Background Aggressive multiple sclerosis (MS) causes early, rapid accumulation of disability with frequent and severe relapses, incomplete recovery from relapses, and/or high T2 lesion burden. Severe relapses are typically treated with high-dose IV corticosteroids followed by plasmapheresis. Patients who do not respond to initial treatment lack universally accepted second-line options. Cyclophosphamide is used in clinical practice for MS patients that do not respond to initial relapse therapy. Despite its use, there is no conclusive data demonstrating its efficacy. Objective To review the UBC MS clinic experience in using cyclophosphamide as a rescue therapy for MS patients. Methods We screened the UBC MS Clinic pharmacy database to identify MS patients followed at the UBC MS clinic that received cyclophosphamide between June 2009 and June 2024 for retrospective chart review. Results The median EDSS score prior to cyclophosphamide therapy was 6 (range 2.5–9). Thirteen out of 23 patients had an improvement in EDSS, including 6/23 with a ≥3 EDSS numerical improvement. Conclusion Rescue cyclophosphamide treatment was associated with improvement in EDSS in MS patients with a relapsing course. Despite the availability of high-efficacy DMTs, this cohort shows there may be a role for cyclophosphamide in the context of severe relapses.
Bibic et al. (Thu,) studied this question.