Venetoclax-based salvage therapy as a bridge to transplant is feasible and effective in patients with relapsed/refractory AML

The B-cell lymphoma 2 inhibitor venetoclax (VEN) in combination with hypomethylating agents has been approved for first-line treatment of patients with acute myeloid leukemia (AML) ineligible for intensive treatment. VEN-containing treatment strategies may also be effective in relapsed/refractory (R...

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Hauptverfasser: Unglaub, Julia Marie (VerfasserIn) , Schlenk, Richard Friedrich (VerfasserIn) , Middeke, Jan Moritz (VerfasserIn) , Krause, Stefan W. (VerfasserIn) , Kraus, Sabrina (VerfasserIn) , Einsele, Hermann (VerfasserIn) , Kramer, Michael (VerfasserIn) , Zukunft, Sven (VerfasserIn) , Kauer, Joseph (VerfasserIn) , Renders, Simon (VerfasserIn) , Katelari, Elena (VerfasserIn) , Schliemann, Christoph (VerfasserIn) , Pabst, Caroline (VerfasserIn) , Luft, Thomas (VerfasserIn) , Dreger, Peter (VerfasserIn) , Röllig, Christoph (VerfasserIn) , Bornhäuser, Martin (VerfasserIn) , Müller-Tidow, Carsten (VerfasserIn) , Sauer, Tim (VerfasserIn)
Dokumenttyp: Article (Journal)
Sprache:Englisch
Veröffentlicht: 17 January 2025
In: Blood advances
Year: 2025, Jahrgang: 9, Heft: 2, Pages: 375-385
ISSN:2473-9537
DOI:10.1182/bloodadvances.2024013086
Online-Zugang:Verlag, kostenfrei, Volltext: https://doi.org/10.1182/bloodadvances.2024013086
Verlag, kostenfrei, Volltext: https://ashpublications.org/bloodadvances/article/9/2/375/517882/Venetoclax-based-salvage-therapy-as-a-bridge-to
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Verfasserangaben:Julia M. Unglaub, Richard F. Schlenk, Jan Moritz Middeke, Stefan W. Krause, Sabrina Kraus, Hermann Einsele, Michael Kramer, Sven Zukunft, Joseph Kauer, Simon Renders, Elena Katelari, Christoph Schliemann, Caroline Pabst, Thomas Luft, Peter Dreger, Christoph Röllig, Martin Bornhäuser, Carsten Müller-Tidow, and Tim Sauer, for the Study Alliance Leukemia

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520 |a The B-cell lymphoma 2 inhibitor venetoclax (VEN) in combination with hypomethylating agents has been approved for first-line treatment of patients with acute myeloid leukemia (AML) ineligible for intensive treatment. VEN-containing treatment strategies may also be effective in relapsed/refractory (R/R) AML; however, comparative studies with conventional therapies for fit patients as a bridge-to-transplant strategy are limited. Using propensity score matching (PSM), we compared 37 patients with R/R AML, who received VEN-based salvage therapy as bridge to allogeneic hematopoietic stem cell transplantation (allo-HCT), with 90 patients from the German Study Alliance Leukemia AML registry, who were treated with non-VEN-containing salvage therapy according to their treating physician’s choice (TPC). The overall response rate among VEN patients was higher than the TPC control cohort (62% vs 42%; P = .049). Overall, 73% of VEN-treated patients vs 63% of TPC patients were bridged to allo-HCT (P = .41). After a median follow-up of 34.3 months for the VEN and 21.0 months for the TPC cohort, the median overall survival (OS) were 15.8 months (95% confidence interval [CI], 10.6 to not evaluable) and 10.5 months (95% CI, 6.8-19.6; P = .15), respectively. PSM revealed a trend toward improved OS for VEN patients (hazard ratio, 0.70; 95% CI, 0.41-1.22; P = .20). Median event-free survival was significantly longer in the VEN cohort (8.0 months) than the TPC cohort (3.7 months; P = .006). Our data suggest that VEN-based salvage therapy is a safe and effective bridge to allo-HCT for this difficult-to-treat AML patient population. 
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