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Pre-clinical Evaluation of Tyrosine Kinase Inhibitors for Treatment of Acute Leukemia
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Published on: September 18, 2013

Clinical Presentation and Treatment Outcomes of Pediatric Acute Promyelocytic Leukemia: A Study From a Developing

Naila Rafiq1, Ghulam Q Pathan1, Sadia Muhammad1

  • 1Departments of Pediatric Hematology Oncology.

Journal of Pediatric Hematology/Oncology
|June 15, 2026
PubMed

Insights

Acute promyelocytic leukemia (APL) is treatable with all-trans retinoic acid (ATRA) and arsenic trioxide (ATO). While this regimen achieves high remission rates in pediatric patients, early deaths from hemorrhage and relapses remain challenges in resource-limited settings.

Area of Science:

  • Hematology
  • Pediatric Oncology
  • Clinical Research

Background:

  • Acute promyelocytic leukemia (APL) is a subtype of acute myeloid leukemia (AML).
  • Early treatment with all-trans retinoic acid (ATRA) and arsenic trioxide (ATO) offers a high cure rate for APL.
  • Resource limitations in low- and middle-income countries (LMICs) can impact treatment outcomes.

Purpose of the Study:

  • To examine the clinical characteristics and outcomes of pediatric APL patients treated with an ATRA-ATO regimen.
  • To evaluate the effectiveness and challenges of this treatment protocol in a resource-constrained environment.

Main Methods:

  • Retrospective review of pediatric patients (1-16 years) diagnosed with APL between January 2019 and December 2022.
  • Diagnosis confirmed by morphology and PML-RARA fluorescence in situ hybridization (FISH).
  • Patients stratified into low-risk (<10×10^9/L white cell count) and high-risk (≥10×10^9/L WCC); survival analyzed using Kaplan-Meier.

Main Results:

  • Fifty pediatric APL patients were evaluated; 68% were high-risk.
  • All patients achieved morphologic and molecular remission.
  • Twenty-two percent of patients died, primarily from hemorrhage; 10% experienced abandonment and 8% relapsed.
  • Two-year event-free survival (EFS) was 60% (81% low-risk, 50% high-risk).

Conclusions:

  • The ATRA-ATO regimen demonstrates high remission rates in pediatric APL.
  • Early mortality due to hemorrhage and treatment abandonment are significant concerns in LMICs.
  • Improving survival necessitates enhanced early detection, referral systems, and supportive care in resource-limited settings.

Keywords:
early mortalityevent-free survivalpediatric acute promyelocytic leukemiaresource-limited settingsrisk-adapted therapy

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