Venetoclax-Based Regimen in Refractory or Relapsed Pediatric Acute Lymphoblastic Leukemia

Area of Science:

• Pediatric Hematology Oncology
• Cancer Therapeutics
• Leukemia Research

Background:

• Pediatric refractory or relapsed acute lymphoblastic leukemia (R/R ALL) presents significant treatment challenges.
• Venetoclax (VEN)-based regimens are emerging as a potential therapeutic option for difficult-to-treat leukemias.

Purpose of the Study:

• To investigate the efficacy and survival outcomes of pediatric R/R ALL treated with a VEN-based regimen.
• To assess the response rates and safety profile of VEN-based therapy in this patient population.

Main Methods:

• Retrospective analysis of pediatric patients with R/R ALL treated with VEN-based regimens.
• Evaluation of complete remission (CR), CR with incomplete recovery (CRi), and objective response rates (ORRs).
• Analysis of survival data and adverse events.

Main Results:

• A high remission rate (77.3%) was observed, with many achieving minimal residual disease (MRD)-negative status.
• Objective response rates were favorable for both B-cell ALL (75%) and T-cell ALL (80%).
• The VEN-based regimen demonstrated effectiveness in early T-cell precursor (ETP) ALL and KMT2A-rearranged ALL, with acceptable hematologic adverse reactions.

Conclusions:

• VEN-based regimens are effective in achieving high remission rates in pediatric R/R ALL.
• The treatment exhibits a favorable safety profile and is particularly beneficial for specific high-risk subtypes like ETP ALL and KMT2A-rearranged ALL.
• VEN-based chemotherapy represents a promising treatment option for pediatric patients with R/R ALL requiring remission induction.