Patient-reported outcomes in a phase II randomised study of regorafenib compared with lomustine in patients with relapsed glioblastoma (the REGOMA trial).


Journal

European journal of cancer (Oxford, England : 1990)
ISSN: 1879-0852
Titre abrégé: Eur J Cancer
Pays: England
ID NLM: 9005373

Informations de publication

Date de publication:
09 2021
Historique:
received: 31 03 2021
revised: 28 06 2021
accepted: 30 06 2021
pubmed: 14 8 2021
medline: 17 12 2021
entrez: 13 8 2021
Statut: ppublish

Résumé

The REGOMA trial showed that regorafenib significantly improved overall survival in patients with recurrent glioblastoma compared with lomustine. Patients treated with regorafenib experienced a higher occurrence of grade 3-4 drug-related adverse events than those receiving the standard treatment. Because this safety profile was expected, it was considered of great importance to assess the patient point of view regarding the disease and treatment impact on different aspects of life and patient well-being. We here report the final results of the health-related quality of life (HRQoL) assessment, a secondary end-point of the study. This trial is registered with ClinicalTrials.gov, NCT02926222. Patient-reported outcomes were assessed, within a prospective, randomised, multicentre, open-label phase II trial, by the European Organisation for Research and Treatment of Cancer core questionnaire and brain module at baseline and every 8-weekly neuroradiological assessment till disease progression. Mixed-effect linear models were fitted for each of the HRQoL domain to examine the change over progression-free time within and between arms. Furthermore, differences were also classified as clinically meaningful changes. To correct for multiple comparisons and avoid type I errors, the level of significance was set at P = 0.01 (2-sided). Of 119 enrolled patients, 56/59 (95%) patients and 58/60 (97%) patients treated with regorafenib and lomustime completed questionnaires at baseline, respectively. No significant differences were observed in any generic or cancer-specific domain during treatment in both arms, or between the two arms, except for the appetite loss and diarrhoea scales which were significantly worse in patients treated with regorafenib. The rate of patients with a clinically meaningful worsening for appetite loss, diarrhoea and for any other domain was not statistically different between the two arms. Regorafenib did not negatively affect HRQoL in patients with recurrent glioblastoma. These data combined with the survival benefit shown in the REGOMA trial support the use of regorafenib as a treatment option for these patients.

Sections du résumé

BACKGROUND
The REGOMA trial showed that regorafenib significantly improved overall survival in patients with recurrent glioblastoma compared with lomustine. Patients treated with regorafenib experienced a higher occurrence of grade 3-4 drug-related adverse events than those receiving the standard treatment. Because this safety profile was expected, it was considered of great importance to assess the patient point of view regarding the disease and treatment impact on different aspects of life and patient well-being. We here report the final results of the health-related quality of life (HRQoL) assessment, a secondary end-point of the study. This trial is registered with ClinicalTrials.gov, NCT02926222.
METHODS
Patient-reported outcomes were assessed, within a prospective, randomised, multicentre, open-label phase II trial, by the European Organisation for Research and Treatment of Cancer core questionnaire and brain module at baseline and every 8-weekly neuroradiological assessment till disease progression. Mixed-effect linear models were fitted for each of the HRQoL domain to examine the change over progression-free time within and between arms. Furthermore, differences were also classified as clinically meaningful changes. To correct for multiple comparisons and avoid type I errors, the level of significance was set at P = 0.01 (2-sided).
RESULTS
Of 119 enrolled patients, 56/59 (95%) patients and 58/60 (97%) patients treated with regorafenib and lomustime completed questionnaires at baseline, respectively. No significant differences were observed in any generic or cancer-specific domain during treatment in both arms, or between the two arms, except for the appetite loss and diarrhoea scales which were significantly worse in patients treated with regorafenib. The rate of patients with a clinically meaningful worsening for appetite loss, diarrhoea and for any other domain was not statistically different between the two arms.
CONCLUSIONS
Regorafenib did not negatively affect HRQoL in patients with recurrent glioblastoma. These data combined with the survival benefit shown in the REGOMA trial support the use of regorafenib as a treatment option for these patients.

