title: Reduced-intensity conditioning therapy with busulfan, fludarabine, and antithymocyte globulin for HLA-haploidentical hematopoietic cell transplantation in acute leukemia and myelodysplastic syndrome.
abstract: Any role for reduced-intensity conditioning (RIC) before hematopoietic cell transplantation (HCT) from a human leukocyte antigen (HLA)-haploidentical donor remains to be defined. We therefore assessed 83 patients (age, 16-70 years): 68 with acute leukemia (including 34 in remission and 34 with refractory disease) and 15 patients with myelodysplastic syndrome, in HCT trials using RIC with busulfan, fludarabine, and antithymocyte globulin. The HLA-haploidentical donors, offspring (n = 38), mothers (n = 24), or siblings (n = 21) of patients, underwent leukapheresis after receiving granulocyte colony-stimulating factor, and donated cells were transplanted without further manipulation. Cyclosporine and methotrexate were given for GVHD prophylaxis. The cumulative incidences of neutrophil engraftment, grade 2 to 4 acute GVHD, chronic GVHD, and transplantation-related mortality after HCT, were 92%, 20%, 34%, and 18%, respectively. After a median follow-up time of 26.6 months (range, 16.8-78.8 months), the event-free and overall survival rates were 56% and 45%, respectively, for patients with acute leukemia in remission; 9% and 9%, respectively, for patients with refractory acute leukemia; and 53% and 53%, respectively, for patients with myelodysplastic syndrome. HCT from an HLA-haploidentical family member resulted in favorable outcomes when RIC containing antithymocyte globulin was performed. This study is registered at www.clinicaltrials.gov as #NCT00521430 and #NCT00732316.
authors.affiliation: Hematology Section, Department of Internal Medicine, University ofUlsan, College of Medicine, Asan Medical Center, Seoul, Korea. khlee2@amc.seoul.kr
authors.forename: Kyoo-Hyung
authors.initials: KH
authors.lastname: Lee
authors.forename: Je-Hwan
authors.initials: JH
authors.lastname: Lee
authors.forename: Jung-Hee
authors.initials: JH
authors.lastname: Lee
authors.forename: Dae-Young
authors.initials: DY
authors.lastname: Kim
authors.forename: Miee
authors.initials: M
authors.lastname: Seol
authors.forename: Young-Shin
authors.initials: YS
authors.lastname: Lee
authors.forename: Young-Ah
authors.initials: YA
authors.lastname: Kang
authors.forename: Mijin
authors.initials: M
authors.lastname: Jeon
authors.forename: Hyun-Ju
authors.initials: HJ
authors.lastname: Hwang
authors.forename: Ah-Rang
authors.initials: AR
authors.lastname: Jung
authors.forename: Sung-Han
authors.initials: SH
authors.lastname: Kim
authors.forename: Sung-Cheol
authors.initials: SC
authors.lastname: Yun
authors.forename: Ho-Jin
authors.initials: HJ
authors.lastname: Shin
mesh_terms: D000208:Acute Disease; D000293:Adolescent; D000328:Adult; D000368:Aged; D000961:Antilymphocyte Serum / Q000008:administration & dosage*; D002066:Busulfan / Q000008:administration & dosage*; D018572:Disease-Free Survival; D005260:Female; D006085:Graft Survival; D006086:Graft vs Host Disease / Q000517:prevention & control; D016179:Granulocyte Colony-Stimulating Factor / Q000494:pharmacology; D006680:HLA Antigens / Q000032:analysis*; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods*; D006648:Histocompatibility; D006801:Humans; D007239:Infections; D053208:Kaplan-Meier Estimate; D007938:Leukemia / Q000401:mortality / Q000601:surgery*; D008297:Male; D008875:Middle Aged; D009190:Myelodysplastic Syndromes / Q000401:mortality / Q000601:surgery*; D036102:Peripheral Blood Stem Cell Transplantation / Q000379:methods*; D012189:Retrospective Studies; D013601:T-Lymphocytes / Q000276:immunology; D014019:Tissue Donors; D019172:Transplantation Conditioning / Q000379:methods*; D016896:Treatment Outcome; D014740:Vidarabine / Q000008:administration & dosage / Q000031:analogs & derivatives*; D055815:Young Adult
chemical_list: D000961:Antilymphocyte Serum; D006680:HLA Antigens; D016179:Granulocyte Colony-Stimulating Factor; D014740:Vidarabine; D002066:Busulfan; C024352:fludarabine