL, daily activities, and work; and
existing support systems for ITP and its management.
Survey Participants and Study Conduct
The I-WISh India-specic analysis is based on data collected as part of I-WISh 1.0, a cross-sectional
survey of adult patients (age 18 years) with ITP and hematologists or hemato-oncologists who treat
patients with ITP. The 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 support networks and physicians who were requested
to disseminate the surveys to patients. Physician surveys were emailed by local eldwork agencies. The
respondents took 30 minutes to complete the questionnaire. Fully deidentied respondent information
was collated and aggregated by local eldwork partners such that the surveys were unlinked and
anonymized. Surveys and details of the survey methods, including how patients and physicians were
identied, have been outlined in the supplementary material and published previously.[8, 9]
To understand the level of agreement that the respondents 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 was considered
“severe”. 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]
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.
Statistical Analyses
Patient and physician survey data were analyzed separately using descriptive statistics. There were no
prespecied hypotheses associated with these exploratory surveys, and as such, data was summarized
Demographic Characteristics and the Diagnostic Journey of
Patients With ITP
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Overall, 21 physicians and 65 patients completed the survey questionnaire between December 2017 and
August 2018. Patients were recruited by either experienced physicians treating ITP (64/65 [98%]) or
patient association groups (1/65 [2%]). Accurate 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).
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Table 1
Patient/physician demographic characteristics and patient diagnostic pathways
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 rst 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 conrmed 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%)
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N = 65
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 qualication
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%)
Chronic lymphocytic leukemia 8 (38%)
Human immunodeciency virus 7 (33%)
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N = 65
Investigation rates (asymptomatic vs high symptom burden)
Spleen evaluation 12 (57%) vs 16
Coomb’s test 5 (24%) vs 13
H. pylori
3 (14%) vs 8
Computed tomography scan 1 (5%) vs 7 (33%)
Platelet specic 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%)
The mean (standard deviation [SD]) age of the patients was 33 (12.62) years, with 39/65 (60%) patients
being male. The symptom burden was moderate to high in 24/57 (42.1%) patients, of whom 17/24 (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 conrmed by physicians specialized in the
management of ITP in 56/65 (86%) patients. Overall, 21/65 (32%) patients expressed a delay in ITP
diagnosis, thereby leading to severe anxiety ( 5 on the Likert scale) in 8/21 (38%) patients.
All physicians included in the survey were either hematologists (n = 13) or hemato-oncologists (n = 8).
More than half of the physicians who participated in the survey (12/21 [57%]) practiced in a private
setting. Of the average caseload, about 87/625 (13.9%) were patients with ITP. ITP was rated as a
“somewhat less important” condition by 14/21 (67%) physicians. Nearly one-fourth (5/21 [24%]) of the
physicians perceived that 26%-50% of patients were misdiagnosed.
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Patient and Physician Perception of ITP Symptoms and
Patient perspective
The most commonly reported symptoms at diagnosis were heavy menstrual bleeding (19/26 [73%]),
fatigue (46/65 [71%]), petechiae (39/65 [60%]), hematoma (30/65 [46%]), and anxiety surrounding
unstable platelet counts (28/65 [43%]). The 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 (35/65 [54%]), heavy menstrual bleeding (10/26 [38%]), anxiety surrounding unstable platelet
counts (23/65 [35%]), petechiae (19/65 [29%]), and hematoma (11/65 [17%]) (Fig.1A). Menorrhagia
(15/19 [79%]), anxiety surrounding unstable platelet counts (17/28 [61%]), and fatigue (27/46 [59%]) were
the most commonly reported severe symptoms at diagnosis (considering symptoms reported by at least
15 patients) (Fig.1A). The key symptoms that patients wanted to be resolved included fatigue (27/65
[42%]), heavy menstrual bleeding (10/26 [38%]), and anxiety surrounding unstable platelet counts (21/65
[32%]) (Supplementary Fig.1).
