A REAL-WORLD, GLOBAL SURVEY OF THE BURDEN OF HEMOPHILIA A AND B ON PATIENTS WITH INHIBITORS
Author(s)
Maria Gheorghe, PhD1, Harika Booragadda, MPH1, Hae Kyung Kim, MSc, PharmD1, Carlos Francisco Estevez, MD1, Reto Wirz, MD2, Nathan Ball, BSc3, Kieran Wynne-Cattanach, MSc3, Ella Morton, BSc3, Sophie Lai, BSc3, Rabiyah Sahar, MSc3, Chris Blazos, BSc3.
1Pfizer Inc., New York, NY, USA, 2Pfizer AG, Zurich, Switzerland, 3Adelphi Real World, Bollington, United Kingdom.
1Pfizer Inc., New York, NY, USA, 2Pfizer AG, Zurich, Switzerland, 3Adelphi Real World, Bollington, United Kingdom.
OBJECTIVES: To describe the physician-reported real-world burden of hemophilia A (HA) and hemophilia B (HB) on male patients with inhibitors.
METHODS: Data were drawn from the Adelphi Real World Disease Specific ProgrammeTM, a cross-sectional survey with retrospective data collection of hemophilia treating physicians in Brazil, France, Germany, India, Italy, Japan, Kingdom of Saudi Arabia, Spain, Taiwan, Turkey, the United Kingdom, and the United States (July 2023-April 2025). Physicians reported data on demographics, treatment satisfaction, healthcare resource utilization (HCRU), clinical outcomes, activities of daily living (ADL), and quality of life (QoL). Analyses were descriptive.
RESULTS: Physicians (n=110) reported data for male hemophilia patients with inhibitors (n=191) (83.2% HA, 16.8% HB), mean (SD) age was 26.2 (15.7) years (6.3% 0-5, 8.9% 6-11, 10.5% 12-17, 74.3% 18-90 years). Overall, 91.6% patients received prophylaxis for hemophilia, 17.6% and 37.0% HA and HB patients respectively received bypassing agents as prophylaxis. Physicians were not completely satisfied with their patient’s prophylaxis for 57.4% HA and 74.1% HB patients. In the 12 months prior to survey, patients underwent median (IQR) 20.0 (12.0-32.0) hemophilia-related tests, 8.0 (4.0-12.0) consultations, and 42.6% were hospitalized. Overall, 77.0% patients required a caregiver, most commonly their parent/guardian (50.8%). Overall, 66.5% patients had experienced joint problems. Physicians reported 35.6% patients had moderate-severe joint problems, 37.2% were expected to require joint surgery, and 66.5% required additional therapy/activities to improve joint health. Physicians described moderate or significant increase in mortality risk due to hemophilia for 64.9% patients. Physicians reported 64.4% patients experienced limitations to ADL, 18.8% changed to lower risk employment, and 66.0% experienced emotional problems due to hemophilia, commonly “fear or worry” (30.4%), “nervousness/anxiety” (30.4%) and “feelings of sadness/depression” (19.4%).
CONCLUSIONS: This global physician-reported study demonstrates an unmet need amongst HA and HB patients with inhibitors, indicated by considerable HCRU, joint problems, and impairment to ADL, work, and QoL.
METHODS: Data were drawn from the Adelphi Real World Disease Specific ProgrammeTM, a cross-sectional survey with retrospective data collection of hemophilia treating physicians in Brazil, France, Germany, India, Italy, Japan, Kingdom of Saudi Arabia, Spain, Taiwan, Turkey, the United Kingdom, and the United States (July 2023-April 2025). Physicians reported data on demographics, treatment satisfaction, healthcare resource utilization (HCRU), clinical outcomes, activities of daily living (ADL), and quality of life (QoL). Analyses were descriptive.
RESULTS: Physicians (n=110) reported data for male hemophilia patients with inhibitors (n=191) (83.2% HA, 16.8% HB), mean (SD) age was 26.2 (15.7) years (6.3% 0-5, 8.9% 6-11, 10.5% 12-17, 74.3% 18-90 years). Overall, 91.6% patients received prophylaxis for hemophilia, 17.6% and 37.0% HA and HB patients respectively received bypassing agents as prophylaxis. Physicians were not completely satisfied with their patient’s prophylaxis for 57.4% HA and 74.1% HB patients. In the 12 months prior to survey, patients underwent median (IQR) 20.0 (12.0-32.0) hemophilia-related tests, 8.0 (4.0-12.0) consultations, and 42.6% were hospitalized. Overall, 77.0% patients required a caregiver, most commonly their parent/guardian (50.8%). Overall, 66.5% patients had experienced joint problems. Physicians reported 35.6% patients had moderate-severe joint problems, 37.2% were expected to require joint surgery, and 66.5% required additional therapy/activities to improve joint health. Physicians described moderate or significant increase in mortality risk due to hemophilia for 64.9% patients. Physicians reported 64.4% patients experienced limitations to ADL, 18.8% changed to lower risk employment, and 66.0% experienced emotional problems due to hemophilia, commonly “fear or worry” (30.4%), “nervousness/anxiety” (30.4%) and “feelings of sadness/depression” (19.4%).
CONCLUSIONS: This global physician-reported study demonstrates an unmet need amongst HA and HB patients with inhibitors, indicated by considerable HCRU, joint problems, and impairment to ADL, work, and QoL.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
PCR85
Topic
Clinical Outcomes, Patient-Centered Research, Real World Data & Information Systems
Topic Subcategory
Patient-reported Outcomes & Quality of Life Outcomes
Disease
Rare & Orphan Diseases, Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)