Outcome and management of patients with hepatocellular carcinoma who achieved a complete response to immunotherapy-based systemic therapy

Bernhard Scheiner; Beodeul Kang; Lorenz Balcar; Iuliana Pompilia Radu; Florian P. Reiter; Gordan Adžić; Jiang Guo; Xu Gao; Xiao Yuan; Long Cheng; Joao Gorgulho; Michael Schultheiss; Frederik Peeters; Florian Hucke; Najib Ben Khaled; Ignazio Piseddu; Alexander Philipp; Friedrich Sinner; Antonio D’Alessio; Katharina Pomej; Anna Saborowski; Melanie Bathon; Birgit Schwacha-Eipper; Valentina Zarka; Katharina Lampichler; Naoshi Nishida; Pei Chang Lee; Anja Krall; Anwaar Saeed; Vera Himmelsbach; Giulia Tesini; Yi Hsiang Huang; Caterina Vivaldi; Gianluca Masi; Arndt Vogel; Kornelius Schulze; Michael Trauner; Angela Djanani; Rudolf Stauber; Masatoshi Kudo; Neehar D. Parikh; Jean François Dufour; Juraj Prejac; Andreas Geier; Bertram Bengsch; Johann von Felden; Marino Venerito; Arndt Weinmann; Markus Peck-Radosavljevic; Fabian Finkelmeier; Jeroen Dekervel; Fanpu Ji; Hung Wei Wang; Lorenza Rimassa; David J. Pinato; Mohamed Bouattour; Hong Jae Chon; Matthias Pinter

doi:10.1097/HEP.0000000000001163

Outcome and management of patients with hepatocellular carcinoma who achieved a complete response to immunotherapy-based systemic therapy

Bernhard Scheiner, Beodeul Kang, Lorenz Balcar, Iuliana Pompilia Radu, Florian P. Reiter, Gordan Adžić, Jiang Guo, Xu Gao, Xiao Yuan, Long Cheng, Joao Gorgulho, Michael Schultheiss, Frederik Peeters, Florian Hucke, Najib Ben Khaled, Ignazio Piseddu, Alexander Philipp, Friedrich Sinner, Antonio D’Alessio, Katharina PomejAnna Saborowski, Melanie Bathon, Birgit Schwacha-Eipper, Valentina Zarka, Katharina Lampichler, Naoshi Nishida, Pei Chang Lee, Anja Krall, Anwaar Saeed, Vera Himmelsbach, Giulia Tesini, Yi Hsiang Huang, Caterina Vivaldi, Gianluca Masi, Arndt Vogel, Kornelius Schulze, Michael Trauner, Angela Djanani, Rudolf Stauber, Masatoshi Kudo, Neehar D. Parikh, Jean François Dufour, Juraj Prejac, Andreas Geier, Bertram Bengsch, Johann von Felden, Marino Venerito, Arndt Weinmann, Markus Peck-Radosavljevic, Fabian Finkelmeier, Jeroen Dekervel, Fanpu Ji, Hung Wei Wang, Lorenza Rimassa, David J. Pinato, Mohamed Bouattour, Hong Jae Chon, Matthias Pinter

Dipartimento di Medicina Traslazionale

Risultato della ricerca: Contributo su rivista › Articolo in rivista › peer review

Abstract

Background and Aims: The outcome of patients with HCC who achieved complete response (CR) to immune-checkpoint inhibitor (ICI)–based systemic therapies is unclear. Approach and Results: Retrospective study of patients with HCC who had CR according to modified Response Evaluation Criteria in Solid Tumors (CR-mRECIST) to ICI-based systemic therapies from 28 centers in Asia, Europe, and the United States. Of 3933 patients with HCC treated with ICI-based noncurative systemic therapies, 174 (4.4%) achieved CR-mRECIST, and 97 (2.5%) had CR according to RECISTv1.1 (CR-RECISTv1.1) as well. The mean age of the total cohort (male, 85%; Barcelona-Clinic Liver Cancer-C, 70%) was 65.9 ± 9.8 years. The majority (83%) received ICI-based combination therapies. Median follow-up was 32.2 (95% CI: 29.9–34.4) months. One- and 3-year overall survival rates were 98% and 86%. One- and 3-year recurrence-free survival rates were excellent in patients with CR-mRECIST-only and CRRECISTv1.1 (78% and 55%; 70% and 42%). Among patients who discontinued ICIs for reasons other than recurrence, those who received immunotherapy for ≥ 6 months after the first mRECIST CR had a longer recurrence-free survival than those who discontinued immunotherapy earlier (p = 0.008). Of 9 patients who underwent curative surgical conversion therapy, 8 (89%) had pathological CR (CR-RECISTv1.1, n = 2/2; CR-mRECIST-only, n = 6/7). Conclusions: Overall survival and recurrence-free survival of patients with CR-mRECIST-only and CR-RECISTv1.1 were excellent, and 6 of 7 patients with CR-mRECIST-only who underwent surgical conversion therapy had pathological CR. Despite potential limitations, these findings support the use of mRECIST in the context of immunotherapy for clinical decision-making. When considering ICI discontinuation, treatment for at least 6 months beyond CR seems advisable.

