title: Engraftment of heavily transfused patients with severe aplastic anemia with a fludarabine-based regimen.
abstract: We have developed a practical conditioning regimen without anti-thymocyte globulin (ATG), irradiation, or other myeloablative alkylating agent for low-income countries in which patients with severe aplastic anemia (SAA), who usually have heavily transfused and a prolonged disease history. The application of ATG, Busulphan, and/or irradiation to cyclophosphamide (Cy) to avoid graft rejection has many short- and long-term complications. In this study, we focused on evaluating a fludarabine-based conditioning regimen, among 83 patients with SAA. Patients were treated with fludarabine (40 mg/m(2) /d; day [-5 to -2]) and cyclophosphamide (50 mg/kg/d; day [-5 to -2]). Altogether, 81 patients indicated initial engraftment, whereas two cases showed primary graft failure. And four of the 81 cases indicated graft rejection during follow-up. Regardless of a high cumulative incidence of acute (55/83; 66.2% grade II-IV; 47/83; 56.6% III-IV) and chronic graft-versus-host disease (50/83; 60.2%), in total, 77 patients showed durable engraftment and transfusion independence, and 64 are alive at a median time of 49 months with an overall survival rate of 66%. In conclusion, this conditioning indicated well toleration, mild toxicity, durable engraftment, excellent survival as well as less cost. Its application might shed new light on SAA at high risk of graft rejection in resource-limited countries.
authors: Department of Hematology, Kunming General Hospital of Chengdu Military Command, Kunming, China. wangsanbinlili@163.net
authors: San-Bin
authors: SB
authors: Wang
authors: Li
authors: L
authors: Li
authors: Xin-Hua
authors: XH
authors: Pan
authors: Deng-Ming
authors: DM
authors: Hu
authors: Li-Hui
authors: LH
authors: Peng
authors: Lin
authors: L
authors: Liu
authors: Zheng-Jun
authors: ZJ
authors: Xie
authors: Bo
authors: B
authors: Yin
authors: Xiao-Juan
authors: XJ
authors: Sun
authors: Jing
authors: J
authors: Yu
authors: Yang
authors: Y
authors: Liang
mesh_terms: D000293:Adolescent; D000328:Adult; D000741:Anemia, Aplastic / Q000401:mortality / Q000601:surgery* / Q000628:therapy; D002648:Child; D002675:Child, Preschool; D003520:Cyclophosphamide / Q000627:therapeutic use*; D004334:Drug Administration Schedule; D004359:Drug Therapy, Combination; D005260:Female; D005500:Follow-Up Studies; D006084:Graft Rejection / Q000517:prevention & control; D006086:Graft vs Host Disease / Q000517:prevention & control; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D053208:Kaplan-Meier Estimate; D008297:Male; D019653:Myeloablative Agonists / Q000627:therapeutic use*; D015996:Survival Rate; D019172:Transplantation Conditioning / Q000379:methods*; D016896:Treatment Outcome; D014740:Vidarabine / Q000031:analogs & derivatives* / Q000627:therapeutic use; D055815:Young Adult
chemical_list: D019653:Myeloablative Agonists; D003520:Cyclophosphamide; D014740:Vidarabine; C024352:fludarabine