EXPERIENCES WITH CLOTTING FACTOR PROPHYLAXIS AND EXPECTATIONS TOWARD EMERGING THERAPIES IN HEMOPHILIA CARE IN BRAZIL: A QUALITATIVE INTERVIEW STUDY

In Brazil, People with Hemophilia (PwH) receive Clotting Factor Concentrates (CFCs) through the public healthcare system. While this model enables universal access to prophylaxis, treatment remains burdensome and complex. This study explored lived experiences of PwH, their families, and Healthcare Professionals (HCPs) regarding CFC prophylaxis and expectations for future therapies. To understand the physical, emotional, and systemic challenges related to current hemophilia care in Brazil and stakeholders’ hopes for less burdensome, equitable treatment options. A targeted thematic analysis was conducted on semi-structured interviews with 43 participants: 23 PwH, 10 family members, and 10 HCPs. Participants were purposively sampled across Brazil to capture geographic and socioeconomic diversity. Interviews were conducted virtually, transcribed verbatim, and analyzed using the framework method. Three overarching themes emerged: (1) Burden of current treatment: Participants emphasized the logistical and emotional toll of regular intravenous prophylaxis, including difficult venous access, time-consuming infusion preparation, cold-chain storage, treatment-related waste, and limitations on mobility and daily activities. The need to self-infuse or depend on caregivers led to stress and fatigue, while some PwH skipped doses when in pain or emotionally overwhelmed. (2) Systemic barriers and healthcare inequities: Despite centralized provision of CFCs, participants reported recurrent medication stockouts at treatment centers, long travel distances to treatment centers, limited access to mental health support, and lack of awareness about hemophilia among non-specialist providers. These challenges were especially acute in remote or low-resource settings, where travel and treatment costs strained families despite the medication being free. (3) Expectations for future therapies: Stakeholders expressed high hopes for long-acting, subcutaneous, and gene therapies to reduce the frequency and burden of treatment. HCPs emphasized the importance of ensuring equitable access, infrastructure for monitoring novel therapies, and education to support adherence. Participants also highlighted the need for individualized treatment plans and stronger psychosocial support. While prophylactic CFC therapy improves clinical outcomes, it imposes a daily burden that undermines quality of life and adherence. Stakeholders' narratives emphasize the urgent need to expand access to simplified therapies and reinforce health literacy strategies. Addressing structural barriers, enhancing patient-centered care, and ensuring equitable implementation of innovations are critical steps for advancing hemophilia care in Brazil.