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Intravenous pamidronate treatment of infants with severe osteogenesis imperfecta
Eva Aström1, Håkan Jorulf, Stefan Söderhäll
1Department of Woman and Child Health, Astrid Lindgren Children's Hospital, Karolinska Institute, Stockholm, Sweden. eva.astrom@karolinska.se
Insights
Early pamidronate treatment for severe osteogenesis imperfecta in infants improved bone density and mobility. While often needing surgery, this early intervention may prevent spinal deformities.
Area of Science:
- Pediatric Orthopedics
- Pediatric Endocrinology
- Bone Metabolism
Background:
- Severe osteogenesis imperfecta (OI) in children necessitates effective treatments.
- Limited data exists on the impact of early-stage intervention with bisphosphonates.
Purpose of the Study:
- To assess the efficacy of initiating intravenous pamidronate treatment in infancy for severe osteogenesis imperfecta.
Main Methods:
- A prospective observational study utilized a historic control group.
- Eleven infants with severe OI received monthly intravenous infusions of disodium pamidronate (APD).
- Participants presented with congenital femoral bowing and vertebral compression fractures.
Main Results:
- Bone mineral density in the lumbar spine increased significantly.
- Bone turnover markers in serum and urine decreased, indicating reduced bone remodeling.
- All treated children achieved the ability to walk independently.
- Vertebral height increased, and no new cases of scoliosis, kyphosis, or basilar impression occurred.
- Five children required tibial rodding for severe deformities; all required femoral rodding for fractures.
Conclusions:
- Intravenous disodium pamidronate (APD) offers effective symptomatic treatment for infants with severe osteogenesis imperfecta.
- Early APD intervention may prevent spinal complications like scoliosis and basilar impression.
- Orthopaedic surgery remains frequently necessary, and long-term monitoring is crucial.
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.
Aim:
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.
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