{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,27]],"date-time":"2026-08-27T06:29:04Z","timestamp":1787812144501,"version":"build-2784847793"},"reference-count":42,"publisher":"Public Library of Science (PLoS)","issue":"4","license":[{"start":{"date-parts":[[2022,4,21]],"date-time":"2022-04-21T00:00:00Z","timestamp":1650499200000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"name":"Novartis Pharma Schweiz AG"}],"content-domain":{"domain":["www.plosone.org"],"crossmark-restriction":false},"short-container-title":["PLoS ONE"],"abstract":"<jats:sec id=\"sec001\"><jats:title>Aims of the study<\/jats:title><jats:p>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.<\/jats:p><\/jats:sec><jats:sec id=\"sec002\"><jats:title>Method<\/jats:title><jats:p>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.<\/jats:p><\/jats:sec><jats:sec id=\"sec003\"><jats:title>Results<\/jats:title><jats:p>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%).<\/jats:p><\/jats:sec><jats:sec id=\"sec004\"><jats:title>Conclusions<\/jats:title><jats:p>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.<\/jats:p><\/jats:sec>","DOI":"10.1371\/journal.pone.0267342","type":"journal-article","created":{"date-parts":[[2022,4,21]],"date-time":"2022-04-21T17:30:23Z","timestamp":1650562223000},"page":"e0267342","update-policy":"https:\/\/doi.org\/10.1371\/journal.pone.corrections_policy","source":"Crossref","is-referenced-by-count":18,"title":["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"],"prefix":"10.1371","volume":"17","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-7757-4753","authenticated-orcid":true,"given":"Alicia","family":"Rov\u00f3","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nathan","family":"Cantoni","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kaveh","family":"Samii","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Axel","family":"R\u00fcfer","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Giedre","family":"Koenen","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sandra","family":"Ivic","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Davide","family":"Cavanna","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Rudolf","family":"Benz","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"340","published-online":{"date-parts":[[2022,4,21]]},"reference":[{"issue":"11","key":"pone.0267342.ref001","doi-asserted-by":"crossref","first-page":"2386","DOI":"10.1182\/blood-2008-07-162503","article-title":"Standardization of terminology, definitions and outcome criteria in immune thrombocytopenic purpura of adults and children: report from an international working group","volume":"113","author":"F Rodeghiero","year":"2009","journal-title":"Blood"},{"key":"pone.0267342.ref002","doi-asserted-by":"crossref","first-page":"880","DOI":"10.3389\/fimmu.2018.00880","article-title":"Emerging concepts in immune thrombocytopenia.","volume":"9","author":"M Swinkels","year":"2018","journal-title":"Front Immunol."},{"issue":"21","key":"pone.0267342.ref003","doi-asserted-by":"crossref","first-page":"2829","DOI":"10.1182\/blood-2017-03-754119","article-title":"Clinical updates in adult immune thrombocytopenia","volume":"129","author":"MP Lambert","year":"2017","journal-title":"Blood"},{"issue":"71","key":"pone.0267342.ref004","doi-asserted-by":"crossref","first-page":"3","DOI":"10.1111\/j.1600-0609.2008.01206.x","article-title":"Immune thrombocytopenic purpura: epidemiology and implications for patients","author":"M. 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