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

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.