Relationship between Frequency of Treatment Administration, Adherence to Treatment, and Quality of Life in Patients with Hemophilia a or Hemophilia B, with or without Inhibitors: A Real-World Survey

Introduction

Despite currently available therapies, patients with hemophilia (PwH) have suboptimal treatment adherence and impaired quality of life (QoL). Frequency of treatment administration may influence both adherence and QoL; however, there is limited evidence about the relationship. We aimed to assess the relationship between frequency of treatment administration, treatment adherence and QoL in patients with hemophilia A (PwHA) or B (PwHB), with or without inhibitors.

Methods

Data were drawn from the Adelphi Real World Hemophilia Disease Specific Programme™, a cross-sectional survey of hemophilia-treating physicians and their next consulting patients in France, Germany, Italy, Spain, Japan, China, the United Kingdom (UK) and the United States (US) from July 2023 to March 2024. Male adolescent (<18 years) or adult (≥18 years) patients diagnosed with moderate to severe hemophilia A or B (baseline clotting factor <5%), with or without inhibitors, were included in this study. Patients were stratified into subgroups by treatment administration frequency: every 1-6 days (Q1-6D), every 7 days (Q1W), and every 28-30 days (Q4W). Physicians reported treatment adherence and satisfaction, annual bleeding rate (ABR), joint health status, aids to improve bleeding and joint health, physical activity level, caregiver involvement, and QoL for their PwHA or B. Adult patients self-reported impact of hemophilia and their quality of life via the EQ-5D Visual Analogue Scale (VAS). Mean (SD) ABR data was reported for patients prescribed treatment for ≥365 days. Analyses were descriptive.

Results

Overall, 214 physicians reported data on 1287 patients (80% PwHA and 20% PwHB), of which 132 (10%) were adolescent, and 1155 (90%) were adult. Prophylactic treatment was received by 708 (55%) of Q1-6D, 236 (18%) of Q1W and 88 (7%) of Q4W. Splitting by hemophilia type, 60% of PwHA and 34% of PwHB received treatment Q1-6D, 12% and 43% of Q1W, and 8% and 1% of Q4W. Of total patients, 289 provided patient-reported data (166 [57%] of Q1-6D, 43 [15%] of Q1W, 34 [12%] of Q4W).

Physicians reported that 92% of patients treated Q4W were fully compliant (take >80% of prescribed dose) with treatment, compared to 90% of Q1W and 81% of Q1-6D.

Physicians also reported that 86% of overall patients take 80%-100% of doses on time. As per treatment administration frequency subgroups, this was 76% patients of Q4W, compared to 87% of Q1-6D and 90% of Q1W.

Mean (standard deviation; SD) ABR was 1.6 (2.3) for patients treated Q1-6D, 0.9 (1.5) for Q1W and 0.8 (1.4) for Q4W. Physicians reported moderate-to-severe joint problems in 13% of patients treated Q1-6D, 18% Q1W and 9% Q4W.

Physicians reported that to reduce overall rate of bleeding, patients avoided playing contact sports (37%), decreased their level of physical activity (28%), and avoided heavy lifting (26%). Physicians reported that 54% adolescents and 60% adults avoided activities or wore protective gear to reduce overall rate of bleeding.

In total, caregiver involvement was reported for 48% of patients treated both Q1W and Q4W versus 43% for Q1-6D; 87% for adolescents and 40% for adults. Use of aids due to joint damage was reported for 23% of patients Q1-6D, 26% for Q1W and 16% for Q4W. This was 15% for adolescent and 24% for adult patients.

Despite high levels of compliance, 85% of overall patients reported that they were worried ‘sometimes’, ‘most of the time’ or ‘all of the time’ about breakthrough bleeds. Patients also reported fear/worry about their hemophilia (43% of Q1-6D, 42% of Q1W and 38% of Q4W). No patients treated Q4W reported worrying ‘all the time’ versus 2% Q1W and 15% Q1-6D. Complete satisfaction in controlling bleeds was reported among 30% patients treated Q1-6D compared to 21% Q1W and 41% Q4W.

Patient-reported mean (SD) EQ-5D-VAS score was 70.9 (16.4) for patients treated Q1-6D and 69.7 (19.0) for Q1W and 82.0 (11.6) for Q4W.

Most patients (95%) treated Q1-6D reported that they often/sometimes avoided physical activity due to their hemophilia, compared to 88% of patients for both Q1W and Q4W.

Conclusion

PwH who received Q4W treatment administrations showed a high level of treatment compliance despite nearly a quarter of them not taking doses on time. PwH who received Q4W also reported a high level of complete satisfaction in controlling bleeds, low ABR and good overall QoL. A novel treatment with lower frequency of administration may increase the compliance to treatment and the QoL of patients.

Cockrell:Sanofi: Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Genentech: Membership on an entity's Board of Directors or advisory committees; Novo Nordisk: Membership on an entity's Board of Directors or advisory committees; Bayer: Membership on an entity's Board of Directors or advisory committees; Biomarin: Membership on an entity's Board of Directors or advisory committees. Afonso:Sanofi: Current Employment, Other: hold stocks and/or stock options in the company. Wynne-Cattanach:Adelphi Real World: Current Employment. Lai:Adelphi Real World: Current Employment. Blazos:Adelphi Real World: Current Employment. Castellano:Adelphi Real World: Current Employment.