title: High-dose cyclophosphamide as salvage therapy for severe aplastic anemia.
abstract: OBJECTIVE
  The treatment options for patients with aplastic anemia who do not respond to conventional immunosuppression are limited. The aim of this study was to evaluate high-dose cyclophosphamide in patients with refractory severe aplastic anemia (SAA).
  
  
  METHODS
  We treated 17 SAA patients with high-dose cyclophosphamide (50 mg/kg/day for 4 consecutive days) who previously did not respond to one or more courses of immunosuppressive therapy. Median age was 31 years (range 6-58); median disease duration was 14 months (range 6-58), and 8 patients met criteria for very severe aplastic anemia (absolute neutrophil count <0.2 x 10(9)/L) at the time of treatment.
  
  
  RESULTS
  At median follow-up of 29 months, 10 patients (59%) are alive. Nine patients (53%) achieved a drug-free remission after high-dose cyclophosphamide; 4 patients achieved a complete remission and 5 patients currently meet criteria for a partial remission but continue to improve. One nonresponder to high-dose cyclophosphamide developed paroxysmal nocturnal hemoglobinuria; another nonresponder developed a myelodysplastic syndrome. In responding patients, median time to 500 neutrophils was 54 days (range 35-119), median time to the last platelet transfusion was 99 days (range 51-751), and median time to the last red cell transfusion was 125 days (range 63-796).
  
  
  CONCLUSIONS
  High-dose cyclophosphamide shows promise for salvaging patients with refractory SAA.
authors.affiliation: The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, MD 21231, USA. rbrodsky@jhmi.edu
authors.forename: Robert A
authors.initials: RA
authors.lastname: Brodsky
authors.forename: Allen R
authors.initials: AR
authors.lastname: Chen
authors.forename: Isadore
authors.initials: I
authors.lastname: Brodsky
authors.forename: Richard J
authors.initials: RJ
authors.lastname: Jones
mesh_terms: D000293:Adolescent; D000328:Adult; D000741:Anemia, Aplastic / Q000150:complications / Q000188:drug therapy* / Q000401:mortality; D002648:Child; D003520:Cyclophosphamide / Q000008:administration & dosage* / Q000633:toxicity; D005260:Female; D006457:Hemoglobinuria, Paroxysmal / Q000209:etiology; D006801:Humans; D007239:Infections / Q000139:chemically induced; D008297:Male; D008875:Middle Aged; D009190:Myelodysplastic Syndromes / Q000209:etiology; D012074:Remission Induction; D016879:Salvage Therapy / Q000009:adverse effects / Q000379:methods*; D016019:Survival Analysis; D016896:Treatment Outcome
chemical_list: D003520:Cyclophosphamide