title: High-dose cyclophosphamide for severe aplastic anemia: long-term follow-up.
abstract: Severe aplastic anemia (SAA) is a life-threatening bone marrow failure disorder that can be treated with bone marrow transplantation, immunosuppressive therapy, and high-dose cyclophosphamide. Here, we report long-term follow-up on 67 SAA patients (44 treatment-naive and 23 refractory) treated with high-dose cyclophosphamide. At 10 years, the overall actuarial survival was 88%, the response rate was 71% with the majority being complete, and the actuarial event-free survival was 58% in 44 treatment-naive SAA patients. Patients with refractory SAA fared less well after high-dose cyclophosphamide therapy; at 10 years, overall actuarial survival, response, and actuarial event-free survival rates were 62%, 48%, and 27%, respectively. High-dose cyclophosphamide is highly effective therapy for severe aplastic anemia. Large randomized controlled trials will be necessary to establish how results of high-dose cyclophosphamide compare with either bone marrow transplantation or standard immunosuppressive regimens, such as antithymocyte globulin and cyclosporine.
authors.affiliation: Division of Hematology,Department of Medicine, Johns Hopkins University School ofMedicine, 720 Rutland Ave, Ross Bldg, Rm 1025, Baltimore, MD 21205, USA. brodsro@jhmi.edu
authors.forename: Robert A
authors.initials: RA
authors.lastname: Brodsky
authors.forename: Allen R
authors.initials: AR
authors.lastname: Chen
authors.forename: Donna
authors.initials: D
authors.lastname: Dorr
authors.forename: Ephraim J
authors.initials: EJ
authors.lastname: Fuchs
authors.forename: Carol Ann
authors.initials: CA
authors.lastname: Huff
authors.forename: Leo
authors.initials: L
authors.lastname: Luznik
authors.forename: B Douglas
authors.initials: BD
authors.lastname: Smith
authors.forename: William H
authors.initials: WH
authors.lastname: Matsui
authors.forename: Steven N
authors.initials: SN
authors.lastname: Goodman
authors.forename: Richard F
authors.initials: RF
authors.lastname: Ambinder
authors.forename: Richard J
authors.initials: RJ
authors.lastname: Jones
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D000741:Anemia, Aplastic / Q000188:drug therapy* / Q000503:physiopathology; D002423:Cause of Death; D002648:Child; D002675:Child, Preschool; D003520:Cyclophosphamide / Q000008:administration & dosage* / Q000009:adverse effects / Q000494:pharmacology / Q000627:therapeutic use*; D004305:Dose-Response Relationship, Drug; D004351:Drug Resistance / Q000187:drug effects; D005260:Female; D005500:Follow-Up Studies; D006410:Hematopoiesis / Q000187:drug effects; D006801:Humans; D008297:Male; D008875:Middle Aged; D016019:Survival Analysis; D013997:Time Factors; D016896:Treatment Outcome; D055815:Young Adult
chemical_list: D003520:Cyclophosphamide