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Br J Haematol. 2009 Apr;145(1):101-6. doi: 10.1111/j.1365-2141.2009.07595.x. Epub 2009 Feb 4.

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

Author information

  • 1Pediatric Hematology and Oncology, Children's Hospital of Philadelphia, University of Pennsylvania School of Medicine, Philadelphia PA 19104, USA. teacheyd@email.chop.edu

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.

PMID:
19208097
[PubMed - indexed for MEDLINE]
PMCID:
PMC2819393
Free PMC Article
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