Crow–Fukase症候群

抄録

Crow–Fukase syndrome is a multisystem disorder characterized by monoclonal plasma cell proliferation and overproduction of vascular endothelial growth factor (VEGF). The prognosis of Crow–Fukase syndrome was poor, with a median survival time of 2–3 years in the absence of arropriate treatment. Based on the plasma cell proliferative disorder, we started an investigator–initiated clinical trial of thalidomide for Crow–Fukase syndrome in 2010. This was a randomized, double–blined, placebo–controlled trial, and the primary endpoint, a dcrease in serum VEGF levels at 24 weeks, was significantly higher in the thalidoimde group (mean : 39%) than in the placebo group (mean : −2%) (P = 0.04). Based on this result, thalidomide was approved for Crow–Fukase syndrome in Feburary 2021. However, it is nesessary to adress new issues such as the development of treatment for thalidomide–refractory cases and equalization of the quality of medical care.