Nephrology Center, Toranomon Hospital, Japan.
Okinaka Memorial Institute, Toranomon Hospital, Japan.
Intern Med. 2021 May 15;60(10):1561-1565. doi: 10.2169/internalmedicine.5992-20. Epub 2020 Dec 22.
Fabry disease (FD) is an X-linked lysosomal storage disorder resulting from a lack of alpha-galactosidase A (AGALA) activity in lysosomes. We herein report a patient with FD revealed by a renal biopsy who survived seven years after the introduction of peritoneal dialysis despite having severe heart failure due to left ventricular hypertrophy (LVH). FD was diagnosed based on a renal biopsy and biochemical analysis showing a low enzymatic activity of AGALA. A microscopic examination at the autopsy revealed marked hypertrophy and vacuolation of cardiac muscle cells. In our case, cardiac involvement determined the prognosis. Peritoneal dialysis is the modality of choice in the long-term management of dialysis patients with FD.
法布里病(FD)是一种 X 连锁溶酶体贮积症,由溶酶体中缺乏α-半乳糖苷酶 A(AGALA)活性引起。我们在此报告一例肾活检证实的 FD 患者,尽管因左心室肥厚(LVH)导致严重心力衰竭,但在引入腹膜透析后存活了七年。FD 的诊断基于肾活检和生化分析,显示 AGALA 的酶活性低。尸检的显微镜检查显示心肌细胞明显肥大和空泡化。在我们的病例中,心脏受累决定了预后。腹膜透析是 FD 透析患者长期管理的首选方式。