TY - JOUR
T1 - IgM anti-MAG± peripheral neuropathy (IMAGiNe) study protocol
T2 - An international, observational, prospective registry of patients with IgM M-protein peripheral neuropathies
AU - the IMAGiNe Consortium
AU - Hamadeh, Tatiana
AU - van Doormaal, Perry T.C.
AU - Pruppers, Mariëlle H.J.
AU - van de Mortel, Johannes P.M.
AU - Hoeijmakers, Janneke G.J.
AU - Cornblath, David R.
AU - Vrancken, Alexander F.J.E.
AU - Faber, Catharina G.
AU - Notermans, Nicolette C.
AU - Merkies, Ingemar S.J.
AU - Basta, Ivana
AU - van den Bergh, Peter Y.K.
AU - Bianco, Mariangela
AU - Briani, Chiara
AU - Cauquil, Cécile
AU - Carr, Aisling S.
AU - Cornblath, David R.
AU - Cutellè, Claudia
AU - D'Sa, Shirley
AU - van Doormaal, Perry T.C.
AU - Dubuisson, Nicolas
AU - Echaniz-Laguna, Andoni
AU - Elsheikh, Bakri H.
AU - Faber, Catharina G.
AU - Goedee, H. Stephan
AU - Hamadeh, Tatiana
AU - Harbo, Thomas
AU - Hoeijmakers, Janneke G.J.
AU - Karlin, Lionel
AU - Khoshnoodi, Mohammad
AU - Labeyrie, Céline
AU - Lunn, Michael P.
AU - Marasca, Manuele
AU - Marques, Wilson
AU - Merkies, Ingemar S.J.
AU - Merle, Hélène
AU - Minnema, Monique C.
AU - van de Mortel, Johannes P.M.
AU - Nascimento, Osvaldo J.M.
AU - Nobile-Orazio, Eduardo
AU - Notermans, Nicolette C.
AU - Palibrk, Aleksa
AU - Pascual-Goñi, Elba
AU - Peric, Stojan
AU - Querol, Luis
AU - Salvalaggio, Alessandro
AU - Stino, Amro M.
AU - Svahn, Juliette
AU - Tejada, Clara
AU - Thomas, Simone
N1 - 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 - Background: International 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. Aims: Functional 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. Methods: The 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. Results: The 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. Conclusion: The 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 - Background: International 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. Aims: Functional 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. Methods: The 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. Results: The 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. Conclusion: The 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 - IgM neuropathies
KW - anti-MAG antibodies
KW - clinimetry
KW - monoclonal gammopathy of undetermined significance
KW - paraproteinemic neuropathies
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U2 - 10.1111/jns.12547
DO - 10.1111/jns.12547
M3 - Article
C2 - 37041730
AN - SCOPUS:85153308364
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 -