Presentation (CTI)

OBJECTIVES: Hemophilia A with inhibitors (Ha-wI) is a condition that significantly impacts the quality of life (QoL) of patients by increasing the risk of severe bleeding that causes frequent hospitalizations, physical limitations and chronic pain, affecting their daily activities and social interaction. The objective of this research is to know the impact of emicizumab on the QoL of patients with severe HA-wI, at a third-level hospital of the Mexican Institute of Social Security in Mexico City.
METHODS: Longitudinal and descriptive, single-center study in patients ≥18 yo with severe HA-wI. For data collection, a survey was designed based on the HAEM-A-QOL, a validated instrument, under dimensions of pain, treatment and general health, adapted to the participants. For the analysis, descriptive statistics were used through frequencies, percentages and intervals. QoL was measured at two time points: during on-demand treatment with bypassing agents and during emicizumab prophylaxis in the same patients (n=18). A comparative analysis of matched samples was performed using Student's t-test to examine differences in QoL.
RESULTS: During on-demand treatment, the average QoL assessed was 29.66 (SD=31.52). During emicizumab prophylaxis, the average QoL increased significantly to 68.06 (SD=80.34). The paired comparison revealed a significant mean difference of 38.41 (68.06 - 29.66) in QoL between the two treatments (t=2.6556, p=0.0129; 95% CI difference: -68.0322 to -8.7811), indicating a substantial improvement with the use of emicizumab, with the dimension of satisfaction with treatment showing the greatest difference of 95% in favor to emicizumab prophylaxis, followed by pain control and physical health.
CONCLUSIONS: Based on Patient Reported Outcomes, Emicizumab has a real and substantial effect on improving HA-wI patients’ QoL compared to on-demand therapy, allowing them to live fuller, more active lives.