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Intravenous bisphosphonate therapy in children with spinal muscular atrophy

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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.

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Authors and Affiliations

  1. Department of Pediatrics, University of Cincinnati College of Medicine, Cincinnati, OH, 45267, USA

    N. Nasomyont & H. Wasserman

  2. Division of Endocrinology, Cincinnati Children’s Hospital Medical Center, 3333 Burnet Ave, ML 7012, Cincinnati, OH, 45229, USA

    N. Nasomyont & H. Wasserman

  3. Division of Biostatistics and Epidemiology, Cincinnati Children’s Hospital Medical Center, Cincinnati, OH, 45229, USA

    L.N. Hornung

Authors
  1. N. Nasomyont
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  2. L.N. Hornung
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  3. H. Wasserman
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Correspondence to H. Wasserman.

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The study was approved by the Institutional Review Board.

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Nasomyont, N., Hornung, L. & Wasserman, H. Intravenous bisphosphonate therapy in children with spinal muscular atrophy. Osteoporos Int 31, 995–1000 (2020). https://doi.org/10.1007/s00198-019-05227-9

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  • DOI: https://doi.org/10.1007/s00198-019-05227-9

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