Limited Efficacy of Venetoclax Combination Regimens in Acute Myeloid Leukemia with Extramedullary Relapse

The recently approved BCL2 inhibitor venetoclax (VEN) has achieved promising outcomes in new and relapsed/refractory (R/R) acute myeloid leukemia (AML). Although its use is not well established in R/R AML with extramedullary disease (EMD), some reports have shown promising outcomes. We retrospectively analyzed 11 patients of R/R AML with EMD (with (n = 4) or without (n = 7) concurrent marrow involvement), who were treated with VEN plus decitabine (n = 9) or low-dose cytarabine (n = 2) between May 2020 and October 2020 in Seoul St. Mary's Hospital. The median number of prior treatment lines was 3 (1-6), and most (n = 9, 81.8%) had multiple sites of EMD. Nine patients (81.8%) received concurrent therapy for extramedullary (EM) involvement sites with radiotherapy (RT) (n = 4), surgery (n = 1), and both of them (n = 4). Among 11 patients, 4 patients (36.4%) had either marrow or EM responses to VEN combination; EM response was seen in 1 patient (9.1%, partial response) who had received concurrent RT (25 Gy, 10 fx) during the 1st cycle of VEN combination, and other 3 patients showed marrow response without EM response. After median follow-up of 27.0 months, the median overall survival was estimated to be 5.4 months. To conclude, VEN combination regimens have shown only modest efficacy in EM recurrence of AML with little impact on eliciting EM response.