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Deoxycholic Acid for Dercum Disease: Repurposing a Cosmetic Agent to Treat a Rare Disease
Channi Silence1, Shauna M Rice1, Andrew Liteplo1
1Ms. Silence, as well as Drs. Liteplo, McFadden, Al Jalbout, Al Saud, and Kourosh, are from the Massachusetts General Hospital, Boston. Ms. Silence and Dr. Kourosh are from the Department of Dermatology. Dr. Kourosh also is from the Harvard Medical School, Boston. Ms. Rice is from the University of Massachusetts Medical School, Worcester. Ms. Gao is from Harvard College, Boston.
Abstract:
Dercum disease is a rare condition characterized by multiple painful fatty tumors distributed throughout the body. There currently are no US Food and Drug Administration-approved treatments for Dercum disease, and the treatments tried have shown little to no efficacy, leaving many patients with a profoundly negative impact on quality of life. We present a case series of 3 patients who were diagnosed with Dercum disease and were treated with deoxycholic acid (DCA), a therapy approved for adipolysis of submental fat. The patients experienced a reduction in tumor size with radiographic evidence as well as a notable reduction in symptoms.
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