Patient preferences and experiences regarding thrombopoietin-receptor agonists for immune thrombocytopenia in The Netherlands (TRAPeze Netherlands study).

OBJECTIVE: Identify patient experience and preference towards thrombopoietin-receptor agonists (TPO-RAs) in treatment of immune thrombocytopenia (ITP) in the Netherlands. METHODS: The Thrombopoietin-Receptor Agonist Patient experience (TRAPeze) survey used a discrete choice experiment (DCE) to elicit patient preferences and a patient burden survey (PBS) to evaluate the clinical and social impact of ITP. TRAPeze collected responses from 6th October to 19th November 2021. RESULTS: Seventy-six respondents completed the DCE: treatment preference appeared to be driven by method of administration (odds ratio [OR] 4.33; 95% confidence interval [CI] 2.88-6.52), frequency of dosing (OR 2.33; 95% CI 1.86-2.92) and drug-food interactions (OR 1.91; 95% CI 1.54-2.37). Respondents preferred therapies delivered orally over subcutaneous injection (OR 4.22; 95% CI 2.76-6.46), dosed once weekly over once daily (OR 2.37; 95% CI 1.58-3.54) and without food restrictions over with restrictions (OR 1.90; 95% CI 1.52-2.38). Sixty-nine respondents completed the DCE and PBS (mean [range] age 53 [19-83] years, 65% female). Seven incomplete PBS responses were excluded from analysis. Respondents were currently, or most recently, receiving eltrombopag (n = 43) or romiplostim (n = 26), of which 30% (n = 21/69) had previously received another TPO-RA. Loss (29%, n = 6/21) and lack (29%, n = 6/21) of response were the most common reasons for switching TPO-RA. Only 28% (n = 18/65) of respondents felt their TPO-RA increased energy levels. CONCLUSION: Patients preferred therapies delivered orally, dosed less frequently and without food restrictions. QoL of ITP patients on TPO-RAs can be improved; the burden analyses presented can inform future efforts towards this.

Treatment preferences towards thrombopoietin-receptor agonists for immune thrombocytopenia and experience of disease (TRAPeze): Italy cohort.

OBJECTIVE: Identify patient preference towards thrombopoietin-receptor agonists (TPO-RAs) and determine the clinical and social impact of immune thrombocytopenia (ITP) in Italy. METHODS: The Thrombopoietin-Receptor Agonist Patient experience (TRAPeze) survey collected responses from Italian residents from 17th January to 28th February 2022. TRAPeze utilized a discrete choice experiment (DCE) to elicit patient preferences towards TPO-RA attributes and a patient burden survey (PBS) to determine ITP disease characteristics and social impact. RESULTS: Seventy-six respondents completed the DCE, of which 69 completed both the DCE and PBS (mean [range] age 45 [18.0-73.0] years, 80% female). TPO-RA attributes with the greatest influence over respondent choice were method of administration (odds ratio [OR] 2.96; 95% confidence interval [CI] 2.16-4.06), drug-food interactions (OR 1.48; 95% CI 1.17-1.86) and frequency of dosing (OR 1.32; 95% CI 1.15-1.52). Respondents were more likely to prefer therapies administered orally over subcutaneous injection (OR 3.76; 95% CI 2.51-5.63), once weekly over once daily (OR 1.83; 95% CI 1.26-2.65), and therapies without food restrictions over with restrictions (OR 1.58; 95% CI 1.17-2.14).The most frequently reported symptoms were bruising (82%), petechiae (65%) and fatigue (64%). Most respondents (84%) felt ITP impacted familial relationships and 71% of employed respondents reported fatigue influencing their ability to work, with 31% reducing working hours. CONCLUSION: Although responses indicated a moderate perception of general health, ITP clearly impacted respondent work and social life. Our findings demonstrate respondents preferred TPO-RAs delivered orally, with less frequent dosing and without food restrictions.

Psychometric Evaluation of ITP Life Quality Index (ILQI) in a Global Survey of Patients with Immune Thrombocytopenia.

