Axitinib plus avelumab in the treatment of recurrent glioblastoma: a stratified, open-label, single-center phase 2 clinical trial (GliAvAx)

Background No treatment demonstrated to improve survival in patients with recurrent glioblastoma (rGB) in a randomized trial. Combining axitinib with the programmed cell death ligand 1 blocking monoclonal antibody avelumab may result in synergistic activity against rGB.Methods Adult patients with rG...

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Main Authors: Bart Neyns, Julia Katharina Schwarze, Gil Awada, Laila Ben Salama, Jennifer De Cremer, Lydia Fischbuch, Laura Seynaeve, Stephanie Du Four, Anne-Marie Vanbinst, Alex Michotte, Hendrik Everaert, Anne Rogiers, Peter Theuns, Johnny Duerinck
Format: Article
Language:English
Published: BMJ Publishing Group 2020-10-01
Series:Journal for ImmunoTherapy of Cancer
Online Access:https://jitc.bmj.com/content/8/2/e001146.full
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author Bart Neyns
Julia Katharina Schwarze
Gil Awada
Laila Ben Salama
Jennifer De Cremer
Lydia Fischbuch
Laura Seynaeve
Stephanie Du Four
Anne-Marie Vanbinst
Alex Michotte
Hendrik Everaert
Anne Rogiers
Peter Theuns
Johnny Duerinck
author_facet Bart Neyns
Julia Katharina Schwarze
Gil Awada
Laila Ben Salama
Jennifer De Cremer
Lydia Fischbuch
Laura Seynaeve
Stephanie Du Four
Anne-Marie Vanbinst
Alex Michotte
Hendrik Everaert
Anne Rogiers
Peter Theuns
Johnny Duerinck
author_sort Bart Neyns
collection DOAJ
description Background No treatment demonstrated to improve survival in patients with recurrent glioblastoma (rGB) in a randomized trial. Combining axitinib with the programmed cell death ligand 1 blocking monoclonal antibody avelumab may result in synergistic activity against rGB.Methods Adult patients with rGB following prior surgery, radiation therapy and temozolomide chemotherapy were stratified according to their baseline use of corticosteroids. Patients with a daily dose of ≤8 mg of methylprednisolone (or equivalent) initiated treatment with axitinib (5 mg oral two times per day) plus avelumab (10 mg/kg intravenous every 2 weeks) (Cohort-1). Patients with a higher baseline corticosteroid dose initiated axitinib monotherapy; avelumab was added after 6 weeks of therapy if the corticosteroid dose could be tapered to ≤8 mg of methylprednisolone (Cohort-2). Progression-free survival at 6 months (6-m-PFS%), per immunotherapy response assessment for neuro-oncology criteria, served as the primary endpoint.Results Between June 2017 and August 2018, 54 patients (27 per cohort) were enrolled and initiated study treatment (median age: 55 years; 63% male; 91% Eastern Cooperative Oncology Group Performance Status 0–1). Seventeen (63%) patients treated in Cohort-2 received at least one dose of avelumab. The 6-m-PFS% was 22.2% (95% CI 6.5% to 37.9%) and 18.5% (95% CI 3.8% to 33.2%) in Cohort-1 and Cohort-2, respectively; median overall survival was 26.6 weeks (95% CI 20.8 to 32.4) in Cohort-1 and 18.0 weeks (95% CI 12.5 to 23.5) in Cohort-2. The best objective response rate was 33.3% and 22.2% in Cohort-1 and Cohort-2, respectively, with a median duration of response of 17.9 and 19.0 weeks. The most frequent treatment-related adverse events were dysphonia (67%), lymphopenia (50%), arterial hypertension and diarrhea (both 48%). There were no grade 5 adverse events.Conclusion The combination of avelumab plus axitinib has an acceptable toxicity profile but did not meet the prespecified threshold for activity justifying further investigation of this treatment in an unselected population of patients with rGB.
