Intravenous pamidronate treatment of infants with severe osteogenesis imperfecta.
OBJECTIVE
Children with the severe forms of osteogenesis imperfecta have in several studies been treated with intravenous pamidronate, but there are only few reports of the effect of early treatment.


OBJECTIVE
To evaluate the effect of treatment started in infancy.


METHODS
In a prospective observational study, with a historic control group, intravenous disodium pamidronate (APD) was given as monthly infusions to 11 children with osteogenesis imperfecta aged 3-13 (median 3.6) months, who had severe osteogenesis imperfecta with congenital bowing of the femora and vertebral compression fractures.


RESULTS
During treatment of children aged between 3 and 6 (median 4.5) years, dual-energy x ray absorptiometry measurements of the lumbar spine showed a gradual increase in bone density. Bone metabolism parameters in serum (alkaline phosphatase, osteocalcin, procollagen 1 carboxy-terminal peptide, collagen 1 teleopeptide) and in urine (deoxypyridinoline) indicated a decrease in bone turnover. An improvement of mobility was seen and at the latest recording, at the age of 3.3-6.5 (median 4.8) years, the children could all walk. Vertebral remodelling was seen, with increased vertebral height, and no child developed scoliosis, kyphosis or basilar impression. All children required femoral intramedullar rods for fractures, and five needed tibial rodding for extreme curvatures that prevented functional standing and walking. No adverse effects were seen on growth, fracture healing or blood chemistry.


CONCLUSIONS
APD is an efficient symptomatic treatment for infants with severe osteogenesis imperfecta, but additional orthopaedic surgery is often needed. Early treatment may prevent scoliosis and basilar impression. Long-term follow-up is important.
Aström
Eva
E
Department of Woman and Child Health, Astrid Lindgren Children's Hospital, Karolinska Institute, Stockholm, Sweden. eva.astrom@karolinska.se
Jorulf
Håkan
H
Söderhäll
Stefan
S
D015502:Absorptiometry, Photon; D001827:Body Height; D001835:Body Weight; D015519:Bone Density / Q000187:drug effects; D050071:Bone Density Conservation Agents / Q000627:therapeutic use*; D002648:Child; D002675:Child, Preschool; D004164:Diphosphonates / Q000627:therapeutic use*; D005260:Female; D006801:Humans; D007223:Infant; D007262:Infusions, Intravenous; D008159:Lumbar Vertebrae / Q000000981:diagnostic imaging / Q000503:physiopathology; D008297:Male; D009048:Motor Skills / Q000187:drug effects; D010013:Osteogenesis Imperfecta / Q000150:complications / Q000000981:diagnostic imaging / Q000188:drug therapy* / Q000503:physiopathology; D000077268:Pamidronate; D011446:Prospective Studies; D012600:Scoliosis / Q000209:etiology / Q000517:prevention & control; D016138:Walking
D050071:Bone Density Conservation Agents; D004164:Diphosphonates; D000077268:Pamidronate