Treatment with sirolimus results in complete responses in patients with autoimmune lymphoproliferative syndrome
David T Teachey, Robert Greiner, Alix Seif, Edward Attiyeh, Jack Bleesing, John Choi, Catherine Manno, Eric Rappaport, Dirk Schwabe, Cecilia Sheen, Kathleen E Sullivan, Hongming Zhuang, Daniel S Wechsler, Stephan A Grupp, David T Teachey, Robert Greiner, Alix Seif, Edward Attiyeh, Jack Bleesing, John Choi, Catherine Manno, Eric Rappaport, Dirk Schwabe, Cecilia Sheen, Kathleen E Sullivan, Hongming Zhuang, Daniel S Wechsler, Stephan A Grupp
Abstract
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.
Conflict of interest statement
Conflict of interest
No author has competing financial interests to declare.
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Source: PubMed