title: Children with myelodysplastic syndrome (MDS) and increasing mixed chimaerism after allogeneic stem cell transplantation have a poor outcome which can be improved by pre-emptive immunotherapy.
abstract: We recently reported that virtually all children with acute leukaemia and myelodysplastic syndrome (MDS) who develop the phenotype of increasing mixed chimaerism (MC) after allogeneic stem cell transplantation (allo-SCT) will relapse. We therefore performed a prospective, multi-centre study focused on children with MDS (n = 65; advanced MDS = 44, refractory cytopenia = 21) after allo-SCT in order to determine to what extent relapse can be prevented by pre-emptive immunotherapy on the basis of increasing MC. Analyses of chimaerism in 44 patients with advanced MDS revealed 31 cases with complete chimaerism (CC)/low-level MC/transient MC, 11 cases with increasing MC and two cases with decreasing MC. The same analyses in 21 MDS patients with refractory cytopenia revealed 17 cases with CC/low-level MC, one case with increasing MC and three cases with decreasing MC. Pre-emptive immunotherapy performed on each patient that showed increasing MC improved event-free survival from 0%, as seen in prior studies, to 50%. We therefore conclude that pre-emptive immunotherapy is an effective treatment option to prevent impending relapse in children with MDS after allo-SCT.
authors.affiliation: Department of Hematology/Oncology, University Children's Hospital, D-60590 Frankfurt, Germany. peter.bader@kgu.de
authors.forename: Peter
authors.initials: P
authors.lastname: Bader
authors.forename: Charlotte
authors.initials: C
authors.lastname: Niemeyer
authors.forename: Andre
authors.initials: A
authors.lastname: Willasch
authors.forename: Hermann
authors.initials: H
authors.lastname: Kreyenberg
authors.forename: Brigitte
authors.initials: B
authors.lastname: Strahm
authors.forename: Bernhard
authors.initials: B
authors.lastname: Kremens
authors.forename: Bernd
authors.initials: B
authors.lastname: Gruhn
authors.forename: Dagmar
authors.initials: D
authors.lastname: Dilloo
authors.forename: Josef
authors.initials: J
authors.lastname: Vormoor
authors.forename: Peter
authors.initials: P
authors.lastname: Lang
authors.forename: Dietrich
authors.initials: D
authors.lastname: Niethammer
authors.forename: Thomas
authors.initials: T
authors.lastname: Klingebiel
authors.forename: James F
authors.initials: JF
authors.lastname: Beck
mesh_terms: D000208:Acute Disease; D000293:Adolescent; D000328:Adult; D000754:Anemia, Refractory, with Excess of Blasts / Q000401:mortality / Q000601:surgery / Q000628:therapy; D001804:Blood Transfusion, Autologous; D002648:Child; D002675:Child, Preschool; D018572:Disease-Free Survival; D005500:Follow-Up Studies; D006801:Humans; D007223:Infant; D007938:Leukemia / Q000401:mortality / Q000601:surgery* / Q000628:therapy; D007951:Leukemia, Myeloid / Q000401:mortality / Q000601:surgery / Q000628:therapy; D017710:Lymphocyte Transfusion / Q000379:methods*; D009190:Myelodysplastic Syndromes / Q000401:mortality / Q000601:surgery* / Q000628:therapy; D011446:Prospective Studies; D033581:Stem Cell Transplantation; D018183:Transplantation Chimera