Ученые определили целевой диапазон концентрации венетоклакса для терапии после трансплантации при лейкозах

Researchers have identified a target venetoclax blood concentration window of 2605 to 4060 ng/mL for post-allogeneic hematopoietic stem cell transplantation (allo-HSCT) maintenance therapy in myelodysplastic syndromes (MDS) and acute myeloid leukemia (AML). According to findings published in Hematological Oncology, the target offers clinicians a benchmark for dosing venetoclax plus decitabine after allo-HCT. Researchers at Tianjin First Central Hospital analyzed 58 patients treated between April 2018 and June 2023, all of whom began maintenance therapy approximately 100 days after transplant. The cohort had a median age of 45 years and included 42 patients with de novo AML and 13 with MDS. Treatment consisted of venetoclax plus decitabine delivered over 2-month cycles, with a median of 5 cycles per patient and doses guided by therapeutic drug monitoring on day 3 of each cycle. At a median follow-up of 574 days, 2-year overall survival and progression-free survival rates were 86.6% and 83.6%, respectively, with a cumulative incidence of relapse of 12.7% and nonrelapse mortality of 4.93%. Hematologic toxicities were the most common adverse events, including neutropenia (n=29), anemia (n=28), and thrombocytopenia (n=24), with no incidence of tumor lysis syndrome or treatment-related deaths. Among 30 patients with therapeutic concentration data, venetoclax levels varied widely, with a median trough of 2901 ng/mL. Patients who sustained remission had significantly higher concentrations than those who relapsed (P =0.019), while concentrations above 4060 ng/mL were strongly linked to treatment-related toxicity (P <0.001). Receiver operating characteristic analysis identified 2605 ng/mL as the threshold below which relapse risk rose and 4060 ng/mL as the point above which toxicity risk climbed sharply. “We have, for the first time, defined a target therapeutic window for venetoclax within this combination, providing a practical basis for individualized dosing in clinical practice,” the authors wrote. They proposed that routine monitoring could let clinicians proactively adjust doses between cycles rather than reacting only after cytopenia develops, potentially reducing treatment interruptions. The authors cautioned that the retrospective, single-center design and modest sample size limit generalizability, and they called for prospective trials to confirm whether monitoring-guided dosing improves outcomes compared with standard empirical approaches. This article originally appeared on Rare Disease Advisor References: Lyu C, Xie T, Pu Y, et al. Defining a therapeutic window for venetoclax in post-transplant maintenance therapy for high-risk acute myeloid leukemia and myelodysplastic syndromes. Hematol Oncol. Published online August 6, 2026. doi:10.1002/hon.70237