Contributions
Abstract: P874
Type: Poster
Abstract Category: Clinical aspects of MS - MS symptoms
Background: Neuromyelitis Optica Spectrum Disorder (NMOSD) is a rare autoimmune disease that frequently causes serious neurological disability. Generation of pathogenic antibodies targeting the water channel, aquaporin 4 (AQP4) is the hallmark of seropositive NMOSD. Many uncertainties remain concerning etiology, epidemiology, pathogenesis, and long-term outcomes. These unmet needs and the imperative to improve therapeutic options motivated the creation of a patient registry and biorepository devoted to solving NMOSD.
Methods: The Collaborative International Research in Clinical and Longitudinal Experience for NMOSD Studies (CIRCLES) registry and biorepository was established through consensus. Using a standardized protocol, patients with NMOSD or comparative controls were recruited into the CIRCLES program through clinical centers and patient advocacy events at five geographically dispersed sites across North America beginning in November 2013. At six-month intervals, demographic information and medical history were collected, including disease phenotype, comorbidity, medication, vaccination, environmental exposure, and activities of daily living. Biospecimens were collected and archived at each interval.
Results: As of April 2016, 562 participants have been enrolled: 440 NMOSD cases and 123 controls (25 comparative disease [21%] and 96 healthy individuals [79%]). Demographic features observed include: gender ratio, 6.1:1 (86% female); median age at enrollment, 43.6 (IQR 32.1, 55.2); racial distribution, 275(63%) white; 76(17%) black/African American; 41(9%) Hispanic/Latino; 37(8%) Asian; 10(2%) other. Biospecimens have been collected on 93% of cases and 97% of controls. Among cases, 82 (19%) have two longitudinal biospecimens, and 61 (14%) have three or more. Preliminary analyses show that compared to healthy controls, NMOSD patients are more likely to have a first degree blood relative diagnosed with an autoimmune disease (49% vs. 37%; P=0.02).
Interpretation: CIRCLES represents a robust registry and biorepository created using standardized methods to facilitate consistency in studying NMOSD. This program has already begun to offer new hypotheses intended to promote solutions to NMOSD.
Prospectus: As CIRCLES expands in geographic diversity and temporal experience, its impact on understanding NMOSD should increase. Thus, the CIRCLES program will accelerate solutions to NMOSD by providing a unique and much needed resource for all NMOSD stakeholders.
Disclosure: Funding for this project is provided by The Guthy-Jackson Charitable Foundation.
Lawrence Cook: Nothing to Disclose
John Rose: Nothing to Disclose
Jessica Alvey: Nothing to Disclose
Anna Jolley: Nothing to Disclose
Rene Kuhn: Nothing to Disclose
Melissa Pederson: Nothing to Disclose
Megan Keneeally: Nothing to Disclose
Renee Rodriguez: Nothing to Disclose
Daniel Behne: Nothing to Disclose
Derek Blackway: Nothing to Disclose
Brian Coords: Nothing to Disclose
Michael Levy: Nothing to Disclose
Jacinta Behne: Nothing to Disclose
Jeffrey Bennett: Nothing to Disclose
May Han: Nothing to Disclose
Maureen Mealy: Nothing to Disclose
Anthony Traboulsee is a consultant for Novartis, Genzyme, Roche and a principal investigator on clinical trials with Biogen, Genzyme, Roche, and Chugai.
Robert Carruthers speakers bureau Biogen, Genzyme, Teva; grant support Novartis, MedImmune, Teva, Guthy-Jackson Charitable Foundation; consulting fees Novartis, Serono, Genzyme
Michael Yeaman is an associate editor for PLoS Pathogens, and study section reviewer for the U.S. National Institutes of Health; holds patents for vaccines and immunotherapies targeting drug-resistant pathogens & infections, anti-infective biologicals, and anti-infective small molecules; is Founder and 170 shareholder of NovaDigm Therapeutics; is Founder and shareholder of Metacin, Inc.; has received research support from the United States Department of Defense, and National Institutes of Health; receives license fees or royalty payments for vaccines (NovaDigm); holds U.S. copyrights and receives license fees or royalty payments for original music compositions and performances in neuroscience research (Metacin); is an advisor to the Guthy-Jackson Charitable Foundation focusing on its mission to solve NMO.
Terrence Blaschke is an adviser to The Guthy-Jackson Charitable Foundation and the Bill & Melinda Gates Foundation
Terry Smith is an advisor to the Guthy-Jackson Charitable Foundation. He is scientific and medical director of the Graves" disease and Thyroid Foundation, and has been a paid consultant for Lipothera, River Vision, Wyeth, Merck, and Novartis. He holds patents for anti-IGF-1 receptor monoclonal therapy in autoimmune diseases.
