1. Lymphoblastic predominance of blastic phase in children with chronic myeloid leukaemia treated with imatinib: A report from the I-CML-Ped Study
- Author
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Petr Sedlacek, Krzysztof Kałwak, Arnaud Petit, Chi Kong Li, Joelle Guilhot, Frédéric Millot, Eveline S. J. M. de Bont, Barbara De Moerloose, Meinolf Suttorp, Andrea Biondi, Deborah Meyran, Srdjana Culic, Birgitte Lausen, Université de Paris (UP), AP-HP Hôpital universitaire Robert-Debré [Paris], Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP), Service d'hématologie-immunologie-oncologie pédiatrique [CHU Trousseau], CHU Trousseau [APHP], Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Sorbonne Université (SU)-Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Sorbonne Université (SU), CIC - Poitiers, Université de Poitiers-Centre hospitalier universitaire de Poitiers (CHU Poitiers)-Direction Générale de l'Organisation des Soins (DGOS)-Institut National de la Santé et de la Recherche Médicale (INSERM), Meyran, D, Petit, A, Guilhot, J, Suttorp, M, Sedlacek, P, De Bont, E, Li, C, Kalwak, K, Lausen, B, Culic, S, de Moerloose, B, Biondi, A, and Millot, F
- Subjects
Male ,0301 basic medicine ,Cancer Research ,[SDV]Life Sciences [q-bio] ,RECOMMENDATIONS ,Tyrosine-kinase inhibitor ,Chronic myeloid leukaemia ,Children ,Accelerated phase ,Blastic phase ,Tyrosine kinase inhibitors ,Haematopoietic stem cell transplantation ,0302 clinical medicine ,hemic and lymphatic diseases ,Cumulative incidence ,Lymphocytes ,Child ,Incidence (epidemiology) ,CHRONIC MYELOGENOUS LEUKEMIA ,3. Good health ,Oncology ,Child, Preschool ,030220 oncology & carcinogenesis ,Imatinib Mesylate ,Female ,PROGNOSTIC DISCRIMINATION ,medicine.drug ,medicine.medical_specialty ,Adolescent ,medicine.drug_class ,Accelerated phase, Blastic phase, Children, Chronic myeloid leukaemia, Haematopoietic stem cell transplantation, Tyrosine kinase inhibitors ,Blastic Phase ,DIAGNOSIS ,03 medical and health sciences ,ADHERENCE ,Leukemia, Myelogenous, Chronic, BCR-ABL Positive ,Internal medicine ,MANAGEMENT ,medicine ,Humans ,neoplasms ,business.industry ,Infant ,Imatinib ,KINASE INHIBITOR ERA ,YOUNGER PATIENTS ,medicine.disease ,RANDOMIZED CML ,Transplantation ,030104 developmental biology ,Blast Crisis ,FOLLOW-UP ,business ,Chronic myelogenous leukemia ,Rare disease - Abstract
Background: Chronic myeloid leukaemia (CML) is a rare disease in children. The frequency and outcome of children evolving to accelerated phase (AP) or blastic phase (BP) under treatment with imatinib is unknown. The aim of the current study is to assess the inci-dence of progression from CML in chronic phase with imatinib frontline in a paediatric setting and describe the management and outcome of these patients.Patients and methods: In the I-CML-Ped Study database (www.clinicaltrials.gov, #NCT01281735), 19 of 339 paediatric patients in chronic phase treated with imatinib in the frontline evolved to CML-AP or CML-BP.Results: With a median follow-up of 38 months (range: 2-190 months), the cumulative inci-dence of progression at 1 and 3 years was 3% (confidence interval [CI] 95%: 1-5%) and 7% (CI 95%: 4-11%), respectively. We observed a large predominance of lymphoid-BP (70%) over myeloid-BP (30%) with imatinib in frontline therapy. Sixteen patients underwent haemato-poietic stem cell transplantation, and eight were treated with a tyrosine kinase inhibitor after transplant. Only the transplanted patients are alive. The 5-year overall survival rate of children with CML-AP/BP is 44%, with no statistical difference between the lymphoid-BP and myeloid-BP outcome.Conclusion: Children evolving to AP or BP under treatment with imatinib have a very poor prognosis with an overall survival under 50%, much worse than children with advanced phase at diagnosis. (c) 2020 Elsevier Ltd. All rights reserved.
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- 2020