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Panobinostat in adults with H3 K27M-mutant diffuse midline glioma: a single-center experience.

Authors :
Neth BJ
Balakrishnan SN
Carabenciov ID
Uhm JH
Daniels DJ
Kizilbash SH
Ruff MW
Source :
Journal of neuro-oncology [J Neurooncol] 2022 Mar; Vol. 157 (1), pp. 91-100. Date of Electronic Publication: 2022 Jan 25.
Publication Year :
2022

Abstract

Introduction: Diffuse midline gliomas (DMG) with the H3 K27M-mutation are a well-described entity with most DMG harboring this mutation, with notable heterogeneity in adults. No therapy has been proven to improve survival in this tumor type. Panobinostat is a histone deacetylase inhibitor that may have therapeutic benefit.<br />Methods: We report our retrospective experience with use of panobinostat in adults (> 18 years) with H3 K27M-mutant DMG treated at Mayo Clinic (Rochester) from January 2016 to August 2020, with follow-up until October 2021. Survival was calculated using the Kaplan-Meier method.<br />Results: 4 patients with H3 K27M-mutant glioma were treated with panobinostat as compassionate use. Patients had a median age of 40 years (range 22-62 years) and 2 were female. Tumor location was midline for all patients, spinal cord (n = 2), brainstem (n = 1), and thalamus (n = 1). All tumors were IDH1/IDH2 wildtype. 3 patients received radiotherapy followed by adjuvant panobinostat. All patients had no other pharmacologic therapy utilized prior to or during panobinostat therapy aside from concurrent dexamethasone utilized in 3 patients. No patient experienced a grade 2 or higher (per CTCAE grade) adverse effect. The median overall survival was 42 months, median progression free survival of 19 months, 2 patients were alive at last follow up (both with spinal cord tumors and received radiation). The best response was stable disease in 2 patients and a partial response in 1 patient.<br />Conclusions: This is the first report of clinical outcomes of panobinostat in adults with H3 K27M-mutant DMG. We showed that it is well-tolerated at the dosage schedule that we describe, with no serious adverse effects throughout the study period.<br /> (© 2022. The Author(s), under exclusive licence to Springer Science+Business Media, LLC, part of Springer Nature.)

Details

Language :
English
ISSN :
1573-7373
Volume :
157
Issue :
1
Database :
MEDLINE
Journal :
Journal of neuro-oncology
Publication Type :
Academic Journal
Accession number :
35076860
Full Text :
https://doi.org/10.1007/s11060-022-03950-8