1. ¹Pediatric Hematology and Oncology, Center for Pediatric and Adolescent Medicine, University of Freiburg, Freiburg, Germany
2. ²Pediatric Hematology-Oncology, University of Pavia, IRCCS Policlinico San Matteo, Pavia, Italy
3. ³Pediatric Hematology and Oncology, Center of Pediatric and Adolescent Medicine, University of Frankfurt am Main, Frankfurt, Germany
4. ⁴Department of Pediatric Hematology and Oncology, University of Münster, Münster, Germany
5. ⁵Department of Pediatric Hematology-Oncology and Endocrinology, University of Essen, Essen, Germany
6. ⁶Department for Hematology and Oncology, Hospital for Pediatric and Adolescent Medicine, Friedrich-Schiller-University, Jena, Germany
7. ⁷Pediatric Hematology and Oncology, Dr von Haunersches Childrens Hospital, Ludwig-Maximilians University, Munich, Germany
8. ⁸Hospital for Pediatric and Adolescent Medicine, University of Erlangen, Erlangen, Germany
9. ⁹Pediatric Hematology and Oncology, Medical School Hannover, Hannover, Germany
10. ¹⁰Department of Pediatric Hematology and Oncology, University Hospital Motol, Prague, Czech Republic
11. ¹¹Institute of Pathology, University of Erlangen, Erlangen, Germany

Correspondence: Dr B Strahm, Pediatric Hematology and Oncology, Center for Pediatric and Adolescent Medicine, University of Freiburg, Mathildenstrasse 1, 79102 Freiburg, Germany. E-mail: brigitte.strahm@uniklinik-freiburg.de

Received 20 November 2006; Revised 5 April 2007; Accepted 23 April 2007; Published online 25 June 2007.

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.