Department of Dermatology, West China Hospital, Sichuan University, Chengdu, China.
Department of Pediatrics, West China Second University Hospital, Sichuan University, Chengdu, China.
Pediatr Dermatol. 2022 Nov;39(6):987-989. doi: 10.1111/pde.15051. Epub 2022 Jun 11.
Methylmalonic acidemia (MMA) is an autosomal recessive genetic disorder caused by decreased activity of methylmalonyl-CoA mutase or metabolic disturbance of its coenzyme cobalamin, cutaneous manifestations are rare clinical signs in this disease. Herein, we describe a Chinese boy with MMA fed with a formula lacking branched-chain amino acids presenting with erythroderma and acrodermatitis enteropathica-like rash, a homozygous nonsense mutation c.742C>T (p.Gln248*) was identified in the MMAA gene. The pedigree exhibited a non-Mendelian inheritance pattern which was attributed to maternal uniparental disomy on chromosome 4q26-q34.1 of the proband, confirmed by chromosomal microarray analysis. Our case highlights the association between skin changes and nutritional deficiency due to therapeutic amino acid restrictions in MMA.
甲基丙二酸血症(MMA)是一种常染色体隐性遗传疾病,由甲基丙二酰辅酶 A 变位酶活性降低或其辅酶钴胺素代谢紊乱引起,皮肤表现是该病罕见的临床体征。本文报道了一例中国男孩,因 MMA 喂养缺乏支链氨基酸的配方奶粉而出现红皮病和肠病性肢端皮炎样皮疹,在 MMAA 基因中发现纯合无义突变 c.742C>T(p.Gln248*)。家系呈现非孟德尔遗传模式,这归因于先证者 4q26-q34.1 号染色体上的母源性单亲二体,通过染色体微阵列分析得到证实。本病例强调了 MMA 因治疗性氨基酸限制导致的皮肤变化与营养缺乏之间的关联。