Department of Pediatric Nephrology, Emma Children's Hospital, Amsterdam UMC, University of Amsterdam, Amsterdam, The Netherlands.
Center for Experimental and Molecular Medicine, Amsterdam UMC, University of Amsterdam, Amsterdam, The Netherlands.
Pediatr Nephrol. 2021 Aug;36(8):2217-2226. doi: 10.1007/s00467-021-05043-6. Epub 2021 Apr 8.
Primary hyperoxaluria type 1 (PH1) is characterized by hepatic overproduction of oxalate and often results in kidney failure. Liver-kidney transplantation is recommended, either combined (CLKT) or sequentially performed (SLKT). The merits of SLKT and the place of an isolated kidney transplant (KT) in selected patients are unsettled. We systematically reviewed the literature focusing on patient and graft survival rates in relation to the chosen transplant strategy.
We searched MEDLINE and Embase using a broad search string, consisting of the terms 'transplantation' and 'hyperoxaluria'. Studies reporting on at least four transplanted patients were selected for quality assessment and data extraction.
We found 51 observational studies from 1975 to 2020, covering 756 CLKT, 405 KT and 89 SLKT, and 51 pre-emptive liver transplantations (PLT). Meta-analysis was impossible due to reported survival probabilities with varying follow-up. Two individual high-quality studies showed an evident kidney graft survival advantage for CLKT versus KT (87% vs. 14% at 15 years, p<0.05) with adjusted HR for graft failure of 0.14 (95% confidence interval: 0.05-0.41), while patient survival was similar. Three other high-quality studies reported 5-year kidney graft survival rates of 48-89% for CLKT and 14-45% for KT. PLT and SLKT yielded 1-year patient and graft survival rates up to 100% in small cohorts.
Our study suggests that CLKT leads to superior kidney graft survival compared to KT. However, evidence for merits of SLKT or for KT in pyridoxine-responsive patients was scarce, which warrants further studies, ideally using data from a large international registry.
原发性高草酸尿症 1 型（PH1）的特征是肝脏草酸过度生成，常导致肾衰竭。推荐进行肝-肾联合移植（CLKT）或序贯移植（SLKT）。SLKT 的优势以及在选定患者中进行孤立肾移植（KT）的位置尚不确定。我们系统地回顾了文献，重点关注与所选移植策略相关的患者和移植物存活率。
我们使用广泛的搜索字符串在 MEDLINE 和 Embase 中进行搜索，该搜索字符串由“移植”和“高草酸尿症”这两个术语组成。选择至少报告了 4 例移植患者的研究进行质量评估和数据提取。
我们从 1975 年至 2020 年共找到了 51 项观察性研究，涵盖了 756 例 CLKT、405 例 KT 和 89 例 SLKT，以及 51 例预防性肝移植（PLT）。由于报道的生存概率随随访时间而异，因此无法进行荟萃分析。两项高质量的单独研究表明，CLKT 与 KT 相比，明显具有肾脏移植物存活优势（15 年时为 87% vs. 14%，p<0.05），调整后的移植物衰竭风险比为 0.14（95%置信区间：0.05-0.41），而患者生存率相似。另外三项高质量研究报道，CLKT 的 5 年肾脏移植物存活率为 48-89%，而 KT 的为 14-45%。在小队列中，PLT 和 SLKT 的 1 年患者和移植物存活率高达 100%。
我们的研究表明，CLKT 可导致肾脏移植物存活率优于 KT。然而，关于 SLKT 的优势或 KT 在吡哆醇反应性患者中的优势的证据很少，这需要进一步的研究，理想情况下使用来自大型国际登记处的数据。
