Journal of Clinical Oncology, 2005 ASCO Annual Meeting Proceedings.
Vol 23, No 16S (June 1 Supplement), 2005: 2539
© 2005 American Society of Clinical Oncology

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Abstract

Successful treatment of Erdheim-Chester disease, a non-langerhans histiocytosis, with Interferon-alpha

Univ of Texas M.D. Anderson Cancer Ctr, Houston, TX; Univ CA Los Angeles, Santa Monica, CA

2539

Background: Erdheim-Chester disease is a rare non-Langerhans histiocytosis, with particular tropism for connective and adipose tissues. The infiltrative process preferentially involves the axial skeleton, pituitary, orbit, retroperitoneum, lung, mediastinum and central nervous system. To date, there is no standard treatment for this rare disorder and about two-thirds of the patients succumb to their disease within three years. Methods: We treated three patients with advanced Erdheim-Chester disease with interferon- at a starting dose of 3 to 6 million units s.c. three times per week. All three patients were middle-aged men who were diagnosed with Erdheim-Chester disease based on clinical, radiological and pathological features. These patients had progressed despite radiation therapy, corticosteroids and chemotherapy. Patients suffered from painful osteosclerosis, diabetes insipidus and/or progression towards blindness due to retro-orbital disease. Results: All three patients demonstrated rapid improvement of symptoms and signs, including resolution of proptosis, improvement in diabetes insipidus, and/or regression of bone lesions accompanied by marked decrease in pain. Because of fatigue, the dose was lowered to one million units three times per week in all patients. This dose was tolerated with almost no side effects. Patients continued to improve on the lower dose and all three individuals have had a long-standing response (2.5+ to 4+ years). Conclusions: Our observations suggest that interferon- has significant beneficial effects in Erdheim-Chester disease.

No significant financial relationships to disclose.

Abstract presentation from the 2005 ASCO Annual Meeting