Blood, 1973, Vol. 42, No. 3, pp. 359-365.
© 1973 American Society of Hematology, Inc.
5-Azacytidine: A New Active Agent for the
Treatment of Acute Leukemia
Myron Karon 1,
Lance Sieger 1,
Suzanne Leimbrock 1,
Jerry Z. Finklestein 1,
Mark E. Nesbit 1, and
Jerry J. Swaney 1
1 Division of Hematology, Department of Pediatrics, Childrens Hospital of Los Angeles.
and the USC School of Medicine; Department of Pediatrics, Harbor General Hospital, and the UCLA
School of Medicine, Torrance, Calif.; the Department of Pediatrics, University of Minnesota School
of Medicine, Minneapolis, Minn.; and Childrens Memorial Hospital and the Department of Pediatrics, Northwestern University, Chicago, Ill.
Thirty-seven children with acute leukemia
were treated with 5-azacytidine in 5-day
courses given every 14 days. Six out of 14
children with acute myelogenous leukemia
who were adequately treated achieved an
M1 marrow. Five of these subsequently
developed complete remissions lasting
8 mo, 6 mo, 3 mo, 2 mo, and 2 mo. Of
22 children with acute lymphocytic leukemia, one achieved an M1 marrow and one
an M2 marrow. The former attained a complete remission which lasted 3 mo. The
maximum tolerated dose is between 150
to 200 mg/sq m on a daily x 5 schedule
given every 14 days. The impressive activity of 5-aza-C in patients with acute
myelogenous leukemia resistant to cytosine arabinoside indicates that this drug
will become an important addition to the
therapeutic armamentaria against this type
of leukemia.
Submitted on January 22, 1973
Revised on March 12, 1973
Accepted on March 22, 1973
