大剂量CTX作为解救方案治疗SAABrodsky.pdfVIP

Experimental Hematology 32 (2004) 435–440 High-dose cyclophosphamide as salvage therapy for severe aplastic anemia a a b a Robert A. Brodsky , Allen R. Chen , Isadore Brodsky , and Richard J. Jones aThe Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Md., USA; bDrexel University College of Medicine, Philadelphia, Pa., USA (Received 13 September 2003; revised 19 December 2003; accepted 3 February 2004) 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). Materials and 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 × 109/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