Treatment of paediatric APL: how does the therapeutic approach differ from adults?

Best Pract Res Clin Haematol. 2014 Mar;27(1):69-78. doi: 10.1016/j.beha.2014.04.007. Epub 2014 Apr 26.

Abstract

Acute promyelocytic leukaemia (APL) in children and adolescents shares many features with APL in adults. There are important distinctions, however, between these age groups in the presentation, complications and treatment outcomes. Paediatric patients are more likely to present with high risk features including elevated WBC count or microgranular variant (M3v). Yet the early death rate is lower in paediatric patients compared to adult patients. Overall outcomes such as CR, OS and EFS appear similar in paediatric and adult patients treated on similar regimens except that very young children may have a higher risk of relapse. While contemporary studies have clearly demonstrated improved survival in adults receiving ATO therapy, currently there is more limited data on the role of ATO in paediatric patients. Here we highlight the similarities and important distinctions between paediatric and adult APL while reviewing available data on treatment of paediatric APL.

Keywords: acute promyelocytic leukaemia; all-trans-retinoic acid; arsenic trioxide; paediatrics.

Publication types

  • Review

MeSH terms

  • Adolescent
  • Adrenal Cortex Hormones / therapeutic use
  • Adult
  • Age Factors
  • Age of Onset
  • Anthracyclines / administration & dosage
  • Anthracyclines / adverse effects
  • Anthracyclines / pharmacology
  • Antineoplastic Combined Chemotherapy Protocols / therapeutic use*
  • Arsenic Trioxide
  • Arsenicals / administration & dosage
  • Arsenicals / pharmacology
  • Biomarkers, Tumor / blood
  • Blood Coagulation Disorders / etiology
  • Blood Coagulation Disorders / mortality
  • Bone Marrow Examination
  • Cell Differentiation / drug effects
  • Child
  • Child, Preschool
  • Heart Diseases / chemically induced
  • Humans
  • Infant
  • Leukemia, Promyelocytic, Acute / diagnosis
  • Leukemia, Promyelocytic, Acute / drug therapy*
  • Leukemia, Promyelocytic, Acute / epidemiology
  • Multicenter Studies as Topic
  • Neoplasm, Residual
  • Neoplasms, Second Primary / drug therapy
  • Neoplasms, Second Primary / epidemiology
  • Oncogene Proteins, Fusion / blood
  • Oxides / administration & dosage
  • Oxides / pharmacology
  • Prognosis
  • Pseudotumor Cerebri / chemically induced
  • Randomized Controlled Trials as Topic
  • Remission Induction
  • Risk Factors
  • Salvage Therapy
  • Tretinoin / administration & dosage
  • Tretinoin / adverse effects
  • Tretinoin / pharmacology

Substances

  • Adrenal Cortex Hormones
  • Anthracyclines
  • Arsenicals
  • Biomarkers, Tumor
  • Oncogene Proteins, Fusion
  • Oxides
  • promyelocytic leukemia-retinoic acid receptor alpha fusion oncoprotein
  • Tretinoin
  • Arsenic Trioxide