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Background <p>The feasibility of trials in liver surgery using a single-component clinical endpoint is low because single endpoints require large samples due to their low incidence. The current study sought to develop and validate a novel composite endpoint of liver surgery (CELS) to facilitate the generation of more feasible and robust high-level evidence in the field of liver surgery.</p> Methods <p>Patients who underwent curative-intent hepatectomy for hepatocellular carcinoma, intrahepatic cholangiocarcinoma, or colorectal liver metastasis were identified using a multi-institutional database. Components of CELS were selected based on perioperative liver surgery-specific complications using univariable logistic regression models. The association of CELS with prolonged length of stay (LOS) and surgery-related death was evaluated and externally validated. Sample sizes were calculated for both individual outcomes and CELS.</p> Results <p>Among 1958 patients, 377 (19.3%) met CELS criteria based on postoperative bile leak (<i>n</i> = 221, 11.3%), post-hepatectomy liver failure (<i>n</i> = 71, 3.6%), post-hepatectomy hemorrhage (<i>n</i> = 38, 1.9%), or intraoperative blood loss of 2000 ml or greater (<i>n</i> = 101, 5.2%). CELS demonstrated favorable discriminative accuracy of surgery-related death (analytic cohort: area under the curve [AUC], 0.79 vs external validation cohort: AUC, 0.85). In addition LOS was longer among the patients with a positive CELS (analytic cohort: 14 vs. 9 days [<i>p</i> &lt; 0.001] vs. the validation cohort: 10 vs. 6 days [<i>p</i> &lt; 0.001]). Relative to individual endpoints, CELS allowed a 45.8–91.6% reduction in sample size.</p> Conclusion <p>CELS effectively predicted surgery-related death and can be used as a standardized, clinically relevant endpoint in prospective trials, facilitating smaller sample sizes and enhancing feasibility compared with single quality outcome&#xa0;metrics.</p>

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A Composite Endpoint of Liver Surgery (CELS): Development and Validation of a Clinically Relevant Endpoint Requiring a Smaller Sample Size

  • 期刊论文

  • 出版模式：

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  • 发表日期：

    2025年1月31日

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  • Jun Kawashima,
  • Miho Akabane,
  • Yutaka Endo,
  • Selamawit Woldesenbet,
  • Mujtaba Khalil,
  • Kota Sahara,
  • Andrea Ruzzenente,
  • Luca Aldrighetti,
  • Todd W. Bauer,
  • Hugo P. Marques,
  • Rita Lopes,
  • Sara Oliveira,
  • Guillaume Martel,
  • Irinel Popescu,
  • Mathew J. Weiss,
  • Minoru Kitago,
  • George Poultsides,
  • Kazunari Sasaki,
  • Shishir K. Maithel,
  • Tom Hugh,
  • Ana Gleisner,
  • Federico Aucejo,
  • Carlo Pulitano,
  • Feng Shen,
  • François Cauchy,
  • Bas Groot Koerkamp,
  • Itaru Endo,
  • Timothy M. Pawlik

摘要

Background

The feasibility of trials in liver surgery using a single-component clinical endpoint is low because single endpoints require large samples due to their low incidence. The current study sought to develop and validate a novel composite endpoint of liver surgery (CELS) to facilitate the generation of more feasible and robust high-level evidence in the field of liver surgery.

Methods

Patients who underwent curative-intent hepatectomy for hepatocellular carcinoma, intrahepatic cholangiocarcinoma, or colorectal liver metastasis were identified using a multi-institutional database. Components of CELS were selected based on perioperative liver surgery-specific complications using univariable logistic regression models. The association of CELS with prolonged length of stay (LOS) and surgery-related death was evaluated and externally validated. Sample sizes were calculated for both individual outcomes and CELS.

Results

Among 1958 patients, 377 (19.3%) met CELS criteria based on postoperative bile leak (n = 221, 11.3%), post-hepatectomy liver failure (n = 71, 3.6%), post-hepatectomy hemorrhage (n = 38, 1.9%), or intraoperative blood loss of 2000 ml or greater (n = 101, 5.2%). CELS demonstrated favorable discriminative accuracy of surgery-related death (analytic cohort: area under the curve [AUC], 0.79 vs external validation cohort: AUC, 0.85). In addition LOS was longer among the patients with a positive CELS (analytic cohort: 14 vs. 9 days [p < 0.001] vs. the validation cohort: 10 vs. 6 days [p < 0.001]). Relative to individual endpoints, CELS allowed a 45.8–91.6% reduction in sample size.

Conclusion

CELS effectively predicted surgery-related death and can be used as a standardized, clinically relevant endpoint in prospective trials, facilitating smaller sample sizes and enhancing feasibility compared with single quality outcome metrics.