Journal Article

DKFZ-2025-02988

http://join2-wiki.gsi.de/foswiki/pub/Main/Artwork/join2_logo100x88.png Pretransplant HMA and Venetoclax in AML patients - a Real-World Analysis from the Cooperative Transplant Study Group.

Rautenberg, C. ; Stelljes, M. ; Bug, G. ; Holtick, U. ; Jost, E. ; Jennrich, S. ; Mohring, A. ; Tischer, J. ; Fraccaroli, A. ; Schliemann, C. ; Schmalbrock, L. K. ; Bullinger, L.DKFZ* ; Metzdorf, J. ; Braitsch, K. ; Verbeek, M. ; Götze, K. ; Acker, F. ; Reinhardt, H. C. ; Kröger, N. ; Schroeder, T.

2025
Elsevier B. V. [Amsterdam]

Abstract: The combination of hypomethylating agents (HMA) and the BCL2 inhibitor venetoclax (VEN) has become standard first-line therapy for AML patients ineligible for intensive treatment and is also used as salvage therapy in relapsed/refractory (R/R) AML. Data on outcomes after subsequent allogeneic stem cell transplantation (allo-SCT) are limited. We retrospectively analyzed 91 patients with newly diagnosed (ND, n=34) or R/R (n=57) AML who received HMA/VEN followed by allo-SCT between 2020 and 2022. Overall response rate (ORR) to HMA/VEN was 67% with no significant difference between ND (73%) and R/R (64%) patients. Pre-transplant MRD negativity was achieved in 49% of responders. Post-transplant 2-year overall survival (OS) and relapse-free survival (RFS) were 52% and 42% for the entire cohort, with similar outcomes for ND (2-year OS 47%, RFS 38%) and R/R (2-year OS 56%, RFS 45%) patients. In patients with high-risk features, such as TP53 mutation or complex karyotype (cKT), ND patients still achieved promising responses (TP53mut 75%, cKT 80%) and acceptable post-transplant outcomes (TP53mut 2-year OS 36%; cKT 2-year OS 45%). In contrast, R/R patients showed lower response rates (TP53mut 14%, cKT 29%) and inferior outcomes (TP53mut 2-year OS 29%; cKT 2-year OS 29%). In conclusion, HMA/VEN is a feasible induction or salvage strategy enabling allo-SCT with encouraging outcomes in high-risk populations. In ND AML, this approach may expand transplant eligibility in older or frail patients. R/R patients with adverse molecular features may require alternative novel therapies or direct transplantation.

Keyword(s): AML ; HMA ; Venetoclax ; allogeneic transplantation ; measurable residual disease

Note: epub

---

Contributing Institute(s):

1. DKTK Koordinierungsstelle Berlin (BE01)

Research Program(s):

1. 899 - ohne Topic (POF4-899) (POF4-899)

Appears in the scientific report 2025

---

Database coverage:
; ; Clarivate Analytics Master Journal List ; Current Contents - Clinical Medicine ; Ebsco Academic Search ; Essential Science Indicators ; IF < 5 ; JCR ; SCOPUS ; Science Citation Index Expanded ; Web of Science Core Collection

---