Unmet Needs in People with Hemophilia Receiving Prophylaxis Treatment: A Real-World Survey

Introduction

Despite receiving prophylactic treatment, patients with hemophilia continue to experience bleeding, chronic pain, and joint damage, which impair their quality of life (QoL).Considering the limited literature that assesses both patient and physician reported QoL information; this study aimed to understand the remaining unmet needs in disease management among adults and adolescents diagnosed with hemophilia.

Methods

Data were drawn from the Adelphi Hemophilia wave III Disease Specific Programme™, a cross-sectional survey of physicians and their patients with hemophilia in France, Germany, Italy, Spain, Japan, China, the United Kingdom (UK) and the United States (US) from July 2023 - March 2024. Physicians reported demographics, treatment information, emotional struggles, joint problems, and physical activity. Patients were then invited to self-report data on emotional problems and main treatment goals. The study included males aged ≥12 years diagnosed with hemophilia A (HA) or B (HB), with or without inhibitors. Analyses were descriptive.

Results

A total of 230 physicians reported data on 1418 patients (mean ± SD age: 30.7±13.8 years) with HA (n = 1134) and HB (n = 284). Of the total population, 94% had no current inhibitors, while 6% had current inhibitors. At the time of survey, most patients with HA were prophylactically treated with standard half-life (SHL) factor (43%), extended half-life (EHL) factor (26%) and non-factor therapy (25%). SHL was the most used prophylaxis for HA patients in China (69%), Italy (47%), the US (45%), Spain (42%), and France (42%). EHL was the most used prophylaxis for HB patients in Japan (89%), France (81%), Germany (74%), Italy (67%), the UK (67%), and Spain (64%).

Besides prophylactic treatment, majority of patients received an on-demand treatment for disease management, except for 19% of HA and 16% of HB patients. Most patients (86%) reported worry about having breakthrough bleeds “sometimes” to “all the time”. Physicians reported similar observations (88%) regarding their patients.

Physicians reported that 83% of patients in China, 76% in Germany, 55% in France, 52% in the US, 39% in Spain and the UK, 31% in Italy, and 29% in Japan had at least one bleeding episode in the last 12 months. At the start of current prophylaxis, physicians assessed that 25% of patients had moderate to severe joint problems. Among patients who received their prophylactic treatment for >12 months, physicians still reported moderate to severe joint problems (15%) at the time of survey. Except for Germany, participants from other countries reported severe joint problems at the time of survey, despite receiving prophylactic treatment (France: 7%, Italy: 6%, US: 3%, the UK: 2%, China: 2%, Spain: 1%, Japan: 1%). Of the 143 adolescents (mean ± SD age: 14.8±1.7 years) and 1275 adults (mean ± SD age: 32.4±13.4 years), physicians reported moderate to severe joint problems in 14% of adolescents and 27% of adults at the start of current prophylaxis, with 9% of adolescents and 15% of adults at the time of survey. In the last 7 days, physicians reported moderate to very severe joint pain in 32% of adults and 30% of adolescents at current consultation.

Although patients were receiving prophylactic treatment, they reported avoiding physical activity often (43%) or sometimes (48%). Most patients in France (56%), China (53%), Germany (48%), and Spain (42%) reported avoiding physical activity often. Due to hemophilia, patients reported fear/worry associated with the disease (43%), nervousness/anxiety (32%), constant lack of energy (26%), tiredness (24%), disease management concerns (21%), and embarrassment (21%). Other than stopping/reducing bleeds, main goal of patients was to improve their QoL (61%), protect joints (45%), and improve physical activity levels (35%). Improving QoL was the main goal of most patients in France (74%), China (63%), the US (60%) and Germany (55%), while the main goal of most patients was joint protection in Italy (70%) and Spain (69%).

Conclusions

Most patients with hemophilia experience bleeding, moderate to severe joint problems, pain, decreased QoL, and avoid physical activity. This suggests that even with available prophylactic therapies, there remain unmet needs for patient treatment and disease management. This shows a need for future treatments aimed at enhancing patients' QoL and physical activity levels while protecting their joints.

Malec:Pfizer: Consultancy; Takeda: Consultancy; Spark Therapeutics: Consultancy; Sobi: Consultancy; CSL Behring: Consultancy, Speakers Bureau; Sanofi: Consultancy, Speakers Bureau; Novo Nordisk: Consultancy. Salehi:Adelphi Real World: Current Employment. Ball:Adelphi Real World: Current Employment. Lai:Adelphi Real World: Current Employment. Afonso:Sanofi: Current Employment, Other: hold stocks and/or stock options in the company.