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A multicenter trial of antithymocyte globulin in aplastic anemia and
related diseases
N Young, P Griffith, E Brittain, G Elfenbein, F Gardner, A Huang, D Harmon, J Hewlett, J Fay and K Mangan
Clinical Hematology Branch, NHLBI, Bethesda, MD 20892.
One hundred fifty patients with bone marrow failure were treated in three
groups with antithymocyte globulin (ATG; Upjohn, Kalamazoo, MI) in a
multicenter trial. Patients were assessed at 3, 6, and 12 months after
initiation of treatment by three criteria: transfusion independence,
clinical improvement, and blood counts. Group I consisted of 77 patients
with acute severe aplastic anemia, randomized to receive either ten or 28
days of ATG. There was no significant difference between the two arms of
this protocol: 47% of all patients were clinically improved and 31% were
transfusion independent at 3 months. Of the severely affected patients, 27%
died before 3 months; most deaths occurred early in treatment. Factors
associated with survival in severely affected patients included male sex,
age less than 40 years, absolute neutrophil count greater than 200/microL,
and idiopathic etiology. Neutrophil counts generally increased by 8 weeks
after treatment, but patients continued to show improvement to 1 year
posttreatment. In Group II, 44 patients with moderate or chronic severe
aplastic anemia were randomized to receive either ten days of ATG or 3
months of high-dose nandrolone decanoate. No patient initially treated with
androgens recovered, but 28% of ATG-treated cases achieved transfusion
independence at 3 months. Group III consisted of patients with a variety of
bone marrow failure syndromes. Patients with pancytopenia and cellular bone
marrow showed response rates similar to those of patients with chronic or
moderate aplastic anemia.
Volume 72,
Issue 6,
pp. 1861-1869,
12/01/1988
Copyright © 1988 by The American Society of Hematology
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