Treatment of Erdheim-Chester Disease with Long-Term High-Dose Interferon-α

Objectives Erdheim-Chester disease (ECD) is a rare non-Langerhans cell histiocytosis, characterized by a foamy CD68+, CD1a− histiocyte tissue infiltration. Efficacy of standard doses of interferon-α-2a (IFNα) has been suggested in a small series but with variation, depending on the organs involved....

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Published in:Seminars in arthritis and rheumatism 2012-06, Vol.41 (6), p.907-913
Main Authors: Hervier, Baptiste, MD, Arnaud, Laurent, Charlotte, Frederic, MD, Wechsler, Bertrand, MD, Piette, Jean Charles, Amoura, Zahir, Haroche, Julien, MD
Format: Article
Language:English
Subjects:
Adult
Aged
Erdheim-Chester disease
Erdheim-Chester Disease - diagnostic imaging
Erdheim-Chester Disease - drug therapy
Female
Follow-Up Studies
histiocytosis
Humans
Interferon-alpha - administration & dosage
Interferon-alpha - therapeutic use
interferon-α
Male
Middle Aged
Prospective Studies
Radiography
Rheumatology
Treatment Outcome
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Summary:Objectives Erdheim-Chester disease (ECD) is a rare non-Langerhans cell histiocytosis, characterized by a foamy CD68+, CD1a− histiocyte tissue infiltration. Efficacy of standard doses of interferon-α-2a (IFNα) 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 IFNα in ECD. Methods Twenty-four ECD patients have received high-dose IFNα (IFNα ≥18 mIU/wk or pegylated-IFNα ≥180 μg/wk). IFNα efficacy was evaluated clinically and morphologically using a standardized protocol (median follow-up 19 months). Results 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 IFNα 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 IFNα 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 IFNα were well-tolerated: 13 (54.2%) patients had side effects but treatment interruption was infrequent ( n = 3, 12.5%). Conclusions High-dose IFNα may be effective in severe ECD. Improvement may be slow, and high-dose IFNα treatment should be prolonged.
ISSN:0049-0172
1532-866X
DOI:10.1016/j.semarthrit.2011.11.004