Venetoclax and Homoharringtonine‐Based Therapy Exhibited a Striking Response in Refractory/Relapsed Early T‐Cell Precursor Acute Lymphoblastic Leukemia

Early T‐cell precursor acute lymphoblastic leukemia (ETP‐ALL) is an aggressive subtype of T‐ALL. Once refractory or relapsed, it is associated with a poor prognosis, with a complete remission (CR) rate of 36%–46% following re‐induction therapy. Previously, we reported a synergistic effect of venetoclax (VEN) and homoharringtonine (HHT) in ETP‐ALL, which potentially elicits notable clinical responses. Herein, we investigated the efficacy and safety of the V‐HAG regimen (VEN, HHT, cytarabine, and granulocyte colony‐stimulating factor [G‐CSF]) in patients with refractory/relapsed ETP‐ALL through a prospective, multicenter, single‐arm, open‐label, phase 2 clinical trial. A total of 18 patients were enrolled, and 100% of these patients achieved CR or CR with incomplete hematological recovery (CRi) after 1 cycle of the V‐HAG regimen as re‐induction therapy. As a follow‐up, both the relapse rate and mortality rate were 33.3%. The 1‐year overall survival and relapse‐free survival were 76.7% (95% confidence interval [CI]: 53.2%‐100.0%) and 55.7% (95% CI: 28.8%–82.6%), respectively. The most common grade 3–4 adverse events were neutropenia (100%), anemia (88.9%), and thrombocytopenia (100%). Notably, the VEN‐ and HHT‐based therapy, V‐HAG regimen, exhibits an extremely high efficacy in the treatment of patients with refractory/relapsed ETP‐ALL with good tolerance, and it provides a promising therapeutic strategy for improving their outcomes.