2014-12-29 17:45
Investigators have just released the interim results of a five-year study of high-dose immunosuppressive therapy (HDIT) and hematopoietic stem cell therapy in 24 individuals with multiple sclerosis. After three years, most of the participants showed improvements in neurological function and sustained remission of active relapsing-remitting MS (RRMS).
The new JAMA Neurology article reports the findings of the Hematopoietic Cell Transplantation for Relapsing-Remitting Multiple Sclerosis (HALT-MS) study. Basically, the idea is that use of HDIT supported by autologous (use of a patient’s own cells) hematopoietic stem cell infusions (transplantation) will stop disease activity in individuals who have poor prognosis RRMS.
In this study, autologous hematopoietic cell transplantation involved removing CD34 stem cells and storing them while the individuals were treated with high-dose immunosuppressive therapy; that is, the chemotherapy drugs carmustine, cytarabine, etoposide, and melphalan. The stored hematopoietic stem cells, which are responsible for the formation of blood, were then transfused into the patient’s bloodstream
At the three-year evaluation point, the authors found that, without the use of any maintenance therapy:
- Overall rate of event-free survival (i.e., survival without loss of neurologic function, new lesions seen on imaging, or clinical relapse) was 78.4 percent
- Progression-free survival was 90.9 percent
- Clinical relapse-free survival was 86.3 percent
- Quality of life, functional scores, and neurological disability all also improved
The authors concluded that the combination of high-dose immunosuppressive therapy and hematopoietic stem cell transplantation “may represent a potential therapeutic option for patients with MS in whom conventional immunotherapy fails.” However, they also emphasized that “longer follow-up is needed to determine the durability of the response.”
This combination therapeutic approach is not without side effects. The most common adverse events were cytopenias (64%), infections (56%), gastrointestinal disorders (36%), metabolism disorders (32%), and nervous system issues (24%), such as headache.
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