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Blood, 1 November 2005, Vol. 106, No. 9, pp. 2992-2994.
Prepublished online as a Blood First Edition Paper on July 14, 2005; DOI 10.1182/blood-2005-06-2238.
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CLINICAL TRIALS AND OBSERVATIONS Brief report
Successful treatment of Erdheim-Chester disease, a nonLangerhans-cell histiocytosis, with interferon-
Fadi Braiteh,
Cynthia Boxrud,
Bita Esmaeli, and
Razelle Kurzrock
From the Phase I Program, Division of Cancer Medicine and the University of Texas Graduate School of Biomedical Sciences at Houston, Texas; the Section of Ophthalmology, Department of Plastic Surgery, M.D. Anderson Cancer Center, Houston, TX; and the Jules Stein Eye Institute, University of California, Los Angeles (UCLA) Santa Monica, CA.
Erdheim-Chester disease is a rare non-Langerhans histiocytosis with multisystem involvement. To date, there is no standard treatment for this disorder, and more than half of the patients succumb within 3 years. Because interferon- promotes the terminal differentiation of histiocytes and dendritic cells, we hypothesized that this molecule would be a useful therapy for Erdheim-Chester disease. We therefore treated 3 patients with advanced disease with interferon- at a starting dose of 3 to 6 x 106 units, which was later reduced, during maintenance, to 1 x 106 units subcutaneous 3 times per week. Marked improvement was noted in all patients, with substantial retro-orbital disease regression within 1 month. Improvement in bone lesions, pain, diabetes insipidus, and other manifestations was gradual over many months. Responses were durable (3+ to 4.5+ years). Our observations suggest that this well-tolerated therapy has a significant effect on the course and outcome of Erdheim-Chester disease.

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