A Historical Cohort Study on the Efficacy of Glucocorticoids and Riboflavin Among Patients with Late-onset Multiple Acyl-CoA Dehydrogenase Deficiency
A Historical Cohort Study on the Efficacy of Glucocorticoids and Riboflavin Among Patients with Late-onset Multiple Acyl-CoA Dehydrogenase Deficiency
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糖皮质激素和核黄素对迟发性多酰基辅酶A脱氢酶缺乏症患者疗效的历史队列研究
DOI：
10.4103/0366-6999.173438
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发表时间：
2016-01-20
影响因子：
6.1
通讯作者：
Wang, Ning
中科院分区：
文献类型：
--
作者：
Liu, Xin-Yi;Wang, Zhi-Qiang;Wang, Ning
背景：迟发性多发性酰基辅酶A脱氢酶缺乏症（MADD）是我国最常见的脂质沉积性肌病。大多数迟发性MADD患者对核黄素反应良好。到目前为止，这些患者往往是糖皮质激素作为一线药物治疗，因为他们被误诊为多发性肌炎没有肌肉活检或基因分析。虽然糖皮质激素似乎可以改善迟发性MADD的脂肪酸代谢，但对其合理性的客观评价以及与核黄素治疗的比较尚不清楚。方法：对45例晚发MADD患者进行历史性队列研究，分为糖皮质激素组和核黄素组。收集基线和1个月随访的详细临床信息。结果：治疗1个月后，核黄素组肌力明显改善（P < 0.05）。两组间肌酶无显著差异。糖皮质激素组痊愈例数明显少于核黄素组（P < 0.05）。另一方面，核黄素组中几乎一半的患者在核黄素治疗1个月后仍表现出高水平的肌酶和较弱的肌肉力量，这意味着1个月的治疗时间可能不够，患者应继续补充核黄素更长的时间。结论：我们的研究结果提供了可靠的证据，核黄素的总体疗效上级糖皮质激素，核黄素治疗晚发MADD患者需要更长的时间。
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.