1. ¹Children's University Hospital, Tübingen, Germany
2. ²Children's University Hospital, Frankfurt, Germany
3. ³Children's University Hospital, Wuerzburg, Germany
4. ⁴Children's University Hospital, TU Muenchen, Germany
5. ⁵St Jude Children's Research Hospital, Memphis, TN, USA

Correspondence: Dr R Handgretinger, St Jude Children's Research Hospital, 332 N. Lauderdale St., Memphis, TN 38105-2794, USA. E-mail: Rupert.Handgretinger@stjude.org

⁶PL and TK contributed equally.

Received 3 June 2003; Accepted 30 June 2003.

Abstract

Transplantation of allogeneic stem cells is currently the only curative treatment for some nonmalignant pediatric diseases. We investigated whether transplantation of purified CD34⁺ stem cells prevents acute and chronic GvHD and reduces transplant-related mortality. A total of 25 pediatric patients with nonmalignant diseases underwent allogeneic transplantation from 26 donors (matched related n=4, matched or partially matched unrelated n=14, mismatched related n=8). All grafts were purified peripheral-blood CD34⁺ stem cells mobilized with G-CSF. Patients received a median of 12.9 10⁶ CD34⁺ progenitor cells with a median of 6.1 10³ contaminating T-lymphocytes per kilogram of body weight. No post transplant immunosuppressive drugs were given for prophylaxis of GvHD. Engraftment was seen in 21 patients. Three patients engrafted after a second transplant and one patient failed to engraft. Two patients had autologous reconstitution 1.5 years post transplant and one of them was successfully retransplanted. No acute GvHD >grade II was seen, and only two patients developed limited, chronic GvHD. In all, 22 patients (88%) are alive with a median follow-up of 3.7 years. In total, 19 patients (76%) are free of disease or of progression. Transplantation of highly purified peripheral-blood CD34⁺ stem cells is associated with low toxicity in patients with nonmalignant diseases.