Observational registry of sorafenib use in clinical practice across Child-Pugh subgroups: The GIDEON study

Jorge A. Marrero, Masatoshi Kudo, Alan P. Venook, Sheng Long Ye, Jean Pierre Bronowicki, Xiao Ping Chen, Lucy Dagher, Junji Furuse, Jean Francois H Geschwind, Laura Ladrón de Guevara, Christos Papandreou, Tadatoshi Takayama, Arun J. Sanyal, Seung Kew Yoon, Keiko Nakajima, Robert Lehr, Stephanie Heldner, Riccardo Lencioni

Research output: Contribution to journalArticle

76 Citations (Scopus)

Abstract

Background & Aims: GIDEON (Global Investigation of therapeutic DEcisions in hepatocellular carcinoma and Of its treatment with sorafeNib) is a prospective, observational registry study evaluating the safety of sorafenib and treatment practices in hepatocellular carcinoma patients. This large global database allowed for assessment of the use and tolerability of sorafenib in patients with liver dysfunction. Methods: Baseline characteristics and medical/treatment history were collected in patients for whom a decision to treat with sorafenib had been made. Adverse event, dosing, and outcomes data were collected during follow-up. Results: In the overall safety population (n. =3202), 1968 patients (61%) had Child-Pugh A status and 666 (21%) had Child-Pugh B. The majority of Child-Pugh A (72%) and Child-Pugh B (70%) patients received an initial sorafenib dose of 800. mg, consistent with the label, and dose reduction rates were 40% and 29%, respectively. The type and incidence of adverse events were generally consistent across Child-Pugh subgroups. The incidence of drug-related adverse events leading to discontinuation was similar between Child-Pugh A and Child-Pugh B patients (17% and 21%). In the intent-to-treat population (n. =3213), median overall survival (months [95% confidence interval]) was longer in Child-Pugh A patients (13.6 [12.8-14.7]) compared with Child-Pugh B patients (5.2 [4.6-6.3]). Conclusions: In clinical practice, the safety profile of sorafenib appeared to be consistent across Child-Pugh A and Child-Pugh B patients. Findings suggest sorafenib may be safely used in some Child-Pugh B patients and indicate the importance of careful patient evaluation when making treatment decisions. Lay summary: The GIDEON (Global Investigation of therapeutic DEcisions in hepatocellular carcinoma and Of its treatment with sorafeNib) study is a large prospective registry of patients with liver cancer who were treated with sorafenib. The aims were to evaluate the safety and tolerability of sorafenib among those in which the liver was not functioning properly. The study showed that the safety profile of sorafenib was consistent across patients with preserved liver function and those in which the liver was not functioning properly, and therefore, suggesting that sorafenib may be a valid treatment for some patients with liver impairment.

Original languageEnglish (US)
JournalJournal of Hepatology
DOIs
StateAccepted/In press - Apr 16 2015

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Registries
Safety
Hepatocellular Carcinoma
Liver
Therapeutics
sorafenib
Incidence
Liver Neoplasms
Drug-Related Side Effects and Adverse Reactions
Population
Observational Studies
Liver Diseases
Decision Making
History
Databases
Confidence Intervals

Keywords

  • Child-Pugh
  • GIDEON
  • HCC
  • Hepatocellular carcinoma
  • Nexavar
  • Sorafenib

ASJC Scopus subject areas

  • Hepatology

Cite this

Observational registry of sorafenib use in clinical practice across Child-Pugh subgroups : The GIDEON study. / Marrero, Jorge A.; Kudo, Masatoshi; Venook, Alan P.; Ye, Sheng Long; Bronowicki, Jean Pierre; Chen, Xiao Ping; Dagher, Lucy; Furuse, Junji; Geschwind, Jean Francois H; de Guevara, Laura Ladrón; Papandreou, Christos; Takayama, Tadatoshi; Sanyal, Arun J.; Yoon, Seung Kew; Nakajima, Keiko; Lehr, Robert; Heldner, Stephanie; Lencioni, Riccardo.

In: Journal of Hepatology, 16.04.2015.

