Presentation (CTI)

OBJECTIVES: To demonstrate the clinical and quality-of-life benefits of emicizumab in Guatemalan patients with hemophilia A, emphasizing its impact on reducing bleeding episodes, improving patient satisfaction, and enhancing overall well-being.
METHODS: A retrospective analysis was conducted on 11 patients (mean age 24.2 years, range 16-34 years) with moderate and severe hemophilia A in Guatemala who received emicizumab treatment. Nearly half of the cohort (46.2%) had inhibitors, and all patients transitioned to emicizumab due to limitations in venous access or suboptimal outcomes with prior prophylactic regimens. The previous prophylactic treatment for most patients was Factor VIII. Patient satisfaction with prior treatment was assessed, along with clinical outcomes, including bleeding episodes, hospitalizations, and quality-of-life improvements before and after treatment initiation.
RESULTS: Patients treated with emicizumab experienced a significant reduction in bleeding episodes, with an average of 1.64 episodes annually prior to treatment. Two patients required hospitalizations during the last year: one was hospitalized once for two weeks, and another twice for one week each. Satisfaction with prior treatment was low, with most patients reporting being "not at all satisfied." In contrast, 61.5% of patients reported being "very satisfied" with emicizumab. Furthermore, all patients (100%) indicated that emicizumab significantly improved their quality of life, demonstrating its effectiveness both clinically and in enhancing patient well-being.
CONCLUSIONS: The use of emicizumab in Guatemalan patients with hemophilia A resulted in improved clinical outcomes and substantial quality-of-life enhancements. These findings highlight the need to expand access to emicizumab as a comprehensive therapeutic option for this population, particularly for those with complex clinical scenarios or poor prior treatment management.