Abstract: P874
Type: Poster
Abstract Category: Clinical aspects of MS - MS symptoms
Background: Neuromyelitis Optica Spectrum Disorder (NMOSD) is a rare autoimmune disease that frequently causes serious neurological disability. Generation of pathogenic antibodies targeting the water channel, aquaporin 4 (AQP4) is the hallmark of seropositive NMOSD. Many uncertainties remain concerning etiology, epidemiology, pathogenesis, and long-term outcomes. These unmet needs and the imperative to improve therapeutic options motivated the creation of a patient registry and biorepository devoted to solving NMOSD.
Methods: The Collaborative International Research in Clinical and Longitudinal Experience for NMOSD Studies (CIRCLES) registry and biorepository was established through consensus. Using a standardized protocol, patients with NMOSD or comparative controls were recruited into the CIRCLES program through clinical centers and patient advocacy events at five geographically dispersed sites across North America beginning in November 2013. At six-month intervals, demographic information and medical history were collected, including disease phenotype, comorbidity, medication, vaccination, environmental exposure, and activities of daily living. Biospecimens were collected and archived at each interval.
Results: As of April 2016, 562 participants have been enrolled: 440 NMOSD cases and 123 controls (25 comparative disease [21%] and 96 healthy individuals [79%]). Demographic features observed include: gender ratio, 6.1:1 (86% female); median age at enrollment, 43.6 (IQR 32.1, 55.2); racial distribution, 275(63%) white; 76(17%) black/African American; 41(9%) Hispanic/Latino; 37(8%) Asian; 10(2%) other. Biospecimens have been collected on 93% of cases and 97% of controls. Among cases, 82 (19%) have two longitudinal biospecimens, and 61 (14%) have three or more. Preliminary analyses show that compared to healthy controls, NMOSD patients are more likely to have a first degree blood relative diagnosed with an autoimmune disease (49% vs. 37%; P=0.02).
Interpretation: CIRCLES represents a robust registry and biorepository created using standardized methods to facilitate consistency in studying NMOSD. This program has already begun to offer new hypotheses intended to promote solutions to NMOSD.
Prospectus: As CIRCLES expands in geographic diversity and temporal experience, its impact on understanding NMOSD should increase. Thus, the CIRCLES program will accelerate solutions to NMOSD by providing a unique and much needed resource for all NMOSD stakeholders.
Disclosure: Funding for this project is provided by The Guthy-Jackson Charitable Foundation.
Lawrence Cook: Nothing to Disclose
John Rose: Nothing to Disclose
Jessica Alvey: Nothing to Disclose
Anna Jolley: Nothing to Disclose
Rene Kuhn: Nothing to Disclose
Melissa Pederson: Nothing to Disclose
Megan Keneeally: Nothing to Disclose
Renee Rodriguez: Nothing to Disclose
Daniel Behne: Nothing to Disclose
Derek Blackway: Nothing to Disclose
Brian Coords: Nothing to Disclose
Michael Levy: Nothing to Disclose
Jacinta Behne: Nothing to Disclose
Jeffrey Bennett: Nothing to Disclose
May Han: Nothing to Disclose
Maureen Mealy: Nothing to Disclose
Anthony Traboulsee is a consultant for Novartis, Genzyme, Roche and a principal investigator on clinical trials with Biogen, Genzyme, Roche, and Chugai.
Robert Carruthers speakers bureau Biogen, Genzyme, Teva; grant support Novartis, MedImmune, Teva, Guthy-Jackson Charitable Foundation; consulting fees Novartis, Serono, Genzyme
Michael Yeaman is an associate editor for PLoS Pathogens, and study section reviewer for the U.S. National Institutes of Health; holds patents for vaccines and immunotherapies targeting drug-resistant pathogens & infections, anti-infective biologicals, and anti-infective small molecules; is Founder and 170 shareholder of NovaDigm Therapeutics; is Founder and shareholder of Metacin, Inc.; has received research support from the United States Department of Defense, and National Institutes of Health; receives license fees or royalty payments for vaccines (NovaDigm); holds U.S. copyrights and receives license fees or royalty payments for original music compositions and performances in neuroscience research (Metacin); is an advisor to the Guthy-Jackson Charitable Foundation focusing on its mission to solve NMO.
Terrence Blaschke is an adviser to The Guthy-Jackson Charitable Foundation and the Bill & Melinda Gates Foundation
Terry Smith is an advisor to the Guthy-Jackson Charitable Foundation. He is scientific and medical director of the Graves" disease and Thyroid Foundation, and has been a paid consultant for Lipothera, River Vision, Wyeth, Merck, and Novartis. He holds patents for anti-IGF-1 receptor monoclonal therapy in autoimmune diseases.