Project Summary
PCORI has identified multiple sclerosis (MS) as an important research topic. Patients, clinicians, and others want to learn how different treatment strategies, aimed at changing specific symptoms or the overall course of MS, affect patients’ symptoms and quality of life. To address this issue, PCORI launched a funding initiative in 2015 on Treatment of Multiple Sclerosis. This research project is one of the studies PCORI awarded as part this program.
This research project is in progress. PCORI will post the research findings on this page within 90 days after the results are final.
What is the research about?
Multiple sclerosis, or MS, is a serious long-term health problem that affects more than 400,000 people in the United States. MS affects the brain and spinal cord. Symptoms include muscle weakness, trouble with coordination and balance, and thinking and memory problems. MS is a leading cause of disability among young adults in the United States. There is no cure for MS, but treatment can help manage symptoms.
Most people with MS have periods of new or worsening symptoms, called relapses, followed by periods of few or no symptoms, called remission. As MS goes on, symptoms slowly get worse without periods of getting better. Early symptoms may include trouble seeing, numbness, or weakness. Over time, patients may have a hard time walking and need a wheelchair.
In the early stages of MS, patients may take standard, first-line medicines to prevent relapses. Some aggressive medicines work better than first-line medicine for reducing relapse. However, aggressive medicines may cause serious side effects, such as higher risk of infections, autoimmune problems, cancers, and liver failure. Doctors don’t know if either method is better for preventing long-term MS disability. This study is comparing standard medicines versus aggressive medicines on delaying MS from reaching the stage where symptoms continue to get worse and lead to disability.
Who can this research help?
Patients with MS and their doctors can use findings from the study to better understand the risks and benefits of using stronger medicines to prevent or delay disability.
What is the research team doing?
The research team is enrolling 900 adults with MS at approximately 45 study sites throughout the United States and following up with them for up to 4.5 years. The team is assigning patients by chance to one of two groups. Half receive standard medicines, and half receive aggressive medicine. The study is comparing the overall effects of these classes of medicine on disability risk. The team is also looking at whether the medicines’ effect on future disability depends on whether patients are at high or low risk for disability at the start of the study.
The team is tracking patients who are at low risk for disability, but who have a relapse or changes on brain scans that suggest that the first medicine isn’t working well. The team reassigns these patients by chance to get either a different standard medicine or an aggressive medicine.
To assess differences in how often patients develop disability during early stages of MS, the team is following patients over time. The team is also tracking differences between groups in quality of life and disabling symptoms such as fatigue, and thinking and memory problems. To track disease progression, the team is using brain scans.
An advisory committee of people with MS, doctors, statistics experts, and patient advocacy groups is giving input on study design.
Research methods at a glance
|
Design Element |
Description |
|---|---|
| Design | Randomized controlled trial |
| Population | Adults ages 18–60 years with relapsing-remitting MS who did not receive chemotherapy previously or take specific MS medicines for a long period of time |
|
Interventions/Comparators |
|
| Outcomes |
Primary: long-term disability as assessed by neurologic examinations Secondary: patient-reported disability, fatigue, health-related quality of life |
 |
Up to 4.5 years of follow-up for primary outcome |
Video
This PCORI-funded study aims to provide clarity for clinicians on which disease modifying therapy should be prescribed for patients living with multiple sclerosis.
Project Details
Drug Interventions
Other Health Services Interventions
Individuals with Disabilities