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More information on HSCT and the long term outcomes below:
Stem Cell Transplants Offer First-Ever MS Treatment That Reverses Disability
Only 2 years after getting a stem cell transplant, half of volunteers showed improvement in their disability scores — a first for any MS therapy.
Dr. Richard K. Burt performed the first hematopoietic stem cell transplant (HSCT) for a multiple sclerosis (MS) patient in the United States at Chicago’s Northwestern Memorial Hospital. Now Burt, Chief of the Division of Medicine-Immunotherapy and Autoimmune Diseases at Northwestern University’s Feinberg School of Medicine, is making headlines again.
Burt and his colleagues published the results of their newest HSCT study earlier this week in the Journal of the American Medical Association. Their results show that HSCT could be the first MS therapy to reverse disability. Though the study group was small, the results have experts hopeful.
For this trial, 151 patients underwent a stem cell transplant. First, their immune systems were tamped down using low-dose chemotherapy. Then, doctors used HSCT therapy, involving an infusion of the patients’ own stem cells, previously harvested from their blood, to reboot their immune systems. After a short stay in the hospital, the volunteers went about their normal lives, needing no “maintenance” drugs.
Over the next several years, the volunteers were periodically given a series of tests to measure their disability. One test, known as the Expanded Disability Status Scale, or EDSS, measures cognition, coordination, and walking, among other things. Participants underwent MRI scans and completed questionnaires to measure their overall quality of life.
The researchers found that at two years post-transplant half of the patients showed a marked improvement in disability. Of the patients who were followed for four years, more than 80 percent remained relapse-free.
Stem Cell Transplant Improves Physical and Cognitive Symptoms In 50% Of MS Patients
By Janet Fang
23 JAN 2015, 21:43
Multiple sclerosis, or MS, is an autoimmune disorder that affects the brain and spinal cord. The immune system destroys the patient's own myelin, the protective membrane wrapped about the nerves, thus disrupting communication with the central nervous system. About 50 percent of patients are unable to walk 25 years after their diagnosis.
Rather than suppress the immune system with a high dose of drugs, a technique called autologous hematopoietic stem cell transplantation (HSCT) hopes to reset the immune system. Hematopoietic simply refers to blood, and autologous means using one’s own cells, instead of those from a donor. So what happens is, after low-dose immunosuppressive drugs are administered to dampen the body’s response, stem cells harvested from the patient’s blood are infused back.
Northwestern’s Richard Burt and colleagues examined the outcomes of 145 MS patients who were treated with this stem cell transplant during trials between 2003 and 2014. Specifically, the patients—who ranged in age from 18 to 60 years old—have relapsing-remitting multiple sclerosis: Symptom flare-ups are followed by periods with little or no symptoms. The patients were periodically tested (2.5 years on average) with the Expanded Disability Status Scale (EDSS), which measures the function of the brainstem and sensory and visual systems, among other things.
The team found significant improvement in 41 patients tested after two years—that’s about 50 percent of the patients who were tested at that interval. For patients tested at four years, they found similar improvement in 23 patients, or 64 percent of the patients tested. Patients who received HSCT showed improvement in physical and cognitive function as well as quality of life, the researchers report. Furthermore, MRI imaging showed a reduction in the volume of brain lesions.
After four years, the relapse-free survival was 80 percent. And the four-year progression-free survival was 87 percent. However, the EDSS score did not improve in patients with secondary-progressive MS or in those who've had MS for more than 10 years.
The results of this preliminary study (observational and without a control group) were published this week in JAMA, the Journal of the American Medical Association. Burt’s team is currently conducting a larger study to compare HSCT with drugs to manage MS that have already been approved by the U.S. Food and Drug Administration. There are currently no therapies for relapsing-remitting multiple sclerosis that can significantly reverse the disability.
Long-term outcomes of autologous hematopoietic stem cell transplantation with reduced-intensity conditioning in multiple sclerosis: physician's and patient's perspectives.
High-dose immunosuppressive therapy (HDIT) with autologous hematopoietic stem cell transplantation (AHSCT) is a promising approach to treatment of multiple sclerosis (MS) patients.
In this paper, we present the long-term outcomes of a prospective single-center study with the analysis of the safety and efficacy of HDIT + AHSCT with reduced-intensity BEAM-like conditioning regimen in 99 MS patients: mean age-35 years old; male/female-39/60; median Expanded Disability Status Scale (EDSS) = 3.5; 43 relapsing/remitting MS, 56 progressive MS. No transplant-related deaths were observed. The mobilization and transplantation procedures were well tolerated.
At 6 months post-transplant, neurological improvement or stabilization was observed in all the patients except one. Cumulative incidence of disease progression was 16.7 % at 8 years after HDIT + AHSCT. Estimated event-free survival at median follow-up of 48.9 months was 80 %: 83.3 % in relapsing/remitting MS vs 75.5 % in progressive MS. Sixty-four patients who did not progress during the first 3 years post-transplant and were monitored for more than 3 years were included in long-term outcome analysis. At the median long-term follow-up of 62 months, 47 % of patients improved by at least 0.5 points on the EDSS scale as compared to baseline and exhibited improvement during the entire period of follow-up; 45 % of patients were stable. No active, new, or enlarging lesions on magnetic resonance imaging were registered in patients without disease progression. AHSCT was accompanied by a significant improvement in patient's quality of life.
Due to the fact that patient selection was quite different to the other studies and that the information about disease activity prior in the disease course and its treatment was inhomogeneous, comparison with the results in the literature should be done with caution. Thus, the risk/benefit ratio of HDIT + AHSCT with reduced-intensity BEAM-like conditioning regimen in our population of MS patients is very favorable. The consistency of our long-term clinical and quality of life results, together with the persistence of improvement, is in favor of the efficacy and safety of this treatment approach in MS patients.