Real-world impact of primary immune thrombocytopenia and treatment with thrombopoietin receptor agonists on quality of life based on patient-reported experience: Results from a questionnaire conducted in Switzerland, Austria, and Belgium

Abstract

Aims of the study Thrombopoietin receptor agonists (TPO-RAs) are approved for immune thrombocytopenia (ITP), but their impact on health-related quality of life (HRQoL) remains poorly investigated in clinical practice. This observational study aimed to gain insight into real-world patient-reported experiences of the burden of ITP and TPO-RAs. Method An online questionnaire of closed questions was used to collect views of patients with primary ITP from Switzerland, Austria, and Belgium, between September 2018 and April 2020. Results Of 46 patients who completed the questionnaire (total cohort), 41% were receiving TPO-RAs. A numerically higher proportion of patients reported being free from symptoms at the time of the questionnaire (54%) than at diagnosis (24%), irrespective of treatment type. Bleeding, the most frequently reported symptom at diagnosis (59%), was reduced at the time of the questionnaire (7%). Conversely, fatigue was reported by approximately 40% of patients at both diagnosis and the time of the questionnaire. Having a normal life and their disease under control was reported by 83% and 76%, respectively, but 41% were worried/anxious about their condition. Nearly 50% reported that ITP impaired their engagement in hobbies/sport or energy levels and 63% reported no impact on employment. When stratified by TPO-RA use, bleeding was better controlled in those receiving TPO-RAs than not (0% vs 11%). A numerically lower proportion receiving TPO-RAs than not reported worry/anxiety about their condition (16% vs 59%) and shifting from full-time to part-time employment (11% vs 22%). Similar proportions were satisfied with their therapy whether they were receiving TPO-RAs or not (89% vs 85%). Conclusions Many factors affect HRQoL in patients with ITP. Of patients receiving TPO-RAs, none experienced bleeding at the time of the questionnaire; they also showed a more positive perspective for some outcomes than those not using TPO-RAs. However, fatigue was not reduced by any treatment.

Health-related quality of life and complications of corticosteroid treatment in patients with immune thrombocytopenia in two teaching hospitals in Ethiopia: A cross-sectional study

“…In this study, the total impact of ITP on HRQoL is 35.41 ± 9.27, and the impact of ITP on work or study is 50.01 ± 2.17, which is greater than the impact of ITP on daily life (31.69 ± 7.38); this is consistent with the ITP World Impact Survey data (8, 39) and survey data reported from Switzerland, Austria, and Belgium (40). Our study is also similar to the studies conducted in China (41), the United States of America (11), and Serbia (9), in which physical function was more impaired than in the other domains of HRQoL.…”

Spotlight on eltrombopag concentration in pediatric immune thrombocytopenia: A single‐center observational study in China

“…During the treatment and management operations, personalized medicine should be prioritized, with consideration for patients' preferences and encouragement for their involvement in the decision‐making process. Studies related to the treatment and management of chronic ITP showed, that both the early administration and prolonged use of effective and well‐tolerated TPO‐RAs have the potential to significantly control bleeding, decrease anxiety about the condition, reduce hospitalization and therapy costs, and improve HRQoL in patients with ITP 26,27 . Other studies showed the possible clinical benefit that can be obtained by combining conventional treatment with TPO‐RAs, in terms of increased response rates and reduced mortality 28 .…”

Burden of immune thrombocytopenia (ITP): Special considerations for refractory ITP

“…Results did not document a reduction of fatigue by the TPO-RAs Romiplostim and Eltrombopag-although they increased platelet counts. 30 In a meta-analysis including nine randomized controlled trials assessing the efficacy and QOL of Romiplostim in adults and children with ITP, no statistically significant difference in HRQoL was found in the study population compared to the control group. 31 Although TPO-RAs increased platelet counts, they did not seem to improve HRQoL.…”