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

Research output: Contribution to journalArticle

22 Citations (Scopus)

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 %, ≥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.

Original languageEnglish
Pages (from-to)297-306
Number of pages10
JournalAnnals of Hematology
Volume94
Issue number2
DOIs
Publication statusPublished - Jan 13 2015

Fingerprint

treosulfan
Hematopoietic Stem Cell Transplantation
Precursor Cell Lymphoblastic Leukemia-Lymphoma
Whole-Body Irradiation
Disease-Free Survival
Pediatrics
Survival
Immunosuppressive Agents
Treatment Failure
Multivariate Analysis

Keywords

  • ALL
  • Paediatrics
  • Stem cell transplantation
  • Toxicity
  • Treosulfan

ASJC Scopus subject areas

  • Hematology
  • Medicine(all)

Cite this

Treosulfan-based conditioning regimens for allogeneic HSCT in children with acute lymphoblastic leukaemia. / Boztug, Heidrun; Zecca, Marco; Sykora, Karl Walter; Veys, Paul; Lankester, Arjan; Slatter, Mary; Skinner, Roderick; Wachowiak, Jacek; Pötschger, Ulrike; Glogova, Evgenia; Peters, Christina.

In: Annals of Hematology, Vol. 94, No. 2, 13.01.2015, p. 297-306.

Research output: Contribution to journalArticle

Boztug, H, Zecca, M, Sykora, KW, Veys, P, Lankester, A, Slatter, M, Skinner, R, Wachowiak, J, Pötschger, U, Glogova, E & Peters, C 2015, 'Treosulfan-based conditioning regimens for allogeneic HSCT in children with acute lymphoblastic leukaemia', Annals of Hematology, vol. 94, no. 2, pp. 297-306. https://doi.org/10.1007/s00277-014-2196-8
Boztug, Heidrun ; Zecca, Marco ; Sykora, Karl Walter ; Veys, Paul ; Lankester, Arjan ; Slatter, Mary ; Skinner, Roderick ; Wachowiak, Jacek ; Pötschger, Ulrike ; Glogova, Evgenia ; Peters, Christina. / Treosulfan-based conditioning regimens for allogeneic HSCT in children with acute lymphoblastic leukaemia. In: Annals of Hematology. 2015 ; Vol. 94, No. 2. pp. 297-306.
@article{10fdf6d445e5474b981956419658620d,
title = "Treosulfan-based conditioning regimens for allogeneic HSCT in children with acute lymphoblastic leukaemia",
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 {\%}, ≥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.",
keywords = "ALL, Paediatrics, Stem cell transplantation, Toxicity, Treosulfan",
author = "Heidrun Boztug and Marco Zecca and Sykora, {Karl Walter} and Paul Veys and Arjan Lankester and Mary Slatter and Roderick Skinner and Jacek Wachowiak and Ulrike P{\"o}tschger and Evgenia Glogova and Christina Peters",
year = "2015",
month = "1",
day = "13",
doi = "10.1007/s00277-014-2196-8",
language = "English",
volume = "94",
pages = "297--306",
journal = "Annals of Hematology",
issn = "0939-5555",
publisher = "Springer Verlag",
number = "2",

}

TY - JOUR

T1 - Treosulfan-based conditioning regimens for allogeneic HSCT in children with acute lymphoblastic leukaemia

AU - Boztug, Heidrun

AU - Zecca, Marco

AU - Sykora, Karl Walter

AU - Veys, Paul

AU - Lankester, Arjan

AU - Slatter, Mary

AU - Skinner, Roderick

AU - Wachowiak, Jacek

AU - Pötschger, Ulrike

AU - Glogova, Evgenia

AU - Peters, Christina

PY - 2015/1/13

Y1 - 2015/1/13

N2 - 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.

AB - 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.

KW - ALL

KW - Paediatrics

KW - Stem cell transplantation

KW - Toxicity

KW - Treosulfan

UR - http://www.scopus.com/inward/record.url?scp=84926614762&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=84926614762&partnerID=8YFLogxK

U2 - 10.1007/s00277-014-2196-8

DO - 10.1007/s00277-014-2196-8

M3 - Article

C2 - 25231927

AN - SCOPUS:84926614762

VL - 94

SP - 297

EP - 306

JO - Annals of Hematology

JF - Annals of Hematology

SN - 0939-5555

IS - 2

ER -