Daily prednisone treatment in Duchenne muscular dystrophy in southwest China
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Published on: April 10, 2019
Jun Hu1,2,3,4,5, Yuanzhen Ye2,3,4,5, Min Kong2,3,4,5
1Department of Pediatrics, Union Hospital, Fujian Medical University, Fuzhou, China.
Insights
Daily prednisone treatment significantly improved muscle strength, function, and quality of life in children with Duchenne muscular dystrophy (DMD). This 12-month study in China found prednisone suitable for managing DMD, slowing progression with manageable side effects.
Area of Science:
- Pediatrics
- Neurology
- Pharmacology
Background:
- Duchenne muscular dystrophy (DMD) is a progressive genetic disorder affecting muscle strength.
- Prednisone is a corticosteroid used to manage inflammatory conditions.
- Understanding prednisone's efficacy and safety in pediatric DMD patients is crucial.
Purpose of the Study:
- To evaluate the influence of daily prednisone treatment on Duchenne muscular dystrophy (DMD).
- To assess efficacy and safety parameters over a 12-month period.
- To conduct a prospective, randomized, placebo-controlled trial in southwestern China.
Main Methods:
- Sixty-six children (4-12 years) with DMD were randomly assigned to prednisone or placebo groups.
- Prednisone dosage was 0.75 mg/kg/day.
- Evaluations included muscle strength, function, quality of life (QoL), quantitative muscle ultrasound (QMUS), and side effects.
Main Results:
- The prednisone group showed significant improvements in muscle strength, function, QoL, and QMUS compared to placebo (P < 0.05).
- No significant differences were observed in body weight, height, BMI, or diastolic blood pressure between groups (P > 0.05).
Conclusions:
- Daily prednisone at 0.75 mg/kg/day is suitable for children with DMD in southwestern China.
- Prednisone treatment slowed disease progression and enhanced QoL and QMUS.
- Observed side effects were moderate and generally well-tolerated.
Introduction:
In this study we aimed to determine the influence of daily prednisone treatment in Duchenne muscular dystrophy (DMD) by performing a prospective, randomized, placebo-controlled trial in southwestern China.
Methods:
Sixty-six children with DMD (4-12 years of age) were divided randomly into prednisone and placebo groups. Efficacy and safety of daily prednisone at 0.75 mg/kg/day were evaluated over 12 months by muscle strength and function, quality of life (QoL), quantitative muscle ultrasound (QMUS), and side effects.
Results:
Significant improvements in muscle strength and function, QoL, and QMUS were observed in the prednisone group compared with the placebo-treated group (P < 0.05). Changes in body weight, height, body mass index, and diastolic blood pressure were similar in both groups (P > 0.05).
Conclusions:
This pilot study in southwestern China found that daily prednisone at 0.75 mg/kg/day is suitable for children with DMD. It slowed disease progression and improved QoL and QMUS. Moderate side effects were generally well tolerated.
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