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Immunosuppression with high-dose i.v. cyclophosphamide and ACTH in progressive multiple sclerosis: cumulative 6-year
J L Carter1, D A Hafler, D M Dawson
1Center for Neurologic Diseases, Brigham and Women's Hospital, Boston, MA 02115.
Insights
High-dose cyclophosphamide and ACTH can stabilize or improve chronic progressive multiple sclerosis (MS) in most patients. However, remissions are not permanent, often requiring retreatment for sustained benefits.
Area of Science:
- Neurology
- Immunology
Background:
- Chronic progressive multiple sclerosis (MS) presents significant management challenges.
- Effective treatment strategies for long-term disease control are needed.
Purpose of the Study:
- To evaluate the efficacy and safety of high-dose intravenous cyclophosphamide and ACTH in chronic progressive MS.
- To identify factors predicting treatment response and analyze long-term outcomes.
Main Methods:
- Retrospective review of 164 patients with chronic progressive MS treated with cyclophosphamide and ACTH.
- Analysis of treatment complications, dosage, response predictors, and disease course post-treatment.
Main Results:
- 81% of patients showed improvement or stabilization at one year post-initial treatment.
- 69% experienced relapse at a mean of 17.6 months; retreatment yielded 70% improvement/stabilization at one year.
- Common side effects included alopecia, nausea, vomiting, and minor infections; no treatment-related deaths or late complications observed.
Conclusions:
- High-dose cyclophosphamide and ACTH is generally well-tolerated and can benefit a majority of chronic progressive MS patients.
- Treatment does not induce permanent remission, necessitating maintenance or retreatment strategies.
- Further research is exploring modified induction regimens and booster injections for sustained remission.
Abstract:
One hundred sixty-four patients with chronic progressive multiple sclerosis (MS) have been treated with a regimen of high-dose IV cyclophosphamide and ACTH over the past 6 years. Their status was reviewed to determine complications associated with treatment, dosage of medication used to induce a remission, factors which may predict a response to therapy, and subsequent course following treatment. One year following initial treatment, 81% of patients were improved or stabilized. Reprogression occurred in 69% of patients at a mean time of 17.6 months. Fifty-eight patients who initially stabilized after treatment and then reprogressed were treated a second time. One year after retreatment, 70% of these patients were improved or stabilized. Alopecia, nausea and vomiting, and minor infections were the most frequent complications. There were no deaths associated with treatment, the complication rate did not change with multiple treatments, and no late complications have yet been observed. Improvement tended to occur in younger patients with shorter disease duration. Although this treatment regimen is generally well tolerated and can favorably affect the course of chronic progressive MS in a majority of patients, a single treatment does not induce a permanent remission, and some form of maintenance treatment or retreatment is required. Current treatment programs involve testing a modified induction regimen and periodic outpatient booster injections to maintain remission.