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Abstract Details

Collaboration and Development of Standard Operating Procedures for Prospective Evaluation and Collection of Biospecimens in Neurosarcoidosis
Autoimmune Neurology
P8 - Poster Session 8 (5:30 PM-6:30 PM)
14-005
Sarcoidosis is a multisystem inflammatory disorder characterized by granulomatous inflammation of affected organs. Central or peripheral nervous system involvement is present in 5-26% of patients with sarcoidosis. There is a need to establish multicenter collaborations to prospectively define clinical profiles and neuroimaging findings and secure biological samples to advance our understanding of the pathogenesis of NS and identify biomarkers of the disease.

To establish a comprehensive research protocol for clinical assessment of patients with neurosarcoidosis (NS) to precisely characterize clinical phenotypes, pathogenic mechanisms, and biomarkers in a longitudinal observational cohort study.

We designed a protocol to recruit newly diagnosed or established patients with sarcoidosis with evidence of active neurological disease (defined by clinical manifestations and imaging or cerebrospinal fluid findings). We used clinical tools to characterize neurological function longitudinally. We evaluated mobility, cognition, processing speed, and disability using the Timed 25-Foot Walk, Montreal Cognitive Assessment, Symbol Digit Modalities Test, and Expanded Disability Status Scale. To comprehensively assess patient-reported outcomes, we evaluated health status, fatigue, pain, bladder and bowel control, mental health, perceived deficits, and walking scale. Abbreviated scale versions were selected when available. We established standard operating procedures for neuroimaging assessment, biospecimen collection, and biobanking.

To date, 24 patients with active NS have been recruited at two sites. 50% of patients are male. The median (IQR) age of all enrollees is 55 years (51-62). 

In this multicenter, prospective, observational, federally funded study of NS, we establish an approach to obtain clinical and biological research data along with the standard of care clinical management to facilitate a reliable characterization of clinical and biological profiles in NS patients. This standardized approach can be applied to future studies of NS patients. 

Authors/Disclosures
Susana C. Dominguez Penuela, MD (Johns Hopkins University School of Medicine)
PRESENTER
Dr. Dominguez Penuela has nothing to disclose.
Paula Barreras, MD (Cedars-Sinai Medical Center) The institution of Dr. Barreras has received research support from Foundation for Sarcoidosis Research. The institution of Dr. Barreras has received research support from American Academy of Neurology.
Greer Waldrop, MD (UCSF) Dr. Waldrop has nothing to disclose.
No disclosure on file
No disclosure on file
Kathryn Fitzgerald, PhD (Johns Hopkins University) The institution of Dr. Fitzgerald has received research support from NIH. The institution of Dr. Fitzgerald has received research support from National MS Society.
Samuel J. Pleasure, MD, PhD (UCSF) Dr. Pleasure has received personal compensation in the range of $10,000-$49,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for JAMA Neurology. The institution of Dr. Pleasure has received research support from NINDS, NIMH, NIDA, National MS Society, Brain Research Foundation, John Merck Fund, Whitehall Foundation, Lupus Research Association, Juvenile Diabetes Research Foundation.
No disclosure on file
Michael R. Wilson, MD, FAAN (University of California San Francisco) Dr. Wilson has received personal compensation in the range of $10,000-$49,999 for serving as an officer or member of the Board of Directors for Delve Bio. Dr. Wilson has stock in Delve Bio. The institution of Dr. Wilson has received research support from Genentech / Roche. The institution of Dr. Wilson has received research support from NIH. The institution of Dr. Wilson has received research support from UCSF Weill Institute for Neurosciences. The institution of Dr. Wilson has received research support from Novartis. The institution of Dr. Wilson has received research support from National Multiple Sclerosis Society. The institution of Dr. Wilson has received research support from Fanconi Anemia Research Foundation. The institution of Dr. Wilson has received research support from Department of Defense. Dr. Wilson has received intellectual property interests from a discovery or technology relating to health care. Dr. Wilson has received intellectual property interests from a discovery or technology relating to health care. Dr. Wilson has received personal compensation in the range of $10,000-$49,999 for serving as a Expert Witness with US Dept of Justice.
Barney J. Stern, MD, FAAN (Johns Hopkins Outpatient Center) The institution of Dr. Stern has received personal compensation in the range of $10,000-$49,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for AAN. The institution of Dr. Stern has received research support from NIH/NINDS. The institution of Dr. Stern has received research support from NIH/NINDS. The institution of Dr. Stern has received research support from NIH/NINDS. Dr. Stern has received publishing royalties from a publication relating to health care. Dr. Stern has a non-compensated relationship as a Board of Directors member with ACGME that is relevant to AAN interests or activities.
Jeffrey M. Gelfand, MD, MAS, FAAN (University of California, San Francisco) Dr. Gelfand has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Arialys. An immediate family member of Dr. Gelfand has received personal compensation in the range of $50,000-$99,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for Headache: The Journal of Head and Face Pain. The institution of Dr. Gelfand has received research support from Genentech/Roche. The institution of Dr. Gelfand has received research support from Vigil Neurosciences. An immediate family member of Dr. Gelfand has received publishing royalties from a publication relating to health care. Dr. Gelfand has received publishing royalties from a publication relating to health care. Dr. Gelfand has received publishing royalties from a publication relating to health care. Dr. Gelfand has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant and Expert, Vaccine Injury Compensation Program with United States Health and Human Services and Department of Justice. Dr. Gelfand has a non-compensated relationship as a Trial Steering Committee Chairperson and member with Roche / Genentech that is relevant to AAN interests or activities.
Carlos A. Pardo-Villamizar, MD (Johns Hopkins U, Med Dept of Neurology) The institution of Dr. Pardo-Villamizar has received research support from National Institutes of Health. The institution of Dr. Pardo-Villamizar has received research support from Bart McLean Fund for Neuroimmunology Research .