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Rituximab in multiple sclerosis: a comparative study on effectiveness, safety, and patient-reported outcomes
Investigator (PI): Piehl, Fredrik
Performing Organization (PO): (Current): Karolinska Institutet, Department of Clinical Neuroscience / 46 (8) 524 800 00
Supporting Agency (SA): Patient-Centered Outcomes Research Institute (PCORI)
Initial Year: 2016
Final Year: 2023
Record Source/Award ID: PCORI/MS-1511-33196
Funding: Total Award Amount: $8,832,957
Award Type: Contract
Award Information: PCORI: More information and project results (when completed)
Abstract: Multiple sclerosis (MS) is a chronic neurological disease that often leads to disability and decreased quality of life. While there is no cure for MS, several different disease-modifying treatments (DMTs) exist. Randomized clinical trials give an estimate of the comparable efficacy of DMTs, but what was rated as the most important outcome by our stakeholders--long-term disability and quality of life--are poorly reflected in these trials. Moreover, clinical trial populations are not representative of the general MS population and the trials do not address the two key decisional dilemmas we propose to study. The result is that many patients are being treated with suboptimal DMTs instead of one that would provide better efficacy, safety, tolerability, and/or patient satisfaction. Rituximab (RTX), while not formally approved for MS, offers several potential advantages over other MS DMTs, as clinical studies have shown that RTX is potent, relatively safe, convenient, and available worldwide, and the duration of benefit extends far beyond the clearance of the drug. Thus, it is often a good choice for patients requiring escalation therapy, or even as a first-line agent in those planning pregnancy or with health care access issues. However, long-term efficacy and safety in RRTRD are still unknown, and these data--together with quality-of-life measurement--needs to be investigated. The two key decisional dilemmas we propose to study are the following: 1) How does RTX compare with other DMTs in those patients who have already had continued disease activity on a previous DMT? and 2) How does RTX compare with other DMTs in those patients faced with choosing their first MS medication? RTX is being increasingly used as treatment for MS in Sweden and the United States. With the help of the largest publically funded population-based registry of MS patients, we propose to utilize the Swedish Neuro Registry to address these aims. As this registry was established in 2001 with the explicit purpose of studying long-term benefits of DMTs, we can in the beginning of this study identify a large cohort that has at least three years of treatment with RTX or other DMTs. By including new patients over a five-year period, we are confident that we can retrieve data from 6,000 patients, of whom close to 4,000 will be followed in a yearly follow-up program over three to nine years after initiation of therapy, triggering inclusion into the study. We will continue to collect yearly standardized measures of disability and quality of life to address these aims. Safety data will be combined with the multiethnic cohort of Kaiser Permanente Southern California. The contribution of the stakeholders will continue to be essential in the design, conduct, dissemination, and implementation of this study. By studying RTX, a drug that is accessible worldwide, and reporting outcomes that matter to patients and stakeholders, results from the proposed study have the potential to improve the care for RRTRD patients throughout the world.
MeSH Terms:
  • Antirheumatic Agents /*therapeutic use
  • California
  • Clinical Trials as Topic
  • Cohort Studies
  • Comparative Effectiveness Research
  • Decision Making
  • Disabled Persons
  • Health Services Accessibility
  • Humans
  • Multiple Sclerosis /*drug therapy
  • Outcome Assessment, Health Care
  • Patient Safety
  • Patient Satisfaction
  • Patient-Centered Care
  • Quality of Life
  • Registries
  • Rituximab /*therapeutic use
  • Self Report
  • Sweden
Country: Sweden
State:
Zip Code: 171 77
UI: 20164128
CTgovId: NCT03193866
Project Status: Ongoing
Record History: ('2019: Project extended to 2023.\nProject start date corrected to 2016 per PCORI, 2/4/2020\n2017: Project extended to 2022.',)