TY - JOUR
T1 - Long-term follow-up of VIALE-A
T2 - Venetoclax and azacitidine in chemotherapy-ineligible untreated acute myeloid leukemia
AU - Pratz, Keith W.
AU - Jonas, Brian A.
AU - Pullarkat, Vinod
AU - Thirman, Michael J.
AU - Garcia, Jacqueline S.
AU - Döhner, Hartmut
AU - Récher, Christian
AU - Fiedler, Walter
AU - Yamamoto, Kazuhito
AU - Wang, Jianxiang
AU - Yoon, Sung Soo
AU - Wolach, Ofir
AU - Yeh, Su Peng
AU - Leber, Brian
AU - Esteve, Jordi
AU - Mayer, Jiri
AU - Porkka, Kimmo
AU - Illés, Árpád
AU - Lemoli, Roberto M.
AU - Turgut, Mehmet
AU - Ku, Grace
AU - Miller, Catherine
AU - Zhou, Ying
AU - Zhang, Meng
AU - Chyla, Brenda
AU - Potluri, Jalaja
AU - DiNardo, Courtney D.
N1 - Publisher Copyright:
© 2024 The Authors. American Journal of Hematology published by Wiley Periodicals LLC.
PY - 2024/4
Y1 - 2024/4
N2 - Venetoclax-azacitidine is approved for treatment of patients with newly diagnosed acute myeloid leukemia (AML) ineligible for intensive chemotherapy based on the interim overall survival (OS) analysis of the VIALE-A study (NCT02993523). Here, long-term follow-up is presented to address survival benefit and long-term outcomes with venetoclax-azacitidine. Patients with newly diagnosed AML who were ineligible for intensive chemotherapy were randomized 2:1 to receive venetoclax-azacitidine or placebo-azacitidine. OS was the primary endpoint; complete remission with/without blood count recovery (CR/CRi) was a key secondary endpoint. This final analysis was conducted when 100% of the predefined 360 OS events occurred. In VIALE-A, 431 patients were enrolled to venetoclax-azacitidine (n = 286) or placebo-azacitidine (n = 145). At 43.2 months median follow-up, median OS was 14.7 months (95% confidence interval [CI], 12.1–18.7) with venetoclax-azacitidine, and 9.6 months (95% CI, 7.4–12.7) with placebo-azacitidine (hazard ratio, 0.58 [95% CI, 0.47–0.72], p <.001); the estimated 24-month OS rate was 37.5% and 16.9%, respectively. Median OS for patients with IDH1/2 mutations and those with measurable residual disease responses was reached in this final analysis. CR/CRi rate was similar to interim analysis. Any-grade hematologic and gastrointestinal adverse events were most common in venetoclax-azacitidine and placebo-azacitidine arms, including thrombocytopenia (47% and 42%) and neutropenia (43% and 29%). No new safety signals were identified. Long-term efficacy and safety confirm venetoclax-azacitidine is an improvement in standard-of-care for patients with AML who are not eligible for intensive chemotherapy because of advanced age or comorbidities.
AB - Venetoclax-azacitidine is approved for treatment of patients with newly diagnosed acute myeloid leukemia (AML) ineligible for intensive chemotherapy based on the interim overall survival (OS) analysis of the VIALE-A study (NCT02993523). Here, long-term follow-up is presented to address survival benefit and long-term outcomes with venetoclax-azacitidine. Patients with newly diagnosed AML who were ineligible for intensive chemotherapy were randomized 2:1 to receive venetoclax-azacitidine or placebo-azacitidine. OS was the primary endpoint; complete remission with/without blood count recovery (CR/CRi) was a key secondary endpoint. This final analysis was conducted when 100% of the predefined 360 OS events occurred. In VIALE-A, 431 patients were enrolled to venetoclax-azacitidine (n = 286) or placebo-azacitidine (n = 145). At 43.2 months median follow-up, median OS was 14.7 months (95% confidence interval [CI], 12.1–18.7) with venetoclax-azacitidine, and 9.6 months (95% CI, 7.4–12.7) with placebo-azacitidine (hazard ratio, 0.58 [95% CI, 0.47–0.72], p <.001); the estimated 24-month OS rate was 37.5% and 16.9%, respectively. Median OS for patients with IDH1/2 mutations and those with measurable residual disease responses was reached in this final analysis. CR/CRi rate was similar to interim analysis. Any-grade hematologic and gastrointestinal adverse events were most common in venetoclax-azacitidine and placebo-azacitidine arms, including thrombocytopenia (47% and 42%) and neutropenia (43% and 29%). No new safety signals were identified. Long-term efficacy and safety confirm venetoclax-azacitidine is an improvement in standard-of-care for patients with AML who are not eligible for intensive chemotherapy because of advanced age or comorbidities.
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U2 - 10.1002/ajh.27246
DO - 10.1002/ajh.27246
M3 - Article
C2 - 38343151
AN - SCOPUS:85185111057
SN - 0361-8609
VL - 99
SP - 615
EP - 624
JO - American journal of hematology
JF - American journal of hematology
IS - 4
ER -