title: 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.
abstract: 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%.
authors.affiliation: Department of Paediatric Haematology and Oncology, Hannover Medical School, Hannover, Germany.
authors.forename: B
authors.initials: B
authors.lastname: Meissner
authors.forename: A
authors.initials: A
authors.lastname: Borkhardt
authors.forename: D
authors.initials: D
authors.lastname: Dilloo
authors.forename: D
authors.initials: D
authors.lastname: Fuchs
authors.forename: W
authors.initials: W
authors.lastname: Friedrich
authors.forename: R
authors.initials: R
authors.lastname: Handgretinger
authors.forename: C
authors.initials: C
authors.lastname: Peters
authors.forename: A
authors.initials: A
authors.lastname: Schrauder
authors.forename: F R
authors.initials: FR
authors.lastname: Schuster
authors.forename: J
authors.initials: J
authors.lastname: Vormoor
authors.forename: B
authors.initials: B
authors.lastname: Maecker
authors.forename: K W
authors.initials: KW
authors.lastname: Sykora
authors.forename: F
authors.initials: F
authors.lastname: Zintl
authors.forename: K
authors.initials: K
authors.lastname: Welte
authors.forename: M
authors.initials: M
authors.lastname: Sauer
mesh_terms: D000293:Adolescent; D002648:Child; D002675:Child, Preschool; D004314:Down Syndrome / Q000150:complications*; D005260:Female; D006086:Graft vs Host Disease / Q000517:prevention & control; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects*; D006801:Humans; D015470:Leukemia, Myeloid, Acute / Q000628:therapy*; D008297:Male; D054198:Precursor Cell Lymphoblastic Leukemia-Lymphoma / Q000628:therapy*; D012008:Recurrence; D012189:Retrospective Studies; D019172:Transplantation Conditioning / Q000009:adverse effects; D017211:Treatment Failure