Pamidronate treatment of severe osteogenesis imperfecta in children under 3 years of age.
Severe osteogenesis imperfecta (OI) is a hereditary disorder characterized by increased bone fragility and progressive bone deformity. Cyclical pamidronate infusions improve clinical outcome in children older than 3 yr of age with severe OI. Because earlier treatment may have potential to prevent deformities and improve functional prognosis in young children, we studied nine severely affected OI patients under 2 yr of age (2.3-20.7 months at entry) for a period of 12 months. Pamidronate was administered i.v. in cycles of 3 consecutive days. Patients received four to eight cycles during the treatment period, with cumulative doses averaging 12.4 mg/kg. Clinical changes were evaluated regularly during treatment, and radiological changes were assessed after 6-12 months of treatment. The control group consisted of six age-matched, severely affected OI patients, who had not received pamidronate treatment. During treatment bone mineral density (BMD) increased between 86-227%. The deviation from normal, as indicated by the z-score, diminished from -6.5 +/- 2.1 to -3.0 +/- 2.1 (P < 0.001). In the control group the BMD z-score worsened significantly. Vertebral coronal area increased in all treated patients (11.4 +/- 3.4 to 14.9 +/- 1.8 cm2; P < 0.001), but decreased in the untreated group (P < 0.05). In the treated patients, fracture rate was lower than in control patients (2.6 +/- 2.5 vs. 6.3 +/- 1.6 fractures/year; P < 0.01). No adverse side-effects were noted, apart from the well known acute phase reaction during the first infusion cycle. Pamidronate treatment in severely affected OI patients under 3 yr of age is safe, increases BMD, and decreases fracture rate.
Plotkin
H
H
Shriners Hospital for Children, and Department of Surgery, McGill University, Montréal, Québec, Canada.
Rauch
F
F
Bishop
N J
NJ
Montpetit
K
K
Ruck-Gibis
J
J
Travers
R
R
Glorieux
F H
FH
D015519:Bone Density / Q000187:drug effects*; D004164:Diphosphonates / Q000627:therapeutic use*; D005260:Female; D050723:Fractures, Bone / Q000517:prevention & control; D006801:Humans; D007223:Infant; D008159:Lumbar Vertebrae / Q000000981:diagnostic imaging; D008297:Male; D010013:Osteogenesis Imperfecta / Q000000981:diagnostic imaging / Q000188:drug therapy* / Q000503:physiopathology; D000077268:Pamidronate; D011859:Radiography
D004164:Diphosphonates; D000077268:Pamidronate