Can chronic myeloid leukaemia in children and adolescents be successfully treated without haematopoietic stem cell transplant? A single centre experience

Br J Haematol. 2016 Jun;173(5):749-53. doi: 10.1111/bjh.13991. Epub 2016 Feb 23.

Abstract

We analysed the long-term outcome of 35 children and adolescents (<20 years at diagnosis) with chronic myeloid leukaemia (CML) in chronic phase: 20 patients had received interferon-alpha and/or tyrosine kinase inhibitors (TKIs), and 15 underwent a haematopoietic stem cell transplant. The 10-year survival probabilities were similar in transplanted and non-transplanted patients (73·3% vs. 72·1%, respectively), whereas the survival probability was significantly lower in patients diagnosed before 1999 compared to those diagnosed afterwards (62·1% vs. 100%, P = 0·0384). The availability of TKIs and the standardized molecular monitoring have significantly improved treatment, management and outcome in children and adolescents with CML.

Keywords: CML; childhood leukaemia; haematopoietic stem cell transplantation; interferon; tyrosine kinase inhibitors.

MeSH terms

  • Adolescent
  • Child
  • Child, Preschool
  • Disease Management
  • Drug Monitoring / methods
  • Female
  • Hematopoietic Stem Cell Transplantation* / statistics & numerical data
  • Humans
  • Interferon-alpha / therapeutic use*
  • Leukemia, Myeloid, Chronic-Phase / mortality
  • Leukemia, Myeloid, Chronic-Phase / therapy*
  • Male
  • Protein Kinase Inhibitors / therapeutic use*
  • Protein-Tyrosine Kinases / antagonists & inhibitors
  • Survival Analysis
  • Treatment Outcome
  • Young Adult

Substances

  • Interferon-alpha
  • Protein Kinase Inhibitors
  • Protein-Tyrosine Kinases