Physician perspective
The most common signs and symptoms reported by physicians, based on the inputs received from their
patients, were similar at diagnosis vs survey completion; these included petechiae (19/21 [90%] vs 19/21
[90%]), bleeding from gums (18/21 [86%] vs 18/21 [86%]), purpura (16/21 [76%] vs 18/21 [86%]),
epistaxis (14/21 [67%] vs 13/21 [62%]), and heavy menstrual bleed (11/21 [52%] vs 12/21 [57%])
(Fig.1B). According to physicians, hematuria, melena, or rectal bleed (17/21 [81%]); profuse bleeding
during surgery (16/21 [76%]); menorrhagia (14/21 [67%]); anxiety surrounding unstable platelet counts
(12/21 [57%]); and hematoma (12/21 [57%]) could have a major negative impact on patient HRQoL
(scored  5 on the Likert scale). According to physicians, about 37% patients experienced fatigue, and the
severity was considered as low ( 4 on the Likert scale) in most patients (17/21 [81%]). Fatigue was
considered to be very severe by 12/21 (57%), 10/21 (48%), and 8/21 (38%) physicians when platelet
counts were below 10×109/L, 30×109/L, and 40×109/L, respectively (Supplementary Fig.2). Overall,
fatigue was considered as a major concern by only 7/21 (33%) physicians.
Impact of ITP on QoL
Based on the ILQI scores, the parameters that signicantly had a negative impact on patient QoL very
often (more than half of the time) were work life/studies (19/50 [38%]), absence of work/education
(16/48 [33%]), and energy levels (19/65 [29%]) (Fig.2A).
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The overall impact on emotional well-being was scored  5 on the Likert scale by 25/66 (38%) patients,
and the top 4 reported reasons with a severe impact were anxiety surrounding unstable platelet counts
(38/65 [58%]), importance of stable platelet counts (38/65 [58%]), uctuation in platelet counts for no
apparent reason (34/65 [52%]), and frustration with ITP symptoms (31/64 [48%]) (Supplementary Fig.3).
Overall, 60/65 (92%) patients did not receive any professional support, of whom 20/60 (33%) expressed a
desire for additional support.
ITP adversely affected the work and nancial situation of patients, with 10/38 (26%) patients reducing
their work hours and 9/36 (25%) seriously considering a reduction in their work hours. Patients reported
an average of 11.1 hours of missed work per week due to the impact of ITP (Supplementary Fig.4). The
total monthly outofpocket expense for a patient with ITP was $211 (~ 16 000 INR), with medicines
accounting for more than 60% of this expense ($132 [~ 9 600 INR]). Patients also spent an average of 6.1
hours/month traveling for their appointments.
Overall, 39/65 (60%) patients expressed the need for support (either ‘rarely’, ‘sometimes’, or ‘often’) for an
average of 33.7 hours/week; homemaking (27/39 [69%]), transportation (26/39 [67%]), healthcare (25/39
[64%]), and management of nances (20/39 [51%]), were the primary reasons for which support was
requested. The key support providers were parents (19/39 [49%]) and spouses (9/39 [23%]).
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 4/20 (21%) of the patients and 5/20 (25%) of the physicians felt that ITP had negatively
impacted patients’ relationship with their spouses. Overall, interference of ITP in the level of patients’
physical activity was reported as severe by 6/20 (30%) of physicians, and 18/20 (90%) physicians 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 (8/18 [44%]) and concerns around increased risk of
bleeding impacting travel plans (9/20 [45%]) was reported by 45% physicians.
Almost all physicians (20/21 [95%]) did not use any QoL tool, but expressed their desire to use a patient
self-assessment questionnaire (12/20 [60%] would use it during every consultation, and 7/20 [35%] would
use it every 6 months). Most physicians (16/21 [76%]) expressed that use of a mobile-based app would
help in recording patient QoL, while 10/21 (48%) physicians expressed that combining paper- and mobile
app-based approach would be the preferred method to use. No major differences were observed in the
response assessments of physicians based on their workload.
