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Intravenous bisphosphonate therapy in children with spinal muscular atrophy

  • N. Nasomyont
  • L.N. Hornung
  • H. WassermanEmail author
Short Communication
  • 33 Downloads

Abstract

Summary

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.

Keywords

Atypical femur fracture, bisphosphonates DXA osteoporosis pediatrics spinal muscular atrophy 

Notes

Compliance with ethical standards

The study was approved by the Institutional Review Board.

Conflicts of interest

None.

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Copyright information

© International Osteoporosis Foundation and National Osteoporosis Foundation 2019

Authors and Affiliations

  1. 1.Department of PediatricsUniversity of Cincinnati College of MedicineCincinnatiUSA
  2. 2.Division of EndocrinologyCincinnati Children’s Hospital Medical CenterCincinnatiUSA
  3. 3.Division of Biostatistics and EpidemiologyCincinnati Children’s Hospital Medical CenterCincinnatiUSA

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