High-dose cyclophosphamide for refractory autoimmune hemolytic anemia

Blood. 2002 Jul 15;100(2):704-6. doi: 10.1182/blood-2002-01-0087.


High-dose cyclophosphamide, without stem cell rescue, has been used successfully to treat aplastic anemia and other autoimmune disorders. To determine the safety and efficacy of high-dose cyclophosphamide among patients with severe refractory autoimmune hemolytic anemia, we treated 9 patients with cyclophosphamide (50 mg. kg(-1). d(-1) for 4 days) who had failed a median of 3 (range, 1-7) other treatments. The median hemoglobin before treatment was 6.7 g/dL (range, 5-10 g/dL). The median time to reach an absolute neutrophil count of 500/microL or greater was 16 days (range, 12-18 days). Six patients achieved complete remission (normal untransfused hemoglobin for age and sex), and none have relapsed after a median follow-up of 15 months (range, 4-29 months). Three patients achieved and continue in partial remission (hemoglobin at least 10 g/dL without transfusion support). High-dose cyclophosphamide was well tolerated and induced durable remissions in patients with severe refractory autoimmune hemolytic anemia.

Publication types

  • Clinical Trial
  • Research Support, Non-U.S. Gov't
  • Research Support, U.S. Gov't, P.H.S.

MeSH terms

  • Adult
  • Anemia, Hemolytic, Autoimmune / drug therapy*
  • Child
  • Cyclophosphamide / administration & dosage*
  • Cyclophosphamide / toxicity
  • Female
  • Follow-Up Studies
  • Humans
  • Male
  • Middle Aged
  • Remission Induction / methods
  • Salvage Therapy
  • Treatment Outcome


  • Cyclophosphamide