Treatment with sirolimus results in complete responses in patients with autoimmune lymphoproliferative syndrome.
نویسندگان
چکیده
We hypothesized that sirolimus, an mTOR inhibitor, may be effective in patients with autoimmune lymphoproliferative syndrome (ALPS) and treated patients who were intolerant to or failed other therapies. Four patients were treated for autoimmune cytopenias; all had a rapid complete or near complete response. Two patients were treated for autoimmune arthritis and colitis, demonstrating marked improvement. Three patients had complete resolution of lymphadenopathy and splenomegaly and all patients had a reduction in double negative T cells, a population hallmark of the disease. Based on these significant responses, we recommend that sirolimus be considered as second-line therapy for patients with steroid-refractory disease.
منابع مشابه
Sirolimus is effective in relapsed/refractory autoimmune cytopenias: results of a prospective multi-institutional trial.
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عنوان ژورنال:
- British journal of haematology
دوره 145 1 شماره
صفحات -
تاریخ انتشار 2009