Intravenous bisphosphonate therapy in children with spinal muscular atrophy

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 i...

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Bibliographic Details
Published in:	Osteoporosis international Vol. 31; no. 5; pp. 995 - 1000
Main Authors:	Nasomyont, N., Hornung, L.N., Wasserman, H.
Format:	Journal Article
Language:	English
Published:	London Springer London 01-05-2020 Springer Nature B.V
Subjects:	Bisphosphonates Bone Density Bone Density Conservation Agents - adverse effects Bone mineral density Child Children Diphosphonates - adverse effects Endocrinology Female Femur Fractures Fractures, Bone Humans Intravenous administration Male Medicine Medicine & Public Health Muscular Atrophy, Spinal Neuromuscular diseases Orthopedics Osteoporosis Osteoporosis - drug therapy Pamidronic acid Patients Pediatrics Retrospective Studies Rheumatology Short Communication Spinal muscular atrophy Zoledronic acid Atypical femur fracture, bisphosphonates spinal muscular atrophy DXA osteoporosis pediatrics
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Summary:	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.
ISSN:	0937-941X 1433-2965
DOI:	10.1007/s00198-019-05227-9