Provided by: Cherie C. Binns RN BS
MSCN
Multiple sclerosis (MS) patients who had autologous hematopoietic stem-cell transplantation had significantly fewer new lesions on MRI than those on mitoxantrone, Italian researchers found.
In a randomized, controlled trial, CD34-positive hematopoietic cell transplant reduced the number of new T2 lesions by 79% compared with mitoxantrone over a 4-year study period (P=0.00016), Giovanni Mancardi, MD, of the University of Genova in Italy, and colleagues reported in Neurology.
The therapy also reduced gadolinium-enhancing lesions and annualized relapse rate, but there was no difference in progression of disability, although the study was not powered to look at the latter finding, the researchers noted.
“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 are not responding well to standard treatments,” Mancardi said in a statement.
The ASTIMS study enrolled 21 patients from seven centers in Italy and Spain from 2004 to 2009 who had relapsing-remitting or secondary progressive MS. They were randomized to intensive immunosuppression followed by either mitoxantrone or autologous hematopoietic stem-cell transplantation every month for 6 months.
The intensive immunosuppression regimen involved mobilization with cyclophosphamide and filgrastim, conditioning with carmustine, cytosine arabinoside, etoposide, melphalan, and anti-thymocyte globulin.
The mean age at transplantation was 35.5 years, and the median Expanded Disability Status Scale (EDSS) score at baseline was 6.
ASTIMS was designed as a phase III study, but became a phase II trial with a primary laboratory endpoint — the cumulative number of new T2 lesions 4 years after randomization — “when it was clear that the number of enrolled patients was lower than expected,” the researchers wrote.
Overall, Mancardi and colleagues found fewer T2 lesions in the stem-cell group than in the mitoxantrone group during follow-up at a median of 2.5 lesions versus a median of eight lesions (rate ratio 0.21, 95% CI 0.10-0.48, P=0.00016).
This effect was evident in the first year and was sustained through 4 years of follow-up, they reported. It was also maintained in all sensitivity analyses.
It also resulted in complete suppression of active inflammatory lesions as measured by gadolinium-enhancing lesions, with no stem-cell patients having new lesions compared with 56% of those on mitoxantrone (P=0.029).
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