Scleromyxedema: long-term follow-up after high-dose melphalan with autologous stem cell transplantation.

BACKGROUND: Scleromyxedema is a cutaneous mucinosis that is often accompanied by severe systemic manifestations. New therapeutic options have been introduced for this condition, but data on the long-term efficacy of treatments are limited. OBJECTIVES: This study was designed to evaluate the long-term efficacy and safety of treatment with high-dose melphalan and autologous peripheral blood stem cell transplantation (PBSCT). METHODS: In an original study published in 2006, seven patients with scleromyxedema were treated with high-dose melphalan and autologous PBSCT between April 2000 and November 2003. This follow-up retrospective study evaluated the long-term efficacy and safety of the treatment in five of the original seven patients. RESULTS: Responses were seen in all five patients. Three patients achieved complete responses, and one achieved a partial response. One patient achieved a complete response followed by a partial response after recurrence. Paraprotein levels were stable in two patients. In one patient, paraprotein level was undetectable 10 years following treatment, and in the other two patients, paraprotein levels decreased significantly following treatment. No long-term complications or adverse effects were noted in any of the patients. CONCLUSIONS: High-dose melphalan with autologous stem cell transplantation appears to be a safe and effective long-term treatment in patients with scleromyxedema. However, further studies are required to investigate this in larger groups of patients.

Scleromyxedema without paraproteinemia.

BACKGROUND: Scleromyxedema is a rare generalized form of lichen myxedematosus, a chronic cutaneous mucinosis of unknown etiology usually associated with a monoclonal gammopathy and underlying systemic disorders. It is characterized by the presence of lichenoid papules and diffuse indurations of the skin. Histologically, mucin deposits are observed in the dermis as variable degrees of fibrosis. Numerous treatment modalities have been reported as producing partial or inconsistent responses associated with significant adverse effects. METHODS: We report an unusual case of scleromyxedema not associated with monoclonal gammopathy in a young patient who was treated with thalidomide. RESULTS: Patient remained stable with maintenance of injuries despite treatment with thalidomide. CONCLUSIONS: Scleromyxedema is a rare presentation for which a defined therapeutic regimen remains to be established. Treatment with thalidomide has proved to be effective in the management of these patients. We suggest that these patients should be followed up with periodic protein electrophoresis with immunofixation for a monoclonal component in blood and urine. As the therapeutic approach to scleromyxedema remains challenging and treatment is based on symptomatic presentation, further clinical studies to substantiate an effective therapeutic regimen with a positive long-term safety and risk profile are required.

Scleromyxedema: successful treatment with thalidomide in two patients.

BACKGROUND: Scleromyxedema is an unusual skin disease characterized by mucinous infiltration of the skin. There is no satisfactory treatment for this disorder. CASE REPORT: We report 2 cases of scleromyxedema, both associated with an IgG-lambda paraprotein. Treatment with thalidomide was started and great improvement of the lesion was noted as shown by a quality of life questionnaire similar to those used in rheumatoid arthritis. TNF-alpha value was elevated in both cases during this treatment. DISCUSSION: We hypothesize that the efficiency of thalidomide could be mediated by its costimulatory effect on T cytotoxic lymphocytes.

Scleromyxedema, a positive effect with thalidomide.

Scleromyxedema is a rare dermatological disorder marked by widespread symmetric 2- to 3-mm, firm, waxy, closely spaced papules involving the hands, forearms, face, neck, upper trunk and thighs. The most common extracutaneous manifestation of scleromyxedema is a benign plasma cell dyscrasia. Treatment of scleromyxedema is limited by the lack of long-term results, toxicity and significant adverse side effects. We report a severe case of scleromyxedema who had marked improvement when treated with thalidomide.