Background: Guillain–Barré syndrome (GBS) and Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) are rare immune-mediated disorders of the peripheral nervous system. They cause significant physical and mental symptoms and functional impairments that impact patients’ daily lives. Current treatment does not effectively prevent the occurrence of these residual symptoms and functional limitations. Patient-Reported Outcomes (PROs) and Patient-Reported Outcome Measures (PROMs) are essential tools that capture patients’ perspectives on their own health, which can be used to assess disease impact and evaluate treatment efficacy. The commonly used Inflammatory Rasch-Built Overall Disability Scale (I-RODS) primarily focuses on activity limitations, social participation, but has been noted to have certain clinimetric shortcomings. This highlights the need for a tailored PROM set that comprehensively assesses relevant aspects of health-related quality of life (HRQL) in patients with GBS or CIDP.
Methods: This prospective mixed-method study consists of a multiphase approach for developing a PROM set in a Dutch adult population with GBS and CIDP. The first phase involves a systematic review and an (online) survey with open-ended questions to identify relevant patient-reported outcomes (PROs), which will be analyzed qualitatively. Subsequently, stakeholder panel meetings will be held with patients and healthcare providers to discuss the identified PROs, including generic and disease specific symptoms and existing suitable PROMs, using the generic Patient Reported Outcomes Measurement Information System (PROMIS) as a basis. Following this, cognitive interviews will be performed to pilot-test a new disease-specific symptom checklist. A Delphi study will then be conducted to achieve consensus on the final PROM set. Finally, a validation study of the selected PROMIS measures and the newly developed disease-specific symptom checklist will be performed. The final phase of the study involves a consensus meeting with the stakeholders to discuss dissemination and implementation strategies for the proposed PROM set.
Discussion: This PROM set is being developed to measure HRQL outcomes relevant for patients with GBS and CIDP. The aim is to develop a tool for clinical practice and research to evaluate the clinical course and effect of treatments from the perspective of patients.
Trial registration: Not applicable.
Keywords: Chronic inflammatory demyelinating polyneuropathy; Guillain–Barré syndrome; Patient-Reported outcome measure; Patient-Reported outcomes.
Relevant COS and Rationale for New Development
We reviewed existing Core Outcome Set (COS) initiatives that may be relevant to our target population and clinical context. One COS referenced in this area focuses specifically on Guillain-Barré Syndrome (GBS) and does not involve patients throughout all stages of development. https://www.comet-initiative.org/Studies/Details/164
Our project includes both GBS and Chronic Inflammatory Demyelinating Polyneuropathy (CIDP), offering a broader scope. In addition to defining a COS, we are developing a comprehensive patient-reported outcome measure (PROM) set, including specific measurement instruments for each selected outcome. This dual approach supports both consensus on what to measure and clarity on how to measure it, enhancing usability in research and clinical settings.
More important, patients have been actively involved at every stage—from initial design to outcome prioritization—and are represented in the steering group. This continuous engagement ensures that the PROM set reflects outcomes that are truly meaningful and relevant to patients.
The need for this new PROM set stems from the absence of a validated, patient-centered framework that addresses both GBS and CIDP. Our work aims to fill this gap by delivering an implementable PROM set that aligns with patient perspectives.
Farah Pelouto¹, Juanita A. Haagsma², Bart C. Jacobs³4, Caroline B. Terwee56
Affiliations:
¹ Department of Neurology, Erasmus MC, University Medical Center Rotterdam, Rotterdam, The Netherlands
² Department of Public Health, Erasmus MC, University Medical Center Rotterdam, Rotterdam, The Netherlands
³ Department of Neurology, Erasmus MC, University Medical Center Rotterdam, Rotterdam, The Netherlands
4 Department of Immunology, Erasmus MC, University Medical Center Rotterdam, Rotterdam, The Netherlands
5 Department of Epidemiology and Data Science, Amsterdam UMC, Vrije Universiteit Amsterdam, Amsterdam, The Netherlands
6 Amsterdam Public Health Research Institute, Methodology, Amsterdam, The Netherlands
Principal Investigator: Bart C. Jacobs
Supervisors: Bart C. Jacobs, Caroline B. Terwee, Juanita A. Haagsma
Disease Category: Neurology
Disease Name: Guillain-Barre syndrome , Chronic inflammatory demyelinating polyneuropathy (CIDP)
Age Range: 16 - 120
Sex: Either
Nature of Intervention: Any
- Clinical experts
- Consumers (patients)
- Patient/ support group representatives
- Researchers
- COS for clinical trials or clinical research
- COS for practice
- Consensus meeting
- Delphi process
- Interview
- Other
- Semi structured discussion
- Survey
- Systematic review
Mixed-method study: systematic review, (online) survey, stakeholder panel meetings, cognitive interviews, Delphi study, and a validation study.