There is significant variability in outcomes evaluated in randomized controlled trials (RCTs) of neuromyelitis optica spectrum disorder (NMOSD). To date, several drugs have been studied and approved for use in patients with NMOSD, and the number of available treatments and trials is expected to increase in coming years. Therefore, defining the appropriate outcomes for future NMOSD clinical trials and identifying the best tools to assess these outcomes is a priority in the field. The main objective is to develop the core outcome set for use in all clinical trials for adult patients with AQP4-IgG-seropositive NMOSD.
The methodological design of this project will be guided by the recommendations from the COMET Initiative and the COSMIN Initiative. The study protocol is developed in accordance with the Core Outcome Set-STAndardised Protocol (COS-STAP) statement, and the final study publications will be written based on the Core Outcome Set-STAndards for Reporting (COS-STAR) statement. The target population of the present study is adult patients with AQP4-IgG-seropositive NMOSD, regardless of their disability.
Potentially relevant COS: There is no identified or developed COS for NMOSD.
- Cristian Eduardo Navarro, MD MSc PhD(c) from Universidad de Antioquia (principal investigator).
- Iván Darío Flórez, MD MSc PhD from Universidad de Antioquia and McMaster University (supervisor).
- Sarah Gorst, BSc MSc PhD from University of Liverpool (supervisor).
- Carlos Enrique Yepes-Delgado, MD MSc PhD from Universidad de Antioquia (supervisor).
- María Isabel Zuluaga, MD from Medicarte S.A (supervisor).
Disease Category: Neurology
Disease Name: Neuromyelitis optica spectrum disorder
Age Range: 18 - 99
Sex: Either
Nature of Intervention: Drug
- Clinical experts
- Conference participants
- Consumers (caregivers)
- Consumers (patients)
- Economists
- Epidemiologists
- Journal editors
- Methodologists
- Patient/ support group representatives
- Policy makers
- Researchers
- COS for clinical trials or clinical research
- COS for practice
- Consensus meeting
- Delphi process
- Interview
- Literature review
- Survey
- Other
This study will be developed in three stages:
- Stage 1: A scoping review with three aims; 1) to determine the list of outcomes which will be part of the COS; 2) to describe the minimal clinically important difference (MID) for each reported outcome; and 3) to explore the perspectives, the values, and the preferences of patients with NMOSD published in qualitative studies.
- Stage 2: A qualitative study with AQP4-IgG-seropositive NMOSD patients, caregivers, and their representatives that explores how patients evaluate treatment outcomes concerning their expectations, prior experiences, level of knowledge, and socio-cultural context. These results will be compared with the results of the scoping review to assess whether the RCTs consider patients' preferences among the outcomes they evaluated.
- Stage 3: A Delphi consensus to define which outcomes will constitute the COS. Following the COS-STAD recommendations, the key stakeholders that will be invited to participate are patients, healthcare professionals, researchers, and policymakers. Quantitative data will be generated by asking the key stakeholders to score the importance of outcomes during an online Delphi survey and by making a final decision as to what outcomes should be part of the COS.
The COS will be disseminated by presentations at conferences, publication in a peer-review journals, publications via academic social media, and identifying NMOSD ambassadors not included in the Delphi panel. This process will be subject to periodic review to ensure strategies are effective and to identify new barriers to implementation.
Our goal is to maintain an updated COS through periodic scoping reviews and ongoing consultation with stakeholders. This approach will allow us to assess the necessity of repeating the voting process, particularly if new outcomes arise for evaluation as our understanding of the disease's natural history, pathophysiology, and treatment impacts evolves.