Literature Review

Are Stem Cell Transplants Superior to Mitoxantrone for Severe MS?



Intense immunosuppression followed by autologous hematopoietic stem cell transplantation (AHSCT) is significantly superior to mitoxantrone in reducing MRI activity in patients with severe multiple sclerosis (MS), according to a study published online ahead of print February 11 in Neurology.

The study involved 21 people whose MS-related disability had increased during the previous year despite treatment with first-line MS drugs. Participants’ average age was 36, and their average disability level required them to use a cane or crutch to walk.

Giovanni L. Mancardi, MD, Director of the Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, and Maternal and Child Health at the University of Genoa, and colleagues conducted a multicenter, phase II trial. They enrolled patients with secondary progressive or relapsing-remitting MS who had a documented increase in the year prior to enrollment on the Expanded Disability Status Scale (EDSS) score and one or more gadolinium-enhancing areas.

The primary end point was the cumulative number of new T2 lesions in the four years following randomization. Secondary end points were the cumulative number of gadolinium-enhancing lesions, relapse rate, and disability progression. A total of 21 patients were randomized. Following immunosupression, 12 patients received mitoxantrone (20 mg every month for six months) and nine patients underwent AHSCT. Seventeen patients had postbaseline evaluable MRI scans.

Intense immunosuppression followed by AHSCT reduced by 79% the number of new T2 lesions, compared with mitoxantrone treatment (2.5 new T2 lesions vs eight new T2 lesions, respectively). AHSCT also reduced gadolinium-enhancing lesions. None of the people who received AHSCT had a new gadolinium-enhancing lesion during the study, while 56% of those taking mitoxantrone had at least one new lesion. Annualized relapse rate was also reduced, but no difference was seen in the progression of disability.

AHSCT “appears to reset the immune system,” said Dr. Mancardi, lead author of the study. “With these results, we can speculate that stem cell treatment may profoundly affect the course of the disease.

“More research is needed with larger numbers of patients who are randomized to receive either the stem cell transplant or an approved therapy, but it’s very exciting to see that this treatment may be so superior to a current treatment for people with severe MS that is not responding well to standard treatments,” Dr. Mancardi said.

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