Lingua originale	Inglese
pagine (da-a)	1714-1727
Numero di pagine	14
Rivista	Hepatology
Volume	81
Numero di pubblicazione	6
DOI	https://doi.org/10.1097/HEP.0000000000001163
Stato di pubblicazione	Pubblicato - 1 giu 2025

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Scheiner, B., Kang, B., Balcar, L., Radu, I. P., Reiter, F. P., Adžić, G., Guo, J., Gao, X., Yuan, X., Cheng, L., Gorgulho, J., Schultheiss, M., Peeters, F., Hucke, F., Khaled, N. B., Piseddu, I., Philipp, A., Sinner, F., D’Alessio, A., ... Pinter, M. (2025). Outcome and management of patients with hepatocellular carcinoma who achieved a complete response to immunotherapy-based systemic therapy. Hepatology, 81(6), 1714-1727. https://doi.org/10.1097/HEP.0000000000001163

@article{6c0a87b3f5d2478db162323805c3be35,

title = "Outcome and management of patients with hepatocellular carcinoma who achieved a complete response to immunotherapy-based systemic therapy",

abstract = "Background and Aims: The outcome of patients with HCC who achieved complete response (CR) to immune-checkpoint inhibitor (ICI)–based systemic therapies is unclear. Approach and Results: Retrospective study of patients with HCC who had CR according to modified Response Evaluation Criteria in Solid Tumors (CR-mRECIST) to ICI-based systemic therapies from 28 centers in Asia, Europe, and the United States. Of 3933 patients with HCC treated with ICI-based noncurative systemic therapies, 174 (4.4\%) achieved CR-mRECIST, and 97 (2.5\%) had CR according to RECISTv1.1 (CR-RECISTv1.1) as well. The mean age of the total cohort (male, 85\%; Barcelona-Clinic Liver Cancer-C, 70\%) was 65.9 ± 9.8 years. The majority (83\%) received ICI-based combination therapies. Median follow-up was 32.2 (95\% CI: 29.9–34.4) months. One- and 3-year overall survival rates were 98\% and 86\%. One- and 3-year recurrence-free survival rates were excellent in patients with CR-mRECIST-only and CRRECISTv1.1 (78\% and 55\%; 70\% and 42\%). Among patients who discontinued ICIs for reasons other than recurrence, those who received immunotherapy for ≥ 6 months after the first mRECIST CR had a longer recurrence-free survival than those who discontinued immunotherapy earlier (p = 0.008). Of 9 patients who underwent curative surgical conversion therapy, 8 (89\%) had pathological CR (CR-RECISTv1.1, n = 2/2; CR-mRECIST-only, n = 6/7). Conclusions: Overall survival and recurrence-free survival of patients with CR-mRECIST-only and CR-RECISTv1.1 were excellent, and 6 of 7 patients with CR-mRECIST-only who underwent surgical conversion therapy had pathological CR. Despite potential limitations, these findings support the use of mRECIST in the context of immunotherapy for clinical decision-making. When considering ICI discontinuation, treatment for at least 6 months beyond CR seems advisable.",

keywords = "RECIST, immune checkpoint inhibitor, liver cancer, systemic therapy",

author = "Bernhard Scheiner and Beodeul Kang and Lorenz Balcar and Radu, \{Iuliana Pompilia\} and Reiter, \{Florian P.\} and Gordan Ad{\v z}i{\'c} and Jiang Guo and Xu Gao and Xiao Yuan and Long Cheng and Joao Gorgulho and Michael Schultheiss and Frederik Peeters and Florian Hucke and Khaled, \{Najib Ben\} and Ignazio Piseddu and Alexander Philipp and Friedrich Sinner and Antonio D{\textquoteright}Alessio and Katharina Pomej and Anna Saborowski and Melanie Bathon and Birgit Schwacha-Eipper and Valentina Zarka and Katharina Lampichler and Naoshi Nishida and Lee, \{Pei Chang\} and Anja Krall and Anwaar Saeed and Vera Himmelsbach and Giulia Tesini and Huang, \{Yi Hsiang\} and Caterina Vivaldi and Gianluca Masi and Arndt Vogel and Kornelius Schulze and Michael Trauner and Angela Djanani and Rudolf Stauber and Masatoshi Kudo and Parikh, \{Neehar D.\} and Dufour, \{Jean Fran{\c c}ois\} and Juraj Prejac and Andreas Geier and Bertram Bengsch and \{von Felden\}, Johann and Marino Venerito and Arndt Weinmann and Markus Peck-Radosavljevic and Fabian Finkelmeier and Jeroen Dekervel and Fanpu Ji and Wang, \{Hung Wei\} and Lorenza Rimassa and Pinato, \{David J.\} and Mohamed Bouattour and Chon, \{Hong Jae\} and Matthias Pinter",

