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

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.

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/m(2) i.v. on days 1-2) followed by G-CSF (n = 38).

The median number of mobilized HSCs per kg was 6.32 × 10(6) (range: 2.64-26.3 × 10(6)). 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).

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.