Inflammatory Neuropathy Consortium base (INCbase): a protocol of a global prospective observational cohort study for the development of a prediction model for treatment response in chronic inflammatory demyelinating polyneuropathy
Milou R. Michael,
Luuk Wieske,
Jeffrey A. Allen,
Michael P. Lunn,
Kathrin Doppler,
Cheng-Yin Tan,
Haruki Koike,
Lars K. Markvardsen,
Mahima Kapoor,
Sung-Tsang Hsieh,
Eduardo Nobile-Orazio,
Bart C. Jacobs,
Yusuf A. Rajabally,
Ivana Basta,
Paolo Ripellino,
Luis Querol,
Filip Eftimov,
on behalf of the INCbase Consortium
Affiliations
Milou R. Michael
Department of Neurology and Neurophysiology, Location AMC, Amsterdam Neuroscience, Amsterdam UMC, University of Amsterdam
Luuk Wieske
Department of Neurology and Neurophysiology, Location AMC, Amsterdam Neuroscience, Amsterdam UMC, University of Amsterdam
Jeffrey A. Allen
Department of Neurology, University of Minnesota
Michael P. Lunn
Centre for Neuromuscular Disease, National Hospital for Neurology and Neurosurgery
Kathrin Doppler
Department of Neurology, University Hospital Würzburg
Cheng-Yin Tan
Division of Neurology, Department of Medicine, University of Malaya
Haruki Koike
Division of Neurology, Department of Internal Medicine, Faculty of Medicine, Saga University
Lars K. Markvardsen
Department of Neurology, Aarhus University Hospital
Mahima Kapoor
Neurology Department, Alfred Hospital
Sung-Tsang Hsieh
Department of Neurology, National Taiwan University Hospital
Eduardo Nobile-Orazio
Neuromuscular and Neuroimmunology Service, Department of Medical Biotechnology and Translational Medicine, IRCCS Humanitas Research Institute, University of Milan
Bart C. Jacobs
Department of Neurology and Immunology, Erasmus MC University Medical Center
Yusuf A. Rajabally
Neuromuscular Service, Neurology, Queen Elizabeth Hospital Birmingham
Ivana Basta
Neurology Clinic, Medical faculty, University Clinical Center of Serbia, University of Belgrade
Paolo Ripellino
Department of Neurology, Neurocenter of Southern Switzerland EOC
Luis Querol
Department of Neurology, Hospital de la Santa Creu i Sant Pau
Filip Eftimov
Department of Neurology and Neurophysiology, Location AMC, Amsterdam Neuroscience, Amsterdam UMC, University of Amsterdam
on behalf of the INCbase Consortium
Department of Neurology and Neurophysiology, Location AMC, Amsterdam Neuroscience, Amsterdam UMC, University of Amsterdam
Abstract Background INCbase is an international, multicenter prospective observational study using a customizable web-based modular registry to study the clinical, biological and electrophysiological variation and boundaries of chronic inflammatory demyelinating polyneuropathy (CIDP). The primary objective of INCbase is to develop and validate a clinical prediction model for treatment response. Methods All patients meeting clinical criteria for CIDP can be included in INCbase. Collected data include demographics, clinical history, diagnostics and various domains of clinical outcomes. Data is collected at a minimum of every 6 months for two years, and more frequently at the discretion of the investigational site to allow for assessment of unexpected changes in treatment response or clinical status. Participants can be enrolled in various sub-studies designed to capture data relevant to specific groups of interest. Data is entered directly into the web-based data entry system by local investigators and/or participants. Collection and local storage of biomaterial is optional. To develop a clinical prediction model for treatment response, newly diagnosed patients with active disease warranting start of first-line treatment will be included. The study population will be split into a development and validation cohort. Univariate and multivariate logistic regression analysis will be used to identify and combine predictors at start of treatment for treatment response at six months. Model performance will be assessed through discrimination and calibration in an external validation cohort. The externally validated prediction model will be made available to researchers and clinicians on the INCbase website. Discussion With this study, we aim to create a clinically relevant and implementable prediction model for treatment response to first line treatments in CIDP. INCbase enrollment started in April 2021, with 29 centers across 8 countries and 303 patients participating to date. This collaborative effort between academia, patient advocacy organizations and pharmaceutical industry will deepen our understanding of how to diagnose and treat CIDP.
