title: Imatinib is effective in children with previously untreated chronic myelogenous leukemia in early chronic phase: results of the French national phase IV trial.
abstract: OBJECTIVE
  Imatinib is the standard of care in adults with chronic myeloid leukemia (CML) in chronic phase (CP). Only a few studies to assess efficacy in children have been performed. We report on the results of the French prospective trial (ClinicalTrials.gov identifier NCT00845221) conducted in children and adolescents with newly diagnosed CML in CP.
  
  
  METHODS
  A total of 44 patients from age 10 months to 17 years with newly diagnosed CML in CP received daily imatinib 260 mg/m(2). Progression-free survival, responses, and tolerance were evaluated.
  
  
  RESULTS
  With a median follow-up times of 31 months (range, 11 to 64 months), the estimated progression-free survival rate at 36 months was 98% (95% CI, 85% to 100%). A complete hematologic response was achieved in 98% of the patients. The rates of complete cytogenetic response (CCyR) and major molecular response (MMR) were 61% and 31% at 12 months, respectively. During follow-up, CCyR and MMR were achieved in 36 children (77%) and 25 children (57%), respectively. Overall, 30% of the patients discontinued imatinib, mainly because of unsatisfactory response. The most common adverse events were neutropenia and musculoskeletal events.
  
  
  CONCLUSIONS
  Imatinib is effective in children with CML in CP with response rates similar to rates reported in adults. The adverse effects are acceptable, but longer follow-up studies are required to fully assess the long-term impact.
authors.affiliation: Centre d’Investigation Clinique 802, Institut National de la Santéet de la Recherche Médicale (INSERM), University Hospital Poitiers, Poitiers, France. f.millot@chu-poitiers.fr
authors.forename: Frédéric
authors.initials: F
authors.lastname: Millot
authors.forename: André
authors.initials: A
authors.lastname: Baruchel
authors.forename: Joelle
authors.initials: J
authors.lastname: Guilhot
authors.forename: Arnaud
authors.initials: A
authors.lastname: Petit
authors.forename: Thierry
authors.initials: T
authors.lastname: Leblanc
authors.forename: Yves
authors.initials: Y
authors.lastname: Bertrand
authors.forename: Françoise
authors.initials: F
authors.lastname: Mazingue
authors.forename: Patrick
authors.initials: P
authors.lastname: Lutz
authors.forename: Cécile
authors.initials: C
authors.lastname: Vérité
authors.forename: Christian
authors.initials: C
authors.lastname: Berthou
authors.forename: Claire
authors.initials: C
authors.lastname: Galambrun
authors.forename: Frédéric
authors.initials: F
authors.lastname: Bernard
authors.forename: Karima
authors.initials: K
authors.lastname: Yacouben
authors.forename: Pierre
authors.initials: P
authors.lastname: Bordigoni
authors.forename: Christine
authors.initials: C
authors.lastname: Edan
authors.forename: Yves
authors.initials: Y
authors.lastname: Reguerre
authors.forename: Gérard
authors.initials: G
authors.lastname: Couillault
authors.forename: Françoise
authors.initials: F
authors.lastname: Méchinaud
authors.forename: Jean-Michel
authors.initials: JM
authors.lastname: Cayuela
authors.forename: François
authors.initials: F
authors.lastname: Guilhot
mesh_terms: D000293:Adolescent; D000970:Antineoplastic Agents / Q000009:adverse effects / Q000494:pharmacology / Q000627:therapeutic use*; D001549:Benzamides; D002648:Child; D002675:Child, Preschool; D018572:Disease-Free Survival; D004305:Dose-Response Relationship, Drug; D064420:Drug-Related Side Effects and Adverse Reactions; D005602:France; D006801:Humans; D000068877:Imatinib Mesylate; D007223:Infant; D015464:Leukemia, Myelogenous, Chronic, BCR-ABL Positive / Q000188:drug therapy* / Q000473:pathology; D010879:Piperazines / Q000009:adverse effects / Q000494:pharmacology / Q000627:therapeutic use*; D011446:Prospective Studies; D011743:Pyrimidines / Q000009:adverse effects / Q000494:pharmacology / Q000627:therapeutic use*; D016019:Survival Analysis
chemical_list: D000970:Antineoplastic Agents; D001549:Benzamides; D010879:Piperazines; D011743:Pyrimidines; D000068877:Imatinib Mesylate