title: A pilot study of reduced toxicity conditioning with BU, fludarabine and alemtuzumab before the allogeneic hematopoietic SCT in children and adolescents.
abstract: We report the results of a pilot study of a BU-fludarabine-alemtuzumab (BFA)-reduced toxicity conditioning (RTC) followed by allogeneic hematopoietic SCT (AlloHSCT) in 12 children and adolescents (<21 years) with malignant and non-malignant diseases. Stem cell sources were: two unrelated cord blood, one unrelated BM, two related and seven unrelated PBSC. Positive CD34 selection was performed in five unrelated PBSC grafts. RCT was carried out with BFA, and GVHD prophylaxis was FK506 and mycophenolate mofetil. The median time for neutrophil and platelet engraftment was 16 and 31 days, respectively. The P of developing ≥ grade II, ≥ grade III aGVHD and cGVHD was 41.6, 25 and 9%, respectively. Only 1 out of 12 developed ≥ grade III toxicity. There was one primary and no secondary graft failure. Mixed donor chimerism on day 100 and 1 year was median 99 and 96%, respectively; ≥ 90% of recipients achieved ≥ 80% donor chimerism. The 3-year overall survival (OS) in all patients was 91.7 ± 8% (100% for malignant vs. 80% for non-malignant diseases, ns). In all, 11 (91%) patients remain alive at median 2.8 (0.3-6.8) years. RTC followed by AlloHSCT, based on BFA conditioning, is feasible and tolerable in children and adolescents, and results in prompt achievement of durable mixed donor chimerism and excellent OS.
authors.affiliation: Department of Pediatric Hematology and Oncology, Collegium Medicum UMK, Bydgoszcz, Poland.
authors.forename: J
authors.initials: J
authors.lastname: Styczynski
authors.forename: B
authors.initials: B
authors.lastname: Tallamy
authors.forename: I
authors.initials: I
authors.lastname: Waxman
authors.forename: C
authors.initials: C
authors.lastname: van de Ven
authors.forename: M C
authors.initials: MC
authors.lastname: Milone
authors.forename: L M
authors.initials: LM
authors.lastname: Shaw
authors.forename: L
authors.initials: L
authors.lastname: Harrison
authors.forename: E
authors.initials: E
authors.lastname: Morris
authors.forename: P
authors.initials: P
authors.lastname: Satwani
authors.forename: M
authors.initials: M
authors.lastname: Bhatia
authors.forename: D
authors.initials: D
authors.lastname: George
authors.forename: M B
authors.initials: MB
authors.lastname: Bradley
authors.forename: J H
authors.initials: JH
authors.lastname: Garvin
authors.forename: J
authors.initials: J
authors.lastname: Schwartz
authors.forename: L A
authors.initials: LA
authors.lastname: Baxter-Lowe
authors.forename: M S
authors.initials: MS
authors.lastname: Cairo
mesh_terms: D000293:Adolescent; D000074323:Alemtuzumab; D000911:Antibodies, Monoclonal; D061067:Antibodies, Monoclonal, Humanized; D000912:Antibodies, Neoplasm; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000627:therapeutic use*; D002066:Busulfan; D002648:Child; D002675:Child, Preschool; D005260:Female; D006085:Graft Survival; D006086:Graft vs Host Disease / Q000145:classification; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects / Q000379:methods* / Q000401:mortality; D006801:Humans; D008297:Male; D010865:Pilot Projects; D016019:Survival Analysis; D018183:Transplantation Chimera; D019172:Transplantation Conditioning / Q000009:adverse effects / Q000379:methods*; D014184:Transplantation, Homologous; D014740:Vidarabine / Q000031:analogs & derivatives; D055815:Young Adult
chemical_list: D000911:Antibodies, Monoclonal; D061067:Antibodies, Monoclonal, Humanized; D000912:Antibodies, Neoplasm; D000074323:Alemtuzumab; D014740:Vidarabine; D002066:Busulfan; C024352:fludarabine