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Wiley, British Journal of Haematology, 1(145), p. 101-106, 2009

DOI: 10.1111/j.1365-2141.2009.07595.x

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

Journal article published in 2009 by David T. Teachey ORCID, Robert Greiner, Alix Seif, Edward Attiyeh, Jack Bleesing, John Choi ORCID, Catherine Manno, Eric Rappaport, Dirk Schwabe, Cecilia Sheen, Kathleen E. Sullivan, Hongming Zhuang, Daniel S. Wechsler, Stephan A. Grupp
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This paper is available in a repository.

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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.