Treatment Patterns and Healthcare Resource Utilization Among Patients With Immune Thrombocytopenia: A Real-World Survey
Author(s)
SANDHYA R. PANCH, MD, MPH1, Maria Belen Rodriguez, MSc, MD2, Susanna Libby, BSc3, Hannah Connolly, MChem3, Imene Gouia, PharmD, MSc4.
1Fred Hutch Cancer Center, University of Washington, Seattle, WA, USA, 2Sanofi, Cambridge, MA, USA, 3Adelphi Real World, Bollington, United Kingdom, 4HEVA, Sanofi, Gentilly, France.
1Fred Hutch Cancer Center, University of Washington, Seattle, WA, USA, 2Sanofi, Cambridge, MA, USA, 3Adelphi Real World, Bollington, United Kingdom, 4HEVA, Sanofi, Gentilly, France.
OBJECTIVES: To characterize treatment patterns and healthcare resource utilization (HCRU) in immune thrombocytopenia (ITP).
METHODS: As part of the Adelphi Real World ITP Disease Specific Programme™ (2020-2021), a survey was conducted across United States, France, Italy, Germany, Spain, United Kingdom, and Japan. Hematologists completed electronic case-record forms for consecutive patients with persistent/chronic ITP on “advanced therapy” beyond corticosteroids and/or intravenous immunoglobulin. Information was obtained on patient demographics, treatment history, reasons for treatment modification/cessation, consultations and hospitalizations, during the previous year. A treatment line was defined as the initiation or alteration of ITP therapy but excluded dose adjustment or interruption.
RESULTS: One hundred and twelve physicians completed data on 292 patients. Mean (SD) patient age was 54.8 (17.0) years; 74.3% were White; 52.7% male; 72.3% had chronic ITP. Thrombopoietin receptor agonists (TPO-RAs) were the predominant advanced therapy at the time of survey, used by 87.7% of patients, followed by rituximab (7.5%). Patients received a mean (SD) of 2.3 (1.0) treatment lines including first- (100.0%), second- (86.0%) and third-line (31.8%) therapies for 9.9 (29.1), 12.5 (18.7), and 14.8 (19.0) months, respectively. Reasons for discontinuation were waning efficacy (steroids: 43.8%; TPO-RAs: 42.8%; rituximab: 42.5%) and lack of initial response (TPO-RAs: 25.7%; rituximab: 27.5%). At time of survey, 9.9% patients received both rescue and maintenance therapy. On average, high-dose steroids were used 2.6 times. During the preceding year, 28.8% patients had ITP-related hospitalization, 57.7% attributable to bleeds. Mean (SD) number of hematologist consultations was 7.6 (6.1). Country-wise data revealed regional patterns.
CONCLUSIONS: Despite available therapies, ITP patients continue to face challenges including bleeding, waning treatment responses, and high HCRU such as frequent hospitalizations and outpatient visits, highlighting the unmet need in ITP. This underscores the need to expand therapeutic options, to research cumulative toxicities from multiple treatments as well as their impact on health-related quality of life.
METHODS: As part of the Adelphi Real World ITP Disease Specific Programme™ (2020-2021), a survey was conducted across United States, France, Italy, Germany, Spain, United Kingdom, and Japan. Hematologists completed electronic case-record forms for consecutive patients with persistent/chronic ITP on “advanced therapy” beyond corticosteroids and/or intravenous immunoglobulin. Information was obtained on patient demographics, treatment history, reasons for treatment modification/cessation, consultations and hospitalizations, during the previous year. A treatment line was defined as the initiation or alteration of ITP therapy but excluded dose adjustment or interruption.
RESULTS: One hundred and twelve physicians completed data on 292 patients. Mean (SD) patient age was 54.8 (17.0) years; 74.3% were White; 52.7% male; 72.3% had chronic ITP. Thrombopoietin receptor agonists (TPO-RAs) were the predominant advanced therapy at the time of survey, used by 87.7% of patients, followed by rituximab (7.5%). Patients received a mean (SD) of 2.3 (1.0) treatment lines including first- (100.0%), second- (86.0%) and third-line (31.8%) therapies for 9.9 (29.1), 12.5 (18.7), and 14.8 (19.0) months, respectively. Reasons for discontinuation were waning efficacy (steroids: 43.8%; TPO-RAs: 42.8%; rituximab: 42.5%) and lack of initial response (TPO-RAs: 25.7%; rituximab: 27.5%). At time of survey, 9.9% patients received both rescue and maintenance therapy. On average, high-dose steroids were used 2.6 times. During the preceding year, 28.8% patients had ITP-related hospitalization, 57.7% attributable to bleeds. Mean (SD) number of hematologist consultations was 7.6 (6.1). Country-wise data revealed regional patterns.
CONCLUSIONS: Despite available therapies, ITP patients continue to face challenges including bleeding, waning treatment responses, and high HCRU such as frequent hospitalizations and outpatient visits, highlighting the unmet need in ITP. This underscores the need to expand therapeutic options, to research cumulative toxicities from multiple treatments as well as their impact on health-related quality of life.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
PCR248
Topic
Patient-Centered Research, Real World Data & Information Systems, Study Approaches
Topic Subcategory
Patient-reported Outcomes & Quality of Life Outcomes
Disease
No Additional Disease & Conditions/Specialized Treatment Areas, Rare & Orphan Diseases, Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)