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Relapse, not regimen-related toxicity, was the major cause of treatment failure in 11 children with Down syndrome undergoing haematopoietic stem cell transplantation for acute leukaemia

Lookup NU author(s): Professor Hermann Josef Vormoor


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We report a retrospective analysis of 11 children with Down syndrome (DS) treated by SCT in eight German/Austrian SCT centres. Indications for transplantation were acute lymphoblastic leukaemia (N=8) and acute myeloid leukaemia (N=3). A reduced intensity conditioning (RIC) containing 2 Gy TBI was given to two patients, another five received a myeloablative regimen with 12 Gy TBI. Treosulphan or busulphan was used in the remaining four children. Four of eleven (36%) patients are alive. All of them were treated with a myeloablative regimen. One of the four surviving children relapsed 9 months after SCT and is currently receiving palliative outpatient treatment. The main cause of death was relapse (5/11). Two children died of regimen-related toxicity (RRT), one from severe exfoliative dermatitis and multiorgan failure after a treosulphan-containing regimen, the other from GvHD-related infections after RIC. Acute GvHD of the skin was observed in 10 of 10 evaluable patients, and chronic GvHD in 4 of 8. Our data show that DS patients can tolerate commonly used, fully myeloablative preparative regimens. The major cause of death is relapse rather than RRT resulting in an event-free survival of 18% and over all survival of 36%.Bone Marrow Transplantation advance online publication, 3 September 2007; doi:10.1038/sj.bmt.1705844.

Publication metadata

Author(s): Meissner B, Borkhardt A, Dilloo D, Fuchs D, Friedrich W, Handgretinger R, Peters C, Schrauder A, Schuster FR, Vormoor J, Maecker B, Sykora KW, Zintl F, Welte K, Sauer M

Publication type: Article

Publication status: Published

Journal: Bone Marrow Transplant

Year: 2007

Volume: 40

Issue: 10

Pages: 945-949

ISSN (print): 0268-3369

ISSN (electronic): 1476-5365


DOI: 10.1038/sj.bmt.1705844

Notes: Journal article


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