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Venetoclax for Children and Adolescents with Acute Lymphoblastic Leukemia and Lymphoblastic Lymphoma

Amber Gibson1, Adriana Trabal1, David McCall1

  • 1Department of Pediatrics, The University of Texas MD Anderson Cancer Center, Houston, TX 77030, USA.

Cancers
|January 11, 2022
PubMed

Insights

This study shows venetoclax is safe and effective for pediatric patients with relapsed/refractory acute lymphoblastic leukemia (ALL) and lymphoblastic lymphoma (LBL). Data support its use in this population, including potential upfront therapy for T-cell ALL/LBL.

Area of Science:

  • Pediatric Hematology Oncology
  • Cancer Therapeutics
  • Clinical Pharmacology

Background:

  • Venetoclax is approved for adult leukemia but its use in pediatric patients with acute lymphoblastic leukemia (ALL) and lymphoblastic lymphoma (LBL) requires further investigation.
  • Robust data on venetoclax safety and efficacy in the pediatric population with relapsed/refractory (R/R) ALL/LBL are limited.

Purpose of the Study:

  • To retrospectively analyze the safety and efficacy of venetoclax in pediatric and adolescent/young adult (AYA) patients diagnosed with ALL or LBL.
  • To evaluate treatment outcomes, including remission rates, survival, and toxicity profiles.

Main Methods:

  • Retrospective analysis of 18 pediatric/AYA patients (aged 6-22 years) with T-cell ALL/LBL or B-cell ALL treated with venetoclax.
  • Evaluation of safety signals, common toxicities (myelosuppression), treatment duration, and prior lines of therapy.
  • Assessment of complete remission (CR) rates, overall survival (OS), and progression-free survival (PFS).

Main Results:

  • No new safety signals for venetoclax were identified; myelosuppression was the most frequent toxicity.
  • Complete remission was achieved in 61% of patients (11/18).
  • Overall survival was 9.14 months and progression-free survival was 7.34 months; 4 patients remain alive on venetoclax combination therapy, and 4 proceeded to stem cell transplantation.

Conclusions:

  • Venetoclax demonstrates a manageable safety profile and significant efficacy in pediatric/AYA patients with R/R ALL/LBL.
  • These findings support the use of venetoclax-based regimens in pediatric patients with R/R ALL/LBL.
  • Further investigation of venetoclax as upfront therapy for T-cell ALL/LBL is warranted.

Keywords:
Bcl-2 inhibitoracute lymphoblastic leukemiaearly precursor T-celllymphoblastic lymphomavenetoclax

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