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series Journal for ImmunoTherapy of Cancer
spelling doaj-art-148c1424e10448fa9ffb5c151ec0bf592025-08-20T02:13:56ZengBMJ Publishing GroupJournal for ImmunoTherapy of Cancer2051-14262020-10-018210.1136/jitc-2020-001146Axitinib plus avelumab in the treatment of recurrent glioblastoma: a stratified, open-label, single-center phase 2 clinical trial (GliAvAx)Bart Neyns0Julia Katharina Schwarze1Gil Awada2Laila Ben Salama3Jennifer De Cremer4Lydia Fischbuch5Laura Seynaeve6Stephanie Du Four7Anne-Marie Vanbinst8Alex Michotte9Hendrik Everaert10Anne Rogiers11Peter Theuns12Johnny Duerinck132 Department of Medical Oncology, Vrije Universiteit Brussel (VUB), Universitair Ziekenhuis Brussel (UZ Brussel), Brussels, BelgiumDepartment of Medical Oncology/Laboratory for Medical and Molecular Oncology (LMMO), Vrije Universiteit Brussel (VUB)/Universitair Ziekenhuis Brussel (UZ Brussel), Brussels, BelgiumDepartment of Medical Oncology/Laboratory of Medical and Molecular Oncology (LMMO), Vrije Universiteit Brussel (VUB), Universitair Ziekenhuis Brussel (UZ Brussel), Brussels, Belgium1 Medical Oncology, Universitair Ziekenhuis Brussel, Brussels, Belgium2 Psychology, Vrije Universiteit Brussel, Brussels, Brussels, Belgium1 Medical Oncology, Universitair Ziekenhuis Brussel, Brussels, Belgium4 Department of Neurology, Vrije Universiteit Brussel (VUB), Universitair Ziekenhuis Brussel (UZ Brussel), Brussels, Belgium4 Neurosurgery, Universitair Ziekenhuis Brussel, Brussels, Belgium8 Department of Radiology, Vrije Universiteit Brussel (VUB), Universitair Ziekenhuis Brussel (UZ Brussel), Brussels, Belgium4 Department of Neurology, Vrije Universiteit Brussel (VUB), Universitair Ziekenhuis Brussel (UZ Brussel), Brussels, Belgium9 Department of Nuclear Medicine, Vrije Universiteit Brussel (VUB), Universitair Ziekenhuis Brussel (UZ Brussel), Brussels, Belgium8 Psychiatry, Centre Hospitalier Universitaire Brugmann, Brussels, Belgium2 Psychology, Vrije Universiteit Brussel, Brussels, Brussels, Belgium1 Department of Neurosurgery, Vrije Universiteit Brussel (VUB), Universitair Ziekenhuis Brussel (UZ Brussel), Brussels, BelgiumBackground No treatment demonstrated to improve survival in patients with recurrent glioblastoma (rGB) in a randomized trial. Combining axitinib with the programmed cell death ligand 1 blocking monoclonal antibody avelumab may result in synergistic activity against rGB.Methods Adult patients with rGB following prior surgery, radiation therapy and temozolomide chemotherapy were stratified according to their baseline use of corticosteroids. Patients with a daily dose of ≤8 mg of methylprednisolone (or equivalent) initiated treatment with axitinib (5 mg oral two times per day) plus avelumab (10 mg/kg intravenous every 2 weeks) (Cohort-1). Patients with a higher baseline corticosteroid dose initiated axitinib monotherapy; avelumab was added after 6 weeks of therapy if the corticosteroid dose could be tapered to ≤8 mg of methylprednisolone (Cohort-2). Progression-free survival at 6 months (6-m-PFS%), per immunotherapy response assessment for neuro-oncology criteria, served as the primary endpoint.Results Between June 2017 and August 2018, 54 patients (27 per cohort) were enrolled and initiated study treatment (median age: 55 years; 63% male; 91% Eastern Cooperative Oncology Group Performance Status 0–1). Seventeen (63%) patients treated in Cohort-2 received at least one dose of avelumab. The 6-m-PFS% was 22.2% (95% CI 6.5% to 37.9%) and 18.5% (95% CI 3.8% to 33.2%) in Cohort-1 and Cohort-2, respectively; median overall survival was 26.6 weeks (95% CI 20.8 to 32.4) in Cohort-1 and 18.0 weeks (95% CI 12.5 to 23.5) in Cohort-2. The best objective response rate was 33.3% and 22.2% in Cohort-1 and Cohort-2, respectively, with a median duration of response of 17.9 and 19.0 weeks. The most frequent treatment-related adverse events were dysphonia (67%), lymphopenia (50%), arterial hypertension and diarrhea (both 48%). There were no grade 5 adverse events.Conclusion The combination of avelumab plus axitinib has an acceptable toxicity profile but did not meet the prespecified threshold for activity justifying further investigation of this treatment in an unselected population of patients with rGB.https://jitc.bmj.com/content/8/2/e001146.full
spellingShingle Bart Neyns
Julia Katharina Schwarze
Gil Awada
Laila Ben Salama
Jennifer De Cremer
Lydia Fischbuch
Laura Seynaeve
Stephanie Du Four
Anne-Marie Vanbinst
Alex Michotte
Hendrik Everaert
Anne Rogiers
Peter Theuns
Johnny Duerinck
Axitinib plus avelumab in the treatment of recurrent glioblastoma: a stratified, open-label, single-center phase 2 clinical trial (GliAvAx)
Journal for ImmunoTherapy of Cancer
title Axitinib plus avelumab in the treatment of recurrent glioblastoma: a stratified, open-label, single-center phase 2 clinical trial (GliAvAx)
title_full Axitinib plus avelumab in the treatment of recurrent glioblastoma: a stratified, open-label, single-center phase 2 clinical trial (GliAvAx)
title_fullStr Axitinib plus avelumab in the treatment of recurrent glioblastoma: a stratified, open-label, single-center phase 2 clinical trial (GliAvAx)
title_full_unstemmed Axitinib plus avelumab in the treatment of recurrent glioblastoma: a stratified, open-label, single-center phase 2 clinical trial (GliAvAx)
title_short Axitinib plus avelumab in the treatment of recurrent glioblastoma: a stratified, open-label, single-center phase 2 clinical trial (GliAvAx)
title_sort axitinib plus avelumab in the treatment of recurrent glioblastoma a stratified open label single center phase 2 clinical trial gliavax
url https://jitc.bmj.com/content/8/2/e001146.full
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