Department of Translational Medicine, University of Ferrara, 44121 Ferrara, Italy.
Department of Internal Medicine, S.S. Annunziata Hospital, Cento (Ferrara), University of Ferrara, 44042 Ferrara, Italy.
Int J Environ Res Public Health. 2021 Mar 23;18(6):3320. doi: 10.3390/ijerph18063320.
Anderson-Fabry disease (FD) is an X-linked lysosomal storage disorder leading to a wide array of clinical manifestations. Among these, gastrointestinal (GI) symptoms such as abdominal pain, bloating, and diarrhea affect about half of the FD adults and more than half of FD children. GI symptoms could be the first manifestation of FD; however, being non-specific, they overlap with the clinical picture of other conditions, such as irritable bowel syndrome and inflammatory bowel disease. This common overlap is the main reason why FD patients are often unrecognized and diagnosis is delayed for many years. The present narrative review is aimed to promote awareness of the GI manifestations of FD amongst general practitioners and specialists and highlight the latest findings of this rare condition including diagnostic tools and therapies. Finally, we will discuss some preliminary data on a patient presenting with GI symptoms who turned to be affected by a variant of uncertain significance of alpha-galactosidase (GLA) gene.
安德森-法布里病(FD)是一种 X 连锁溶酶体贮积症,可导致广泛的临床表现。其中,胃肠道(GI)症状如腹痛、腹胀和腹泻影响约一半的 FD 成年患者和一半以上的 FD 儿童患者。GI 症状可能是 FD 的首发表现;然而,由于其非特异性,它们与其他疾病的临床表现重叠,如肠易激综合征和炎症性肠病。这种常见的重叠是 FD 患者经常未被识别且诊断被延迟多年的主要原因。本综述旨在提高全科医生和专家对 FD 的 GI 表现的认识,并强调这种罕见疾病的最新发现,包括诊断工具和治疗方法。最后,我们将讨论一些初步数据,一名出现 GI 症状的患者被发现携带 GLA 基因的意义未明的变异。