Neurology 2021 02 14;96(6):e817-e830. Epub 2020 Dec 14.
From the Division of Immunotherapy (R.K.B., X.H., K.Q., I.A.), Department of Medicine, Department of Neurology (R.B., T.S.), Department of Preventive Medicine (I.H.), and Department of Pathology and Cell and Developmental Biology (T.S.), Northwestern University, Chicago, IL; and Department of Neurology (J.R.), University of Utah, Salt Lake City.
Objective: To test the hypothesis that autologous nonmyeloablative hematopoietic stem cell transplantation (HSCT) is safe and shows efficacy in the treatment of stiff-person spectrum disorder (SPSD).
Methods: Twenty-three participants were treated in a prospective open-label cohort study of safety and efficacy. After stem cell mobilization with cyclophosphamide (2 g/m) and filgrastim (5-10 µg/kg/d), participants were treated with cyclophosphamide (200 mg/kg) divided as 50 mg/kg IV on day -5 to day -2; rabbit anti-thymocyte globulin (thymoglobulin) given intravenously at 0. Read More