Loos Sebastian, Kemper Markus J, Schmaeschke Kaja, Herden Uta, Fischer Lutz, Hoppe Bernd, Levart Tanja Kersnik, Grabhorn Enke, Schild Raphael, Oh Jun, Brinkert Florian
Department of Pediatric Nephrology, University Children's Hospital, University Medical Center Hamburg-Eppendorf, Hamburg, Germany.
Department of Pediatrics, Asklepios Klinik Nord Heidberg, Hamburg, Germany.
Front Pediatr. 2023 Mar 17;11:1157215. doi: 10.3389/fped.2023.1157215. eCollection 2023.
Combined or sequential liver and kidney transplantation (CLKT/SLKT) restores kidney function and corrects the underlying metabolic defect in children with end-stage kidney disease in primary hyperoxaluria type 1 (PH1). However, data on long-term outcome, especially in children with infantile PH1, are rare.
All pediatric PH1-patients who underwent CLKT/SLKT at our center were analyzed retrospectively.
Eighteen patients (infantile PH1 = 10, juvenile PH1 = 8) underwent transplantation (CLKT = 17, SLKT = 1) at a median age of 5.4 years (1.5-11.8). Patient survival was 94% after a median follow-up of 9.2 years (6.4-11.0). Liver and kidney survival-rates after 1, 10, and 15 years were 90%, 85%, 85%, and 90%, 75%, 75%, respectively. Age at transplantation was significantly lower in infantile than juvenile PH1 (1.6 years (1.4-2.4) vs. 12.8 years (8.4-14.1), = 0.003). Median follow-up was 11.0 years (6.8-11.6) in patients with infantile PH1 vs. 6.9 years (5.7-9.9) in juvenile PH1 ( = 0.15). At latest follow-up kidney and/or liver graft loss and/or death showed a tendency to a higher rate in patients with infantile vs. juvenile PH1 (3/10 vs. 1/8, = 0.59).
In conclusion, the overall patient survival and long-term transplant outcome of patients after CLKT/SLKT for PH1 is encouraging. However, results in infantile PH1 tended to be less optimal than in patients with juvenile PH1.
肝肾联合移植或序贯肝肾移植（CLKT/SLKT）可恢复1型原发性高草酸尿症（PH1）终末期肾病患儿的肾功能，并纠正潜在的代谢缺陷。然而，关于长期预后的数据，尤其是婴儿型PH1患儿的数据很少。
对在本中心接受CLKT/SLKT的所有儿科PH1患者进行回顾性分析。
18例患者（婴儿型PH1 = 10例，青少年型PH1 = 8例）接受了移植（CLKT = 17例，SLKT = 1例），中位年龄为5.4岁（1.5 - 11.8岁）。中位随访9.2年（6.4 - 11.0年）后患者生存率为94%。1年、10年和15年后肝脏和肾脏的生存率分别为90%、85%、85%和90%、75%、75%。婴儿型PH1患者的移植年龄显著低于青少年型PH1患者（1.6岁（1.4 - 2.4岁）对12.8岁（8.4 - 14.1岁），P = 0.003）。婴儿型PH1患者的中位随访时间为11.0年（6.8 - 11.6年），青少年型PH1患者为6.9年（5.7 - 9.9年）（P = 0.15）。在最近一次随访时，婴儿型PH1患者与青少年型PH1患者相比，肾脏和/或肝脏移植丢失和/或死亡的发生率有升高趋势（3/10对1/8，P = 0.59）。
总之，PH1患者接受CLKT/SLKT后的总体患者生存率和长期移植预后令人鼓舞。然而，婴儿型PH1的结果往往不如青少年型PH1患者理想。
