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October 6, 1962

Clinical Evaluation of Cyclophosphamide: A New Agent for the Treatment of Children with Acute Leukemia

Author Affiliations

Houston, Tex.; Atlanta, Ga.; St. Louis

JAMA. 1962;182(1):30-37. doi:10.1001/jama.1962.03050400032006

Forty-four children with advanced acute leukemia were treated with cyclophosphamide (Cytoxan ). Complete remissions were observed in 8 and partial remissions in 5. The complete remissions were independent of antecedent or concomitant steroid therapy and persisted from 3 to 15 months. Three of these patients developed leukemic involvement of the central nervous system, though otherwise in complete remission, indicating that cyclophosphamide did not inhibit this complication. The previous favorable response of the patients achieving complete remission to the steroids 6-mercaptopurine and methotrexate ( Amethopterin) eliminated the possibility of any cross-resistance between these agents and cyclophosphamide. The most common side effect was leukopenia which was readily reversible by interruption of therapy. Hemorrhagic cystitis, alopecia, hepatitis, anorexia, diarrhea, nausea, vomiting, and dizziness were other reported side reactions. Cyclophosphamide appears to be an important addition to the chemotherapy of children with acute leukemia.