A Historical Cohort Study on the Efficacy of Glucocorticoids and Riboflavin Among Patients with Late-onset Multiple AcyI-CoA Dehydrogenase Deficiency

作     者：Xin-Yi Liu Zhi-Qiang Wang Dan-Ni Wang Min-Ting Lin Ning Wang Liu Xin-Yi;Wang Zhi-Qiang;Wang Dan-Ni;Lin Min-Ting;Wang Ning

作者机构：Department of Neurology and Institute of Neurology First Affiliated Hospital Fujian Medical University Fuzhou Fujian 350005 China Fujian Key Laboratory of Molecular Neurology Fuzhou Fujian 350005 China 

出 版 物：《Chinese Medical Journal》 (中华医学杂志（英文版）)

年 卷 期：2016年第129卷第2期

页      面：142-146页

核心收录：

学科分类：0710[理学-生物学] 090603[农学-临床兽医学] 071010[理学-生物化学与分子生物学] 1002[医学-临床医学] 07[理学] 09[农学] 0906[农学-兽医学] 

基　　金：grants from the National Natural Science Foundation of China，National Key Clinical Specialty Discipline Construction Program and Fujian Key Clinical Specialty Discipline Construction Program 

主　　题：Glucocorticoids Historical Cohort Study Late-onset Multiple Acyl-CoA Dehydrogenase Deficiency Lipid StorageMyopathy Riboflavin 

摘      要：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 l-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.