Abstract
Background
Scoliosis in patients with Duchenne muscular dystrophy (DMD) is usually progressive and is treated with surgery. However, it is unclear whether the existing evidence is sufficiently scientifically rigorous to support a recommendation for spinal surgery for most patients with DMD and scoliosis. This is an updated review, and an updated search was undertaken in which no new studies were found for inclusion.
Objectives
To determine the effectiveness and safety of spinal surgery in patients with DMD with scoliosis. We intended to test whether spinal surgery is effective in increasing survival and improving respiratory function, quality of life, and overall functioning, and whether spinal surgery is associated with severe adverse effects.
Search methods
On 16 June 2015 we searched the Cochrane Neuromuscular Disease Group Specialized Register, the Cochrane Central Register of Controlled Trials (CENTRAL), MEDLINE, EMBASE, and CINAHL Plus. We also searched ProQuest Dissertation and Thesis database (January 1980 to June 2015), the National Institutes of Health Clinical Trials Database (6 January 2015), and the WHO International Clinical Trials Registry Platform (17 June 2015), and checked references. We imposed no language restrictions.
Selection criteria
We planned to include controlled clinical trials using random or quasi-random allocation of treatment evaluating all forms of spinal surgery for scoliosis in patients with DMD in the review. The control interventions would have been no treatment, non-operative treatment, or a different form of spinal surgery.
Data collection and analysis
We used standard methodological procedures expected by The Cochrane Collaboration. Two review authors independently examined the search results and evaluated the study characteristics against inclusion criteria in order to decide which studies to include in the review.
Main results
Of the 49 relevant studies we found, none met the inclusion criteria for the review because they were not clinical trials, but prospective or retrospective reviews of case series.
Authors’ conclusions
Since no randomized controlled clinical trials were available to evaluate the effectiveness of scoliosis surgery in patients with DMD, we can make no good evidence-based conclusion to guide clinical practice. Patients with scoliosis should be informed as to the uncertainty of benefits and potential risks of surgery for scoliosis. Randomized controlled trials are needed to investigate the effectiveness of scoliosis surgery, in terms of quality of life, functional status, respiratory function, and life expectancy.
Plain language summary
Surgery for curvature of the spine in patients with Duchenne muscular dystrophy
Review question
What is the effectiveness and safety of spinal surgery to treat scoliosis in patients with Duchenne muscular dystrophy (DMD)?
Background
Scoliosis, or curvature of the spine, is common in patients with DMD. It is usually progressive, and surgery is often performed to halt its progression, improve cosmetic appearance, facilitate care, preserve upper limb and respiratory function, and hopefully increase life expectancy. We wished to learn whether spinal surgery was better or worse than the alternatives.
Study characteristics
We found no randomized controlled trials.
Key results and quality of the evidence
We found 49 relevant studies, however they were not clinical trials but prospective or retrospective reviews of case series. The quality of evidence was very low because no clinical trial was available. This is an updated review, and an updated search was undertaken in which no new studies were found.
Conclusion
No randomized controlled clinical trials are available to evaluate the effectiveness of scoliosis surgery in patients with DMD. Randomized controlled clinical trials are needed in this group of patients to evaluate the benefits and risks of different surgical treatments.
The evidence is current to 5 January 2015.