Management of Goals and Treatment Options in ITP
ITP diagnosis to treatment required an average of 0.9 months, with over half of the patients (34/65 [52%])
undergoing a period of “wait and watch.” The important treatment goals for patients were healthy blood
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counts (42/65 [65%]), improvement in QoL (35/65 [54%]), prevention of episodes on worsening of ITP
(33/65 [51%]), reduction in spontaneous bleeding (18/65 [28%]), and an overall improvement in
symptoms (17/65 [26%]) (Fig.3A). A majority of patients (41/65 [63%]) strongly agreed that their current
treatment was helping them reach their treatment goals.
A once-daily oral pill was preferred by 49/65 (75%) patients. At the time of survey completion, the most
frequently administered treatments were corticosteroids (38/65 [58%]), androgens (9/65 [14%]), anti-CD20
(9/65 [14%]), thrombopoietin receptor agonists (TPO-RAs; 8/65 [12%]), and other immunosuppressants
(7/65 [11%]); the average duration of these medications was 4.7 months. When the symptom burden was
low, most patients reported undergoing treatment with corticosteroids (27/33 [82%]), and as the burden
increased to moderate and above, corticosteroid use decreased slightly (17/24 [71%]). The use of
androgens (8/33 [24%] to 11/24 [46%]), anti-CD20 (9/33 [27%] to 11/24 [46%]), and TPO-RAs (3/33 [9%]
to 7/24 [29%]) increased with increasing symptom burden. Data on treatment satisfaction were based on
a low patient number (data not presented here).
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 monitoring was done more routinely in newly diagnosed patients (every 15 days)
compared with patients with chronic ITP (every 1.7 months). The major treatment goals for physicians
were reduction in spontaneous bleeding (17/21 [81%]), better QoL (14/21 [67%]), symptom improvement
(9/21 [43%]), healthy blood counts (6/21 [29%]), and reduction in fatigue symptom (5/21 [24%]). Nearly
90% of physicians (18/21 [86%]) believed that they discussed and agreed on treatment goals with their
patients, and 14/21 (67%) physicians aimed to limit the immunosuppressive effect of the treatment
The most important attributes while making treatment decisions for patients with ITP were offering cure
or sustained 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 physicians preferred oral treatment
options as the rst line of treatment. Corticosteroids (19/21 [90%]) and intravenous immunoglobulins
(IVIgs; 16/21 [76%]) were the preferred treatments in newly diagnosed patients with ITP. TPO-RAs (19/21
[90%]) and anti-CD20 (17/21 [81%]), followed by androgens (16/21 [76%]), were the preferred treatment
options in patients with persistent and chronic ITP (Fig.4; Supplementary Fig.5).
For patients relapsing for the rst time, corticosteroids (14/21 [67%]), followed by androgens (8/21 [38%])
and IVIgs/anti-CD20 (7/21 [33%] each), were preferred, while during second relapse other
immunosuppressants (9/21 [43%]) followed by corticosteroids (8/21 [38%] were preferred; by the third
relapse, TPO-RAs (11/21 [52%]), followed by anti-CD20 therapy (8/21 [38%]), were the preferred treatment
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Based on physician perspective, patients treated with TPO-RAs had the least incidence of side effects. A
total of 13/21 (62%) physicians agreed that they were satised with the current treatment options
available. Lack of ecacy (21/21 [100%]), followed by side effects (19/21 [90%]), and cost/coverage
(17/21 [81%]), was the most important reason for a change in therapy.
Patient and Physician Relationship
When compared with patients, a lower proportion of physicians were completely satised 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, 9/21 (43%) physicians expressed that patients faced at
least some level of diculty in accessing information. About half of the physicians indicated that they
provided disease management–related information in a leaet format explaining the contents of the
leaet (9/21 [43%]). From the patient perspective, 40/65 (62%) had not received any information from
their HCP. For patients who recieved information from their HCP, it was either through a leaet (10/25
[40%]) or through HCPs showing the website content during consultation (8/25 [32%]) or by being
directed to the website for accessing information about the disease (7/25 [28%]). A large proportion of
patients did not have any contact with patient support groups (61/65 [94%]).
Among 64/65 (98%) patients 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.
