engleski

Our experience in bisphosphonate therapy for osteogenesis imperfecta

Severe osteogenesis imperfecta (OI) is a hreditary disorder characterized by increased bone fragility and progressive bone deformity. Secondary osteoporosis is an important feature of OI. So far, no effective medical treatment is available. Antiresorptive activity of the aminobisphosphonates may improve clinical outcome in children. Aim is to assess the clinical impact of the administration of bisphosphonates in Croatian OI patients. We introduced threrapy in 1998 ecouraged by parents from Croatian OI Society. Here we report results of 1-3 years treatment with intravenous pamidronate in six children (four girls) of age 3 months - 11 years at entry, with severe OI. Pamidronate was administered in cycles as monthly infusion at a daily dose of 1 - 1,5 mg/kg during 6 months, following pause for three months, or the same dose for three days every four months. All took 500-1000 mg calcium from food or supplements and 1000 IU of vitamin D daily. During treatment DEXA measurements showed a gradual increase in bone density in all patients. Biochemically assessed bone turnover rate fell. Number of confirmed fractures decreased in all. The reduction in pain and improvement in well being and ability were impressive in two boys who had been confined to a wheelchair and now they walk using crutches. No adverse side-effects were noted, apart from the well known acute phase reaction during the first infusion cycle in two children. Although the bisphosphonates do not correct basic abnormalities in OI, they significantly alter the natural course of the disease and improve patients quality of life. For the time being they seem not only effective but also avoid of any adverse effects on bone growth and remodeling.

osteogenesis imperfecta; bisphosphonates; osteoporosis; collagen

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