Scleromyxedema: role of high-dose melphalan with autologous stem cell transplantation.
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.
Donato
Michele L
ML
Department of Blood and Bone Marrow Transplantation, The University of Texas M. D. Anderson Cancer Center, Houston, TX 77030-4009, USA.
Feasel
Adrienne M
AM
Weber
Donna M
DM
Prieto
Victor G
VG
Giralt
Sergio A
SA
Champlin
Richard E
RE
Duvic
Madeleine
M
D000328:Adult; D000368:Aged; D018906:Antineoplastic Agents, Alkylating / Q000008:administration & dosage*; D004305:Dose-Response Relationship, Drug; D005260:Female; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D008297:Male; D020714:Maximum Tolerated Dose; D008558:Melphalan / Q000008:administration & dosage*; D008875:Middle Aged; D009230:Myxedema / Q000473:pathology / Q000628:therapy*; D010166:Palliative Care; D012074:Remission Induction; D045745:Scleroderma, Limited / Q000473:pathology / Q000628:therapy*; D012595:Scleroderma, Systemic / Q000473:pathology / Q000628:therapy*; D014182:Transplantation, Autologous; D016896:Treatment Outcome
D018906:Antineoplastic Agents, Alkylating; D008558:Melphalan