Toggle Main Menu Toggle Search

Open Access padlockePrints

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

Lookup NU author(s): Professor Roderick Skinner

Downloads


Licence

This work is licensed under a Creative Commons Attribution-NonCommercial 4.0 International License (CC BY-NC 4.0).


Abstract

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 %, a parts per thousand yensecond 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.


Publication metadata

Author(s): Boztug H, Zecca M, Sykora KW, Veys P, Lankester A, Slatter M, Skinner R, Wachowiak J, Potschger U, Glogova E, Peters C, EBMT Paediat Dis Working Party

Publication type: Article

Publication status: Published

Journal: Annals of Hematology

Year: 2015

Volume: 94

Issue: 2

Pages: 297-306

Print publication date: 01/02/2015

Online publication date: 19/09/2014

Acceptance date: 19/08/2014

Date deposited: 23/12/2015

ISSN (print): 0939-5555

ISSN (electronic): 1432-0584

Publisher: Springer

URL: http://dx.doi.org/10.1007/s00277-014-2196-8

DOI: 10.1007/s00277-014-2196-8


Altmetrics

Altmetrics provided by Altmetric


Share