Danazol for the treatment of refractory autoimmune thrombocytopenia in systemic lupus erythematosus.

STUDY OBJECTIVE

to determine the efficacy of danazol therapy in patients with systemic lupus erythematosus with severe autoimmune thrombocytopenia refractory to other therapies.

DESIGN

noncontrolled clinical trial, with a minimum of 8 weeks of therapy, the maximum determined by clinical response.

SETTING

referral-based rheumatology clinic at an army medical center.

PATIENTS

sequential sample of six patients with systemic lupus erythematosus with severe autoimmune thrombocytopenia refractory to high-dose glucocorticoids. Four patients also failed splenectomy, or cytotoxic drugs, or both.

INTERVENTIONS

danazol, 200 mg four times per day, was added to the previous therapeutic regimen for at least 2 months.

MEASUREMENTS AND MAIN RESULTS

all six patients had normal platelet counts within 6 weeks of starting danazol treatment. After resolution of thrombocytopenia for at least 1 month, immunosuppressive medications were tapered; one patient had a relapse. During an average follow-up of 12 months, the danazol dose was lowered in the five remaining patients but could not be discontinued without recurrence of thrombocytopenia. During danazol therapy, platelet-bound IgG antibodies and circulating immune complexes did not decrease significantly. Danazol was well tolerated.

CONCLUSIONS

danazol appears to be a useful adjunctive treatment for refractory autoimmune thrombocytopenia associated with systemic lupus erythematosus.