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

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.