Phase I/II study of the deacetylase inhibitor panobinostat after allogeneic stem cell transplantation in patients with high-risk MDS or AML (PANOBEST trial)

  • Maintenance therapy after allogeneic hematopoietic stem cell transplantation (HSCT) for acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) is conceptually attractive to prevent relapse, but has been hampered by the limited number of suitable anti-leukemic agents. The deacetylase inhibitor (DACi) panobinostat demonstrated moderate anti-leukemic activity in a small subset of patients with advanced AML and high-risk MDS in phase I/II trials.1, 2 It also displays immunomodulatory activity3 that may enhance leukemia-specific cytotoxicity4 and mitigate graft versus host disease (GvHD), but conversely could impair T- and NK cell function.5, 6 We conducted this open-label, multi-center phase I/II trial (NCT01451268) to assess the feasibility and preliminary efficacy of prolonged prophylactic administration of panobinostat after HSCT for AML or MDS. The study protocol was approved by an independent ethics committee and conducted in compliance with the Declaration of Helsinki. All patients provided written informed consent. ...
Author:Gesine Bug, Andreas BurchertORCiDGND, Eva-Maria Wagner, Nicolaus Kröger, Tobias Berg, Saskia Güller, Stephan Klaus Metzelder, Andrea Wolf, Sabine HüneckeGND, Peter BaderORCiDGND, Johannes Schetelig, Hubert ServeORCiDGND, Oliver Gerhard Ottmann
Pubmed Id:
Parent Title (English):Leukemia
Publisher:Springer Nature
Place of publication:London
Document Type:Article
Year of Completion:2017
Date of first Publication:2017/07/28
Publishing Institution:Universitätsbibliothek Johann Christian Senckenberg
Release Date:2018/03/01
Tag:Acute myeloid leukaemia; Molecularly targeted therapy; Myelodysplastic syndrome; Phase I trials; Stem-cell therapies
Page Number:3
First Page:2523
Last Page:2525
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Institutes:Medizin / Medizin
Dewey Decimal Classification:6 Technik, Medizin, angewandte Wissenschaften / 61 Medizin und Gesundheit / 610 Medizin und Gesundheit
Licence (German):License LogoCreative Commons - Namensnennung 4.0