title: Social media methods for studying rare diseases.
abstract: For pediatric rare diseases, the number of patients available to support traditional research methods is often inadequate. However, patients who have similar diseases cluster "virtually" online via social media. This study aimed to (1) determine whether patients who have the rare diseases Fontan-associated protein losing enteropathy (PLE) and plastic bronchitis (PB) would participate in online research, and (2) explore response patterns to examine social media's role in participation compared with other referral modalities. A novel, internet-based survey querying details of potential pathogenesis, course, and treatment of PLE and PB was created. The study was available online via web and Facebook portals for 1 year. Apart from 2 study-initiated posts on patient-run Facebook pages at the study initiation, all recruitment was driven by study respondents only. Response patterns and referral sources were tracked. A total of 671 respondents with a Fontan palliation completed a valid survey, including 76 who had PLE and 46 who had PB. Responses over time demonstrated periodic, marked increases as new online populations of Fontan patients were reached. Of the responses, 574 (86%) were from the United States and 97 (14%) were international. The leading referral sources were Facebook, internet forums, and traditional websites. Overall, social media outlets referred 84% of all responses, making it the dominant modality for recruiting the largest reported contemporary cohort of Fontan patients and patients who have PLE and PB. The methodology and response patterns from this study can be used to design research applications for other rare diseases.
authors.affiliation: Congenital Heart Center, C.S. Mott Children's Hospital, kurts@med.umich.edu.
authors.forename: Kurt R
authors.initials: KR
authors.lastname: Schumacher
authors.affiliation: College of Pharmacy, and.
authors.forename: Kathleen A
authors.initials: KA
authors.lastname: Stringer
authors.affiliation: Congenital Heart Center, C.S. Mott Children's Hospital.
authors.forename: Janet E
authors.initials: JE
authors.lastname: Donohue
authors.affiliation: Congenital Heart Center, C.S. Mott Children's Hospital.
authors.forename: Sunkyung
authors.initials: S
authors.lastname: Yu
authors.affiliation: Congenital Heart Center, C.S. Mott Children's Hospital.
authors.forename: Ashley
authors.initials: A
authors.lastname: Shaver
authors.affiliation: Congenital Heart Center, C.S. Mott Children's Hospital.
authors.forename: Regine L
authors.initials: RL
authors.lastname: Caruthers
authors.affiliation: University of Michigan School of Public Health, University of Michigan, Ann Arbor, Michigan.
authors.forename: Brian J
authors.initials: BJ
authors.lastname: Zikmund-Fisher
authors.affiliation: Congenital Heart Center, C.S. Mott Children's Hospital.
authors.forename: Carlen
authors.initials: C
authors.lastname: Fifer
authors.affiliation: Congenital Heart Center, C.S. Mott Children's Hospital.
authors.forename: Caren
authors.initials: C
authors.lastname: Goldberg
authors.affiliation: Congenital Heart Center, C.S. Mott Children's Hospital.
authors.forename: Mark W
authors.initials: MW
authors.lastname: Russell
mesh_terms: D000293:Adolescent; D029481:Bronchitis, Chronic / Q000175:diagnosis* / Q000453:epidemiology / Q000628:therapy*; D054626:Consumer Health Information; D005260:Female; D018729:Fontan Procedure / Q000009:adverse effects*; D006306:Health Surveys; D006801:Humans; D008297:Male; D011504:Protein-Losing Enteropathies / Q000175:diagnosis / Q000453:epidemiology / Q000628:therapy*; D035583:Rare Diseases; D035842:Research Subjects; D061108:Social Media; D011795:Surveys and Questionnaires; D014481:United States
keywords: Fontan; rare disease; social media
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