TY - JOUR
T1 - OptimisAtion of Diagnostic Accuracy in idioPathic inflammaTory myopathies (ADAPT study)
T2 - A protocol for a prospective diagnostic accuracy study of multimodality testing in patients suspected of a treatable idiopathic inflammatory myopathy
AU - Walter, Hannah A. W.
AU - Kamperman, Renske G.
AU - Raaphorst, Joost
AU - Verhamme, Camiel
AU - Koelman, Johannes H. T. M.
AU - Potters, Wouter V.
AU - Hemke, Robert
AU - Smithuis, Frank F.
AU - Aronica, Eleonora
AU - van Leeuwen, Ester M. M.
AU - Baars, Paul A.
AU - de Visser, Marianne
AU - van Schaik, Ivo N.
AU - Bossuyt, Patrick M. M.
AU - van der Kooi, Anneke J.
N1 - Funding Information: Funding The authors have not declared a specific grant for this research from any funding agency in the public, commercial or not-for-profit sectors. Competing interests INvS chaired a steering committee for a CSL-Behring study investigating the safety and efficacy of SCIg in CIDP and received departmental honoraria for serving on scientific advisory boards for CSL-Behring and Kedrion. He received departmental research support from The Netherlands Organisation for Scientific Research, and from the Dutch Prinses Beatrix Spierfonds. All lecturing and consulting fees for INvS were donated to the Stichting Klinische Neurologie, a local foundation that supports research in the field of neurological disorders. He served on the editorial board of the Cochrane Neuromuscular Disease Group, was a member of the organising committee of the Inflammatory Neuropathy Consortium (INC), a standing committee of the Peripheral Nerve Society and was a member of the Scientific Board of the Kreuth III meeting on the optimal use of plasma-derived medicinal products, especially coagulation factors and normal immunoglobulins organised under the auspices of the European Directorate for the Quality of Medicines & HealthCare (EDQM). AJvdK received departmental honoraria for serving on a scientific advisory board for ArgenX. JR received departmental research support from the Dutch Prinses Beatrix Spierfonds, Dutch ALS foundation and Sanquin Plasma Products. MdV is a member of the Data Monitoring Committee of Novartis Pharma AG and chair of the Independent Data Monitoring Committee of Dynacure. Publisher Copyright: ©
PY - 2021/12/1
Y1 - 2021/12/1
N2 - Introduction Idiopathic inflammatory myopathies (IIMs) excluding inclusion body myositis (IBM) are a group of heterogeneous autoimmune disorders characterised by subacute-onset and progressive proximal muscle weakness, which are frequently part of a multisystem autoimmune disorder. Reaching the diagnosis can be challenging, and no gold standard for the diagnosis of IIM exists. Diagnostic modalities include serum creatine kinase activity, muscle imaging (MRI or ultrasound (US)), electromyography (EMG), myositis autoantibody testing and muscle biopsy. Several diagnostic criteria have been developed for IIMs, varying in reported sensitivity and specificity. Hypothesis We hypothesise that an evidence-based diagnostic strategy, using fewer and preferably the least invasive diagnostic modalities, can achieve the accuracy of a complete panel of diagnostic tests, including MRI, US, EMG, myositis-specific autoantibody testing and muscle biopsy. Methods and analysis The OptimizAtion of Diagnostic Accuracy in idioPathic inflammaTory myopathies study is a prospective diagnostic accuracy study with an over-complete study design. 100 patients suspected of an IIM excluding IBM will be included. A reference diagnosis will be assigned by an expert panel using all clinical information and all results of all ancillary tests available, including 6 months of follow-up. Several predefined diagnostic strategies will be compared against the reference diagnosis to find the optimal diagnostic strategy. Ethics and dissemination Ethical approval was obtained from the medical ethics committee of the Academic Medical Centre, University of Amsterdam, The Netherlands (2019-814). The results will be distributed through conference presentations and peer-reviewed publications. Trial registration number Netherlands trial register; NL8764.
AB - Introduction Idiopathic inflammatory myopathies (IIMs) excluding inclusion body myositis (IBM) are a group of heterogeneous autoimmune disorders characterised by subacute-onset and progressive proximal muscle weakness, which are frequently part of a multisystem autoimmune disorder. Reaching the diagnosis can be challenging, and no gold standard for the diagnosis of IIM exists. Diagnostic modalities include serum creatine kinase activity, muscle imaging (MRI or ultrasound (US)), electromyography (EMG), myositis autoantibody testing and muscle biopsy. Several diagnostic criteria have been developed for IIMs, varying in reported sensitivity and specificity. Hypothesis We hypothesise that an evidence-based diagnostic strategy, using fewer and preferably the least invasive diagnostic modalities, can achieve the accuracy of a complete panel of diagnostic tests, including MRI, US, EMG, myositis-specific autoantibody testing and muscle biopsy. Methods and analysis The OptimizAtion of Diagnostic Accuracy in idioPathic inflammaTory myopathies study is a prospective diagnostic accuracy study with an over-complete study design. 100 patients suspected of an IIM excluding IBM will be included. A reference diagnosis will be assigned by an expert panel using all clinical information and all results of all ancillary tests available, including 6 months of follow-up. Several predefined diagnostic strategies will be compared against the reference diagnosis to find the optimal diagnostic strategy. Ethics and dissemination Ethical approval was obtained from the medical ethics committee of the Academic Medical Centre, University of Amsterdam, The Netherlands (2019-814). The results will be distributed through conference presentations and peer-reviewed publications. Trial registration number Netherlands trial register; NL8764.
KW - Neuromuscular disease
KW - Neuropathology
KW - Radiology & imaging
KW - Rheumatology
KW - Ultrasound
UR - http://www.scopus.com/inward/record.url?scp=85122195441&partnerID=8YFLogxK
UR - https://www.scopus.com/inward/record.uri?partnerID=HzOxMe3b&scp=85122195441&origin=inward
UR - https://www.ncbi.nlm.nih.gov/pubmed/34903547
U2 - https://doi.org/10.1136/bmjopen-2021-053594
DO - https://doi.org/10.1136/bmjopen-2021-053594
M3 - Article
C2 - 34903547
SN - 2044-6055
VL - 11
JO - BMJ Open
JF - BMJ Open
IS - 12
M1 - e053594
ER -