gms | German Medical Science

Objectives: Different regimens of steroids are currently the main component of drug therapy for Duchenne muscular dystrophy (DMD), prolonging ambulation, preserving upper limb and respiratory function and avoiding scoliosis. A multilevel contracture release has been shown to effect ambulation positively in DMD, with patients operated on early in the course of the disease benefiting most. However, an independent effect of steroid treatment has not been evaluated and, to date, international guidelines do not include multilevel surgery.

Methods: We analysed all DMD patients who consulted our outpatient clinic between 2013 & 2017. Ambulatory patients had been offered steroid treatment. Patients with initial contractures of the hip, knee and ankle joints and shortening of the iliotibial band were offered bilateral hip and knee releases, aponeurectomy of the IT band and Achilles tendon lengthening. If they had sufficient quadriceps strength, i.e. total muscle force of >/=3/5 MRC, and were able to rise from a supine position to standing in <5s. Patients received soft casts for one week and were mobilised out of bed on postoperative day one. They underwent inpatient physical therapy (PT) for 46 weeks. None of them was splinted after surgery.

Results: 86 patients were included. Mean age was 16.3 yrs. All patients received nonstandardised PT. 44 had been treated with glucocorticoids (GC) for a mean of 5.6 yrs. Median age at loss of ambulation was 12yrs in patients treated with GC as opposed to 9 yrs in those without GC. 27 patients underwent Rideau's multilevel contracture release at a median age of 7.1 yrs (410). These patients lost ambulation at a median age of 12 yrs. We found a significant additive effect of both therapies: while patients having received neither GC nor surgery lost ambulation at a median age of 9 yrs, those with GC and surgery were able to walk independently until a median age of 14 yrs, hence 2 yrs longer than with only one of the two treatment options.

Conclusion: Standard GC treatment and early multilevel contracture release in lower limbs have a positive effect on prolongation of ambulation in DMD. Combination of both therapies is significantly more effective than each single therapy.