JavaScript is NOT enabled !

Please enable JavaScript.

Article

PubMed

This record comes from PubMed

Treosulfan-based conditioning regimens for allogeneic haematopoietic stem cell transplantation in children with non-malignant diseases

Slatter, M A
Author Slatter, M A Paediatric Immunology Department, Paediatric HSCT, Great North Children's Hospital, Newcastle upon Tyne, UK
Boztug, H
Author Boztug, H Department of Paediatrics, St. Anna Kinderspital and Children's Cancer Research Institute, Medical University of Vienna, Vienna, Austria
Pötschger, U
Author Pötschger, U Department of Paediatrics, St. Anna Kinderspital and Children's Cancer Research Institute, Medical University of Vienna, Vienna, Austria
Sykora, K-W
Author Sykora, K-W Department of Paediatric Hematology/Oncology, Hannover Medical School, Hannover, Germany
Lankester, A
Author Lankester, A Department of Paediatrics, Leiden University Medical Center, RC Leiden, The Netherlands
Yaniv, I
Author Yaniv, I Sackler Faculty of Medicine, Paediatric Hematology Oncology Schneider Children's Medical Center of Israel, Tel Aviv University, Tel Aviv, Israel
Sedlacek, P
Author Sedlacek, P Department of Paediatric Hematology and Oncology Teaching Hospital Motol, 2nd Medical School, Charles University Motol Prague, Motol Prague, Czech Republic
Glogova, E
Author Glogova, E Department of Paediatrics, St. Anna Kinderspital and Children's Cancer Research Institute, Medical University of Vienna, Vienna, Austria
Veys, P
Author Veys, P Paediatric HSCT, Great Ormond Street Hospital for Children National Health Service Trust, London, UK
Gennery, A R
Author Gennery, A R Paediatric Immunology Department, Paediatric HSCT, Great North Children's Hospital, Newcastle upon Tyne, UK
Peters, C
Author Peters, C ORCID Department of Paediatrics, St. Anna Kinderspital and Children's Cancer Research Institute, Medical University of Vienna, Vienna, Austria
EBMT Inborn Errors and Paediatric Diseases Working Parties
Author EBMT Inborn Errors and Paediatric Diseases Working Parties

Bone marrow transplantation. 2015 Dec ; 50 (12) : 1536-41. [epub] 20150810

Bone Marrow Transplant
ISSN 1476-5365 | 0268-3369
Source

Language English Country England, Great Britain Media print-electronic

Document type Journal Article, Multicenter Study

Persistent link https://www.medvik.cz/link/pmid26259076

PubMed 26259076
DOI 10.1038/bmt.2015.171
PII: bmt2015171
Knihovny.cz E-resources

MeSH
Allografts MeSH
Busulfan administration & dosage adverse effects analogs & derivatives MeSH
Child MeSH
Infant MeSH
Humans MeSH
Survival Rate MeSH
Adolescent MeSH
Child, Preschool MeSH
Disease-Free Survival MeSH
Transplantation Conditioning methods MeSH
Retrospective Studies MeSH
Risk Factors MeSH
Stem Cell Transplantation * MeSH
Age Factors MeSH
Check Tag
Child MeSH
Infant MeSH
Humans MeSH
Adolescent MeSH
Male MeSH
Child, Preschool MeSH
Female MeSH
Publication type
Journal Article MeSH
Multicenter Study MeSH
Names of Substances
Busulfan MeSH
treosulfan MeSH Browser

An increasing number of children with non-malignant diseases can be cured by allogeneic haematopoietic stem cell transplantation (HSCT). Treosulfan (L-treitol-1,4-bis-methanesulfonate) is being used more frequently for conditioning, owing to its' lower toxicity profile compared with conventional myeloablative regimens. A retrospective analysis was performed of children registered in the EBMT database, who received treosulfan before HSCT between January 2005 and 2010, to identify possible dose-related toxicity and determine the incidence of engraftment, treatment-related mortality and overall survival (OS). Results from 316 transplants from 11 different countries are presented. Ninety-five (30%) were under 1 year of age at the time of transplant. OS was 83% and event-free survival was 76%; 3-year OS and event-free survival of infants below 1 year were 79% and 73%, respectively. No association was found with age at transplant, dose of treosulfan given, other agents used in combination with treosulfan, donor type, stem cell source, or second or subsequent transplant. In this report of the largest number of children to date receiving treosulfan for non-malignant diseases, treosulfan is shown to be a safe and effective agent even for those under 1 year of age at the time of transplant. Further prospective studies are needed using precisely defined protocols with pharmacokinetic monitoring and detailed chimerism analysis. In addition, long-term studies will be vital to determine long-term effects, for example, on fertility in comparison with other regimens.

Department of Paediatric Hematology and Oncology Teaching Hospital Motol 2nd Medical School Charles University Motol Prague Motol Prague Czech Republic

Department of Paediatric Hematology Oncology Hannover Medical School Hannover Germany

Department of Paediatrics Leiden University Medical Center RC Leiden The Netherlands

Department of Paediatrics St Anna Kinderspital and Children's Cancer Research Institute Medical University of Vienna Vienna Austria

Paediatric HSCT Great Ormond Street Hospital for Children National Health Service Trust London UK

Paediatric Immunology Department Paediatric HSCT Great North Children's Hospital Newcastle upon Tyne UK

Sackler Faculty of Medicine Paediatric Hematology Oncology Schneider Children's Medical Center of Israel Tel Aviv University Tel Aviv Israel

See more in PubMed

Blood. 2004 Jan 15;103(2):725-31 PubMed

Drug Discov Today. 2014 Oct;19(10):1572-86 PubMed

Bone Marrow Transplant. 2001 Dec;28(11):1001-11 PubMed

Bone Marrow Transplant. 2001 Nov;28(10):909-15 PubMed