note = "Publisher Copyright: Copyright {\textcopyright} 2024 American Association for the Study of Liver Diseases.",

year = "2025",

month = jun,

day = "1",

doi = "10.1097/HEP.0000000000001163",

language = "English",

volume = "81",

pages = "1714--1727",

journal = "Hepatology",

issn = "0270-9139",

publisher = "Lippincott Williams and Wilkins",

number = "6",

}

Scheiner, B, Kang, B, Balcar, L, Radu, IP, Reiter, FP, Adžić, G, Guo, J, Gao, X, Yuan, X, Cheng, L, Gorgulho, J, Schultheiss, M, Peeters, F, Hucke, F, Khaled, NB, Piseddu, I, Philipp, A, Sinner, F, D’Alessio, A, Pomej, K, Saborowski, A, Bathon, M, Schwacha-Eipper, B, Zarka, V, Lampichler, K, Nishida, N, Lee, PC, Krall, A, Saeed, A, Himmelsbach, V, Tesini, G, Huang, YH, Vivaldi, C, Masi, G, Vogel, A, Schulze, K, Trauner, M, Djanani, A, Stauber, R, Kudo, M, Parikh, ND, Dufour, JF, Prejac, J, Geier, A, Bengsch, B, von Felden, J, Venerito, M, Weinmann, A, Peck-Radosavljevic, M, Finkelmeier, F, Dekervel, J, Ji, F, Wang, HW, Rimassa, L, Pinato, DJ, Bouattour, M, Chon, HJ & Pinter, M 2025, 'Outcome and management of patients with hepatocellular carcinoma who achieved a complete response to immunotherapy-based systemic therapy', Hepatology, vol. 81, n. 6, pagg. 1714-1727. https://doi.org/10.1097/HEP.0000000000001163

TY - JOUR

T1 - Outcome and management of patients with hepatocellular carcinoma who achieved a complete response to immunotherapy-based systemic therapy

AU - Scheiner, Bernhard

AU - Kang, Beodeul

AU - Balcar, Lorenz

AU - Radu, Iuliana Pompilia

AU - Reiter, Florian P.

AU - Adžić, Gordan

AU - Guo, Jiang

AU - Gao, Xu

AU - Yuan, Xiao

AU - Cheng, Long

AU - Gorgulho, Joao

AU - Schultheiss, Michael

AU - Peeters, Frederik

AU - Hucke, Florian

AU - Khaled, Najib Ben

AU - Piseddu, Ignazio

AU - Philipp, Alexander

AU - Sinner, Friedrich

AU - D’Alessio, Antonio

AU - Pomej, Katharina

AU - Saborowski, Anna

AU - Bathon, Melanie

AU - Schwacha-Eipper, Birgit

AU - Zarka, Valentina

AU - Lampichler, Katharina

AU - Nishida, Naoshi

AU - Lee, Pei Chang

AU - Krall, Anja

AU - Saeed, Anwaar

AU - Himmelsbach, Vera

AU - Tesini, Giulia

AU - Huang, Yi Hsiang

AU - Vivaldi, Caterina

AU - Masi, Gianluca

AU - Vogel, Arndt

AU - Schulze, Kornelius

AU - Trauner, Michael

AU - Djanani, Angela

AU - Stauber, Rudolf

AU - Kudo, Masatoshi

AU - Parikh, Neehar D.

AU - Dufour, Jean François

AU - Prejac, Juraj

AU - Geier, Andreas

AU - Bengsch, Bertram

AU - von Felden, Johann

AU - Venerito, Marino

AU - Weinmann, Arndt

AU - Peck-Radosavljevic, Markus

AU - Finkelmeier, Fabian

AU - Dekervel, Jeroen

AU - Ji, Fanpu

AU - Wang, Hung Wei

AU - Rimassa, Lorenza

AU - Pinato, David J.

