Division of Transplant Surgery, Department of Surgery, Medical College of Wisconsin, Milwaukee, Wisconsin; The Transplant Center, Froedtert and The Medical College of Wisconsin, Milwaukee, Wisconsin.
Division of Hematology and Oncology, Department of Medicine, Medical College of Wisconsin, Milwaukee, Wisconsin.
J Surg Res. 2019 Oct;242:23-30. doi: 10.1016/j.jss.2019.04.023. Epub 2019 May 3.
Orthotopic liver transplantation (OLT) after neoadjuvant therapy (NT) in well-selected patients with unresectable hilar cholangiocarcinoma (CCA) achieves excellent recurrence-free survival. Current criteria for NT-OLT exclude patients with locally advanced hilar and intrahepatic CCA from potential cure. We sought to evaluate the efficacy of NT in downstaging locally advanced CCA, and examine outcomes after OLT.
Among 24 patients referred for unresectable hilar and intrahepatic CCA from January 2013 through August 2017, 18 met center-specific inclusion criteria for the NT-OLT treatment protocol: hilar tumor size ≤3.5 cm or intrahepatic ≤8 cm, and regional lymphadenopathy but without distant metastasis. Median follow-up was 22.1 mo from diagnosis.
Of 18 patients who initiated NT, 11 were removed from the protocol due to tumor progression (n = 6) or uncontrolled infection and failure-to-thrive (n = 5). Median NT duration tended to be shorter for patients progressing to dropout than for those surviving to OLT (5.5 versus 13.5 mo, P = 0.109). Among five patients who received OLT, 1-y post-OLT patient survival was 80%: three survive recurrence-free (14.5-29.2 mo post-OLT); one developed an isolated tumor recurrence in a single portacaval lymph node at 12 mo post-OLT; and one experienced non-tumor-related death. All dropout patients died at a median of 14.4 mo after diagnosis.
This is the first prospective study to show successful NT downstaging of unresectable locally advanced hilar and intrahepatic CCA before OLT. NT-OLT for select patients with locally advanced hilar and intrahepatic CCA achieved acceptable short-term recurrence-free survival.
在经过新辅助治疗（NT）的选择后，原位肝移植（OLT）可使不可切除的肝门部胆管癌（CCA）患者获得极好的无复发生存率。目前用于 NT-OLT 的标准将局部晚期肝门部和肝内 CCA 患者排除在潜在治愈之外。我们试图评估 NT 在降级局部晚期 CCA 中的疗效，并检查 OLT 后的结果。
2013 年 1 月至 2017 年 8 月期间，24 例患者因不可切除的肝门部和肝内 CCA 被转诊，其中 18 例符合 NT-OLT 治疗方案的中心特定纳入标准：肝门部肿瘤大小≤3.5cm 或肝内≤8cm，且存在区域淋巴结病但无远处转移。中位随访时间为诊断后 22.1 个月。
18 例开始接受 NT 的患者中，有 11 例因肿瘤进展（n=6）或无法控制的感染和生长不良（n=5）而退出方案。进展至退出组的患者 NT 持续时间中位数明显短于存活至 OLT 组（5.5 个月与 13.5 个月，P=0.109）。在接受 OLT 的 5 例患者中，1 年 OLT 后患者存活率为 80%：3 例无复发生存（OLT 后 14.5-29.2 个月）；1 例在 OLT 后 12 个月时在单个门腔静脉淋巴结中出现孤立性肿瘤复发；1 例因非肿瘤相关原因死亡。所有退出患者均在诊断后 14.4 个月中位时间内死亡。
这是第一项前瞻性研究，表明在 OLT 之前，NT 可成功降级不可切除的局部晚期肝门部和肝内 CCA。NT-OLT 治疗局部晚期肝门部和肝内 CCA 的选择患者获得了可接受的短期无复发生存率。
