Safety and efficacy of hematopoietic stem cells mobilization in patients with multiple sclerosis

Abstract

Introduction: Multiple sclerosis (MS) is a T-cell-mediated chronic inflammatory disorder of the central nervous system. Several agents have been approved for the treatment of MS; however, their efficacy is limited and short term. Autologous hematopoietic stem cell (HSC) transplantation may remain an encouraging option for some MS patients who failed prior conventional treatment. Objective: To assess the safety and effectiveness of HSCs mobilization in patients with MS. Material and methods: Thirty-nine patients (20 females and 19 males) with relapsing-remitting MS at median age of 40 years (range: 25-63) were included in this study. As a stem cell mobilization, they received either granulocyte colony-stimulating factor (G-CSF) alone (10 μg/kg s.c. daily; n = 1) or cyclophosphamide (CY; 2.0 g/m2 i.v. on days 1-2) followed by G-CSF (n = 38). Results: The median number of mobilized HSCs per kg was 6.32 × 106 (range: 2.64-26.3 × 106). One apheresis was sufficient for collection of HSCs in 30 out of 39 MS patients (77%). Two aphereses were required for seven patients, three for one and four for one (17, 3, and 3%; respectively). Side effects of HSCs mobilization have been reported for eight patients (30%) and they were following: Staphylococcus epidermidis bacteremia (n= 1), fever of unknown origin (n = 3), diarrhea (n = 3), and headache (n = 1). Conclusions: Mobilization using CY and/or G-CSF resulted in effective mobilization in all MS patients. This procedure was found to be safe. No fatal outcome has been reported.