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 Blood, 15 January 2006, Vol. 107, No. 2, pp. 463-466.
Prepublished online as a Blood First Edition Paper on September 22, 2005; DOI 10.1182/blood-2004-12-4870.

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CLINICAL TRIALS AND OBSERVATIONS

Scleromyxedema: role of high-dose melphalan with autologous stem cell transplantation

Michele L. Donato, Adrienne M. Feasel, Donna M. Weber, Victor G. Prieto, Sergio A. Giralt, Richard E. Champlin, and Madeleine Duvic

From the Departments of Blood and Bone Marrow Transplantation, Dermatology, Myeloma, and Dermatopathology, The University of Texas M. D. Anderson Cancer Center, Houston, TX.

Scleromyxedema, the most severe manifestation of the spectrum of lichen myxedematosus, is characterized by cutaneous mucinosis, extracutaneous manifestations, and a monoclonal gammopathy. Seven of 8 patients evaluated at our center were treated with high-dose melphalan (180 mg/m2 intravenously) and autologous peripheral blood stem cell transplantation, with marked improvement of gastrointestinal, central nervous system, pulmonary manifestations, and Karnofsky performance status. Five patients obtained a cutaneous complete remission and 2 patients had partial remissions. Three patients with slight progression in the skin at 12, 8, and 4 months after treatment received a second cycle of high-dose melphalan and had further symptomatic improvement. The lichen myxedematosus–scleromyxedema spectrum appears to be a continuum that requires the presence of a serum paraprotein and differs in severity of skin lesions, extracutaneous manifestations, and performance status. High-dose melphalan followed by autologous transplantation appears effective for improving the symptoms and systemic manifestations of scleromyxedema.


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Blood 2006 107: 415. [Full Text] [PDF]



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