Multicenter retrospective analysis of the safety and efficacy of regorafenib after progression on sorafenib in Korean patients with hepatocellular carcinoma

Changhoon Yoo, Joong Won Park, Yoon Jun Kim, Do Young Kim, Su Jong Yu, Tae Seop Lim, Su Jin Lee, Baek Yeol Ryoo, Ho Yeong Lim

Research output: Contribution to journalArticleResearchpeer-review

Abstract

Background The pivotal RESORCE trial showed that regorafenib was effective as second-line therapy for patients with advanced HCC who progressed on first-line sorafenib. Real-world data are needed to assess clinical outcomes and adverse events in the setting of daily practice. Methods Between April 2017 and August 2017, the Named Patient Program (NPP) was activated to provide controlled, pre-approval access of regorafenib in Korea. This analysis is a multicenter retrospective study of patients who received regorafenib under the NPP. Results A total of 49 patients entered into this NPP, and 40 patients received regorafenib in five Korean institutions. All but one patient received regorafenib as second-line therapy after progression on sorafenib, and 36 (90%) and 34 (85%) patients were classified as Child-Pugh A and BCLC stage C, respectively. The response rate was 10% (n = 4). The median progression-free survival (PFS) was 3.7 months (95% CI, 2.5–4.9 months), and the median overall survival (OS) was not reached. The 1 year OS rate was 54.6%. The time-to-progression (TTP) on prior sorafenib was significantly associated with PFS and OS. The most common grade 3–4 toxicities were hand-foot skin reaction (n = 3, 8%), hypertension (n = 2, 5%), and increased aspartate aminotransferase (n = 2, 5%). Conclusion Regorafenib was well-tolerated and effective in patients with advanced HCC who progressed on first-line sorafenib, with efficacy and safety outcomes consistent with those of the previous RESORCE trial. TTP on first-line sorafenib may predict the efficacy of subsequent regorafenib.

Original languageEnglish
Pages (from-to)567-572
Number of pages6
JournalInvestigational New Drugs
Volume37
Issue number3
DOIs
StatePublished - 15 Jun 2019

Fingerprint

Hepatocellular Carcinoma
Safety
Disease-Free Survival
regorafenib
sorafenib
Survival
Korea
Aspartate Aminotransferases
Multicenter Studies
Foot
Survival Rate
Retrospective Studies
Hand
Hypertension
Skin
Therapeutics

Keywords

  • Hepatocellular carcinoma
  • Regorafenib
  • Sorafenib

Cite this

Yoo, Changhoon ; Park, Joong Won ; Kim, Yoon Jun ; Kim, Do Young ; Yu, Su Jong ; Lim, Tae Seop ; Lee, Su Jin ; Ryoo, Baek Yeol ; Lim, Ho Yeong. / Multicenter retrospective analysis of the safety and efficacy of regorafenib after progression on sorafenib in Korean patients with hepatocellular carcinoma. In: Investigational New Drugs. 2019 ; Vol. 37, No. 3. pp. 567-572.
@article{e8c6dfb9ba844b26a0d727283ccfaade,
title = "Multicenter retrospective analysis of the safety and efficacy of regorafenib after progression on sorafenib in Korean patients with hepatocellular carcinoma",
abstract = "Background The pivotal RESORCE trial showed that regorafenib was effective as second-line therapy for patients with advanced HCC who progressed on first-line sorafenib. Real-world data are needed to assess clinical outcomes and adverse events in the setting of daily practice. Methods Between April 2017 and August 2017, the Named Patient Program (NPP) was activated to provide controlled, pre-approval access of regorafenib in Korea. This analysis is a multicenter retrospective study of patients who received regorafenib under the NPP. Results A total of 49 patients entered into this NPP, and 40 patients received regorafenib in five Korean institutions. All but one patient received regorafenib as second-line therapy after progression on sorafenib, and 36 (90{\%}) and 34 (85{\%}) patients were classified as Child-Pugh A and BCLC stage C, respectively. The response rate was 10{\%} (n = 4). The median progression-free survival (PFS) was 3.7 months (95{\%} CI, 2.5–4.9 months), and the median overall survival (OS) was not reached. The 1 year OS rate was 54.6{\%}. The time-to-progression (TTP) on prior sorafenib was significantly associated with PFS and OS. The most common grade 3–4 toxicities were hand-foot skin reaction (n = 3, 8{\%}), hypertension (n = 2, 5{\%}), and increased aspartate aminotransferase (n = 2, 5{\%}). Conclusion Regorafenib was well-tolerated and effective in patients with advanced HCC who progressed on first-line sorafenib, with efficacy and safety outcomes consistent with those of the previous RESORCE trial. TTP on first-line sorafenib may predict the efficacy of subsequent regorafenib.",
keywords = "Hepatocellular carcinoma, Regorafenib, Sorafenib",
author = "Changhoon Yoo and Park, {Joong Won} and Kim, {Yoon Jun} and Kim, {Do Young} and Yu, {Su Jong} and Lim, {Tae Seop} and Lee, {Su Jin} and Ryoo, {Baek Yeol} and Lim, {Ho Yeong}",
year = "2019",
month = "6",
day = "15",
doi = "10.1007/s10637-018-0707-5",
language = "English",
volume = "37",
pages = "567--572",
journal = "Investigational New Drugs",
issn = "0167-6997",
publisher = "Kluwer Academic Publishers",
number = "3",

