Study of medical management for patients with Duchenne muscular dystrophy in Japan: what was changed by a practical guideline

Abstract

In order to clarify the changes in the medical management of patients with Duchenne muscular dystrophy after publication of a practical guideline, questionnaire surveys targeting Japanese certified neurologists and child neurologists before and four years after publication of the guideline were conducted. The practical guideline was evaluated by the majority of specialists as their preferred information source. After the guideline, a more aggressive trend was seen in steroid treatment, rehabilitation, regular assessment of spinal deformity, etc., and significant differences were observed in respiratory care and nutritional management. Although there were concerns that it would be hard to produce useful guidelines due to the lack of high-level evidence for a rare disease, guidelines including expert recommendations have proved to be an important tool for disseminating best practicable care. We are planning to revise the guideline based on the results of the analysis.