engleski

Bisphosphonate therapy for osteogenesis imperfecta

Severe osteogenesis imperfecta (OI) is a hereditary disorder characterised by increased bone fragility and progressive bone deformity. So far, no effective medical treatment is avaliable. As secondary osteoporosis is an important feature of OI, antiresorptive activity of the aminobisphosphonates may improve clinical outcome in children. Aimwas to asses the clinical impact of the administration of bisphosponates in Croatian OI patients. We report results of 1-3 years treatment with intravenous pamidronate (APD) in seven children (four girls) of age 3 months - 11 years at entry, with severe OI. Pamidronate was administered in cycles as monthly infusions 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. Following tratmend DEXA measurements showed a gradual increase in bone density in all patients. 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. Acute phase reactions were noted during first infusion cycle in two children and asymptomatic hypocalcaemia in three children. Three children gained excessive weight. Although bisphosponates 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 devoid of any adverse effects on bone growth and remodelling.

osteogenesis imperfecta ; bisphosphonates

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