TY - JOUR
T1 - IgM anti-MAG(+/-) peripheral neuropathy (IMAGiNe) study protocol: An international, observational, prospective registry of patients with IgM M-protein peripheral neuropathies
AU - Hamadeh, T.
AU - van Doormaal, P.T.C.
AU - Pruppers, M.H.J.
AU - van de Mortel, J.P.M.
AU - Hoeijmakers, J.G.J.
AU - Cornblath, D.R.
AU - Vrancken, A.F.J.E.
AU - Faber, C.G.
AU - Notermans, N.C.
AU - Merkies, I.S.J.
AU - IMAGiNe Consortium
N1 - Funding Information:
The consortium recognizes and thanks the Foundation for Peripheral Neuropathy (FPN) and Mr. Louis T. Mazawey for their valuable support and contributions to the study. Additionally, we extend our thanks to the study participants for volunteering their medical information for scientific use, as well as all involved physicians and support personnel at the consortia sites for making this research possible.
Publisher Copyright:
© 2023 The Authors. Journal of the Peripheral Nervous System published by Wiley Periodicals LLC on behalf of Peripheral Nerve Society.
PY - 2023/6
Y1 - 2023/6
N2 - BackgroundInternational consensus on IgM +/- anti-MAG +/- PNP (IgM PNP) is lacking. Despite increasing interest in clinical trials, validated disease-specific measures are needed to adequately capture limitations and changes over time. The IMAGiNe (IgM +/- anti-myelin associated glycoprotein [MAG] peripheral neuropathy) study surges as an international collaboration to create a standardized registry of patients with IgM +/- anti-MAG PNP. The consortium, which currently consists of 11 institutions from 7 countries, presents here the IMAGiNe study design and protocol. AimsFunctional outcome measures will be constructed at the level of impairment, as well as activity and participation. We aim to describe the natural history of the cohort, the role of anti-MAG antibodies, the presence of clinical subtypes, and potential biomarkers. MethodsThe IMAGiNe study is a prospective, observational cohort study with a 3-year follow-up. At each assessment, researchers collect clinical data and subjects complete a list of preselected outcome measures. Among these, the "Pre-Rasch-built Overall Disability Scale (Pre-RODS)" questionnaire will be submitted to Rasch analysis to assess classic and modern clinimetric requirements. ResultsThe final measures will include the IgM-PNP-specific RODS and Ataxia Rating Scale (IgM-PNP-ARS). Descriptions of the disease course, clinical heterogeneity, treatment regimes, variations in laboratory values, and antibody titers will help reach consensus on diagnosis and follow-up strategies. ConclusionThe constructed interval scales will be cross-culturally valid and suitable for use in future clinical trials and daily practice. The ultimate goals are to improve functional individualized assessment, reach international consensus, and lay the foundations for successful designs in future studies.
AB - BackgroundInternational consensus on IgM +/- anti-MAG +/- PNP (IgM PNP) is lacking. Despite increasing interest in clinical trials, validated disease-specific measures are needed to adequately capture limitations and changes over time. The IMAGiNe (IgM +/- anti-myelin associated glycoprotein [MAG] peripheral neuropathy) study surges as an international collaboration to create a standardized registry of patients with IgM +/- anti-MAG PNP. The consortium, which currently consists of 11 institutions from 7 countries, presents here the IMAGiNe study design and protocol. AimsFunctional outcome measures will be constructed at the level of impairment, as well as activity and participation. We aim to describe the natural history of the cohort, the role of anti-MAG antibodies, the presence of clinical subtypes, and potential biomarkers. MethodsThe IMAGiNe study is a prospective, observational cohort study with a 3-year follow-up. At each assessment, researchers collect clinical data and subjects complete a list of preselected outcome measures. Among these, the "Pre-Rasch-built Overall Disability Scale (Pre-RODS)" questionnaire will be submitted to Rasch analysis to assess classic and modern clinimetric requirements. ResultsThe final measures will include the IgM-PNP-specific RODS and Ataxia Rating Scale (IgM-PNP-ARS). Descriptions of the disease course, clinical heterogeneity, treatment regimes, variations in laboratory values, and antibody titers will help reach consensus on diagnosis and follow-up strategies. ConclusionThe constructed interval scales will be cross-culturally valid and suitable for use in future clinical trials and daily practice. The ultimate goals are to improve functional individualized assessment, reach international consensus, and lay the foundations for successful designs in future studies.
KW - anti-MAG antibodies
KW - clinimetry
KW - IgM neuropathies
KW - monoclonal gammopathy of undetermined significance
KW - paraproteinemic neuropathies
KW - DEMYELINATING POLYNEUROPATHY
KW - UNDETERMINED SIGNIFICANCE
KW - MONOCLONAL GAMMOPATHY
KW - OUTCOME MEASURES
KW - SCALE
KW - PARAPROTEINEMIA
KW - DISABILITY
KW - ANTIBODIES
KW - RITUXIMAB
KW - NAARDEN
U2 - 10.1111/jns.12547
DO - 10.1111/jns.12547
M3 - Article
C2 - 37041730
SN - 1085-9489
VL - 28
SP - 269
EP - 275
JO - Journal of the Peripheral Nervous System
JF - Journal of the Peripheral Nervous System
IS - 2
ER -