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.

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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

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article

Patient survey in immune thrombocytopenia (ITP): Identifying unmet needs related to treatment and disease control in patients living in the United States

Nichola Cooper, Stephen J. Morgan, Matias Cordoba, E. P. Reineke et al.
2 citations
British Journal of Haematology
Platelet Disorders and Treatments
3.54
article

Patient survey in immune thrombocytopenia (ITP): Identifying unmet needs related to treatment and disease control in patients living in the United States

Nichola Cooper, Stephen J. Morgan, Matias Cordoba, E. P. Reineke, Terry Gernsheimer, Imène Gouia, Caroline Kruse, Jean‐Pascal Roussy, Lisa Anne Schmitt, Julie Laurent, Marleni Arvelo‐Saillant
article en
2 citations

Abstract

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.

British Journal of HaematologyVol. 207(3)
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)
Sanofi
Openalex Percentile: Top 7%
Platelet Disorders and Treatments
3.54
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