AU - Bouattour, Mohamed

AU - Chon, Hong Jae

AU - Pinter, Matthias

PY - 2025/6/1

Y1 - 2025/6/1

N2 - Background and Aims: The outcome of patients with HCC who achieved complete response (CR) to immune-checkpoint inhibitor (ICI)–based systemic therapies is unclear. Approach and Results: Retrospective study of patients with HCC who had CR according to modified Response Evaluation Criteria in Solid Tumors (CR-mRECIST) to ICI-based systemic therapies from 28 centers in Asia, Europe, and the United States. Of 3933 patients with HCC treated with ICI-based noncurative systemic therapies, 174 (4.4%) achieved CR-mRECIST, and 97 (2.5%) had CR according to RECISTv1.1 (CR-RECISTv1.1) as well. The mean age of the total cohort (male, 85%; Barcelona-Clinic Liver Cancer-C, 70%) was 65.9 ± 9.8 years. The majority (83%) received ICI-based combination therapies. Median follow-up was 32.2 (95% CI: 29.9–34.4) months. One- and 3-year overall survival rates were 98% and 86%. One- and 3-year recurrence-free survival rates were excellent in patients with CR-mRECIST-only and CRRECISTv1.1 (78% and 55%; 70% and 42%). Among patients who discontinued ICIs for reasons other than recurrence, those who received immunotherapy for ≥ 6 months after the first mRECIST CR had a longer recurrence-free survival than those who discontinued immunotherapy earlier (p = 0.008). Of 9 patients who underwent curative surgical conversion therapy, 8 (89%) had pathological CR (CR-RECISTv1.1, n = 2/2; CR-mRECIST-only, n = 6/7). Conclusions: Overall survival and recurrence-free survival of patients with CR-mRECIST-only and CR-RECISTv1.1 were excellent, and 6 of 7 patients with CR-mRECIST-only who underwent surgical conversion therapy had pathological CR. Despite potential limitations, these findings support the use of mRECIST in the context of immunotherapy for clinical decision-making. When considering ICI discontinuation, treatment for at least 6 months beyond CR seems advisable.

AB - Background and Aims: The outcome of patients with HCC who achieved complete response (CR) to immune-checkpoint inhibitor (ICI)–based systemic therapies is unclear. Approach and Results: Retrospective study of patients with HCC who had CR according to modified Response Evaluation Criteria in Solid Tumors (CR-mRECIST) to ICI-based systemic therapies from 28 centers in Asia, Europe, and the United States. Of 3933 patients with HCC treated with ICI-based noncurative systemic therapies, 174 (4.4%) achieved CR-mRECIST, and 97 (2.5%) had CR according to RECISTv1.1 (CR-RECISTv1.1) as well. The mean age of the total cohort (male, 85%; Barcelona-Clinic Liver Cancer-C, 70%) was 65.9 ± 9.8 years. The majority (83%) received ICI-based combination therapies. Median follow-up was 32.2 (95% CI: 29.9–34.4) months. One- and 3-year overall survival rates were 98% and 86%. One- and 3-year recurrence-free survival rates were excellent in patients with CR-mRECIST-only and CRRECISTv1.1 (78% and 55%; 70% and 42%). Among patients who discontinued ICIs for reasons other than recurrence, those who received immunotherapy for ≥ 6 months after the first mRECIST CR had a longer recurrence-free survival than those who discontinued immunotherapy earlier (p = 0.008). Of 9 patients who underwent curative surgical conversion therapy, 8 (89%) had pathological CR (CR-RECISTv1.1, n = 2/2; CR-mRECIST-only, n = 6/7). Conclusions: Overall survival and recurrence-free survival of patients with CR-mRECIST-only and CR-RECISTv1.1 were excellent, and 6 of 7 patients with CR-mRECIST-only who underwent surgical conversion therapy had pathological CR. Despite potential limitations, these findings support the use of mRECIST in the context of immunotherapy for clinical decision-making. When considering ICI discontinuation, treatment for at least 6 months beyond CR seems advisable.

KW - RECIST

KW - immune checkpoint inhibitor

KW - liver cancer

KW - systemic therapy

UR - https://www.scopus.com/pages/publications/85210310317

U2 - 10.1097/HEP.0000000000001163

DO - 10.1097/HEP.0000000000001163

M3 - Article

SN - 0270-9139

VL - 81

SP - 1714

EP - 1727

JO - Hepatology

JF - Hepatology

IS - 6

ER -

Outcome and management of patients with hepatocellular carcinoma who achieved a complete response to immunotherapy-based systemic therapy

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