Millot, FredericMaledon, NatachaGuilhot, JoelleKalwak, KrzysztofSuttorp, Meinolf2023-06-142023-06-142019-07Millot, F. vd. (2019). ''Favourable outcome of de novo advanced phases of childhood chronic myeloid leukaemia''. European Journal of Cancer, 115, 17-23.0959-80491879-0852https://doi.org/10.1016/j.ejca.2019.03.020https://www.sciencedirect.com/science/article/pii/S0959804919302229http://hdl.handle.net/11452/33036Background: Chronic myeloid leukaemia (CML) is very rare in children. The aim of the study is to report the experience within the I-CML-Ped study in children and adolescents presenting at diagnosis with advanced phase disease and to describe their characteristics and outcomes. Methods: Of 479 children and adolescents enrolled in the international registry for childhood chronic myeloid leukaemia (I-CML-Ped Study; www.clinicaltrials.gov NCT01281735), 36 children (7.5%) presented at initial diagnosis with CML in advanced phase according to the European Leukemia Net criteria. Results: Nineteen (4%) patients were diagnosed in accelerated phase (CML-AP), and among the 17 patients (3.5%) diagnosed in blastic phase (CML-BP), 70% presented with lymphoid immunophenotype. Initial treatment of CML-AP/CML-BP consisted of tyrosine kinase inhibitors (TKIs) with or without chemotherapy, leading to complete haematologic response in 33 of 36 (92%) patients. Seventeen patients proceeded to haematopoietic stem cell transplantation. At the last follow-up, 18 of 19 patients with de novo CML-AP are alive in at least major molecular response (MMR) (n = 16), in progression (n = 1) or in molecular relapse (n = 1) and 13 of 17 patients with de novo CML-BP are alive in at least MMR. Five-year overall survival rates are 94% (95% confidence interval [CI]: 66%-99%) and 74% (95% CI: 44%-89%) for patients diagnosed in CML-AP and CML-BP, respectively. Conclusion: Children with advanced phase at diagnosis of CML seem to have a better survival rate than that reported for advanced phases evolving under TKI treatment.eninfo:eu-repo/semantics/openAccessOncologyChronic myeloid leukaemiaImatinibTyrosine kinase inhibitorsStem cell transplantationChildrenChronic myelogenous leukemiaTyrosine kinase inhibitorsLymphoid blast crisisInternational registryPrognostic-factorsInterphase-fishCml patientsRecommendationsChildrenSurvivalAdolescentAge of onsetCancer survivorsChildChild, preschoolDatabases, factualDisease progressionFemaleHematopoietic stem cell transplantationHumansLeukemia, myelogenous, chronic, BCR-ABL positiveMaleMolecular targeted therapyNeoplasm stagingProtein kinase inhibitorsRegistriesTime factorsTreatment outcomeArticle0004717539000042-s2.0-85065258653172311531082688OncologyChronic Myeloid Leukemia; Imatinib; Protein Tyrosine Kinase InhibitorImatinibProtein tyrosine kinase inhibitorProtein kinase inhibitorAdolescentAdultAdvanced cancerArticleCancer chemotherapyChildChildhood leukemiaChronic myeloid leukemiaControlled studyFemaleFollow upHematopoietic stem cell transplantationHumanImmunophenotypingInfantLeukemia relapseMajor clinical studyMaleOverall survivalPriority journalProspective studyRetrospective studyTreatment responseAdverse eventCancer stagingCancer survivorChronic myeloid leukemiaClinical trialComparative studyDisease exacerbationFactual databaseHematopoietic stem cell transplantationMolecularly targeted therapyMortalityMulticenter studyOnset agePathologyPreschool childRegisterTime factorTreatment outcome