Hiroyuki Muranushi, Junya Kanda, Tadakazu Kondo, Yasuyuki Arai, Ikue Okamura-Shiki, Takashi Ikeda, Hiroki Amagase, Takehiro Okuda, Daishi Nakagawa, Takeshi Maeda, Yasunori Ueda, Kazunori Imada, Kazuhiro Yago, Tomoharu Takeoka, Mutsumi Okada, Yasuko Miyahara, MItsumasa Watanabe, Nobuyoshi Arima, Toshiyuki Kitano, Masakatsu Hishizawa, Katsuhiro Io, Satoko Oka, Kosuke Asagoe, Takashi Akasaka, Kouhei Yamashita, Akifumi Takaori-Kondo
The present retrospective multicenter study in Japan evaluated the role of venetoclax plus azacitidine (V/A) before first allogeneic hematopoietic stem cell transplantation (SCT) in 144 transplant-eligible acute myeloid leukemia (AML). Thirty-seven patients received V/A prior to SCT, mainly as salvage therapy for refractory and adverse-risk AML. Patients were classified into four groups according to remission status at SCT and V/A use. The 1-year overall survival (OS), cumulative incidence of relapse (CIR) and non-relapse mortality (NRM) of those who achieved complete remission (CR) with intensive chemotherapy (IC) were 93%, 5% and 4%, respectively. The 1-year OS, CIR and NRM of those who were refractory/intolerant to IC and achieved CR with V/A were 93%, 7% and 7%, respectively. The 1-year OS, CIR and NRM of those who were refractory to IC and proceeded directly to SCT were 52%, 54% and 13%, respectively. The 1-year OS, CIR and NRM of those who were refractory/intolerant to IC and refractory to V/A were 41%, 39% and 29%, respectively. Overall, use of V/A prior to SCT salvaged some AML patients who were refractory/intolerant to IC, and the transplant outcomes of these patients were favorable. Further studies are needed to optimize treatment for AML prior to SCT.