Treatment of Erdheim-Chester disease with long-term high-dose interferon-α.

[erdheim-chester disease]

Erdheim-Chester disease (ECD ) is a rare non-Langerhans cell histiocytosis , characterized by a foamy CD 68 +, CD 1 a- histiocyte tissue infiltration . Efficacy of standard doses of interferon-α-2 a (IFN α ) has been suggested in a small series but with variation , depending on the organs involved . Our aim was to report our single -center experience about the use of high -dose IFN α in ECD .Twenty -four ECD patients have received high -dose IFNα (IFNα ≥18 mIU /wk or pegylated-IFNα ≥180 μg /wk ). IFN α efficacy was evaluated clinically and morphologically using a standardized protocol (median follow-up 19 months ).Indication for treatment was central nervous system and /or heart involvement (n = 20 ), exophthalmos (n = 1 ), and standard-dose IFNα inefficacy (n = 3 ). High -dose IFN α was effective in 16 patients (67 %) with improvement (n = 11 , 46 %) and stabilization (n = 5 , 21 %). Late and gradual improvement was observed during prolonged follow-up in most patients . The efficacy of high -dose IFN α was dependent on the organs involved : central nervous system and heart improvement or stabilization occurred in 7 /11 (64 %) and 11 /14 (79 %) patients , respectively . Six patients (25 %) worsened . High doses of IFN α were well-tolerated : 13 (54 .2 %) patients had side effects but treatment interruption was infrequent (n = 3 , 12 .5 %).High -dose IFN α may be effective in severe ECD . Improvement may be slow , and high -dose IFNα treatment should be prolonged .

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