University of Cambridge School of Clinical Medicine, Cambridge, UK
The University of Notre Dame Australia School of Medicine, Fremantle, Australia.
BMJ Case Rep. 2024 Mar 15;17(3):e259192. doi: 10.1136/bcr-2023-259192.
Multiple acyl-coenzyme A dehydrogenase deficiency (MADD) is a rare metabolic disorder which typically manifests with muscle weakness. However, despite late-onset MADD being treatable, it is often misdiagnosed, due in part to the heterogeneity of presentations. We report a case of late-onset MADD manifesting first as a sensory neuropathy before progressing to myopathic symptoms and acute metabolic decompensation. Early diagnostic workup with acylcarnitine profiling and organic acid analysis was critical in patient outcome; metabolic decompensation and myopathic symptoms were completely reversed with riboflavin supplementation and dietary modification, although sensory neuropathy persisted. Clinical consideration of MADD as part of the differential diagnosis of neuropathy with myopathy is crucial for a timely diagnosis and treatment of MADD.
多种酰基辅酶 A 脱氢酶缺乏症(MADD)是一种罕见的代谢紊乱疾病,通常表现为肌肉无力。然而,尽管迟发性 MADD 可治疗,但由于表现形式的异质性,常被误诊。我们报告了一例迟发性 MADD 的病例,其最初表现为感觉性神经病,然后进展为肌病症状和急性代谢失代偿。酰基辅酶 A 谱分析和有机酸分析的早期诊断工作对患者的预后至关重要;补充核黄素和饮食调整完全逆转了代谢失代偿和肌病症状,尽管感觉性神经病仍持续存在。将 MADD 作为伴有肌病的神经病鉴别诊断的一部分进行临床考虑,对于及时诊断和治疗 MADD 至关重要。