Pediatrics, "Grigore T. Popa" University of Medicine and Pharmacy, 700115 Iasi, Romania.
Faculty of Medicine and Pharmacy, University of Oradea, 410087 Oradea, Romania.
Genes (Basel). 2023 Feb 17;14(2):516. doi: 10.3390/genes14020516.
Fabry disease is an X-linked lysosomal storage disease, second in prevalence after Gaucher disease. The onset of symptoms occurs in childhood or adolescence with palmo-plantar burning pains, hypo hidrosis, angiokeratomas, and corneal deposits. In the absence of diagnosis and treatment, the disease will progress to the late phase, characterized by progressive cardiac, cerebral and renal damage, and possible death. We present the case of an 11-year-old male boy who was transferred to the Pediatric Nephrology Department for palmo-plantar burning pain and end stage renal disease. Following the evaluations for the etiology of end stage renal disease we excluded the vasculitis, the neurologic diseases, extrapulmonary tuberculosis. Because of suggestive aspect at CT scan and lack of etiologic diagnosis of renal insufficiency we performed lymph node and kidney biopsy, with a surprising result for storage disease. The specific investigation confirmed the diagnosis.
法布里病是一种 X 连锁溶酶体贮积病,其发病率仅次于戈谢病。症状发作发生在儿童或青少年时期,伴有手掌-足底烧灼感、少汗、血管角质瘤和角膜沉积物。如果不进行诊断和治疗,疾病将进展到晚期,特征为进行性心脏、大脑和肾脏损伤,可能导致死亡。我们报告了一例 11 岁男性患儿,因手掌-足底烧灼感和终末期肾病转入儿科肾病科。在排除血管炎、神经系统疾病和肺外结核等终末期肾病病因后,我们进行了 CT 扫描检查和淋巴结及肾脏活检,结果提示贮积病,特异性检查证实了这一诊断。