Treosulfan-based conditioning regimens for allogeneic HSCT in children with acute lymphoblastic leukaemia

Autor: Boztug, H., Zecca, M., Sykora, K.W., Veys, P., Lankester, A., Slatter, M., Skinner, R., Wachowiak, J., Potschger, U., Glogova, E., Peters, C., EBMT Paediat Dis Working Party
Jazyk: angličtina
Rok vydání: 2015
Předmět:
Diarrhea
Male
Oncology
medicine.medical_specialty
Transplantation Conditioning
Adolescent
Treosulfan
Vomiting
medicine.medical_treatment
Graft vs Host Disease
Hematopoietic stem cell transplantation
Disease-Free Survival
hemic and lymphatic diseases
Internal medicine
Antineoplastic Combined Chemotherapy Protocols
medicine
Humans
Transplantation
Homologous
Child
Busulfan
Retrospective Studies
Stomatitis
Dose-Response Relationship
Drug
Toxicity
business.industry
Remission Induction
Hematopoietic Stem Cell Transplantation
Stem cell transplantation
Infant
Retrospective cohort study
Paediatrics
Hematology
General Medicine
Precursor Cell Lymphoblastic Leukemia-Lymphoma
Total body irradiation
Surgery
Transplantation
Treatment Outcome
surgical procedures
operative
Child
Preschool
Multivariate Analysis
Female
business
ALL
medicine.drug
Zdroj: Annals of Hematology, 94(2), 297-306
Popis: Standard myeloablative conditioning regimens for children with acute lymphoblastic leukaemia are based on total body irradiation (TBI). However, TBI causes profound short-term and long-term side effects, provoking the necessity for alternative regimens. Treosulfan combines a potent immunosuppressive and antileukaemic effect with myeloablative activity and low toxicity profile. We retrospectively studied toxicity and outcome of 71 paediatric patients with acute lymphoblastic leukaemia (ALL) undergoing haematopoietic stem cell transplantation (HSCT) following treosulfan-based conditioning aiming to identify risk factors for treatment failure and dose-depending outcome differences. Early regimen-related toxicity was low. No case of veno-occlusive disease was reported. There was no association of toxicity with age or number of HSCT. Event-free survival (EFS) of infants was significantly better compared to older children. Overall survival (OS) at 3 years was 51 % and not significantly influenced by number of HSCT (first HSCT 54 %, ≥second HSCT 44 %, p = 0.71). In multivariate analysis, OS and EFS were significantly worse for patients transplanted without complete remission (p = 0.04 and 0.004). Treatment-related mortality was low at 14 %. We conclude that treosulfan-based conditioning is a safe and efficacious approach for paediatric ALL.
Databáze: OpenAIRE
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