[New strategy of treatment for POEMS syndrome--autologous peripheral blood stem cell transplantation and thalidomide therapy].
POEMS (polyneuropathy, organomegaly, endocrinopathy, M-protein, and skin changes) syndrome is a rare cause of demyelinating and axonal mixed neuropathy with multiorgan involvement. This syndrome is potentially fatal; progressive neuropathy and/or massive peripheral edema or pleural effusion/ascites deteriorate patients' quality of life. Serious complications such as multiorgan failure due to capillary leak syndrome and thromboembolic events may occur, resulting in poor prognosis. The pathogenesis of POEMS syndrome is not well understood, but overproduction of vascular endothelial growth factor (VEGF), probably secreted by plasmacytoma, may be responsible for most of its characteristic symptoms. Many case reports and series have described patients who have been treated with irradiation, resection of plasmacytoma, chemotherapies, corticosteroids, plasmapheresis, and intravenous immunoglobulin infusion; however, there is no established treatment regimen. In suitable cases, high-dose chemotherapies with autologous peripheral blood stem cell transplantation is highly recommended because this treatment could result in obvious improvement in neuropathy as well as other symptoms, with a significant decrease in the serum VEGF levels. However, from pooled data of published experience, the transplant-related mortality is reported is 5%. At present, indications of this treatment have not yet been established and long-term prognosis is unclear. Treatments with thalidomide or lenalidomide, and anti-VEGF monoclonal antibody (bevacizumab) should be considered as future therapies.
Kuwabara
Satoshi
S
Department of Neurology, Graduate School of Medicine, Chiba University, 1-8-1 Inohana, Chuo-ku, Chiba 260-8670, Japan.
D020533:Angiogenesis Inhibitors / Q000627:therapeutic use*; D000911:Antibodies, Monoclonal / Q000627:therapeutic use; D061067:Antibodies, Monoclonal, Humanized; D000068258:Bevacizumab; D003131:Combined Modality Therapy; D006801:Humans; D016878:POEMS Syndrome / Q000209:etiology / Q000628:therapy*; D036102:Peripheral Blood Stem Cell Transplantation; D010954:Plasmacytoma / Q000378:metabolism; D017410:Practice Guidelines as Topic; D013792:Thalidomide / Q000627:therapeutic use*; D014182:Transplantation, Autologous; D042461:Vascular Endothelial Growth Factor A / Q000378:metabolism
D020533:Angiogenesis Inhibitors; D000911:Antibodies, Monoclonal; D061067:Antibodies, Monoclonal, Humanized; D042461:Vascular Endothelial Growth Factor A; D000068258:Bevacizumab; D013792:Thalidomide