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.
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.
Bader
Peter
P
Department of Hematology/Oncology, University Children's Hospital, D-60590 Frankfurt, Germany. peter.bader@kgu.de
Niemeyer
Charlotte
C
Willasch
Andre
A
Kreyenberg
Hermann
H
Strahm
Brigitte
B
Kremens
Bernhard
B
Gruhn
Bernd
B
Dilloo
Dagmar
D
Vormoor
Josef
J
Lang
Peter
P
Niethammer
Dietrich
D
Klingebiel
Thomas
T
Beck
James F
JF
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