Intravenous bisphosphonate therapy in children with spinal muscular atrophy
| Autor: | Lindsey Hornung, Halley Wasserman, Nat Nasomyont |
|---|---|
| Rok vydání: | 2019 |
| Předmět: |
0301 basic medicine
Male medicine.medical_specialty Endocrinology Diabetes and Metabolism Osteoporosis Population 030209 endocrinology & metabolism Muscular Atrophy Spinal 03 medical and health sciences Fractures Bone 0302 clinical medicine Bone Density Internal medicine Medicine Humans education Child Retrospective Studies Bone mineral Femur fracture education.field_of_study Bone Density Conservation Agents Diphosphonates business.industry medicine.disease SMA Rheumatology Zoledronic acid Anesthesia Orthopedic surgery Female 030101 anatomy & morphology business medicine.drug |
| Zdroj: | Osteoporosis international : a journal established as result of cooperation between the European Foundation for Osteoporosis and the National Osteoporosis Foundation of the USA. 31(5) |
| ISSN: | 1433-2965 |
| Popis: | 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.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.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 had2 years of follow-up, fracture rate decreased from 1.4 to 0.1 fracture/year. An atypical femur fracture was observed in one patient.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. |
| Databáze: | OpenAIRE |
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