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Published on: October 3, 2011
Intravenous bisphosphonate therapy in children with spinal muscular atrophy
N Nasomyont1,2, L N Hornung3, H Wasserman4,5
1Department of Pediatrics, University of Cincinnati College of Medicine, Cincinnati, OH, 45267, USA.
Insights
Intravenous bisphosphonates (IV BP) show promise for treating low bone density and reducing fractures in children with spinal muscular atrophy (SMA). While generally safe, careful monitoring is advised due to a rare risk of atypical femur fractures.
Area of Science:
- Pediatric Endocrinology
- Neuromuscular Disorders
- Bone Metabolism
Background:
- Children with spinal muscular atrophy (SMA) face a high risk of fragility fractures and low bone mineral density (BMD).
- Intravenous bisphosphonates (IV BP) are utilized for disuse osteoporosis in pediatric patients.
- Limited data exists on the safety and efficacy of IV BP specifically in the SMA population.
Purpose of the Study:
- To evaluate the safety and efficacy of intravenous bisphosphonates (IV BP) for treating disuse osteoporosis and low bone mineral density (BMD) in pediatric patients with spinal muscular atrophy (SMA).
Main Methods:
- Retrospective chart review of pediatric patients with SMA receiving IV BP for low BMD or disuse osteoporosis between 2010 and 2018.
- Analysis of patient demographics, IV BP types (pamidronate, zoledronic acid), infusion details, adverse events, BMD changes, and fracture rates.
Main Results:
- Eight pediatric patients with SMA received 39 IV BP infusions; 75% had SMA type 1.
- Acute phase reactions occurred after 38% of initial infusions and 3% of subsequent infusions.
- Bone mineral density (BMD) improved at 1 year; fracture rate decreased from 1.4 to 0.1 fractures/year in patients with over 2 years of follow-up. One atypical femur fracture was noted.
Conclusions:
- Intravenous bisphosphonate (IV BP) therapy appears safe and effective in reducing fracture rates in children with spinal muscular atrophy (SMA).
- Minimal acute side effects were observed.
- Continued caution is warranted due to the potential for atypical femur fractures in this population.
Abstract:
This is the first report on safety and efficacy of intravenous bisphosphonates (IV BP) for treatment of disuse osteoporosis and low bone mineral density (BMD) in children with spinal muscular atrophy (SMA). IV BP appears to be safe and effective in fracture rate reduction. However, caution is necessary given the occurrence of an atypical femur fracture.
Introduction:
Children with SMA are at high risk for fragility fractures and low BMD. IV BP have been used for treatment of disuse osteoporosis in pediatrics. However, safety and efficacy of IV BP in the SMA population has not been reported.
Methods:
Retrospective chart review of IV BP for treatment of disuse osteoporosis and low BMD in children with SMA at a tertiary pediatric center from 2010 to 2018 RESULTS: Eight patients (50% female; 75% SMA type 1; median age at first infusion 6.7 years) receiving a total of 39 infusions (54% pamidronate, 46% zoledronic acid) were included in this report. Acute phase reactions occurred following 38% and 3% of initial and subsequent infusions, respectively. BMD trended toward improvement at 1 year post-treatment. Among six patients who had > 2 years of follow-up, fracture rate decreased from 1.4 to 0.1 fracture/year. An atypical femur fracture was observed in one patient.
Conclusion:
These findings suggest that in children with SMA, IV BP therapy appears to be safe with minimal acute side effects and effective to reduce fracture rate. Caution is still needed given the occurrence of an atypical femur fracture in SMA population.
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