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.
Br J Haematol
; 205(1): 268-279, 2024 Jul.
Article
en En
| MEDLINE
| ID: mdl-38803040
ABSTRACT
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 BCRABL1 transcripts ranging between 0.001% and 33%. The protocol included a BCRABL1-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 BCRABL1 <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.
Palabras clave
Texto completo:
1
Colección:
01-internacional
Asunto principal:
Leucemia Mielógena Crónica BCR-ABL Positiva
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Trasplante de Células Madre Hematopoyéticas
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Acondicionamiento Pretrasplante
Límite:
Adolescent
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Child
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Child, preschool
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Female
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Humans
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Male
Idioma:
En
Revista:
Br J Haematol
Año:
2024
Tipo del documento:
Article
País de afiliación:
Austria