Introduction: Haemophilia A and B has been associated with increased prevalence of low bone mineral density (BMD). However, no study has so far evaluated the effects of anti-osteoporotic therapy on BMD.
Methods/design: The primary endpoint of this prospective study was to estimate the effect of 12-month therapy of oral ibandronate 150 mg/month on BMD in patients with haemophilia A and B. Secondary endpoint was its effect on bone turnover markers (BTM), including serum C-terminal telopeptide of type one collagen (sCTX) and tartrate-resistant acid phosphatase band 5b (as markers of bone resorption), osteocalcin and bone-specific alkaline phosphatase (as markers of bone formation). We present an interim analysis of the first 12 months of the study.
Adult patients with T-score <−2.5 S.D. or Z-score <−2 and/or increased risk of fracture according to FRAX model were included. All received 1000 mg/day calcium carbonate with 800 IU/day cholecalciferol.
Results: Ten males (aged 43.7±13.8 years, seven with haemophilia A) were included. Ibandronate resulted in a significant increase in lumbar BMD (from 0.885±0.162 to 0.926±0.177 g/cm2, (+4.9%), P=0.011). No significant change in BMD of total hip (from 0.717±0.128 to 0.729±0.153 g/cm2, P=0.963) or neck (0.741±0.135 to 0.761±0.146 g/cm2, P=0.952) was noticed.
Ibandronate led to a significant decrease in sCTX (from 0.520±0.243 to 0.347±0.230 ng/ml, −29.9%, P=0.042). No significant change in the other BTM was observed. Ibandronate was generally well-tolerated. No fractures were reported.
Conclusions: In the first study conducted so far evaluating the effect of bisphosphonates in patients with haemophilia and increased fracture risk, ibandronate significantly improved BMD in lumbar spine and reduced bone resorption. Its effect on hip BMD and bone formation markers was not significant.
27 Apr - 01 May 2013
European Society of Endocrinology