Treatment with sirolimus results in complete responses in patients with autoimmune lymphoproliferative syndrome

DT Teachey, R Greiner, A Seif, E Attiyeh… - British journal of …, 2009 - Wiley Online Library
DT Teachey, R Greiner, A Seif, E Attiyeh, J Bleesing, J Choi, C Manno, E Rappaport…
British journal of haematology, 2009Wiley Online Library
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 …
Summary
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.
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