A REAL-WORLD, GLOBAL SURVEY OF THE DISEASE BURDEN OF TREATED HEMOPHILIA B MALE PATIENTS
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 real-world physician- and patient-reported disease burden within the treated hemophilia B population, globally.
METHODS: Data were drawn from the Adelphi Real World Disease Specific ProgrammeTM, a cross-sectional survey with retrospective data collection of physicians and males living with hemophilia B (MlwHB) 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 patient demographics, clinical outcomes and treatment barriers. Patients reported data on treatment satisfaction, and impact of treatment via the Hemophilia Quality of Life Questionnaire (Haem-A-QoL) (scored 0 to 100, low to high impairment). Analyses were descriptive.
RESULTS: As reported by physicians (n=256), treated MlwHB (n=622) mean (SD) age was 25.2 (15.0) years, mean (SD) BMI was 22.4 (4.6) kg/m2 and 40.1% had severe hemophilia (clotting factor level <1%). At time of survey, 89.5% were prescribed prophylaxis (Ppx), and median (IQR) duration of this treatment was 3.5 (1.5-6.4) years. Physicians were not completely satisfied with Ppx for 47.8% MlwHB, most commonly due to administration/dosing related concerns (73.1%), including “too many infusions/injections” (34.2%), “lengthy infusions/injections” (24.2%) and “difficult to administer” treatment (21.9%). Physicians reported 53.5% of MlwHB experienced ≥1 breakthrough bleeding episode in the 12 months prior to survey and 47.8% suffered joint problems, with hemophilic arthropathy among 51.0% of these. Physicians reported 52.6% of patients required additional therapies, most commonly physical therapy (31.4%) and thermotherapy (10.5%). Among patients with self-reported data (n=122), 73.8% were not completely satisfied with current treatment; mean (SD) Haem-A-QoL treatment domain score was 47.1 (20.2) (n=67), and 90.2% experienced difficulty performing daily activities.
CONCLUSIONS: Results suggest that burden remains high for treated MlwHB through impaired joint health, difficulty with activities, treatment dissatisfaction, and patient-reported Haem-A-QoL treatment domain score, indicating unmet need that novel therapies could address.
METHODS: Data were drawn from the Adelphi Real World Disease Specific ProgrammeTM, a cross-sectional survey with retrospective data collection of physicians and males living with hemophilia B (MlwHB) 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 patient demographics, clinical outcomes and treatment barriers. Patients reported data on treatment satisfaction, and impact of treatment via the Hemophilia Quality of Life Questionnaire (Haem-A-QoL) (scored 0 to 100, low to high impairment). Analyses were descriptive.
RESULTS: As reported by physicians (n=256), treated MlwHB (n=622) mean (SD) age was 25.2 (15.0) years, mean (SD) BMI was 22.4 (4.6) kg/m2 and 40.1% had severe hemophilia (clotting factor level <1%). At time of survey, 89.5% were prescribed prophylaxis (Ppx), and median (IQR) duration of this treatment was 3.5 (1.5-6.4) years. Physicians were not completely satisfied with Ppx for 47.8% MlwHB, most commonly due to administration/dosing related concerns (73.1%), including “too many infusions/injections” (34.2%), “lengthy infusions/injections” (24.2%) and “difficult to administer” treatment (21.9%). Physicians reported 53.5% of MlwHB experienced ≥1 breakthrough bleeding episode in the 12 months prior to survey and 47.8% suffered joint problems, with hemophilic arthropathy among 51.0% of these. Physicians reported 52.6% of patients required additional therapies, most commonly physical therapy (31.4%) and thermotherapy (10.5%). Among patients with self-reported data (n=122), 73.8% were not completely satisfied with current treatment; mean (SD) Haem-A-QoL treatment domain score was 47.1 (20.2) (n=67), and 90.2% experienced difficulty performing daily activities.
CONCLUSIONS: Results suggest that burden remains high for treated MlwHB through impaired joint health, difficulty with activities, treatment dissatisfaction, and patient-reported Haem-A-QoL treatment domain score, indicating unmet need that novel therapies could address.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
PCR137
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)