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This prospective study of 40 adult males with severe hemophilia A or B compared bone mineral density (BMD) changes over one year across five prophylactic treatment groups: emicizumab (with/without inhibitors), FVIII, FIX, or investigational agents. Baseline hip BMD was significantly lower than age-matched norms, but after one year, BMD changes did not differ significantly across treatment groups, with emicizumab showing non-inferiority to FVIII. The study suggests bleed control, rather than the specific factor replacement strategy, is key to bone health in hemophilia.
Emicizumab prophylaxis for hemophilia demonstrates comparable BMD outcomes to FVIII prophylaxis at one year, suggesting bleeding control is the primary driver of bone health, rather than the specific factor replacement strategy.
INTRODUCTION Haemophilia, an X-linked disorder, is linked to reduced bone mineral density (BMD). Emicizumab, a factor VIII (FVIII) mimetic, provides a model to study the role of FVIII in bone health. AIM To evaluate BMD in adults with severe haemophilia under different prophylactic regimens, focusing on whether emicizumab maintains bone health comparable to FVIII prophylaxis. METHODS Forty male adults with severe haemophilia A or B were prospectively enrolled at a single centre (2019-2024) and assigned to five prophylactic groups: emicizumab (with/without inhibitors), FVIII, factor IX (FIX) or investigational agents. Annual assessments included dual-energy X-ray absorptiometry, joint status, mobility and bone turnover markers. RESULTS Of the 40 patients, 18 received FVIII, 10 FIX, 4 haemophilia A with inhibitors on emicizumab, 4 haemophilia A without inhibitors on emicizumab and 4 investigational therapies. At baseline, spine BMD z-scores were comparable to age-matched norms, whereas hip BMD was significantly lower (median -0.8; p < 0.001). After 1 year, spine and hip BMD changes did not differ significantly across treatment groups (p = 0.9010 and p = 0.8073, respectively). Lower annual bleeding rate, middle age and BMI extremes were associated with BMD improvement. Emicizumab-treated patients, with or without inhibitors, showed non-inferior BMD outcomes compared to those receiving FVIII, with all 95% confidence intervals within the predefined margin (±0.5). CONCLUSION BMD outcomes did not differ across regimens. Emicizumab was comparable to FVIII, suggesting bleed control, not factor replacement, is key to bone health in haemophilia. PLAIN LANGUAGE SUMMARY People with haemophilia often have problems with joint bleeding, which can limit movement and reduce bone strength. Different treatments are now available to prevent bleeding, including factor replacement and newer medicines such as emicizumab. It is not clear whether these treatments affect bone health differently. In this study, we followed 40 adults with haemophilia and measured their bone health for 1 year. We found that bone outcomes were similar across all treatment groups. Emicizumab worked just as well as factor VIII in maintaining bone strength. This means that good bleeding control may be the key to keeping bones healthy in haemophilia, regardless of the treatment type.