Research output: Contribution to journalArticle

Marrero, JA, Kudo, M, Venook, AP, Ye, SL, Bronowicki, JP, Chen, XP, Dagher, L, Furuse, J, Geschwind, JFH, de Guevara, LL, Papandreou, C, Takayama, T, Sanyal, AJ, Yoon, SK, Nakajima, K, Lehr, R, Heldner, S & Lencioni, R 2015, 'Observational registry of sorafenib use in clinical practice across Child-Pugh subgroups: The GIDEON study', Journal of Hepatology. https://doi.org/10.1016/j.jhep.2016.07.020
Marrero, Jorge A. ; Kudo, Masatoshi ; Venook, Alan P. ; Ye, Sheng Long ; Bronowicki, Jean Pierre ; Chen, Xiao Ping ; Dagher, Lucy ; Furuse, Junji ; Geschwind, Jean Francois H ; de Guevara, Laura Ladrón ; Papandreou, Christos ; Takayama, Tadatoshi ; Sanyal, Arun J. ; Yoon, Seung Kew ; Nakajima, Keiko ; Lehr, Robert ; Heldner, Stephanie ; Lencioni, Riccardo. / Observational registry of sorafenib use in clinical practice across Child-Pugh subgroups : The GIDEON study. In: Journal of Hepatology. 2015.
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abstract = "Background & Aims: GIDEON (Global Investigation of therapeutic DEcisions in hepatocellular carcinoma and Of its treatment with sorafeNib) is a prospective, observational registry study evaluating the safety of sorafenib and treatment practices in hepatocellular carcinoma patients. This large global database allowed for assessment of the use and tolerability of sorafenib in patients with liver dysfunction. Methods: Baseline characteristics and medical/treatment history were collected in patients for whom a decision to treat with sorafenib had been made. Adverse event, dosing, and outcomes data were collected during follow-up. Results: In the overall safety population (n. =3202), 1968 patients (61{\%}) had Child-Pugh A status and 666 (21{\%}) had Child-Pugh B. The majority of Child-Pugh A (72{\%}) and Child-Pugh B (70{\%}) patients received an initial sorafenib dose of 800. mg, consistent with the label, and dose reduction rates were 40{\%} and 29{\%}, respectively. The type and incidence of adverse events were generally consistent across Child-Pugh subgroups. The incidence of drug-related adverse events leading to discontinuation was similar between Child-Pugh A and Child-Pugh B patients (17{\%} and 21{\%}). In the intent-to-treat population (n. =3213), median overall survival (months [95{\%} confidence interval]) was longer in Child-Pugh A patients (13.6 [12.8-14.7]) compared with Child-Pugh B patients (5.2 [4.6-6.3]). Conclusions: In clinical practice, the safety profile of sorafenib appeared to be consistent across Child-Pugh A and Child-Pugh B patients. Findings suggest sorafenib may be safely used in some Child-Pugh B patients and indicate the importance of careful patient evaluation when making treatment decisions. Lay summary: The GIDEON (Global Investigation of therapeutic DEcisions in hepatocellular carcinoma and Of its treatment with sorafeNib) study is a large prospective registry of patients with liver cancer who were treated with sorafenib. The aims were to evaluate the safety and tolerability of sorafenib among those in which the liver was not functioning properly. The study showed that the safety profile of sorafenib was consistent across patients with preserved liver function and those in which the liver was not functioning properly, and therefore, suggesting that sorafenib may be a valid treatment for some patients with liver impairment.",
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author = "Marrero, {Jorge A.} and Masatoshi Kudo and Venook, {Alan P.} and Ye, {Sheng Long} and Bronowicki, {Jean Pierre} and Chen, {Xiao Ping} and Lucy Dagher and Junji Furuse and Geschwind, {Jean Francois H} and {de Guevara}, {Laura Ladr{\'o}n} and Christos Papandreou and Tadatoshi Takayama and Sanyal, {Arun J.} and Yoon, {Seung Kew} and Keiko Nakajima and Robert Lehr and Stephanie Heldner and Riccardo Lencioni",
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T1 - Observational registry of sorafenib use in clinical practice across Child-Pugh subgroups

T2 - The GIDEON study

AU - Marrero, Jorge A.

AU - Kudo, Masatoshi

AU - Venook, Alan P.

AU - Ye, Sheng Long

AU - Bronowicki, Jean Pierre

AU - Chen, Xiao Ping

AU - Dagher, Lucy

AU - Furuse, Junji

AU - Geschwind, Jean Francois H

AU - de Guevara, Laura Ladrón

AU - Papandreou, Christos

AU - Takayama, Tadatoshi

AU - Sanyal, Arun J.

