title: A Historical Cohort Study on the Efficacy of Glucocorticoids and Riboflavin Among Patients with Late-onset Multiple Acyl-CoA Dehydrogenase Deficiency.
abstract: BACKGROUND
  Late-onset multiple acyl-CoA dehydrogenase deficiency (MADD) is the most common type of lipid storage myopathies in China. Most patients with late-onset MADD are well responsive to riboflavin. Up to now, these patients are often treated with glucocorticoids as the first-line drug because they are misdiagnosed as polymyositis without muscle biopsy or gene analysis. Although glucocorticoids seem to improve the fatty acid metabolism of late-onset MADD, the objective evaluation of their rationalization on this disorder and comparison with riboflavin treatment are unknown.
  
  
  METHODS
  We performed a historical cohort study on the efficacy of the two drugs among 45 patients with late-onset MADD, who were divided into glucocorticoids group and riboflavin group. Detailed clinical information of baseline and 1-month follow-up were collected.
  
  
  RESULTS
  After 1-month treatment, a dramatic improvement of muscle strength was found in riboflavin group (P < 0.05). There was no significant difference in muscle enzymes between the two groups. Significantly, the number of patients with full recovery in glucocorticoids group was less than the number in riboflavin group (P < 0.05). On the other hand, almost half of the patients in riboflavin group still presented high-level muscle enzymes and weak muscle strength after 1-month riboflavin treatment, meaning that 1-month treatment duration maybe insufficient and patients should keep on riboflavin supplement for a longer time.
  
  
  CONCLUSIONS
  Our results provide credible evidences that the overall efficacy of riboflavin is superior to glucocorticoids, and a longer duration of riboflavin treatment is necessary for patients with late-onset MADD.
authors.forename: Xin-Yi
authors.initials: XY
authors.lastname: Liu
authors.forename: Zhi-Qiang
authors.initials: ZQ
authors.lastname: Wang
authors.forename: Dan-Ni
authors.initials: DN
authors.lastname: Wang
authors.forename: Min-Ting
authors.initials: MT
authors.lastname: Lin
authors.affiliation: Department of Neurology and Institute of Neurology, First Affiliated Hospital, Fujian Medical University; Fujian Key Laboratory of Molecular Neurology, Fuzhou, Fujian 350005, China.
authors.forename: Ning
authors.initials: N
authors.lastname: Wang
mesh_terms: D000293:Adolescent; D000328:Adult; D017668:Age of Onset; D002648:Child; D015331:Cohort Studies; D005260:Female; D005938:Glucocorticoids / Q000627:therapeutic use*; D006801:Humans; D008052:Lipid Metabolism, Inborn Errors / Q000628:therapy; D008297:Male; D054069:Multiple Acyl Coenzyme A Dehydrogenase Deficiency / Q000188:drug therapy*; D009136:Muscular Dystrophies / Q000628:therapy; D012256:Riboflavin / Q000627:therapeutic use*; D055815:Young Adult
chemical_list: D005938:Glucocorticoids; D012256:Riboflavin
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