Arnon Ronen, Kerkar Nanda, Davis Michael K, Anand Ravinder, Yin Wanrong, González-Peralta Regino P
Department of Pediatrics, Mount Sinai Medical Center, Mount Sinai School of Medicine, New York, NY 10029, USA.
Pediatr Transplant. 2010 Sep 1;14(6):796-805. doi: 10.1111/j.1399-3046.2010.01339.x. Epub 2010 Jun 27.
Metabolic diseases are the second largest indication for LT in children after BA. There are limited data on the long-term post-transplant outcome in this unique group of patients. Therefore, our aim was to assess post-liver transplant outcomes and to evaluate risk factors for mortality and graft loss in children with metabolic disorders in comparison to those with non-metabolic diagnoses. We reviewed all patients enrolled in the SPLIT registry. Between 1995 and 2008, 446 of 2997 (14.9%) children enrolled in SPLIT underwent liver transplant for metabolic diseases. One-yr and five-yr patient survival for children with metabolic diseases was 94.6% and 88.9% and for those with other diseases 90.7% and 86.1% (log-rank p = 0.05), respectively. One-yr and five-yr graft survival for children with metabolic disorders was 90.8% and 83.8%, and for those with other diseases 85.4% and 78.0% (log-rank p = 0.005), respectively. Children with metabolic diseases were less likely to experience gastrointestinal complications (5.6% vs. 10.7%, p = 0.001), portal vein thrombosis (2.9% vs. 5.2%, p = 0.04), and reoperations within 30 days post-transplant (33.4% vs. 37.8%, p = 0.05) than those with other indications. In conclusion, children who underwent liver transplant for metabolic disease had similarly excellent patient survival as, and better graft survival than, those who received a liver allograft for other indications.
代谢性疾病是儿童肝移植仅次于胆道闭锁的第二大适应症。关于这一独特患者群体移植后的长期结局的数据有限。因此，我们的目的是评估肝移植后的结局，并比较代谢紊乱儿童与非代谢性诊断儿童的死亡和移植物丢失风险因素。我们回顾了所有纳入SPLIT登记处的患者。1995年至2008年期间，在SPLIT登记的2997名儿童中有446名（14.9%）因代谢性疾病接受了肝移植。代谢性疾病儿童的1年和5年患者生存率分别为94.6%和88.9%，其他疾病儿童的1年和5年患者生存率分别为90.7%和86.1%（对数秩检验p=0.05）。代谢紊乱儿童的1年和5年移植物生存率分别为90.8%和83.8%，其他疾病儿童的1年和5年移植物生存率分别为85.4%和78.0%（对数秩检验p=0.005）。与其他适应症的儿童相比，代谢性疾病儿童发生胃肠道并发症（5.6%对10.7%，p=0.001）、门静脉血栓形成（2.9%对5.2%，p=0.04）以及移植后30天内再次手术（33.4%对37.8%，p=0.05）的可能性较小。总之，因代谢性疾病接受肝移植的儿童与因其他适应症接受肝移植的儿童相比，患者生存率同样出色，移植物生存率更高。
