title: Transplantation of highly purified peripheral-blood CD34+ progenitor cells from related and unrelated donors in children with nonmalignant diseases.
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 x 10(6) CD34(+) progenitor cells with a median of 6.1 x 10(3) 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.
authors.affiliation: Children's University Hospital, Tübingen, Germany.
authors.forename: P
authors.initials: P
authors.lastname: Lang
authors.forename: T
authors.initials: T
authors.lastname: Klingebiel
authors.forename: P
authors.initials: P
authors.lastname: Bader
authors.forename: J
authors.initials: J
authors.lastname: Greil
authors.forename: M
authors.initials: M
authors.lastname: Schumm
authors.forename: P-G
authors.initials: PG
authors.lastname: Schlegel
authors.forename: M
authors.initials: M
authors.lastname: Eyrich
authors.forename: S
authors.initials: S
authors.lastname: Mueller-Weihrich
authors.forename: D
authors.initials: D
authors.lastname: Niethammer
authors.forename: R
authors.initials: R
authors.lastname: Handgretinger
mesh_terms: D000293:Adolescent; D000740:Anemia / Q000401:mortality / Q000628:therapy*; D018952:Antigens, CD34; D001782:Blood Donors; D002648:Child; D002675:Child, Preschool; D006085:Graft Survival; D006086:Graft vs Host Disease; D006801:Humans; D007107:Immune System / Q000502:physiology; D007223:Infant; D008212:Lymphocyte Depletion; D036102:Peripheral Blood Stem Cell Transplantation / Q000379:methods* / Q000592:standards; D012038:Regeneration; D016019:Survival Analysis; D014184:Transplantation, Homologous
chemical_list: D018952:Antigens, CD34