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

Blood. 2006 Jan 15;107(2):463-6. doi: 10.1182/blood-2004-12-4870. Epub 2005 Sep 22.

Abstract

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/m(2) 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.

Publication types

  • Research Support, N.I.H., Extramural

MeSH terms

  • Adult
  • Aged
  • Antineoplastic Agents, Alkylating / administration & dosage*
  • Dose-Response Relationship, Drug
  • Female
  • Hematopoietic Stem Cell Transplantation*
  • Humans
  • Male
  • Maximum Tolerated Dose
  • Melphalan / administration & dosage*
  • Middle Aged
  • Myxedema / pathology
  • Myxedema / therapy*
  • Palliative Care
  • Remission Induction
  • Scleroderma, Limited / pathology
  • Scleroderma, Limited / therapy*
  • Scleroderma, Systemic / pathology
  • Scleroderma, Systemic / therapy*
  • Transplantation, Autologous
  • Treatment Outcome

Substances

  • Antineoplastic Agents, Alkylating
  • Melphalan