title: Fludarabine-based disease-specific conditioning or conventional myeloablative conditioning in hematopoietic stem cell transplantation for treatment of non-malignant diseases.
abstract: Fludarabine-based conditioning (FBC) was given to 24 patients and conventional myeloablative conditioning (MC) to 33 patients, most children, before hematopoietic stem cell transplantation (HSCT) for non-malignant diseases. The donors were human leukocyte antigen (HLA)-A, -B, -DRbeta1-identical related (33%) or unrelated (67%). In the FBC group, two grafts failed versus three in the MC group; all were successfully regrafted. Fever was more common in the MC patients (P=0.003). Bacteremia occurred in 25% of the FBC group and 50% in the MC group (P=0.1). In the FBC group, platelet engraftment was faster and transfusions were fewer (P<0.05). Mucositis and renal function were similar in the two groups. The MC group had higher maximum bilirubin (P=0.03) and less often normal spirometry (P=0.04) after HSCT. A 7-year-old girl in the MC group had permanent alopecia. No patients had severe acute graft-versus-host disease (GVHD). Chronic GVHD was rare. Complete donor CD3+ chimerism was more common in the MC group (P=0.01), but CD33+ engraftment was better with FBS (P=0.03). Treatment-related mortality was 4 and 15%, and 5-year survival was 89 and 85% in the FBC and MC groups. Although survival was similar, FBC is a promising alternative to MC in non-malignant disorders.
authors.affiliation: Division of Clinical Immunology, Karolinska Institutet, Karolinska University Hospital, Huddinge, Stockholm, Sweden. Olle.Ringden@ki.se
authors.forename: O
authors.initials: O
authors.lastname: Ringdén
authors.forename: M
authors.initials: M
authors.lastname: Remberger
authors.forename: P
authors.initials: P
authors.lastname: Svenberg
authors.forename: B-M
authors.initials: BM
authors.lastname: Svahn
authors.forename: G
authors.initials: G
authors.lastname: Dahllöf
authors.forename: B
authors.initials: B
authors.lastname: Gustafsson
authors.forename: Z
authors.initials: Z
authors.lastname: Hassan
authors.forename: B
authors.initials: B
authors.lastname: Omazic
authors.forename: M
authors.initials: M
authors.lastname: Uzunel
authors.forename: J
authors.initials: J
authors.lastname: Aschan
authors.forename: L
authors.initials: L
authors.lastname: Barkholt
authors.forename: J
authors.initials: J
authors.lastname: Winiarski
authors.forename: P
authors.initials: P
authors.lastname: Ljungman
authors.forename: J
authors.initials: J
authors.lastname: Mattsson
mesh_terms: D000293:Adolescent; D000328:Adult; D000741:Anemia, Aplastic / Q000628:therapy*; D000970:Antineoplastic Agents / Q000494:pharmacology*; D002648:Child; D002675:Child, Preschool; D005260:Female; D006086:Graft vs Host Disease; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods*; D006453:Hemoglobinopathies / Q000628:therapy*; D006801:Humans; D007153:Immunologic Deficiency Syndromes / Q000628:therapy*; D007223:Infant; D008297:Male; D008875:Middle Aged; D019172:Transplantation Conditioning; D014740:Vidarabine / Q000031:analogs & derivatives* / Q000494:pharmacology
chemical_list: D000970:Antineoplastic Agents; D014740:Vidarabine; C024352:fludarabine