Zaidan M, Palsson R, Merieau E, Cornec-Le Gall E, Garstka A, Maggiore U, Deteix P, Battista M, Gagné E-R, Ceballos-Picot I, Duong Van Huyen J-P, Legendre C, Daudon M, Edvardsson V O, Knebelmann B
Department of Nephrology-Transplantation, Necker Hospital, APHP, Paris, France; Paris Descartes University, Sorbonne Paris Cité, Paris, France; Département Biologie cellulaire, INSERM U1151, Institut Necker Enfants Malades, Paris, France.
Am J Transplant. 2014 Nov;14(11):2623-32. doi: 10.1111/ajt.12926. Epub 2014 Oct 10.
Adenine phosphoribosyltransferase (APRT) deficiency is a rare autosomal recessive enzyme defect of purine metabolism that usually manifests as 2,8-dihydroxyadenine (2,8-DHA) nephrolithiasis and more rarely chronic kidney disease. The disease is most often misdiagnosed and can recur in the renal allograft. We analyzed nine patients with recurrent 2,8-DHA crystalline nephropathy, in all of whom the diagnosis had been missed prior to renal transplantation. The diagnosis was established at a median of 5 (range 1.5-312) weeks following the transplant procedure. Patients had delayed graft function (n=2), acute-on-chronic (n=5) or acute (n=1) allograft dysfunction, whereas one patient had normal graft function at the time of diagnosis. Analysis of allograft biopsies showed birefringent 2,8-DHA crystals in renal tubular lumens, within tubular epithelial cells and interstitium. Fourier transformed infrared microscopy confirmed the diagnosis in all cases, which was further supported by 2,8-DHA crystalluria, undetectable erythrocyte APRT enzyme activity, and genetic testing. With allopurinol therapy, the allograft function improved (n=7), remained stable (n=1) or worsened (n=1). At last follow-up, two patients had experienced allograft loss and five had persistent chronic allograft dysfunction. 2,8-DHA nephropathy is a rare but underdiagnosed and preventable disorder that can recur in the renal allograft and may lead to allograft loss.
腺嘌呤磷酸核糖转移酶(APRT)缺乏症是一种罕见的嘌呤代谢常染色体隐性酶缺陷疾病,通常表现为2,8 - 二羟基腺嘌呤(2,8 - DHA)肾结石,较少见的表现为慢性肾病。该疾病常被误诊,且可在肾移植受者中复发。我们分析了9例复发性2,8 - DHA结晶性肾病患者,所有患者在肾移植前均未得到诊断。诊断在移植手术后中位时间为5(范围1.5 - 312)周时确立。患者出现移植肾功能延迟(n = 2)、慢性基础上急性发作(n = 5)或急性(n = 1)移植肾功能障碍,而1例患者在诊断时移植肾功能正常。对移植肾活检分析显示,在肾小管管腔、肾小管上皮细胞内及间质中有双折射的2,8 - DHA晶体。傅里叶变换红外显微镜检查在所有病例中均确诊,2,8 - DHA结晶尿、红细胞APRT酶活性检测不到以及基因检测进一步支持了该诊断。使用别嘌醇治疗后,移植肾功能改善(n = 7)、保持稳定(n = 1)或恶化(n = 1)。在最后一次随访时,2例患者移植肾失功,5例患者持续存在慢性移植肾功能障碍。2,8 - DHA肾病是一种罕见但诊断不足且可预防的疾病,可在肾移植受者中复发,并可能导致移植肾失功。