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Rationale and efficacy of interleukin-1 targeting in Erdheim-Chester disease.

Abstract
Erdheim-Chester disease (ECD) pathophysiology remains largely unknown. Its treatment is not codified and usually disappointing. Interferon (IFN)-α therapy lacks efficacy for some life-threatening manifestations and has a poor tolerance profile. Because interleukin (IL)-1Ra synthesis is naturally induced after stimulation by IFN-α, we hypothesized that recombinant IL-1Ra (anakinra) might have some efficacy in ECD. We treated 2 patients who had poor tolerance or contraindication to IFN-α with anakinra as a rescue therapy and measured their serum C-reactive protein, IL-1β, IL-6, and monocytic membranous IL-1α (mIL-1α) levels before, under, and after therapy. Another untreated ECD patient and 5 healthy subjects were enrolled as controls. After treatment, fever and bone pains rapidly disappeared in both patients, as well as eyelid involvement in one patient. In addition, retroperitoneal fibrosis completely or partially regressed, and C-reactive protein, IL-6, and mIL-1α levels decreased to within the normal and control range. Beside injection-site reactions, no adverse event was reported. Therefore, our results support a central role of the IL-1 network, which seemed to be overstimulated in ECD. Its specific blockade using anakinra thereby opens new pathophysiology and therapeutic perspectives in ECD.
AuthorsAchille Aouba, Sophie Georgin-Lavialle, Christian Pagnoux, Nicolas Martin Silva, Amédée Renand, Françoise Galateau-Salle, Sophie Le Toquin, Henri Bensadoun, Frederique Larousserie, Stéphane Silvera, Nicole Provost, Sophie Candon, Raphaèle Seror, Mathilde de Menthon, Olivier Hermine, Loïc Guillevin, Boris Bienvenu
JournalBlood (Blood) Vol. 116 Issue 20 Pg. 4070-6 (Nov 18 2010) ISSN: 1528-0020 [Electronic] United States
PMID20724540 (Publication Type: Case Reports, Journal Article)
Chemical References
  • Interleukin 1 Receptor Antagonist Protein
  • Interleukin-1alpha
  • C-Reactive Protein
Topics
  • Adult
  • C-Reactive Protein (metabolism)
  • Drug Tolerance
  • Erdheim-Chester Disease (diagnostic imaging, drug therapy, metabolism)
  • Female
  • Humans
  • Interleukin 1 Receptor Antagonist Protein (pharmacology, therapeutic use)
  • Interleukin-1alpha (metabolism)
  • Male
  • Middle Aged
  • Monocytes (drug effects, metabolism)
  • Radiography
  • Treatment Outcome

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