To the best of our knowledge, this questionnaire-based survey is the rst 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 number 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 doctortopopulation 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
Commission, and endorsed by WHO.[7, 13] Moreover, in India, the population-to-specialist ratio is high,[7]
which further increases the patient burden of hematologists and hematooncologists. With such a high
patient burden and an estimated average primary care physician consultation time of ~ 2 minutes in
India,[5] physicians tend to primarily 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.
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Heavy menstrual bleeding, fatigue, and anxiety surrounding unstable platelet counts were predominantly
reported as severe by patients at both diagnosis and survey completion. Physician perspectives on the
frequency 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. This
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.[14] A high frequency of
menorrhagia, irondeciency anemia, and other nutritional anemias found among Indian patients could be
an important contributing factor for fatigue.[15–17] Similar to fatigue events, menorrhagia also impacts a
number of HRQoL measures[18, 19] and was reported by a majority of women (> 70%) in this analysis. The
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).[20, 21] 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
frequently (doctor shopping), it could result in an overall increase in healthcare cost. Therefore,
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 61–16% from diagnosis to survey completion. This 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.[22]
The assessment and improvement of HRQoL parameters 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.[23] In this subgroup 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 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, inability 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 bleeding and taking medications
abroad. The 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.[23–25] Although a few public health insurance programs in
India cover nonmedical expenditure, such as transportation, lodging, and food costs, for patients and
caregivers, there is no provision for incurring the loss of pay suffered by patients or their spouses,[25]
thereby increasing the socioeconomic burden of the disease. The lack of patient support groups and
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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 treating 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.[26] However, in India, engagement in patient support groups is low. The major constraints in
ensuring higher engagement rates could be the lack of awareness, lack of time, or anxiety around
discussing the negative aspects of the disease publicly.[26] There is a need to consider a holistic approach
toward assessment of symptom burden and impact of ITP on QoL in routine clinical practice in India.
Physician ability to effectively and compassionately communicate the nature of disease and
management options is important to build trust in a patient–physician relationship, and shared
decisionmaking is a key element in improving patient–physician communication.[27] Although nearly 90%
of physicians in our study mentioned that they had included their patients’ perspective during
decisionmaking, the implementation of a participatory decisionmaking model in ITP, which has been in
place for cancer management for a considerable period of time,[28] may not be feasible in the Indian
context. This could be due to the existing gaps in patient knowledge of the disease and effectiveness of
available treatment options.[29] 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 ensuring 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 important treatment goals for both patients and
physicians, underlining the importance of assessing HRQoL among patients with ITP. This 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 physicians.[9]
Overall, the survey data outcomes and driven conclusions must be interpreted with caution, given the
small sample size of the respondents, specically the patient group. 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 considered 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.
Based on the overall respondent assessment, the study highlights the need for education/training on all
aspects of disease management—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.
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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
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
Availability of data and materials:Not applicable
Competing interests:Dr. Shashank Udupi is a full-time employee of Novartis Healthcare Private Limited.
None of the other authors had any conict of interest to declare.
Funding:This study was sponsored by Novartis Pharmaceuticals Corporation.
Authors' contributions:All authors contributed in the interpretation of data, writing and critically reviewing
the manuscript, and approval of the nal draft.
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.
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Figure 1
Frequency and severity of symptoms of ITP at diagnosis and survey completion – Patient and physician
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Figure 2
Impact of ITP on QoL – Patient and physician perspective
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Figure 3
Treatment goals
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Figure 4
Management of ITP – Prescribed treatments
Supplementary Files
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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.
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.
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.
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.
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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.
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.
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Over the last decade, there have been numerous developments and changes in treatment practices for the management of patients with immune thrombocytopenia (ITP). This article is an update of the International Consensus Report published in 2010. A critical review was performed to identify all relevant articles published between 2009 and 2018. An expert panel screened, reviewed, and graded the studies and formulated the updated consensus recommendations based on the new data. The final document provides consensus recommendations on the diagnosis and management of ITP in adults, during pregnancy, and in children, as well as quality-of-life considerations.
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.
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.
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.