Seventy-nine cases of autoimmune thrombocytopenia seen by the Baylor H
ematology section of The Methodist Hospital between 1991 and 1996 were
retrospectively reviewed to assess the effectiveness of danazol in th
e treatment of autoimmune thrombocytopenia. Among the 42 patients who
received danazol, the mean initial platelet count prior to treatment w
as 24.3 +/- 17.4 (SD) x 10(9)/L. with a mean duration of disease of 53
months. Most cases were idiopathic, but some patients had underlying
secondary disorders (rheumatoid arthritis, systemic lupus erythematosu
s, HIV, and/or Evans' syndrome). Overall 57% of the patients treated w
ith danazol had an excellent or a good response with three patients wh
o had unmaintained remission for >11 months. Minimal side effects were
noted. Fifty percent of the patients with associated secondary disord
ers achieved an excellent or good response. The lytic component of all
three Evans' syndrome cases was well controlled with danazol. In two
cases, danazol was effective where a variety of other treatment regime
ns were not. An excellent or a good response was found in 58%, 62%, an
d 53% in patients >65 years old, between 45 and 65 years old, and <45
years old, respectively. Response rates were similar in males and fema
les. Seventy percent of the nonsplenectomized patients had an excellen
t or a good response compared to 33% in postsplenectomy patients. Over
all in view of its favorable side-effect profile, it is rational early
on to attempt to abrogate the need for splenectomy, it may salvage sp
lenectomy failures, and there is a reasonable response rate in those r
efractory to multiple prior therapies.