Hemopoietic stem cell transplantation can be considered a treatment option for patients with aggressive, refractory multiple sclerosis, a single-center study suggested.
Among a group of 35 patients given stem cell transplants, progression-free survival was 44% at 15 years for those who had active lesions on MRI and 10% for those who did not have active lesions (P=0.01), according to Vasilios K. Kimiskidis, MD, and colleagues from the Aristotle University in Thessaloniki, Greece.
This meant that the best results were seen in patients with inflammatory central nervous system involvement, the researchers explained in the March 22nd issue of “Neurology”.
“The debulking of autoreactive clones, complete suppression of inflammation, and, possibly, the qualitative immune changes induced by [hemopoietic stem cell transplantation] may all play a beneficial role in slowing down disease progression,” they observed.
Between 1995 and 2001, Kimiskidis and colleagues did stem cell transplants in select patients who had progressed at least one point on the 10-point Expanded Disability Status Scale during the previous year.
Patients’ median age was 40, and median duration of disease was seven years.
The most common type of multiple sclerosis in this cohort was secondary progressive, followed by primary progressive. One patient had aggressive relapsing-progressive, or malignant, multiple sclerosis.
Previous treatments included steroids, interferon-beta, and azathioprine.
Median baseline disability scores ranged from 4.5 to 8 (median 6), and active lesions were present on MRI in 12 patients.
Clinical improvements ranging from 0.5 to 5.5 points (median 1) on the disability scale were seen among 16 patients, with improvements lasting for a median of two years.
In two of the patients, the improvements persisted for more than seven years, and the response was considered lifesaving in the patient with malignant disease.
Overall progression-free survival at 15 years was 25%.
Median progression-free survival in secondary and relapsing disease was 5.4 years, and 1.5 years in primary progressive disease.
Significant and sustained reductions also were seen in the number and volume of gadolinium-enhancing lesions seen on MRI, the researchers reported.
Better outcomes appeared to be associated with age younger than 35 and a shorter duration of disease.
Five patients died — one from aspergillosis two months after the transplant, another from a pulmonary hemorrhage 2.5 years after the procedure, a third at 12 years from prostate cancer. Two patients died from complications related to their multiple sclerosis at 10 and 14 years.
Kimiskidis and colleagues acknowledged that their phase 1-2 study was methodologically weak, being from a single center and having a limited number of patients, and provided only class IV evidence.
“The critical question whether [hemopoietic stem cell transplantation] is superior to conventional treatments cannot be answered on the basis of our uncontrolled data,” they wrote.
Nonetheless, they argued in favor of the treatment in patients with rapidly progressive disease, noting that “the final status of our patients may be considered satisfactory, although disease progression, with or without an initial improvement, occurred in a significant proportion of patients.”
They noted that the European Group for Blood and Marrow Transplantation also has found good results in more than 175 patients, particularly among patients who were younger, less disabled, and with shorter disease duration.
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