Annals of Hematology

, Volume 94, Issue 2, pp 297–306 | Cite as

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

  • Heidrun Boztug
  • Marco Zecca
  • Karl-Walter Sykora
  • Paul Veys
  • Arjan Lankester
  • Mary Slatter
  • Roderick Skinner
  • Jacek Wachowiak
  • Ulrike Pötschger
  • Evgenia Glogova
  • Christina Peters
  • on behalf of the EBMT paediatric diseases working party
Original Article


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.


Treosulfan ALL Paediatrics Stem cell transplantation Toxicity 



We thank Arnaud Dalassier for data correction and management.

Conflict of interest

The EBMT received an unrestricted grant for the retrospective data collection and analysis from medac GmbH. CP received travel grants and study support from medac, Pierre Fabre, Sanofi and Novartis. HB received a travel grant from Pierre Fabre. KWS received travel grants and study support from medac.


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Copyright information

© Springer-Verlag Berlin Heidelberg 2014

Authors and Affiliations

  • Heidrun Boztug
    • 1
  • Marco Zecca
    • 2
  • Karl-Walter Sykora
    • 3
  • Paul Veys
    • 4
  • Arjan Lankester
    • 5
  • Mary Slatter
    • 6
  • Roderick Skinner
    • 7
  • Jacek Wachowiak
    • 8
  • Ulrike Pötschger
    • 1
  • Evgenia Glogova
    • 1
  • Christina Peters
    • 1
  • on behalf of the EBMT paediatric diseases working party
  1. 1.St. Anna Kinderspital and Children’s Cancer Research InstituteDepartment of Paediatrics, Medical University of ViennaViennaAustria
  2. 2.Paediatric Haematology/OncologyFondazione IRCCS, Policlinico San Matteo FoundationPaviaItaly
  3. 3.Department of Paediatric Hematology/OncologyHannover Medical SchoolHannoverGermany
  4. 4.Great Ormond Street Hospital for Children National Health Service TrustLondonUK
  5. 5.Department of PaediatricsLeiden University Medical CenterLeidenThe Netherlands
  6. 6.Paediatric Immunology DepartmentGreat North Children’s HospitalNewcastle upon TyneUK
  7. 7.Department of Paediatric and Adolescent Haematology and Oncology, and Children’s HSCT UnitGreat North Children’s Hospital, Royal Victoria InfirmaryNewcastle upon TyneUK
  8. 8.Department of Paediatric Haematology, Oncology, and Haematopoietic Stem Cell TransplantationUniversity of Medical SciencesPoznańPoland

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