title: Reduced intensity conditioning in unrelated donor transplantation for refractory cytopenia in childhood.
abstract: Myelodysplastic syndromes (MDS) are a heterogenous group of acquired hematopoietic stem cell disorders. Refractory cytopenia (RC) is the most common subtype of childhood MDS and hematopoietic stem cell transplantation (HSCT) is the only curative treatment. HSCT following a myeloablative preparative regimen is associated with a low probability of relapse and considerable transplant-related mortality. In the present European Working Groups of MDS pilot study, we investigated whether a reduced intensity conditioning regimen (RIC) is able to offer reduced toxicity without increased rates of graft failure or relapse. Nineteen children with RC were transplanted from an unrelated donor following RIC consisting of fludarabine, thiotepa and anti-thymocyte globulin. Three patients experienced graft failure. Neutrophil and platelet engraftment occurred at a median time of 23 and 30 days, respectively. Cumulative incidence of grade II-IV and grade III and IV acute graft-versus-host disease (GVHD) was 0.48 and 0.13, respectively; three patients developed extensive chronic GVHD. Although infections were the predominant complications, only one patient with extensive chronic GVHD died from infectious complications. Overall and event-free survival at 3 years were 0.84 and 0.74, respectively. In conclusion, our results were comparable to those of patients treated with myeloablative HSCT. Long-term follow-up is needed to demonstrate the expected reduction in long-term sequelae.
authors.affiliation: Pediatric Hematology and Oncology, Center for Pediatric and Adolescent Medicine, University of Freiburg, Mathildenstrasse 1, 79102 Freiburg, Germany. brigitte.strahm@uniklinik-freiburg.de
authors.forename: B
authors.initials: B
authors.lastname: Strahm
authors.forename: F
authors.initials: F
authors.lastname: Locatelli
authors.forename: P
authors.initials: P
authors.lastname: Bader
authors.forename: K
authors.initials: K
authors.lastname: Ehlert
authors.forename: B
authors.initials: B
authors.lastname: Kremens
authors.forename: F
authors.initials: F
authors.lastname: Zintl
authors.forename: M
authors.initials: M
authors.lastname: Führer
authors.forename: D
authors.initials: D
authors.lastname: Stachel
authors.forename: K-W
authors.initials: KW
authors.lastname: Sykora
authors.forename: P
authors.initials: P
authors.lastname: Sedlacek
authors.forename: I
authors.initials: I
authors.lastname: Baumann
authors.forename: C M
authors.initials: CM
authors.lastname: Niemeyer
mesh_terms: D000293:Adolescent; D000753:Anemia, Refractory / Q000628:therapy*; D002648:Child; D002675:Child, Preschool; D005260:Female; D006085:Graft Survival; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods*; D006801:Humans; D007223:Infant; D053208:Kaplan-Meier Estimate; D008297:Male; D010865:Pilot Projects; D019172:Transplantation Conditioning / Q000379:methods*; D014184:Transplantation, Homologous