Haematopoietic stem cell transplantation after reduced intensity conditioning in children and adolescents with chronic myeloid leukaemia: A prospective multicentre trial of the I-BFM Study Group
Language English Country England, Great Britain Media print-electronic
Document type Journal Article, Multicenter Study, Clinical Trial
PubMed
38803040
DOI
10.1111/bjh.19535
Knihovny.cz E-resources
- Keywords
- allogeneic stem cell transplantation, children and adolescents, chronic myeloid leukaemia, reduced intensity conditioning,
- MeSH
- Leukemia, Myelogenous, Chronic, BCR-ABL Positive * therapy mortality MeSH
- Child MeSH
- Protein Kinase Inhibitors therapeutic use MeSH
- Humans MeSH
- Adolescent MeSH
- Graft vs Host Disease etiology prevention & control MeSH
- Child, Preschool MeSH
- Transplantation Conditioning * methods MeSH
- Prospective Studies MeSH
- Hematopoietic Stem Cell Transplantation * methods MeSH
- Treatment Outcome MeSH
- Check Tag
- Child MeSH
- Humans MeSH
- Adolescent MeSH
- Male MeSH
- Child, Preschool MeSH
- Female MeSH
- Publication type
- Journal Article MeSH
- Clinical Trial MeSH
- Multicenter Study MeSH
- Names of Substances
- Protein Kinase Inhibitors MeSH
This prospective multicentre trial evaluated the safety and the efficacy of a thiotepa/melphalan-based reduced intensity conditioning (RIC) haematopoietic stem cell transplantation (HSCT) in children and adolescents with chronic myeloid leukaemia (CML) in chronic phase (CP). Thirty-two patients were transplanted from matched siblings or matched unrelated donors. In 22 patients, HSCT was performed due to insufficient molecular response or loss of response to first- or second-generation tyrosine kinase inhibitor (TKI), with pretransplant BCR::ABL1 transcripts ranging between 0.001% and 33%. The protocol included a BCR::ABL1-guided intervention with TKI retreatment in the first year and donor lymphocyte infusions (DLI) in the second-year post-transplant. All patients engrafted. The 1-year transplant-related mortality was 3% (confidence interval [CI]: 0%-6%). After a median follow-up of 6.3 years, 5-year overall survival and event-free survival are 97% (CI: 93%-100%) and 91% (CI: 79%-100%) respectively. The current 5-year leukaemia-free survival with BCR::ABL1 <0.01% is 97% (CI: 88%-100%) and the current TKI- and DLI-free survival is 95% (CI: 85%-100%). The incidence of chronic graft-versus-host disease (GvHD) was 32%, being severe in four patients (13%). At last follow-up, 31 patients are GvHD-free and have stopped immunosuppression. RIC HSCT following pretreatment with TKI is feasible and effective in children and adolescents with CP-CML with an excellent disease-free and TKI-free survival.
Children's Cancer Research Institute Medical University of Vienna Vienna Austria
Department of Hematology Oncology Schneider Children's Medical Center of Israel Petah Tikva Israel
Department of Paediatric Haematology and Oncology St Anna Children's Hospital Vienna Austria
Department of Paediatrics and Adolescent Medicine Medical University of Vienna Vienna Austria
Department of Pediatric Hematology and Oncology Hannover Medical School Hannover Germany
Department of Pediatrics Medical University of Innsbruck Innsbruck Austria
Paediatric Haemato Oncology University of Helsinki Helsinki Finland
Pediatric Transplant Unit Fondazione IRCCS San Gerardo Dei Tintori Monza Italy
School of Medicine and Surgery University of Milano Bicocca Milan Italy
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