Nephrology Department, Hôtel-Dieu de France, Saint Joseph University, Beirut, Lebanon.
Pathology Department, Hôtel-Dieu de France, Saint Joseph University, Beirut, Lebanon.
Nephron. 2021;145(5):503-507. doi: 10.1159/000516281. Epub 2021 May 27.
Adenine phosphoribosyltransferase (APRT) deficiency is a rare disorder caused by an autosomal recessive genetic disease leading to the deposition of 2,8-dihydroxyadenine (2,8-DHA) in the kidney. The disease remains under-recognized, oftentimes diagnosed in late stages of renal insufficiency or a failed kidney allograft with biopsy-proven disease recurrence. Here, we present the case of a 59-year-old middle eastern male patient diagnosed with 2,8-DHA nephropathy after a very unusual presentation, and we show how the initiation of an appropriate therapy slowed down his evolution toward kidney replacement therapies. His disease was found to be secondary to a specific APRT gene variant c.188G>A p (Gly63Asp) also described in 4 other patients, all from middle eastern origins.
腺嘌呤磷酸核糖基转移酶(APRT)缺陷是一种罕见的疾病,由常染色体隐性遗传疾病引起,导致肾脏中沉积 2,8-二羟腺嘌呤(2,8-DHA)。这种疾病仍然认识不足,通常在肾功能不全的晚期或移植肾活检证实疾病复发时才被诊断出来。在这里,我们介绍了一名 59 岁的中东男性患者的病例,他以非常不寻常的表现被诊断为 2,8-DHA 肾病,我们展示了如何启动适当的治疗来减缓他向肾脏替代治疗的进展。他的疾病被发现是由 APRT 基因的特定变体 c.188G>A p (Gly63Asp)引起的,该变体也在另外 4 名来自中东的患者中被描述。