What is the role of high-dose cyclophosphamide in the management of aplastic anemia?

Updated: Jan 29, 2021
  • Author: Sameer Bakhshi, MD; Chief Editor: Emmanuel C Besa, MD  more...
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At present, the use of high-dose cyclophosphamide should be limited to clinical trials. [90] Preliminary data suggested that high-dose cyclophosphamide may result in durable remissions in some patients with aplastic anemia. However, some of these patients develop PNH and cytogenetic abnormalities on follow-up. A prospective, randomized study comparing high-dose cyclophosphamide plus cyclosporine with ATG plus cyclosporine in 31 patients with previously untreated severe aplastic anemia was terminated early because of very high mortality and fungal infections in the cyclophosphamide arm. [105]

A long-term follow-up study of high-dose cyclophosphamide treatment showed better results in treatment-naive patients than in those with refractory disease. At 10 years, in 44 treatment-naive SAA patients and 23 patients with refractory SAA, overall actuarial survival was 88% versus 62%, the response rate was 71% versus 48%, and the actuarial event-free survival was 58% versus 27%, respectively. 

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