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

• Published in: British journal of haematology Vol. 173; no. 5; pp. 749 - 753
• Main Authors: Giona, Fiorina, Moleti, Maria L., De Benedittis, Daniela, Santopietro, Michelina, Nanni, Mauro, Testi, Anna M., Orlando, Sonia, Iori, Anna P., Piciocchi, Alfonso, Gottardi, Enrico, Barberi, Walter, Diverio, Daniela, Saglio, Giuseppe, Foà, Robin
• Format: Journal Article
• Language: English
• Published: England 01.06.2016
• Subjects: Adolescent; Child; Child, Preschool; childhood leukaemia; CML; Disease Management; Drug Monitoring - methods; Female; haematopoietic stem cell transplantation; Hematopoietic Stem Cell Transplantation - statistics & numerical data; Humans; interferon; 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; tyrosine kinase inhibitors; Young Adult; tyrosine kinase inhibitors; childhood leukaemia; CML; interferon; haematopoietic stem cell transplantation
• ISSN: 0007-1048; 1365-2141
• DOI: 10.1111/bjh.13991

Summary 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.