Sirolimus is effective in relapsed/refractory autoimmune cytopenias: results of a prospective multi-institutional trial.

@article{Bride2016SirolimusIE,
  title={Sirolimus is effective in relapsed/refractory autoimmune cytopenias: results of a prospective multi-institutional trial.},
  author={Karen L Bride and Tiffaney L. Vincent and Kim Smith-Whitley and Michele P Lambert and Jack Jan Hendrik Bleesing and Alix E Seif and Catherine S. Manno and James Casper and Stephan A. Grupp and David Teachey},
  journal={Blood},
  year={2016},
  volume={127 1},
  pages={17-28}
}
Patients with autoimmune multilineage cytopenias are often refractory to standard therapies requiring chronic immunosuppression with medications with limited efficacy and high toxicity. We present data on 30 patients treated on a multicenter prospective clinical trial using sirolimus as monotherapy. All children (N = 12) with autoimmune lymphoproliferative syndrome (ALPS) achieved a durable complete response (CR), including rapid improvement in autoimmune disease, lymphadenopathy, and… CONTINUE READING
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