LongHest project: A prospective, observational study of extended half‐life treatment in the musculoskeletal health of patients with severe haemophilia A

BACKGROUND: Prophylactic treatment is the gold standard in the treatment of patients with haemophilia. Prophylaxis with extended half‐life (EHL) treatment has shown long‐term safety and efficacy in patients with haemophilia. AIM: To evaluate the efficacy of prophylaxis with EHL treatment in the frequency of haemarthrosis and musculoskeletal health in adult patients with severe haemophilia A. METHODS: Prospective cohort study. Forty‐six patients with severe haemophilia A were recruited. The frequency of haemarthrosis (self‐reports), joint condition (Haemophilia Joint Health Score), pain intensity (visual analogue scale), range of motion (goniometry), and strength (dynamometry) and muscle activation (surface electromyography) were evaluated. Three assessments were carried out: at baseline (T0), at 6 months (T1) and at 12 months following treatment (T2). RESULTS: There were significant changes in the within‐subject effect in the frequency of haemarthrosis in elbow (F(1.05;96.20) = 3.95; P < .001) and knee (F(1.73;157.99) = 9.96; P < .001). Significant within‐subject effect in elbow pain intensity (F(2;182) = 63.51; P < .001) was found. The mean values of the frequency haemarthrosis in elbow (from .66±1.01 to .04±.20) and knees (from .55±.68 to .33±.53) decrease after the period study. The intensity of elbow pain and (from 3.08±1.69 to 2.67±1.73), decrease after the 12‐month follow‐up period. CONCLUSIONS: Prophylaxis with extended half‐life treatment reduces the frequency of haemarthrosis in elbow and knee in adult patients with haemophilia. EHL treatment reduces the intensity of elbow pain in patients with haemophilic arthropathy.