1. ¹Department of Paediatric Haematology and Oncology, Hannover Medical School, Hannover, Germany
2. ²Department of Paediatric Haematology and Oncology, Dr von Haunersches Kinderspital, University of Munich, Munich, Germany
3. ³Clinic of Paediatric Haematology, Oncology and Clinical Immunology, HHU Duesseldorf, Duesseldorf, Germany
4. ⁴Department of Paediatric Haematology and Oncology, Friedrich-Schiller-University Jena, Jena, Germany
5. ⁵Department of Paediatrics, University of Ulm, Ulm, Germany
6. ⁶Department of Paediatric Haematology and Oncology, University Children's Hospital, Eberhard Karls University, Tuebingen, Germany
7. ⁷Department of Paediatric Haematology and Oncology, St Anna Children's Hospital, Vienna, Austria
8. ⁸Department of Paediatrics, University Medical Center Schleswig-Holstein, Campus Kiel, Kiel, Germany
9. ⁹Department of Paediatric Haematology and Oncology, University Children's Hospital, Muenster, Germany

Correspondence: Dr M Sauer, Department of Paediatric Haematology and Oncology Children's Hospital–OE 6780, Hannover Medical University, Carl-Neuberg-Strasse 1, Hannover 30625, Germany. E-mail: sauer.martin@mh-hannover.de

Received 7 March 2007; Revised 22 May 2007; Accepted 21 June 2007; Published online 3 September 2007.

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%.