TREATMENT OF HISTIOCYTOSIS-X WITH SUPPRESSIN A: RESULTS FROM A RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED STUDY

Abstract

We have previously described that thymic hormones might be useful in the treatment of histiocytosis-X. Based on those data, the Office of Orphan Products Development at the FDA is sponsoring a randomized, double-blind, placebo-controlled clinical study to determine if Suppressin A, a mixture of thymic hormones that specifically induce the differentiation of suppressor T-lymphocytes, is biologically active in histiocytosis-X. Patients are randomized to receive either Suppressin A or placebo for 42 days, after which they are re-evaluated and the code broken. To help in the evaluation of biological activity, four distinct clinical scoring systems were created: 1)organ specific; 2)whole body; 3)vital systems (respiratory, hepatic and hematopoetic function); and 4)overall performance status. The results to date from the initial 13 patients who have completed the six-week treatment course in this on-going study are:These data indicate biological activity of Suppressin A in the treatment of histiocytosis-X. These early trends of significance need to be confirmed through further patient recruitment and study.