William J. von Liebig Transplant Center, Mayo Clinic, Rochester, Minnesota 55905, USA.
Gastroenterology. 2012 Jul;143(1):88-98.e3; quiz e14. doi: 10.1053/j.gastro.2012.04.008. Epub 2012 Apr 12.
BACKGROUND & AIMS: Excellent single-center outcomes of neoadjuvant chemoradiation and liver transplantation for unresectable perihilar cholangiocarcinoma caused the United Network of Organ Sharing to offer a standardized model of end-stage liver disease (MELD) exception for this disease. We analyzed data from multiple centers to determine the effectiveness of this treatment and the appropriateness of the MELD exception.
We collected and analyzed data from 12 large-volume transplant centers in the United States. These centers met the inclusion criteria of treating 3 or more patients with perihilar cholangiocarcinoma using neoadjuvant therapy, followed by liver transplantation, from 1993 to 2010 (n = 287 total patients). Center-specific protocols and medical charts were reviewed on-site.
The patients completed external radiation (99%), brachytherapy (75%), radiosensitizing therapy (98%), and/or maintenance chemotherapy (65%). Seventy-one patients dropped out before liver transplantation (rate, 11.5% in 3 months). Intent-to-treat survival rates were 68% and 53%, 2 and 5 years after therapy, respectively; post-transplant, recurrence-free survival rates were 78% and 65%, respectively. Patients outside the United Network of Organ Sharing criteria (those with tumor mass >3 cm, transperitoneal tumor biopsy, or metastatic disease) or with a prior malignancy had significantly shorter survival times (P < .001). There were no differences in outcomes among patients based on differences in surgical staging or brachytherapy. Although most patients came from 1 center (n = 193), the other 11 centers had similar survival times after therapy.
Patients with perihilar cholangiocarcinoma who were treated with neoadjuvant therapy followed up by liver transplantation at 12 US centers had a 65% rate of recurrence-free survival after 5 years, showing this therapy to be highly effective. An 11.5% drop-out rate after 3.5 months of therapy indicates the appropriateness of the MELD exception. Rigorous selection is important for the continued success of this treatment.
新辅助放化疗和肝移植治疗不可切除的肝门部胆管癌取得了卓越的单中心疗效，促使器官共享联合网络为此疾病提供了终末期肝病模型（MELD）评分例外标准。我们分析了多中心数据，以确定该治疗方法的有效性和 MELD 评分例外的适宜性。
我们收集并分析了美国 12 家大容量移植中心的数据。这些中心符合纳入标准，即从 1993 年至 2010 年，采用新辅助治疗（n = 287 例患者）治疗 3 例或 3 例以上肝门部胆管癌患者，随后进行肝移植。对中心特定的方案和病历进行了现场审查。
患者均完成了外照射（99%）、近距离放疗（75%）、增敏治疗（98%）和/或维持化疗（65%）。71 例患者在肝移植前退出（3 个月内的退出率为 11.5%）。意向治疗生存率分别为治疗后 2 年和 5 年的 68%和 53%；移植后无复发生存率分别为 78%和 65%。不符合器官共享联合网络标准（肿瘤直径＞3cm、腹膜后肿瘤活检或转移疾病）或有既往恶性肿瘤的患者生存时间明显缩短（P＜0.001）。根据手术分期或近距离放疗的不同，患者结局无差异。尽管大多数患者来自 1 个中心（n = 193），但其他 11 个中心治疗后的生存时间相似。
在 12 家美国中心接受新辅助治疗后进行肝移植的肝门部胆管癌患者，5 年后无复发生存率为 65%，表明该治疗方法非常有效。治疗 3.5 个月后有 11.5%的患者退出，表明 MELD 评分例外是适宜的。严格的选择对于该治疗方法的持续成功至关重要。
