Favourable outcome of de novo advanced phases of childhood chronic myeloid leukaemia

Eur J Cancer. 2019 Jul;115:17-23. doi: 10.1016/j.ejca.2019.03.020. Epub 2019 May 10.

Abstract

Background: Chronic myeloid leukaemia (CML) is very rare in children. The aim of the study is to report the experience within the I-CML-Ped study in children and adolescents presenting at diagnosis with advanced phase disease and to describe their characteristics and outcomes.

Methods: Of 479 children and adolescents enrolled in the international registry for childhood chronic myeloid leukaemia (I-CML-Ped Study; www.clinicaltrials.govNCT01281735), 36 children (7.5%) presented at initial diagnosis with CML in advanced phase according to the European LeukemiaNet criteria.

Results: Nineteen (4%) patients were diagnosed in accelerated phase (CML-AP), and among the 17 patients (3.5%) diagnosed in blastic phase (CML-BP), 70% presented with lymphoid immunophenotype. Initial treatment of CML-AP/CML-BP consisted of tyrosine kinase inhibitors (TKIs) with or without chemotherapy, leading to complete haematologic response in 33 of 36 (92%) patients. Seventeen patients proceeded to haematopoietic stem cell transplantation. At the last follow-up, 18 of 19 patients with de novo CML-AP are alive in at least major molecular response (MMR) (n = 16), in progression (n = 1) or in molecular relapse (n = 1) and 13 of 17 patients with de novo CML-BP are alive in at least MMR. Five-year overall survival rates are 94% (95% confidence interval [CI]: 66%-99%) and 74% (95% CI: 44%-89%) for patients diagnosed in CML-AP and CML-BP, respectively.

Conclusion: Children with advanced phase at diagnosis of CML seem to have a better survival rate than that reported for advanced phases evolving under TKI treatment.

Keywords: Children; Chronic myeloid leukaemia; Imatinib; Stem cell transplantation; Tyrosine kinase inhibitors.

Publication types

  • Comparative Study
  • Multicenter Study

MeSH terms

  • Adolescent
  • Age of Onset
  • Cancer Survivors*
  • Child
  • Child, Preschool
  • Databases, Factual
  • Disease Progression
  • Female
  • Hematopoietic Stem Cell Transplantation* / adverse effects
  • Hematopoietic Stem Cell Transplantation* / mortality
  • Humans
  • Leukemia, Myelogenous, Chronic, BCR-ABL Positive / mortality
  • Leukemia, Myelogenous, Chronic, BCR-ABL Positive / pathology
  • Leukemia, Myelogenous, Chronic, BCR-ABL Positive / therapy*
  • Male
  • Molecular Targeted Therapy
  • Neoplasm Staging
  • Protein Kinase Inhibitors / adverse effects
  • Protein Kinase Inhibitors / therapeutic use*
  • Registries
  • Time Factors
  • Treatment Outcome

Substances

  • Protein Kinase Inhibitors

Associated data

  • ClinicalTrials.gov/NCT01281735