Osteogenesis imperfecta (OI) is a heritable disease of connective tissue, characterized by increased bone fragility.
Bisphosphonates currently seems to be the most promising
therapy, at least in children. We tested IV
neridronate, an amino-
bisphosphonate structurally similar to
alendronate and
pamidronate in adults with OI. Twenty-three men and 23 premenopausal women with OI were randomized to either iv
neridronate (100 mg infused intravenously for 30 minutes every 3 months) or no treatment with a ratio of 2 to 1. Control patients were given the same
bisphosphonate therapy at the end of the first year. Clinical evaluation included bone densitometry measurements using dual energy X-ray absorptiometry (DXA), fasting serum and urinary biochemistry every 6 months, and radiographs of the spine taken at baseline and after 12 and 24 months of follow-up. Spine and hip bone
mineral density rose by 3.0 +/- 4.6% (SD) and by 4.3 +/- 3.9%, respectively, within the first 12 months of treatment, whereas small insignificant changes were observed in the control group. During the second year of follow-up, additional 3.91% and 1.49% increases were observed at the spine and hip, respectively. Markers of skeletal turnover significantly fell during
neridronate treatment. Fracture incidence during
neridronate treatment was significantly lower than before
therapy and compared with controls.
Neridronate iv infusions, administered quarterly, significantly increase bone mineral density and lowered the risk of clinical fracture in adults with OI.
Bisphosphonate therapy seems to provide clinical benefits, not only to children with OI, but also to adult patients.