Treatment of myeloid malignancies relapsing after allogeneic hematopoietic stem cell transplantation with venetoclax and hypomethylating agents—a retrospective multicenter analysis on behalf of the German Cooperative Transplant Study Group

• DOI: 10.1007/s00277-020-04321-x
• Publication Date: 2020-11-16T05:05:24Z
• Authors: Esther Schuler; Eva-Maria Wagner-...; Salem Ajib; Gesine Bug; Martina Crysandt; Sabine Dressler; Andreas Hausmann; Daniela Heidenreich; Klaus Hirschbühl; Matthias Hoepting; Edgar Jost; Jennifer Kaivers; Stefan Klein; Michael Koldehoff; Lambros Kordelas; Oliver Kriege; Lutz P. Müller; Christina Rautenberg; Judith Schaffrath; Christoph Schmid; Daniel Wolff; Rainer Haas; Martin Bornhäuser; Thomas Schroeder; Guido Kobbe
• Keywords: Venetoclax; Decitabine; Salvage therapy; Azacitidine; Hypomethylating agent; Hematology

Abstract Treatment of relapse after allogeneic hematopoietic stem cell transplantation (alloHSCT) remains a great challenge. Aiming to evaluate the combination venetoclax and hypomethylating agents (HMAClax) for treatment myeloid malignancies alloHSCT, we retrospectively collected data from 32 patients treated at 11 German centers. Venetoclax was applied with azacitidine ( n = 13) or decitabine 19); received DLI in addition. HMAClax first salvage therapy 8 patients. The median number cycles per patient 2 (1–19). All but 1 had grade 3/4 neutropenia. Hospital admission infections necessary 23 (72%); 5 these were fatal. In 30 evaluable patients, overall response rate (ORR) 47% (14/30, 3 CR MRD neg , CR, CRi, MLFS, PR). ORR 86% versus 35% later p 0.03). 6 molecular (MR), 67% 42% hematological (HR) 24, n.s.). After follow-up 8.4 months, 25 (78%) died 7 alive. Estimated survival 3.7 months. Median 5.7 3.4 months n.s.) MR (not reached) compared HR (3.4 0.024). This retrospective case series shows that is utilized various different combinations, schedules, doses. Toxicity substantial who receive venetoclax/HMA combinations as derive greatest benefit.