}

Multicenter retrospective analysis of the safety and efficacy of regorafenib after progression on sorafenib in Korean patients with hepatocellular carcinoma. / Yoo, Changhoon; Park, Joong Won; Kim, Yoon Jun; Kim, Do Young; Yu, Su Jong; Lim, Tae Seop; Lee, Su Jin; Ryoo, Baek Yeol; Lim, Ho Yeong.

In: Investigational New Drugs, Vol. 37, No. 3, 15.06.2019, p. 567-572.

Research output: Contribution to journalArticleResearchpeer-review

TY - JOUR

T1 - Multicenter retrospective analysis of the safety and efficacy of regorafenib after progression on sorafenib in Korean patients with hepatocellular carcinoma

AU - Yoo, Changhoon

AU - Park, Joong Won

AU - Kim, Yoon Jun

AU - Kim, Do Young

AU - Yu, Su Jong

AU - Lim, Tae Seop

AU - Lee, Su Jin

AU - Ryoo, Baek Yeol

AU - Lim, Ho Yeong

PY - 2019/6/15

Y1 - 2019/6/15

N2 - Background The pivotal RESORCE trial showed that regorafenib was effective as second-line therapy for patients with advanced HCC who progressed on first-line sorafenib. Real-world data are needed to assess clinical outcomes and adverse events in the setting of daily practice. Methods Between April 2017 and August 2017, the Named Patient Program (NPP) was activated to provide controlled, pre-approval access of regorafenib in Korea. This analysis is a multicenter retrospective study of patients who received regorafenib under the NPP. Results A total of 49 patients entered into this NPP, and 40 patients received regorafenib in five Korean institutions. All but one patient received regorafenib as second-line therapy after progression on sorafenib, and 36 (90%) and 34 (85%) patients were classified as Child-Pugh A and BCLC stage C, respectively. The response rate was 10% (n = 4). The median progression-free survival (PFS) was 3.7 months (95% CI, 2.5–4.9 months), and the median overall survival (OS) was not reached. The 1 year OS rate was 54.6%. The time-to-progression (TTP) on prior sorafenib was significantly associated with PFS and OS. The most common grade 3–4 toxicities were hand-foot skin reaction (n = 3, 8%), hypertension (n = 2, 5%), and increased aspartate aminotransferase (n = 2, 5%). Conclusion Regorafenib was well-tolerated and effective in patients with advanced HCC who progressed on first-line sorafenib, with efficacy and safety outcomes consistent with those of the previous RESORCE trial. TTP on first-line sorafenib may predict the efficacy of subsequent regorafenib.

AB - Background The pivotal RESORCE trial showed that regorafenib was effective as second-line therapy for patients with advanced HCC who progressed on first-line sorafenib. Real-world data are needed to assess clinical outcomes and adverse events in the setting of daily practice. Methods Between April 2017 and August 2017, the Named Patient Program (NPP) was activated to provide controlled, pre-approval access of regorafenib in Korea. This analysis is a multicenter retrospective study of patients who received regorafenib under the NPP. Results A total of 49 patients entered into this NPP, and 40 patients received regorafenib in five Korean institutions. All but one patient received regorafenib as second-line therapy after progression on sorafenib, and 36 (90%) and 34 (85%) patients were classified as Child-Pugh A and BCLC stage C, respectively. The response rate was 10% (n = 4). The median progression-free survival (PFS) was 3.7 months (95% CI, 2.5–4.9 months), and the median overall survival (OS) was not reached. The 1 year OS rate was 54.6%. The time-to-progression (TTP) on prior sorafenib was significantly associated with PFS and OS. The most common grade 3–4 toxicities were hand-foot skin reaction (n = 3, 8%), hypertension (n = 2, 5%), and increased aspartate aminotransferase (n = 2, 5%). Conclusion Regorafenib was well-tolerated and effective in patients with advanced HCC who progressed on first-line sorafenib, with efficacy and safety outcomes consistent with those of the previous RESORCE trial. TTP on first-line sorafenib may predict the efficacy of subsequent regorafenib.

KW - Hepatocellular carcinoma

KW - Regorafenib

KW - Sorafenib

UR - http://www.scopus.com/inward/record.url?scp=85058111487&partnerID=8YFLogxK

U2 - 10.1007/s10637-018-0707-5

DO - 10.1007/s10637-018-0707-5

M3 - Article

VL - 37

SP - 567

EP - 572

JO - Investigational New Drugs

JF - Investigational New Drugs

SN - 0167-6997

IS - 3

ER -