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Published on: January 12, 2019
Efficacy of Zoledronic Acid on Bone Mineral Density in Boys With Duchenne Muscular Dystrophy: A Prospective
Santashree Chatterjee1, Renu Suthar2, Tulika Singh3
1Department of Pediatrics, Post Graduate Institute of Medical Education and Research (PGIMER), Chandigarh, India.
Insights
Intravenous zoledronic acid (ZA) improved bone mineral density (BMD) and reduced bone turnover in boys with Duchenne muscular dystrophy (DMD). The treatment was safe and well-tolerated, showing no serious adverse events.
Area of Science:
- Pediatric Endocrinology
- Bone Metabolism
- Neuromuscular Disorders
Background:
- Duchenne muscular dystrophy (DMD) is associated with low bone mineral density (BMD) and increased fracture risk.
- Evaluating interventions to improve bone health in DMD patients is crucial.
Purpose of the Study:
- To assess the efficacy and safety of intravenous zoledronic acid (ZA) in improving BMD and reducing bone turnover in North Indian boys with DMD.
Main Methods:
- Prospective study of boys aged 5-18 years with genetically confirmed DMD and fracture history or low BMD.
- ZA administered in three doses over 3 months; outcomes assessed at 12 months.
Main Results:
- Significant improvement in lumbar spine (0.67 z-score) and femur neck (0.49 z-score) BMD.
- Reduced serum C-terminal telopeptide of type 1 collagen (CTX) levels by 63.3%.
- ZA was well-tolerated with no serious adverse events; functional status and quality of life remained stable.
Conclusions:
- Intravenous ZA is effective in improving BMD and reducing bone turnover in boys with DMD.
- The treatment demonstrates a favorable safety profile in this pediatric population.
Background:
To evaluate the efficacy and safety of intravenous zoledronic acid (ZA) in improving bone mineral density (BMD) and reducing bone turnover in boys with Duchenne muscular dystrophy (DMD) from North India.
Methods:
In this prospective study, boys aged 5-18 years with genetically confirmed diagnosis of DMD, and either a history of low-impact long-bone or vertebral compression fractures (VCFs), or a lumbar spine/right femur neck dual-energy x-ray absorptiometry (DXA) BMD ≤ -2 z-score for age were enrolled. ZA was administered in three doses (total 0.125 mg/kg/year) at 3-month intervals. Outcomes assessed at 12 ± 2 months included changes in lumbar spine and femur neck BMD z-scores, VCFs number and grade, serum C-terminal telopeptide of type 1 collagen (CTX) levels, back pain, functional status, and quality of life.
Results:
Fifty-five boys (mean age 10.7 ± 2.4 years) were enrolled. Seven boys (13%) had 8 long-bone fractures, and 33 (60%) had 86 VCFs on spine radiographs. Lumbar spine BMD z-score improved by 0.67 (95% confidence interval: 0.42-0.91; P = 0.001), and femur neck z-score by 0.49 (95% confidence interval: 0.06-0.93; P < 0.001). VCFs prevalence remained unchanged, though 26.7% developed new grade 1 VCFs. Mean serum CTX levels decreased from 0.488 to 0.179 ng/mL post ZA therapy (P < 0.001). ZA was well tolerated with no serious adverse events. Functional status and quality-of-life scores remained stable.
Conclusions:
Intravenous ZA significantly improved BMD and reduced bone turnover in boys with DMD without major adverse effects.
