Factors contributing to the development of hemophilic arthropathy: A real-world study
摘要
Hemophilia A is a rare X-linked bleeding disorder that often leads to hemophilic arthropathy (HA), a major cause of disability and an important healthcare burden worldwide. To identify the factors associated with HA among Colombian patients with severe hemophilia A and to evaluate how current clinical practice aligns with international care recommendations. This study employed a convergent mixed-methods design combining a targeted literature review (TLR), an analysis of 90 anonymized physician-collected patient diaries (PDs), and semistructured interviews with hematologists. Associations were assessed using odds ratios (ORs) and mean differences. The findings were triangulated to inform expert prioritization of risk and protective factors. PDs were mainly obtained from adults (38.9%) and children (3–11 years) (31.1%). Key gaps in clinical practice included the limited use of genetic testing (31.1%) and laboratory monitoring (< 20%). Physical activity (OR: 0.16; 95% CI: 0.06–0.47) and primary prophylaxis (OR: 0.07 vs. secondary; OR: 0.10 vs. on-demand) were considered protective factors for joint damage. HA bleeding episodes during the prior 12 months (OR: 4.6; 95% CI: 1.8–11.8) and primary prophylaxis (OR: 0.2 vs. secondary prophylaxis; OR: 0.3 vs. tertiary prophylaxis) were associated with HA. Patients with joint damage/HA had a relative delay in prophylaxis initiation compared with those without the conditions (median, 155/191 vs. 50/60.5 months, respectively). In this study, early prophylaxis and physical activity were revealed as protective factors for HA. Gaps remain in local care, underscoring the need to adopt guidelines and personalized treatment to improve patient outcomes.