Abstract
Venetoclax is approved for adult patients with chronic lymphocytic leukemia and acute myeloid leukemia. Expanding its use to the pediatric population is currently under investigation, but more robust data are needed. We retrospectively analyzed the safety and efficacy of venetoclax in children/AYA with ALL/LBL. We identified 18 patients (T-cell ALL, n = 7; T-cell LBL, n = 6; B-cell ALL, n = 5) aged 6-22 years. No new venetoclax safety signals were identified; the most common toxicity was myelosuppression. No deaths occurred within 30 days from the start of the therapy. A mean of 2.6 (range 0-8) prior lines of therapy were given. The mean duration of venetoclax was 4.06 months (range 0.2-24.67 months). Complete remission was achieved in 11 (61%) patients. Of the eight patients who remain alive, four are continuing on venetoclax combination therapy, and four proceeded to hematopoietic stem cell transplantation. Three patients who initially achieved CR, later relapsed, and are deceased. Nine patients are deceased, and one patient was lost to follow-up. Overall survival is 9.14 months (range 1.1-33.1), and progression-free survival is 7.34 months (range 0.2-33.1). This is the largest cohort of pediatric/AYA patients who received venetoclax for ALL/LBL. Our data support the consideration of venetoclax-based regimens in pediatric patients with R/R ALL/LBL and its investigation as upfront therapy for T-cell ALL/LBL.
Highlights
Pediatric acute lymphoblastic leukemia (ALL) and lymphoblastic lymphoma (LBL) have remarkable outcomes, with event-free survival (EFS) >90% for B-cell ALL, 85–89% for T-cell ALL, and 80–85% for T-cell LBL patients [1,2,3,4,5,6]
Given the encouraging effects of venetoclax combinations with chemotherapy in patients with lymphoid malignancies and the sparsity of reported outcomes in T-cell LBL, and pediatrics/AYA in general, we retrospectively reviewed our institutional experience of venetoclax use in pediatric/AYA patients at The University of Texas MD Anderson Cancer
Response criteria were established per the Revised Recommendations of the International Working Group Response Criteria in Acute Leukemia, and responses for lymphomas were according to the Malignant Lymphomas Imaging Working Group [29]
Summary
Pediatric acute lymphoblastic leukemia (ALL) and lymphoblastic lymphoma (LBL) have remarkable outcomes, with event-free survival (EFS) >90% for B-cell ALL, 85–89% for T-cell ALL, and 80–85% for T-cell LBL patients [1,2,3,4,5,6]. For B-cell ALL, relapse is seen in >10% of patients, and 2% of patients never achieve first remission due to refractory disease [7,8]. When focusing on T-cell ALL, seen in 15% of the pediatric ALL population, and the early thymic precursor (ETP) phenotype, the relapse rate is up to 18% [9,10,11]. Prognosis becomes especially poor for patients with relapsed T-cell ALL/LBL, with a dismal response to salvage therapy and a 3-year EFS
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