Venetoclax-based salvage therapy as bridge-to-transplant is feasible and effective in patients with relapsed/refractory AML.
Julia Marie UnglaubRichard F SchlenkJan M MiddekeStefan W KrauseSabrina KrausHermann EinseleMichael KramerSven ZukunftJoseph KauerSimon RendersElena KatelariChristoph SchliemannCaroline PabstThomas LuftPeter DregerChristoph RölligMartin BornhäuserCarsten Muller-TidowTim SauerPublished in: Blood advances (2024)
The BCL2-inhibitor Venetoclax (VEN) in combination with hypomethylating agents (HMA) has been approved for first-line treatment of acute myeloid leukemai (AML) patients ineligible for intensive treatment. Emerging Data suggest that VEN containing treatment strategies may also be effective in relapsed/refractory (R/R) AML, however, comparative studies with conventional treatment strategies for medically fit patients as a bridge-to transplant strategy are limited. Using propensity score matching (PSM) analysis, we compared 37 R/R AML patients, who received VEN-based salvage therapy as bridge to allogeneic hematopoietic cell transplantation (allo-HCT) with 90 patients from the German Study Alliance Leukemia (SAL) AML registry, who were treated with non-VEN-containing salvage therapy according to their treating physician's choice (TPC) including intensive and non-intensive protocols. The overall response rate (ORR=CR+CRi) among all VEN patients was significantly higher compared to the TPC control cohort (62% vs. 42%; p=0.049). Overall, 73% of VEN-treated patients vs. 63% of TPC patients were successfully bridged to allo-HCT (p =0.41). After a median follow-up of 34.3 months for the VEN cohort and 21.0 months for the TPC cohort, the median overall-survival (OS) was 15.8 months (95%-CI, 10.6-NE) and 10.5 months (95%-CI, 6.8-19.6) (p=0.15), respectively. PSM revealed a trend towards improved OS for VEN patients (HR 0.70; 95%-CI, 0.41-1.22; p=0.20). Median event free survival (EFS) was significantly longer in the VEN cohort (8.0 months) compared to the TPC cohort (3.7 months) (p=0.006). In summary, our data suggests that VEN-based salvage therapy is a safe and effective bridge to allo-HCT for this difficult-to-treat AML patient population.
Keyphrases
- end stage renal disease
- acute myeloid leukemia
- ejection fraction
- chronic kidney disease
- peritoneal dialysis
- prognostic factors
- primary care
- emergency department
- acute lymphoblastic leukemia
- bone marrow
- immune response
- mesenchymal stem cells
- intensive care unit
- cell death
- hepatitis b virus
- high dose
- diffuse large b cell lymphoma
- hodgkin lymphoma
- smoking cessation
- combination therapy
- case report
- acute respiratory distress syndrome
- pi k akt
- respiratory failure