Identifiants

pubmed: 34388515
pii: S0959-8049(21)00454-8
doi: 10.1016/j.ejca.2021.06.055
pii:
doi:

Substances chimiques

Phenylurea Compounds 0
Pyridines 0
regorafenib 24T2A1DOYB
Lomustine 7BRF0Z81KG

Banques de données

ClinicalTrials.gov
['NCT02926222']

Types de publication

Clinical Trial, Phase II Journal Article Multicenter Study Randomized Controlled Trial Research Support, Non-U.S. Gov't

Langues

eng

Sous-ensembles de citation

IM

Pagination

179-190

Informations de copyright

Copyright © 2021 Elsevier Ltd. All rights reserved.

Déclaration de conflit d'intérêts

Conflict of interest statement GL has declared a consulting or advisory role for funding from Bayer, AbbVie, Orbus Therapeutics and BrainFarm; travel funding from Roche and Bayer; RR has declared research funding from Mundipharma, Novocure, UCB; BD has declared personal fees from EISAI, ELI LILLY, ASTRA ZENECA, MSD, ROCHE, AMGEN, personal grants and non-financial support from IPSEN, SANOFI and BAYER; VZ has declared consulting or advisory role funding from Bristol-Myers Squibb and Merck, Speakers Bureau funding from Bayer, Roche, Bristol-Myers Squibb, Astellas Pharma, Servier, AstraZeneca and Lilly, travel and accommodation funding from Bayer, Roche and Servier. All remaining authors have declared no conflict of interest to declare.

Auteurs

Giuseppe Lombardi (G)

Department of Oncology, Oncology 1, Veneto Institute of Oncology IOV - IRCCS, Padua, Italy.

Paola Del Bianco (P)

Clinical Research Unit, Veneto Institute of Oncology IOV - IRCCS, Padua, Italy.

Alba A Brandes (AA)

Medical Oncology Department, AUSL/IRCCS, Neurological Science Institute, Bologna, Italy.

Marica Eoli (M)

Molecolar Neuro-Oncology Unit, IRCCS Foundation, "Carlo Besta" Neurological Institute, Milan, Italy.

Roberta Rudà (R)

Department of Neuro-Oncology, University of Turin and City of Health and Science Hospital, Turin, Italy.

Toni Ibrahim (T)

Osteoncology and Rare Tumors Center, IRST-IRCCS "Dino Amadori", Meldola, Italy.

Ivan Lolli (I)

Medical Oncology Unit, IRCCS Saverio de Bellis Hospital, Castellana Grotte, Bari, Italy.

Simona Rizzato (S)

Department of Oncology, Santa Maria della Misericordia University Hospital, Udine, Italy.

Bruno Daniele (B)

Oncology Unit, Ospedale del Mare, Napoli, Italy.

Andrea Pace (A)

Neuroncology Unit, IRCCS Regina Elena Cancer Institute, Roma, Italy.

Francesco Pasqualetti (F)

Radiotherapy Unit, University Hospital S. Chiara, Pisa, Italy.

Mario Caccesse (M)

Department of Oncology, Oncology 1, Veneto Institute of Oncology IOV - IRCCS, Padua, Italy.

Eleonora Bergo (E)

Department of Oncology, Oncology 1, Veneto Institute of Oncology IOV - IRCCS, Padua, Italy.

Giovanna Magni (G)

Clinical Research Unit, Veneto Institute of Oncology IOV - IRCCS, Padua, Italy.

Gian Luca De Salvo (GL)

Clinical Research Unit, Veneto Institute of Oncology IOV - IRCCS, Padua, Italy. Electronic address: gianluca.desalvo@iov.veneto.it.

Vittorina Zagonel (V)

Department of Oncology, Oncology 1, Veneto Institute of Oncology IOV - IRCCS, Padua, Italy.

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Classifications MeSH