Background & Aims

The United Network for Organ Sharing (UNOS) grants priority listing for liver transplant for patients with hepatocellular carcinoma after successful down-staging to Milan criteria. We evaluated the national experience on down-staging by comparing 2 down-staging groups: tumor burden meeting UNOS down-staging (UNOS-DS) inclusion criteria, and all-comers (AC)-DS with initial tumor burden beyond UNOS-DS criteria vs patients always within Milan criteria.

Methods

We performed a retrospective analysis of the UNOS database of 23,398 patients listed for liver transplant who had submitted a hepatocellular carcinoma Model for End-Stage Liver Disease exception application from 2010 to 2019, classified as always within Milan (n = 20,579), UNOS-DS (n = 2151), and AC-DS (n = 668).

Results

The 2-year cumulative probabilities of dropout were 19% for Milan, 25% for UNOS-DS (P < .001), and 30% for AC-DS (P < .001). In multivariate analysis of the down-staging groups, factors predicting dropout included Model for End-Stage Liver Disease at listing (hazard ratio [HR], 1.06; P < .001) and initial total tumor diameter (HR, 1.04; P = .002). Compared with α-fetoprotein (AFP) level ≤20 ng/mL, AFP levels of 21 to 100, 101 to 1000, and greater than 1000 ng/mL were associated with a higher risk of dropout (HRs, 1.63, 2.06, and 4.58, respectively; P < .001). A subset of all-comers with AFP levels greater than 100 ng/mL had a 2-year probability of dropout of 52% vs 26% for all others beyond Milan criteria (P < .001).

Conclusions

All-comers had a significantly higher risk for waitlist dropout compared with the UNOS-DS and Milan groups after initial successful down-staging to Milan criteria. In particular, the subgroup of AC-DS with an AFP level greater than 100 ng/mL had a greater than 50% probability of dropout in the next 2 years. These observations suggest a high likelihood of failure when expanding the indications for down-staging.

中文翻译：

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肝细胞癌降期候补名单结果的国家经验：所有患者的高辍学率

背景与目标

器官共享联合网络 (UNOS) 授予肝细胞癌患者成功降期至米兰标准后进行肝移植的优先名单。我们通过比较 2 个降期组来评估国家降期经验：符合 UNOS 降期 (UNOS-DS) 纳入标准的肿瘤负荷，以及初始肿瘤负荷超出 UNOS-DS 标准的所有人 (AC)-DS与始终符合米兰标准的患者相比。

方法

我们对 UNOS 数据库进行了回顾性分析，该数据库包含 23,398 名在 2010 年至 2019 年提交肝细胞癌终末期肝病模型例外申请的肝移植患者，归类为始终在米兰 (n = 20,579)，UNOS-DS (n = 2151)，和 AC-DS (n = 668)。

结果

Milan 的 2 年累积辍学概率为 19%，UNOS-DS 为 25% ( P < .001)，AC-DS 为 30% ( P < .001)。在降期组的多变量分析中，预测退出的因素包括上市时的终末期肝病模型（风险比 [HR]，1.06；P < .001）和初始肿瘤总直径（HR，1.04；P  = .001）。 002). 与甲胎蛋白 (AFP) 水平≤20 ng/mL 相比，AFP 水平为 21 至 100、101 至 1000 和大于 1000 ng/mL 与更高的脱落风险相关（HRs，1.63、2.06 和 4.58 ，分别为；P < .001)。AFP 水平大于 100 ng/mL 的所有参与者的子集在 2 年内辍学的概率为 52%，而对于所有其他超出米兰标准的参与者，这一比例为 26%（P <.001）。

结论

在最初成功降级至米兰标准后，与 UNOS-DS 和米兰组相比，所有参与者的候补名单退出风险明显更高。特别是，AFP 水平大于 100 ng/mL 的 AC-DS 亚组在未来 2 年内退出的概率大于 50%。这些观察结果表明，在扩大降期适应症时失败的可能性很高。