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Benefits of pamidronate in children with osteogenesis imperfecta: an open prospective study
Véronique Forin1, Asma Arabi, Vincent Guigonis
1Service d'Orthopédie Pédiatrique, Hôpital Armand Trousseau, Assistance Publique Hôpitaux de Paris, Université Paris VI, Paris, France. veronique.forin@trs.ap-hop-paris.fr
Insights
Pamidronate treatment significantly reduced fracture rates and pain in children with osteogenesis imperfecta (OI). This bisphosphonate therapy also improved bone mineral density (BMD) and decreased bone remodeling markers in pediatric patients.
Area of Science:
- Pediatric Endocrinology
- Orthopedics
- Pharmacology
Background:
- Osteogenesis imperfecta (OI) is a genetic disorder characterized by fragile bones and frequent fractures.
- Current treatments aim to reduce fracture incidence and improve bone quality.
Purpose of the Study:
- To evaluate the efficacy of pamidronate in managing osteogenesis imperfecta in pediatric patients.
- To assess the impact of pamidronate on fracture rates, bone pain, and bone metabolism markers.
Main Methods:
- A cohort of 29 pediatric patients with OI received cyclic pamidronate infusions.
- Treatment involved varying doses based on age (0.5 mg/kg/day for infants, 1 mg/kg/day for children) over 3-day cycles.
- Fracture rates, bone mineral density (BMD), and biochemical markers were assessed before and during treatment over a median follow-up of 16 months.
Main Results:
- Pamidronate significantly decreased fracture incidence from 1.5 to 0.5 per year in infants and 2.0 to 1.0 per year in children.
- Bone pain reduced after the first cycle, and bone remodeling markers (alkaline phosphatase, N-telopeptide) decreased significantly.
- Bone mineral density (BMD) increased substantially in the spine (55.4%) and femoral neck (16%), with improved Z-scores.
Conclusions:
- Pamidronate is effective in increasing BMD, reducing bone turnover, alleviating pain, and lowering fracture rates in children with OI.
- The treatment demonstrated a favorable safety profile, with no adverse effects on growth or fracture healing observed.
Objectives:
To study the efficacy of pamidronate in children with osteogenesis imperfecta (OI).
Patients And Methods:
Twenty-nine patients (median age 8.7 years), were given pamidronate in cyclic infusions of 3 days. Patients received 3-13 cycles (median 6), at a dose of 0.5 mg/kg/day in infants (below 2 years of age) and 1 mg/kg/day in children (2 years and older). The interval time between cycles was 2 months in infants and 4 months in children. The median follow-up was 16 months. All patients received daily supplementation of calcium, vitamin D and physical rehabilitation. Assessments were performed at baseline and before each cycle. Fracture rate under treatment was compared to the one in the pre-treatment period.
Results:
Pain decreased after the first infusion cycle (P < 0.0001). The median of fracture incidence decreased from 15 to 0.5 per year in infants and from 2.0 to 1 per year in children (P = 0.04). Alkaline phosphatase decreased by 31.2% and N-telopeptide collagen cross-links decreased by 61.8% (P < 0.001). Bone mineral density (BMD) of the spine increased by a median of 55.4% (P < 0.001). Z-scores increased from a median of -4.7 to -2.6 (P < 0.001). The femoral neck, BMD increased by a median of 16%. The area of the first four lumbar vertebrae increased by a median of 21.5% (P < 0.001). No adverse effect on growth or on fracture healing was observed. Side effects were symptomatic hypocalcemia in one infant, and the transient acute phase reaction.
Conclusion:
Pamidronate increases BMD, decreases bone remodeling markers, pain and fracture rate in infants and children with OI.
