Beyzaei Zahra, Shamsaeefar Alireza, Ghatei Kiana, Kazemi Kurosh, Nikeghbalian Saman, Bahador Ali, Dehghani Masoud, Malekhosseini Seyed-Ali, Geramizadeh Bita
Transplant Research Center, Shiraz University of Medical Sciences, Shiraz, Iran.
Shiraz Transplant Center, Abu-Ali-Sina Hospital, Shiraz University of Medical Sciences, Shiraz, Iran.
Pediatr Transplant. 2025 Jun;29(4):e70102. doi: 10.1111/petr.70102.
Primary hyperoxalurias (PHs) are rare inborn errors of metabolism caused by a deficiency of hepatic enzymes, leading to excessive urinary oxalate excretion and the overproduction of oxalate, which accumulates in various organs. If left untreated, PHs can cause serious morbidity, end-stage kidney disease (ESKD), and mortality. Liver transplantation (LT) is a recognized treatment option for children with these diseases. This study aimed to analyze the outcome of PHs disease post-LT from a single center in Iran.
This retrospective, single-center study was conducted at the Shiraz Transplant Center from 2012 to 2023, focusing on liver transplant recipients with PH. We evaluated long-term outcomes and post-transplantation results for both deceased donor liver transplantation (DDLT) and living donor liver transplantation (LDLT). Biochemical lab results (pre- and post-transplantation), perioperative data, surgical procedures, transplantation outcomes, and recipient and donor characteristics were reported. Kaplan-Meier survival analysis was used to assess graft and patient survival.
33 recipients with LT (LDLT, n = 6; DDLT, n = 27) were included. The median age at the time of transplantation was 8 years (range: 3-18 years). Following liver transplantation, all of the patients had normalization of liver enzymes. Urine oxalate levels gradually decreased from 198 to 51 (< 45 mg/1.73 m/day). Among the 33 patients, eight experienced episodes of acute rejection, and five developed chronic rejection. Eight patients underwent kidney transplantation before liver transplantation, while 21 patients initially received liver transplantation. 26 patients survived and remained in good health during a median follow-up period of 7 years (range: 1.5-11 years). For patients with PHs, the survival rates at 6 months, 1 year, 3 years, and 5 years were 100%, 97%, 94%, and 85%, respectively. The graft survival for patients was 100%, 100%, 97%, and 97% at 6 months, 1 year, 3 years, and 5 years, respectively.
PHs is a rare metabolic disorder, and LT significantly improves both survival and quality of life for affected patients. In our cohort, the majority of patients exhibited favorable long-term outcomes, along with a notable reduction in urine oxalate levels post-transplantation. However, challenges persist, including graft shortages and the risk of renal graft loss due to oxalosis, which continue to affect overall treatment outcomes. These findings highlight the importance of close monitoring and multidisciplinary care in managing PH patients' post-transplant.
原发性高草酸尿症（PHs）是一种罕见的先天性代谢紊乱疾病，由肝脏酶缺乏引起，导致尿草酸排泄过多和草酸盐过度生成，并在各个器官中蓄积。若不治疗，PHs可导致严重发病、终末期肾病（ESKD）和死亡。肝移植（LT）是治疗这些疾病患儿的一种公认的治疗选择。本研究旨在分析伊朗一个单一中心肝移植后PHs疾病的治疗结果。
本回顾性单中心研究于2012年至2023年在设拉子移植中心进行，重点关注肝移植受者中的PH患者。我们评估了尸体供肝肝移植（DDLT）和活体供肝肝移植（LDLT）的长期结果和移植后结果。报告了生化实验室结果（移植前后）、围手术期数据、手术程序、移植结果以及受者和供者特征。采用Kaplan-Meier生存分析评估移植物和患者的生存率。
纳入33例接受肝移植的受者（LDLT，n = 6；DDLT，n = 27）。移植时的中位年龄为8岁（范围：3 - 18岁）。肝移植后，所有患者的肝酶均恢复正常。尿草酸水平从198逐渐降至51（<45mg/1.73m²/天）。在33例患者中，8例发生急性排斥反应，5例发生慢性排斥反应。8例患者在肝移植前接受了肾移植，而21例患者最初接受了肝移植。26例患者存活，在中位随访期7年（范围：1.5 - 11年）内保持健康。对于PHs患者，6个月、1年、3年和5年的生存率分别为100%、97%、94%和85%。患者的移植物生存率在6个月、1年、3年和5年时分别为100%、100%、97%和97%。
PHs是一种罕见的代谢紊乱疾病，肝移植显著提高了受影响患者的生存率和生活质量。在我们的队列中，大多数患者表现出良好的长期结果，移植后尿草酸水平显著降低。然而，挑战依然存在，包括移植物短缺以及由于草酸沉着症导致肾移植物丢失的风险，这些继续影响总体治疗结果。这些发现凸显了在管理PH患者移植后密切监测和多学科护理的重要性。
