Advances and perspectives in treatment for refractory neuropathies; with special foci upon immune-mediated neuropathies and Crow-Fukase syndrome

Abstract

There are significant advances in immune-modulating treatments for Guillain-Barré syndrome (GBS) and chronic inflammatory demyelinating polyneuropathy (CIDP) in the past 20 years. GBS, however, is still a serious disease with a mortality rate of 8% and 20% of the patients being unable to walk independently a year after onset. For CIDP and related disorders such as multifocal motor neuropathy, and demyeinating neuropathy with antimyelin-associated-glycoprotein (MAG) antibody, treatments should be based on individual pathophysiology. Rituximab could be a promising agent for the subtypes of CIDP refractory to conventional immune treatments. Crow-Fukase syndrome is a rare cause of demyelinating neuropathy with multiorgan involvement. Overproduction of vascular endothelial growth factor (VEGF), probably mediated by monoclonal proliferation of plasma cells, is likely to be responsible for most of the characteristic symptoms. There is no established treatment regimen for Crow-Fukase syndrome. In appropriate candidates, 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. Indication of this treatment has not yet been established, and long-term prognosis is unclear at present. Treatments that should be considered as future therapy against Crow-Fukase syndrome include thalidomide, and anti-VEGF monoclonal antibody (bevacizumab).