Commentary 10.1172/JCI131332
1Hematology Division, Brigham and Women’s Hospital, Boston, Massachusetts, USA.
2Harvard Medical School, Boston, Massachusetts, USA.
Address correspondence to: Nancy Berliner, Brigham and Women’s Hospital, 75 Francis Street, Boston, Massachusetts 02115, USA. Phone: 617.732.5840; Email: nberliner@bwh.harvard.edu.
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1Hematology Division, Brigham and Women’s Hospital, Boston, Massachusetts, USA.
2Harvard Medical School, Boston, Massachusetts, USA.
Address correspondence to: Nancy Berliner, Brigham and Women’s Hospital, 75 Francis Street, Boston, Massachusetts 02115, USA. Phone: 617.732.5840; Email: nberliner@bwh.harvard.edu.
Find articles by
Berliner, N.
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JCI
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PubMed
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First published September 16, 2019 - More info
Idiopathic multicentric Castleman disease (iMCD) is a rare hematologic illness of systemic inflammation and organ dysfunction, with unknown etiology. Although therapies targeting IL-6 have been proven effective, a subset of patients with iMCD are resistant to this approach. In this issue of the JCI, Fajgenbaum et al. performed an in-depth analysis of serum inflammatory markers in three iMCD patients refractory to IL-6 blockade, and identified activation of the mTOR pathway associated with symptom flares. Treatment with sirolimus, an mTOR inhibitor, induced remission in all three patients. This study models a precision medicine approach to discovering therapies for rare diseases.
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