INTRODUCTION: Immune thrombocytopenia (ITP) is an autoimmune disorder caused by immunologic destruction of otherwise normal platelets. Patients and physicians differ in their views pertaining to the limitations imposed on patients' daily lives by ITP and its treatment. Poor understanding of ITP symptoms can result in misdiagnosis and complex treatment patterns, and affect patient health-related quality of life (HRQoL). The ITP Life Quality Index (ILQI) is a 10-item patient-reported outcome measure developed for clinical practice to aid discussions between patients and physicians. This research aimed to validate the psychometric properties of the ILQI using data collected in the ITP World Impact Survey (I-WISh). METHODS: I-WISh data containing responses to the ILQI from 1507 patients with ITP across 13 countries worldwide was subject to psychometric analysis to evaluate the structure, reliability and validity of the ILQI and assess scoring cut-offs. RESULTS: The ILQI has an overarching unidimensional structure, supporting a total score including all 10 items. Reliability was supported (Cronbach's alpha = 0.90). ILQI scores monotonically increased with ITP severity. ILQI scores correlated with measures of fatigue and emotional well-being, supporting construct validity. Differential item functioning (DIF) analyses showed that ILQI item responses were interpreted similarly between the USA and other Western countries. It was suggested that previous clinical cut-off score of 20 for "impaired HRQoL" was reduced to 17 and a cut-off of 23-25 (rather than 30) was suggested to assess "significantly impaired HRQoL". CONCLUSION: The validity and reliability of the ILQI to assess HRQoL of patients with ITP is supported. The revised cut-off scores for the ILQI will aid patient-centric decision-making.

Patient preferences and experiences regarding thrombopoietin-receptor agonists for immune thrombocytopenia in the United Kingdom and Ireland (TRAPeze UK & IE study).

OBJECTIVES: To establish the experiences with and preferences towards existing thrombopoietin-receptor agonist (TPO-RA) treatments of individuals with immune thrombocytopenia (ITP) in the UK and Ireland, based on treatment attributes. METHODS: Responses from UK and Ireland individuals with ITP were collected in a pan-European online survey (TRAPeze, [Thrombopoietin-Receptor Agonist Patient experience survey]) from 18 September 2020 to 18 February 2021. TRAPeze was a survey of treatment preference regarding TPO-RAs (using a discrete choice experiment design), participant demographics, disease characteristics, treatment history, overall satisfaction with therapy, direct healthcare resource utilization and wider social impact. RESULTS: The survey was completed by 32 UK respondents. Characteristics with the greatest influence on preference towards TPO-RA treatments were method of administration (odds ratio (OR) 5.6, 95% confidence interval (CI) 3.2-10.1) and drug-food interactions (OR 3.2, 95% CI 1.8-5.7). Particularly, participants were more likely to select an oral tablet over a subcutaneous injection (OR 7.4, 95% CI 3.6-15.1) and a treatment without food restrictions rather than with food restrictions (OR 3.6, 95% CI 1.8-6.8). CONCLUSION: This is the first study to quantify the preference of individuals with ITP towards TPO-RA treatment attributes and demonstrates preference for orally administered treatments, without drug-food interactions.

Qualitative study to support the content validity of the immune thrombocytopenia (ITP) Life Quality Index (ILQI).

Immune thrombocytopenia (ITP) is an acquired immune-mediated disorder. Bleeding is the primary symptom that presents in varying severities. ITP has a negative impact on health-related quality of life (HRQoL). The ITP Life Quality Index (ILQI) was developed as a 10-item patient-reported outcome measure to assess impact on HRQoL in ITP. The objective of the present study was to confirm the content validity of the ILQI with a qualitative interview study in the UK involving 15 adult participants with ITP. Combined concept elicitation (CE) and cognitive debriefing (CD) interviews were conducted to explore the symptoms and impacts associated with ITP and confirm content validity of the draft ILQI. The CE phase elicited 14 ITP symptom concepts, including: bruising (all 15 patients, 100%), fatigue (14, 93·3%) and bleeding gums/blood blisters (13, 86·7%). Impacts included decreased ability to participate in sport (all 15 patients, 100%) and anxiety (12, 80%). The CD phase resulted in an adjustment to the ILQI recall period from 1 week to 'the past month'. Updates were made to improve relevance and response options. The qualitative interviews support the content validity of the ILQI and confirm that the concepts assessed are relevant and consistently understood and interpreted by adult patients with ITP.

Immune thrombocytopenia (ITP) World Impact Survey (iWISh): Patient and physician perceptions of diagnosis, signs and symptoms, and treatment.

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, for example, 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.

Immune thrombocytopenia (ITP) World Impact Survey (I-WISh): Impact of ITP on health-related quality of life.

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. The 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. Note, 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.