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A randomized controlled trial of early surgery in Duchenne muscular dystrophy
A Y Manzur1, S A Hyde, E Rodillo
1Hammersmith Neuromuscular Centre, Department of Paediatrics, Royal Postgraduate Medical School, London, U.K.
Insights
Early surgery for contractures in young boys with Duchenne muscular dystrophy corrected deformities but did not improve strength or function. Some patients experienced faster functional decline post-operation.
Area of Science:
- Pediatric Orthopedics
- Neuromuscular Disorders
- Rehabilitative Medicine
Background:
- Contractures are common in Duchenne muscular dystrophy (DMD).
- Early intervention aims to manage functional limitations in pediatric patients with DMD.
Purpose of the Study:
- To evaluate the efficacy of early surgical intervention for contractures in boys with Duchenne muscular dystrophy.
Main Methods:
- A randomized controlled trial involving 20 boys (age 4-6 years) with DMD.
- Surgical procedures included hip flexor release, iliotibial band excision, and bilateral tendo Achilles lengthening.
- Quantitative assessments of muscle strength and function were conducted for at least 12 months post-randomization.
Main Results:
- Surgery successfully corrected deformities in all patients.
- No significant beneficial effect on muscle strength or overall function was observed.
- Data indicated a more rapid deterioration of function in some surgically treated patients during the second year.
- Sequential muscle ultrasound and biopsy suggested continued disease progression post-surgery.
Conclusions:
- Early surgical treatment for contractures in young DMD patients does not improve strength or function.
- The surgical approach may not alter the natural disease course and could potentially accelerate functional decline.
- This type of surgery is not recommended as a routine treatment for Duchenne muscular dystrophy contractures.
Abstract:
We performed a randomized controlled trial of early surgical treatment of contractures in 20 boys with Duchenne muscular dystrophy, age 4-6 yr. Surgery consisted of release of hip flexors, removal of iliotibial bands, and lengthening of tendo Achilles bilaterally. All patients were monitored for at least 12 months post-randomization, and assessed quantitatively for muscle strength and function. Surgery corrected the deformities, but had no beneficial effect on strength or function. Indeed, data in the second year showed more rapid deterioration of function in some of the operated boys. There appeared to be continued evolution of pathology following surgery, as assessed by sequential muscle ultrasound and muscle biopsy. We cannot recommend this type of surgery as a routine treatment.