AU - Yoon, Seung Kew

AU - Nakajima, Keiko

AU - Lehr, Robert

AU - Heldner, Stephanie

AU - Lencioni, Riccardo

PY - 2015/4/16

Y1 - 2015/4/16

N2 - Background & Aims: GIDEON (Global Investigation of therapeutic DEcisions in hepatocellular carcinoma and Of its treatment with sorafeNib) is a prospective, observational registry study evaluating the safety of sorafenib and treatment practices in hepatocellular carcinoma patients. This large global database allowed for assessment of the use and tolerability of sorafenib in patients with liver dysfunction. Methods: Baseline characteristics and medical/treatment history were collected in patients for whom a decision to treat with sorafenib had been made. Adverse event, dosing, and outcomes data were collected during follow-up. Results: In the overall safety population (n. =3202), 1968 patients (61%) had Child-Pugh A status and 666 (21%) had Child-Pugh B. The majority of Child-Pugh A (72%) and Child-Pugh B (70%) patients received an initial sorafenib dose of 800. mg, consistent with the label, and dose reduction rates were 40% and 29%, respectively. The type and incidence of adverse events were generally consistent across Child-Pugh subgroups. The incidence of drug-related adverse events leading to discontinuation was similar between Child-Pugh A and Child-Pugh B patients (17% and 21%). In the intent-to-treat population (n. =3213), median overall survival (months [95% confidence interval]) was longer in Child-Pugh A patients (13.6 [12.8-14.7]) compared with Child-Pugh B patients (5.2 [4.6-6.3]). Conclusions: In clinical practice, the safety profile of sorafenib appeared to be consistent across Child-Pugh A and Child-Pugh B patients. Findings suggest sorafenib may be safely used in some Child-Pugh B patients and indicate the importance of careful patient evaluation when making treatment decisions. Lay summary: The GIDEON (Global Investigation of therapeutic DEcisions in hepatocellular carcinoma and Of its treatment with sorafeNib) study is a large prospective registry of patients with liver cancer who were treated with sorafenib. The aims were to evaluate the safety and tolerability of sorafenib among those in which the liver was not functioning properly. The study showed that the safety profile of sorafenib was consistent across patients with preserved liver function and those in which the liver was not functioning properly, and therefore, suggesting that sorafenib may be a valid treatment for some patients with liver impairment.

AB - Background & Aims: GIDEON (Global Investigation of therapeutic DEcisions in hepatocellular carcinoma and Of its treatment with sorafeNib) is a prospective, observational registry study evaluating the safety of sorafenib and treatment practices in hepatocellular carcinoma patients. This large global database allowed for assessment of the use and tolerability of sorafenib in patients with liver dysfunction. Methods: Baseline characteristics and medical/treatment history were collected in patients for whom a decision to treat with sorafenib had been made. Adverse event, dosing, and outcomes data were collected during follow-up. Results: In the overall safety population (n. =3202), 1968 patients (61%) had Child-Pugh A status and 666 (21%) had Child-Pugh B. The majority of Child-Pugh A (72%) and Child-Pugh B (70%) patients received an initial sorafenib dose of 800. mg, consistent with the label, and dose reduction rates were 40% and 29%, respectively. The type and incidence of adverse events were generally consistent across Child-Pugh subgroups. The incidence of drug-related adverse events leading to discontinuation was similar between Child-Pugh A and Child-Pugh B patients (17% and 21%). In the intent-to-treat population (n. =3213), median overall survival (months [95% confidence interval]) was longer in Child-Pugh A patients (13.6 [12.8-14.7]) compared with Child-Pugh B patients (5.2 [4.6-6.3]). Conclusions: In clinical practice, the safety profile of sorafenib appeared to be consistent across Child-Pugh A and Child-Pugh B patients. Findings suggest sorafenib may be safely used in some Child-Pugh B patients and indicate the importance of careful patient evaluation when making treatment decisions. Lay summary: The GIDEON (Global Investigation of therapeutic DEcisions in hepatocellular carcinoma and Of its treatment with sorafeNib) study is a large prospective registry of patients with liver cancer who were treated with sorafenib. The aims were to evaluate the safety and tolerability of sorafenib among those in which the liver was not functioning properly. The study showed that the safety profile of sorafenib was consistent across patients with preserved liver function and those in which the liver was not functioning properly, and therefore, suggesting that sorafenib may be a valid treatment for some patients with liver impairment.

KW - Child-Pugh

KW - GIDEON

KW - HCC

KW - Hepatocellular carcinoma

KW - Nexavar

KW - Sorafenib

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