Workshop reportReport of the second Outcome Measures in Myotonic Dystrophy type 1 (OMMYD-2) international workshop San Sebastian, Spain, October 16, 2013
Introduction
For this second workshop, a total of 46 attendees (clinicians and researchers working in myotonic dystrophy type 1 (DM1)) from France, Canada, USA, the UK, Germany, Sweden, England, Hungary, the Netherlands and Italy met in San Sebastian, Spain on October 16, 2013 (see participants' list at the end of the report). The overall goal of this second Outcome Measures in myotonic dystrophy type 1 (OMMYD-2) workshop was to continue to select the best outcome measures (OM) to be used in clinical trials based on metrological properties and experience of experts. However, data concerning the validity and the reliability of these OM in the DM1 population are scarce. The more specific objective of the OMMYD community is therefore to document the metrological properties of the selected OM with DM1 patients between workshops. Attendees were divided into six Special Interest Groups (SIG): (1) Cognitive Functions; (2) Patient-reported Outcome Measures (in replacement of Quality of Life from OMMYD-1); (3) Functional Capacity Outcome Measures (in replacement of Upper and Lower Extremity Functions from OMMYD-1); (4) Muscle Testing and Training; (5) Disease Severity Index; and (6) Sleepiness/Fatigue/Apathy (new SIG).
The OMMYD conference is based on the methodology developed by the OMERACT group in the rheumatology field for the development and selection of outcome measures [1]. Criteria for the endorsement of a measure workshop are based on the OMERACT Filter. The three component criteria are:
- 1.
Truth: is the measure truthful, does it measure what it intends to measure? Is the result unbiased and relevant? This criterion captures the issues of face, content, construct and criterion validity.
- 2.
Discrimination: does the measure discriminate between situations that are of interest? The situations can be states at one time (for classification or prognosis) or states at different times (to measure change). This criterion captures the issues of reliability and sensitivity to change.
- 3.
Feasibility: can the measure be applied easily, given constraints of time, money, and interpretability? This criterion addresses the pragmatic reality of the use of the measure, one that may be decisive in determining a measure's success.
Section snippets
Session 1. Cognitive functions Special Interest Group
The session was chaired by Dr. Giovanni Meola from the University of Milan, Italy.
Session 2. Patient-reported outcome measure Special Interest Group
The session was chaired by Chad Heatwole, MD, MSCI from the University of Rochester, USA.
Session 3. Functional capacity outcome measures Special Interest Group
The session was chaired by Professor Mario Leone from the Interdisciplinary Research Group on Neuromuscular Disorders and director of the Centre for Interdisciplinary Research on Quality and Healthy Lifestyle, Université du Québec à Chicoutimi, Canada.
Session 4. Muscle testing and training Special Interest Group
The session was chaired by Professor Luc J. Hébert from the Université Laval, Québec, Canada.
Session 5. DSI-DM1 Special Interest Group
The session was chaired by Professor Cynthia Gagnon from the Interdisciplinary Research Group on Neuromuscular Disorders, Université de Sherbrooke, Canada.
First draft of the DSI-DM1 was presented at OMMYD-1 (November 30, 2011) and it includes a total of 39 items. Of these items, 6 were considered as risk factors and then kept separately from the scale, 8 items were excluded, and 13 items require more discussion. This second meeting was based on this preliminary selection and commentaries. All
Session 6. Sleepiness/fatigue/apathy Special Interest Group
The session was chaired by Dr. Luc Laberge from ÉCOBES – Recherche et transfert, Cégep de Jonquière, Saguenay, Canada.
Research agenda for the OMMYD working groups
As discuss above, SIGs pointed out a lack of data concerning some specific instruments or methods. They agreed together to accomplish some small projects prior to OMMYD-3.
Cognitive-Special Interest Group
Giovanni Meola (Italy)
Stefan Winblad (Sweden)
Mario Gomez-Pereira (France)
Louis Richer (Canada)
Nicolas Sergeant (France)
Sarah Howe (Canada, reporter)
Chris Turner (United Kingdom)
Fransisco Jose Fernando-Gomez (Spain)
Asger Frost (Denmark)
Geneviève Gourdon (France)
Patient-reported outcome measure-Special Interest Group
Chad Heatwole (USA)
Valeria A. Sansone (Italy)
Michael Rose (United Kingdom)
Nicholas Johnson (USA)
Annie Plourde (Canada)
Cindy Nowinsky (USA)
Upper and lower extremities-Special Interest Group
Mario Leone (Canada)
Benedikt Schoser (Germany)
Marie Kierkegaard (Sweden)
Edith H. Cup (Netherlands)
Acknowledgments
This meeting was made possible by the financial support of the Association Française contre les Myopathies (AFM) (MED2 2013) and the Marigold Foundation. We would like to thank Sarah Howe for her clerical support during Cognitive SIG session. We also thank Isabelle Côté for having coordinated the writing of this report and for the conference. The chair of the Muscle SIG would like to acknowledge the great work done by Émilie Petitclerc, PT, MSc(c) who has conducted a thorough literature review
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