title: Can chronic myeloid leukaemia in children and adolescents be successfully treated without haematopoietic stem cell transplant? A single centre experience.
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.
authors.affiliation: Haematology, Department of Cellular Biotechologies and Haematology, "Sapienza" University, Rome, Italy.
authors.forename: Fiorina
authors.initials: F
authors.lastname: Giona
authors.affiliation: Haematology, Department of Cellular Biotechologies and Haematology, "Sapienza" University, Rome, Italy.
authors.forename: Maria L
authors.initials: ML
authors.lastname: Moleti
authors.affiliation: Haematology, Department of Cellular Biotechologies and Haematology, "Sapienza" University, Rome, Italy.
authors.forename: Daniela
authors.initials: D
authors.lastname: De Benedittis
authors.affiliation: Haematology, Department of Cellular Biotechologies and Haematology, "Sapienza" University, Rome, Italy.
authors.forename: Michelina
authors.initials: M
authors.lastname: Santopietro
authors.affiliation: Haematology, Department of Cellular Biotechologies and Haematology, "Sapienza" University, Rome, Italy.
authors.forename: Mauro
authors.initials: M
authors.lastname: Nanni
authors.affiliation: Haematology, Department of Cellular Biotechologies and Haematology, "Sapienza" University, Rome, Italy.
authors.forename: Anna M
authors.initials: AM
authors.lastname: Testi
authors.affiliation: GIMEMA Foundation, Rome, Italy.
authors.forename: Sonia
authors.initials: S
authors.lastname: Orlando
authors.affiliation: Haematology, Department of Cellular Biotechologies and Haematology, "Sapienza" University, Rome, Italy.
authors.forename: Anna P
authors.initials: AP
authors.lastname: Iori
authors.affiliation: GIMEMA Foundation, Rome, Italy.
authors.forename: Alfonso
authors.initials: A
authors.lastname: Piciocchi
authors.affiliation: Department of Clinical and Biological Sciences, University of Turin at Orbassano, Turin, Italy.
authors.forename: Enrico
authors.initials: E
authors.lastname: Gottardi
authors.affiliation: Haematology, Department of Cellular Biotechologies and Haematology, "Sapienza" University, Rome, Italy.
authors.forename: Walter
authors.initials: W
authors.lastname: Barberi
authors.affiliation: Haematology, Department of Cellular Biotechologies and Haematology, "Sapienza" University, Rome, Italy.
authors.forename: Daniela
authors.initials: D
authors.lastname: Diverio
authors.affiliation: Department of Clinical and Biological Sciences, University of Turin at Orbassano, Turin, Italy.
authors.forename: Giuseppe
authors.initials: G
authors.lastname: Saglio
authors.affiliation: Haematology, Department of Cellular Biotechologies and Haematology, "Sapienza" University, Rome, Italy.
authors.forename: Robin
authors.initials: R
authors.lastname: Foà
mesh_terms: D000293:Adolescent; D002648:Child; D002675:Child, Preschool; D019468:Disease Management; D016903:Drug Monitoring / Q000379:methods; D005260:Female; D018380:Hematopoietic Stem Cell Transplantation / Q000706:statistics & numerical data; D006801:Humans; D016898:Interferon-alpha / Q000627:therapeutic use*; D015466:Leukemia, Myeloid, Chronic-Phase / Q000401:mortality / Q000628:therapy*; D008297:Male; D047428:Protein Kinase Inhibitors / Q000627:therapeutic use*; D011505:Protein-Tyrosine Kinases / Q000037:antagonists & inhibitors; D016019:Survival Analysis; D016896:Treatment Outcome; D055815:Young Adult
chemical_list: D016898:Interferon-alpha; D047428:Protein Kinase Inhibitors; D011505:Protein-Tyrosine Kinases
keywords: CML; childhood leukaemia; haematopoietic stem cell transplantation; interferon; tyrosine kinase inhibitors