An institutional experience of pre‐emptive liver transplantation for pediatric primary hyperoxaluria type 1
An institutional experience of pre‐emptive liver transplantation for pediatric primary hyperoxaluria type 1
复制标题
抢先性肝移植治疗小儿原发性高草酸尿症1型的机构经验
作者：
S. Khorsandi;M. Samyn;Akhila V. Hassan;H. Vilca‐Melendez;S. Waller;R. Shroff;G. Koffman;W. V. van’t Hoff;A. Baker;A. Dhawan;N. Heaton
原发性高尿酸1型（PH 1）是一种遗传性代谢疾病，最终导致ESRF。预先肝移植（pLTx）治疗代谢缺陷，避免了肾移植（KTx）的需要。报告了儿科PH 1 LTx的机构经验，并与文献进行了比较。在2004年至2015年期间，8名儿童接受了PH 1的pLTx。3例接受pLTx，中位GFR为40（30-46）mL/min/1.73 m2，5例接受序贯肝肾联合移植（cLKTx）;在cLKTx时均接受RRT。在一例pLTx病例中，八年半后需要KTx。pLTx是在年龄较大（中位数8岁与2岁）和较大的儿童（中位数27与7.75 kg），有一个温和的PH 1表型。在儿科PH 1中，理想情况下，应在肾和肾外系统性草酸盐中毒并发症发生之前进行pLTx，pLTx可以“早期”或“晚期”使用。早期是指肾功能得以保留，目的是避免肾脏替代。然而，在晚期（GFR < 30 mL/min/1.73 m2），目的是稳定肾功能并延迟对KTx的需求。最终，移植策略取决于PH 1表型，疾病阶段，儿童大小和器官可用性。
Primary hyperoxaluria type 1 (PH1) is an inherited metabolic disease that culminates in ESRF. Pre‐emptive liver transplantation (pLTx) treats the metabolic defect and avoids the need for kidney transplantation (KTx). An institutional experience of pediatric PH1 LTx is reported and compared to the literature. Between 2004 and 2015, eight children underwent pLTx for PH1. Three underwent pLTx with a median GFR of 40 (30–46) mL/min/1.73 m2 and five underwent sequential combined liver‐kidney transplantation (cLKTx); all were on RRT at the time of cLKTx. In one case of pLTx, KTx was required eight and a half yr later. pLTx was performed in older (median 8 vs. 2 yr) and larger children (median 27 vs. 7.75 kg) that had a milder PH1 phenotype. In pediatric PH1, pLTx, ideally, should be performed before renal and extrarenal systemic oxalosis complications have occurred, and pLTx can be used “early” or “late.” Early is when renal function is preserved with the aim to avoid renal replacement. However, in late (GFR < 30 mL/min/1.73 m2), the aim is to stabilize renal function and delay the need for KTx. Ultimately, transplant strategy depends on PH1 phenotype, disease stage, child size, and organ availability.