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Observational Study
. 2022 Apr 21;17(4):e0267342.
doi: 10.1371/journal.pone.0267342. eCollection 2022.

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

Alicia Rovó  1 , Nathan Cantoni  2 , Kaveh Samii  3 , Axel Rüfer  4 , Giedre Koenen  5 , Sandra Ivic  5 , Davide Cavanna  5 , Rudolf Benz  6
Affiliations
Observational Study

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

Alicia Rovó et al. PLoS One. .

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.

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Conflict of interest statement

ARovó was not paid for her work on this study. She has acted as a consultant, taken part in advisory boards, and received honoraria and research funding from Novartis. She has also acted as a consultant, taken part in advisory boards, and received honoraria and/or research funding from Alexion, AstraZeneca, Celgene, CSL Behring, and OrPha Swiss. NC was not paid for his work on the study. He has acted as a consultant for Celgene and Novartis and has received honoraria (consultancy honoraria and travel grants) from AbbVie, Alexion, Amgen, BMS, Celgene, Gilead, Incyte, Janssen-Cilag, Novartis, OrPha Swiss, Pfizer, Roche, Sandoz, Sanofi-Aventis, Shire, Takeda, as well as research funding from AstraZeneca, CSL Behring, Novartis, Pierre Fabre, and Shire (Baxter Oncology). KS was not paid for his work on this study. He has acted as a consultant and taken part in advisory boards organized by Novartis. He has also taken part in advisory boards organized by Alexion, Celgene, Janssen-Cilag, OrPha Swiss, and Takeda. ARüfer was not paid for his work on the study. He has taken part in advisory boards organized by Amgen, BMS/Celgene, Janssen-Cilag, Novartis, OrPha Swiss, and Takeda. GK was an employee of Novartis Pharma Schweiz AG at the time of the study, and is now an employee of Novartis AG. SI, and DC are employees of Novartis Pharma Schweiz AG. RB was not paid for his work on the study. He has acted as a consultant, taken part in advisory boards, and received research funding from Novartis. He has also taken part in advisory boards and received honoraria and travel support to educational events from AbbVie, Alexion, Celgene, OrPha Swiss, and Takeda. This does not alter our adherence to PLOS ONE policies on sharing data and materials.

Figures

Fig 1
Fig 1. Symptoms at diagnosis and at the time of the questionnaire (total cohort).
Data in the figure are presented as the proportion of patients reporting each symptom within the total cohort (N = 46). Patients indicated their symptoms through a multiple-choice question and could select more than one symptom. For patients who indicated they experienced bleeding, they were prompted in a subsequent question to indicate the type of bleeding that they had experienced. †Bleeding included cutaneous bleeding (purpura, petechiae, haematoma, prolonged bleeding from cuts), mucosal bleeding (nosebleeds, heavy menstrual bleeding), and internal bleeding (organ bleeding, blood in urine or stool). Of 27 patients who reported bleeding at diagnosis, 26 reported cutaneous bleeding (96%), 18 reported mucosal bleeding (67%), and 3 reported internal bleeding (11%). Of the 3 patients who reported bleeding at the time of the questionnaire, 3 reported cutaneous bleeding (100%), 2 reported mucosal bleeding (67%), and 1 reported internal bleeding (33%).
Fig 2
Fig 2. Symptoms at the time of questionnaire according to TPO-RA usage.
Data in the figure are presented as the proportion of patients reporting each symptom within the total cohort (N = 46). Patients indicated their symptoms through a multiple-choice question and could select more than one symptom. Patients who responded that they had experienced bleeding were prompted in a subsequent question to indicate the type of bleeding that they had experienced. †Bleeding included cutaneous bleeding (purpura, petechiae, haematoma, prolonged bleeding from cuts), mucosal bleeding (nosebleeds, heavy menstrual bleeding), and internal bleeding (organ bleeding, blood in urine or stool). Of the three patients who reported bleeding in the cohort not using TPO-RAs, three reported cutaneous bleeding (100%), two reported mucosal bleeding (67%), and one reported internal bleeding (33%). Abbreviation: TPO-RA, thrombopoietin receptor agonist.
Fig 3
Fig 3. Patients’ feelings about ITP in the total cohort.
Patients could select a single response per statement. Data are presented as the proportion of patients per category for each statement (N = 46 for all figures). Abbreviation: ITP, immune thrombocytopenia.
Fig 4
Fig 4. Impact of ITP on daily activities in the total cohort.
Patients could select a single response per statement. Data are presented as the proportion of patients per category for each statement (N = 46).
See this image and copyright information in PMC

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