Arnon Ronen, Annunziato Rachel, Miloh Tamir, Wasserstein Melissa, Sogawa Hiroshi, Wilson Monique, Suchy Frederick, Kerkar Nanda
Department of Pediatrics, Mount Sinai School of Medicine, New York, NY, USA.
Pediatr Transplant. 2011 Jun;15(4):400-5. doi: 10.1111/j.1399-3046.2011.01497.x. Epub 2011 Apr 19.
Patients with HT-1 can develop progressive liver disease and have a high incidence of HCC. LT is indicated in patients with fulminant liver failure, HCC or decompensated chronic liver disease refractory to NTBC. To determine the need for LT and outcomes after LT in children with HT-1. Children with HT-1 who had LT between 10/1987 and 5/2008 were identified from the UNOS database. Of 11,467 children in the UNOS database, 125 (1.1%) required LT secondary to HT-1. Mean age at LT was two and half yr (s.d. ± 3.6 yr). Mean age at LT during the first 10 yr of the study (1.82, s.d. ± 2.86 yr) was significantly lower than in the last decade (3.70, s.d. ± 4.42 yr), p = 0.01. Nearly half of the patients (58, 46.4%) were transplanted between 1988 and 1992. Overall, one- and five-yr patient survival was 90.4% and 90.4%, respectively. LT is a valuable option for children with HT-1 with fulminant liver failure or when medical treatment fails. The rate of LT for children with HT-1 has decreased and age at transplant increased over the last decade most probably reflecting the effect of early diagnosis and treatment with NTBC.
HT-1患者可发展为进行性肝病，且肝癌发病率较高。对于暴发性肝衰竭、肝癌或对NTBC难治的失代偿性慢性肝病患者，肝移植是一种治疗选择。目的是确定HT-1儿童患者肝移植的必要性及肝移植后的预后情况。从UNOS数据库中识别出1987年10月至2008年5月期间接受肝移植的HT-1儿童患者。在UNOS数据库的11467名儿童中，125名（1.1%）因HT-1需要进行肝移植。肝移植时的平均年龄为两岁半（标准差±3.6岁）。研究前10年肝移植时的平均年龄（1.82岁，标准差±2.86岁）显著低于过去十年（3.70岁，标准差±4.42岁），p = 0.01。近一半患者（58名，46.4%）在1988年至1992年期间接受移植。总体而言，患者1年和5年生存率分别为90.4%和90.4%。对于患有暴发性肝衰竭或药物治疗失败的HT-1儿童患者，肝移植是一种有价值的选择。在过去十年中，HT-1儿童患者的肝移植率有所下降，移植年龄有所增加，这很可能反映了NTBC早期诊断和治疗的效果。
