Real-world use of venetoclax in the treatment of paediatric and teenage/young adult haematological malignancies

Br J Haematol. 2024 Dec;205(6):2355-2362. doi: 10.1111/bjh.19791. Epub 2024 Sep 26.

Abstract

Early-phase trials of venetoclax in children and teenagers/young adults with leukaemia have yielded promising results, but there remains a paucity of real-world data. To address this, we report a cohort of 41 children treated with venetoclax for a range of haematological malignancies, demonstrating complete remission in 43.6%, with most achieving minimal residual disease (MRD) negativity. Venetoclax was particularly effective as a bridge to transplant, with bridging successful in 75% of patients. Patients with MRD <1% at initiation of venetoclax were more likely to achieve MRD negativity (81.8% vs. 34.5%, p = 0.007) and had improved overall survival (54.5% vs. 17.9%, p = 0.004).

Keywords: acute leukaemia; childhood leukaemia; clinical studies; leukaemia therapy; myeloid leukaemia.

MeSH terms

  • Adolescent
  • Adult
  • Antineoplastic Agents / therapeutic use
  • Bridged Bicyclo Compounds, Heterocyclic* / administration & dosage
  • Bridged Bicyclo Compounds, Heterocyclic* / therapeutic use
  • Child
  • Child, Preschool
  • Female
  • Hematologic Neoplasms* / drug therapy
  • Hematopoietic Stem Cell Transplantation
  • Humans
  • Male
  • Neoplasm, Residual*
  • Remission Induction
  • Sulfonamides* / administration & dosage
  • Sulfonamides* / therapeutic use
  • Young Adult

Substances

  • venetoclax
  • Bridged Bicyclo Compounds, Heterocyclic
  • Sulfonamides
  • Antineoplastic Agents