title: Impact of early molecular response in children with chronic myeloid leukemia treated in the French Glivec phase 4 study.
abstract: Studies in adults have shown that an early molecular response to imatinib predicts clinical outcome in chronic myeloid leukemia (CML). We investigated the impact of the BCR-ABL1 transcript level measured 3 months after starting imatinib in a cohort of 40 children with CML. Children with a BCR-ABL1/ABL ratio higher than 10% at 3 months after the start of imatinib had a larger spleen size and a higher white blood cell count compared with those with BCR-ABL1/ABL ≤10%. Children with BCR-ABL1/ABL ≤10% 3 months after starting imatinib had higher rates of complete cytogenetic response and major molecular response at 12 months compared with those with BCR-ABL1/ABL >10%. With a median follow-up of 71 months (range, 22-96 months), BCR-ABL1/ABL ≤10% correlated with better progression-free survival. Thus, early molecular response at 3 months predicts outcome in children treated with imatinib for CML. This trial was registered at www.clinicaltrials.gov as #NCT00845221.
authors.affiliation: Centre d'Investigation Clinique 1402 INSERM, Centre Hospitalier Universitaire de Poitiers, Poitiers, France;
authors.forename: Frédéric
authors.initials: F
authors.lastname: Millot
authors.affiliation: Centre d'Investigation Clinique 1402 INSERM, Centre Hospitalier Universitaire de Poitiers, Poitiers, France;
authors.forename: Joelle
authors.initials: J
authors.lastname: Guilhot
authors.affiliation: Pediatric Department, Assistance Publique-Hôpitaux de Paris and University Paris Diderot, Assistance Publique-Hôpital Saint Louis and Robert Debré, Paris, France;
authors.forename: André
authors.initials: A
authors.lastname: Baruchel
authors.affiliation: Hematology Unit, Hôpital Trousseau, Paris, France;
authors.forename: Arnaud
authors.initials: A
authors.lastname: Petit
authors.affiliation: Hematology Unit, Institut d'Hématologie et d'Oncologie Pédiatrique, Hospices Civils de Lyon, Lyon, France;
authors.forename: Yves
authors.initials: Y
authors.lastname: Bertrand
authors.affiliation: Pediatric Oncology Unit, University Hospital, Lille, France;
authors.forename: Françoise
authors.initials: F
authors.lastname: Mazingue
authors.affiliation: Pediatric Oncology Unit, University Hospital, Strasbourg, France;
authors.forename: Patrick
authors.initials: P
authors.lastname: Lutz
authors.affiliation: Pediatric Oncology Unit, University Hospital, Bordeaux, France;
authors.forename: Cecile
authors.initials: C
authors.lastname: Vérité
authors.affiliation: Hematology Department, University Hospital, Brest, France;
authors.forename: Christian
authors.initials: C
authors.lastname: Berthou
authors.affiliation: Hematology Unit, University Hospital, Marseille, France;
authors.forename: Claire
authors.initials: C
authors.lastname: Galambrun
authors.affiliation: Pediatric Oncology Unit, University Hospital, Montpellier, France;
authors.forename: Nicolas
authors.initials: N
authors.lastname: Sirvent
authors.affiliation: Hematology Unit, Hôpital Robert Debré, Paris, France;
authors.forename: Karima
authors.initials: K
authors.lastname: Yakouben
authors.affiliation: Hematology Unit, University Hospital, Nancy, France;
authors.forename: Claudine
authors.initials: C
authors.lastname: Schmitt
authors.affiliation: Pediatric Oncology Unit, University Hospital, Rennes, France;
authors.forename: Virginie
authors.initials: V
authors.lastname: Gandemer
authors.affiliation: Pediatric Oncology Unit, University Hospital, Angers, France;
authors.forename: Yves
authors.initials: Y
authors.lastname: Reguerre
authors.affiliation: Pediatric Oncology Unit, University Hospital, Dijon, France;
authors.forename: Gérard
authors.initials: G
authors.lastname: Couillault
authors.affiliation: Pediatric Oncology Unit, University Hospital, Nantes, France; and.
authors.forename: Françoise
authors.initials: F
authors.lastname: Mechinaud
authors.affiliation: Laboratory of Molecular Hematology, University Hospital Saint-Louis, Assistance Publique-Hôpitaux de Paris and EA3518 Unit, University Paris Diderot, Paris, France.
authors.forename: Jean-Michel
authors.initials: JM
authors.lastname: Cayuela
mesh_terms: D000293:Adolescent; D001549:Benzamides / Q000627:therapeutic use; D002648:Child; D002675:Child, Preschool; D020732:Cytogenetic Analysis; D018572:Disease-Free Survival; D005602:France; D016044:Fusion Proteins, bcr-abl / Q000235:genetics; D006801:Humans; D000068877:Imatinib Mesylate; D007223:Infant; D015464:Leukemia, Myelogenous, Chronic, BCR-ABL Positive / Q000188:drug therapy* / Q000235:genetics*; D010879:Piperazines / Q000627:therapeutic use; D011743:Pyrimidines / Q000627:therapeutic use
chemical_list: C581634:BCR-ABL1 fusion protein, human; D001549:Benzamides; D010879:Piperazines; D011743:Pyrimidines; D000068877:Imatinib Mesylate; D016044:Fusion Proteins, bcr-abl