Patient survey in immune thrombocytopenia (<scp>ITP</scp>): Identifying unmet needs related to treatment and disease control in patients living in the United States
Immune thrombocytopenia (ITP) is a chronic disease with primary therapeutic goals of platelet count recovery to safe levels to minimize active/future bleeding, alongside easing additional symptoms negatively impacting overall patient well-being with consequent improvement in physical fatigue/energy levels, daily/work-related activities and social/emotional health. Documentation of this rare disease is important for evaluating real-world experiences in treatment satisfaction, expectations and unmet needs in disease management. This cross-sectional, real-world evidence survey was conducted from 9 February 2023 to 4 April 2023, by the Platelet Disorder Support Association and Sanofi in US adults diagnosed with ITP for ≥1 year. Results showed that although most patients receive and adhere to treatment, lack of sustained efficacy fuels a need for more effective therapy. Patients desired long-term ITP control and were most concerned with increasing/stabilizing platelet counts and decreasing fatigue and bleeding severity. Better treatment was needed to ease burdensome health-related quality of life symptoms, especially physical fatigue and anxiety, and decrease effects on daily life and activities. Additionally, involvement in shared decision-making and engagement in educational, digital healthcare and psychological support were preferred. Overall, ITP patients desired long-term disease management options that improved platelet counts, minimized bleeding and reduced physical fatigue and anxiety, while also engaging in patient support options.
Authors
- Nichola Cooper (ORCID: https://orcid.org/0000-0002-9845-8292)
- Stephen J. Morgan (ORCID: https://orcid.org/0000-0003-4496-1121)
- Matias Cordoba (ORCID: https://orcid.org/0009-0007-8882-8863)
- E. P. Reineke (ORCID: https://orcid.org/0000-0003-0748-0898)
- Terry Gernsheimer (ORCID: https://orcid.org/0000-0002-2671-5608)
- Imène Gouia
- Caroline Kruse (ORCID: https://orcid.org/0000-0003-4259-0988)
- Jean‐Pascal Roussy
- Lisa Anne Schmitt (ORCID: https://orcid.org/0000-0002-0000-1770)
- Julie Laurent (ORCID: https://orcid.org/0000-0002-1705-7631)
- Marleni Arvelo‐Saillant
Institutions
- GTx (United States) (US)
- University of Washington (US)
- Dignity Health (US)
- Sanofi (France) (FR)
- AVEO Oncology (United States) (US)
- Cambridge Scientific (United States) (US)
- Fred Hutch Cancer Center (US)
- Institute of Public Affairs (PL)
- Ohio Physical Therapy Association (US)
- Imperial College London (GB)
Publication Details
- Journal
- British Journal of Haematology
- Published
- 2025-08-17
- DOI
- https://doi.org/10.1111/bjh.20257
- Citations
- 2
- Primary Topic
- Platelet Disorders and Treatments
- Type
- article
- Field-Weighted Citation Impact
- 3.54
Funders
- Sanofi