Best practice in Duchenne muscular dystrophy

摘要

The authors of the Lancet article have clearly put in a great deal of hard work and effort in order to produce their extensive document outlining care guidelines for DMD. However, the recent editorial by Professor Dubowitz highlights some very important considerations. There is no doubt that an article such as the Lancet care pathway will influence commissioners who ultimately hold the purse strings for service development. The DMD care pathway is complex and aims to make recommendations for all eventualities. Because of the dearth of good quality scientific research the authors have used a novel consensus approach to arrive at a conclusion for best practice. Unfortunately, this has led to some sweeping statements, which lack evidence. One such statement which causes concern is: 'Surgery in a patient who has DMD should be done in a full-service hospital that has experience of patients with DMD'. Many neuromuscular centres, such as ours, are based in rehabilitation hospitals that do not have 'full hospital services' but which do have many decades of experience in caring for these patients which has included surgical procedures such as diagnostic muscle biopsy and TA lengthening. The implication of this statement is that our practice is unsafe. However, in 25 years our centre had excellent results and no serious complications from spinal surgery.