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Prospective multicenter observational cohort study of comparative effectiveness of disease modifying treatments for myasthenia gravis (MG)
Investigator (PI): Sanders, Donald B; Narayanaswami, Pushpa
Performing Organization (PO): (Current): Duke University, School of Medicine, Department of Neurology / (919) 684-0053
Supporting Agency (SA): Patient-Centered Outcomes Research Institute (PCORI)
Initial Year: 2017
Final Year: 2021
Record Source/Award ID: PCORI/CER-1609-35953 ; PCORI/APDTO-1609-35953
Funding: Total Award Amount: $2,517,288
Award Type: Contract
Award Information: PCORI: More information and project results (when completed)
Abstract: Myasthenia gravis (MG) is an autoimmune disease that produces weakness of voluntary muscles that can lead to death. Eye muscles are most commonly affected, producing double vision and droopy eyelids. Involvement of other muscles may lead to limb weakness and difficulty swallowing, chewing, talking, and breathing. MG is caused by an immune system attack on specific muscle proteins critical for communication between motor nerves and muscles. These muscle proteins are located at the neuromuscular junction; the most commonly effected protein is the acetylcholine receptor. Because of the autoimmune attack, nerve to muscle communication is impaired resulting in poor muscle contraction and weakness. MG is typically treated initially with drugs that improve the communication at the neuromuscular junction and temporarily relieve weakness, but that do not affect the underlying immune attack (symptomatic therapy). Other therapies that alter immune system function, such as steroids, may also be used (immunosuppressives). Most patients require immunosuppressive therapy, which may be lifelong. Unfortunately, the best approach to treat patients with symptomatic or immunosuppressive therapy is unknown. A study comparing different treatment approaches will greatly improve our ability to treat patients with this potentially fatal disease. We propose to compare the outcomes of different approaches used to treat patients with MG to determine the best treatment regimens from both the physician's and the patient's point of view. We will observe patients with MG treated at 10 clinics in North America and evaluate their outcome after 2 years. We will determine outcomes to treatment that follows recently published international consensus guidance statements for treating MG, as well as to treatment with individual immunosuppressive drugs. At the end of the project we hope to be able to determine which treatments are best and whether adhering to the treatment guidance statements improves outcomes. It is important for patients with MG to be managed in a manner that is most associated with good outcomes. Treating patients appropriately will improve their health, reduce complications, and improve their quality of life. Key stakeholders--including patients with MG, neurologists who specialize in treating MG, payer representatives, and patient organizations--will provide critical input into the design and execution of this study. Among these stakeholders is the Myasthenia Gravis Foundation of America the largest patient organization for patients with MG in North America. These stakeholders will work closely together with us throughout the project to ensure that the study is focused on outcomes that are important to patients.
MeSH Terms:
  • Cohort Studies
  • Comparative Effectiveness Research
  • Foundations
  • Humans
  • Immune System
  • Immunosuppressive Agents /therapeutic use
  • Multicenter Studies as Topic
  • Muscle Contraction
  • Muscle Weakness
  • Muscles /physiopathology
  • Myasthenia Gravis /*therapy
  • Neurology
  • North America
  • Observational Studies as Topic
  • Outcome Assessment (Health Care)
  • Program Development
  • Program Evaluation
  • Quality of Life
Country: United States
State: North Carolina
Zip Code: 27710
UI: 20181375
Project Status: Ongoing
Record History: ('2018: Project extended to 2021.',)