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

Research output: Contribution to journalArticle

97 Citations (Scopus)

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.

Original languageEnglish (US)
Pages (from-to)101-106
Number of pages6
JournalBritish Journal of Haematology
Volume145
Issue number1
DOIs
StatePublished - Apr 1 2009

Fingerprint

Autoimmune Lymphoproliferative Syndrome
Sirolimus
Therapeutics
Splenomegaly
Colitis
Arthritis
Steroids
T-Lymphocytes

All Science Journal Classification (ASJC) codes

  • Hematology

Cite this

Teachey, David T. ; Greiner, Robert ; Seif, Alix ; Attiyeh, Edward ; Bleesing, Jack ; Choi, John ; Manno, Catherine ; Rappaport, Eric ; Schwabe, Dirk ; Sheen, Cecilia ; Sullivan, Kathleen E. ; Zhuang, Hongming ; Wechsler, Daniel S. ; Grupp, Stephan A. / Treatment with sirolimus results in complete responses in patients with autoimmune lymphoproliferative syndrome. In: British Journal of Haematology. 2009 ; Vol. 145, No. 1. pp. 101-106.
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author = "Teachey, {David T.} and Robert Greiner and Alix Seif and Edward Attiyeh and Jack Bleesing and John Choi and Catherine Manno and Eric Rappaport and Dirk Schwabe and Cecilia Sheen and Sullivan, {Kathleen E.} and Hongming Zhuang and Wechsler, {Daniel S.} and Grupp, {Stephan A.}",
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Teachey, DT, Greiner, R, Seif, A, Attiyeh, E, Bleesing, J, Choi, J, Manno, C, Rappaport, E, Schwabe, D, Sheen, C, Sullivan, KE, Zhuang, H, Wechsler, DS & Grupp, SA 2009, 'Treatment with sirolimus results in complete responses in patients with autoimmune lymphoproliferative syndrome', British Journal of Haematology, vol. 145, no. 1, pp. 101-106. https://doi.org/10.1111/j.1365-2141.2009.07595.x

Treatment with sirolimus results in complete responses in patients with autoimmune lymphoproliferative syndrome. / Teachey, David T.; Greiner, Robert; Seif, Alix; Attiyeh, Edward; Bleesing, Jack; Choi, John; Manno, Catherine; Rappaport, Eric; Schwabe, Dirk; Sheen, Cecilia; Sullivan, Kathleen E.; Zhuang, Hongming; Wechsler, Daniel S.; Grupp, Stephan A.

In: British Journal of Haematology, Vol. 145, No. 1, 01.04.2009, p. 101-106.

Research output: Contribution to journalArticle

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