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.

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

GOTTARDI, ENRICO MARCO;SAGLIO, Giuseppe;
2016

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.
173
5
749
753
www.blackwellpublishing.com/journals/BJH
Childhood leukaemia; CML; Haematopoietic stem cell transplantation; Interferon; Tyrosine kinase inhibitors; Adolescent; Child; Child, Preschool; Disease Management; Drug Monitoring; Female; Humans; Interferon-alpha; Leukemia, Myeloid, Chronic-Phase; Male; Protein Kinase Inhibitors; Protein-Tyrosine Kinases; Survival Analysis; Treatment Outcome; Young Adult; Hematopoietic Stem Cell Transplantation; Hematology
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; Foã , Robin
File in questo prodotto:
Non ci sono file associati a questo prodotto.

I documenti in IRIS sono protetti da copyright e tutti i diritti sono riservati, salvo diversa indicazione.

Utilizza questo identificativo per citare o creare un link a questo documento: http://hdl.handle.net/2318/1650478
Citazioni
  • ???jsp.display-item.citation.pmc??? 0
  • Scopus 1
  • ???jsp.display-item.citation.isi??? 0
social impact