Social media methods for studying rare diseases.

Area of Science:

• Pediatric Cardiology
• Rare Diseases
• Digital Health Research

Background:

• Traditional research faces challenges in recruiting sufficient pediatric rare disease patients.
• Patients with rare conditions often form online communities via social media.
• Fontan-associated protein losing enteropathy (PLE) and plastic bronchitis (PB) are rare conditions affecting Fontan patients.

Purpose of the Study:

• To assess patient participation in online research for PLE and PB.
• To compare social media's effectiveness against other referral methods for recruitment.
• To explore response patterns in online rare disease research.

Main Methods:

• An internet-based survey was developed to collect data on PLE and PB.
• The study was accessible online via web and Facebook portals for one year.
• Recruitment relied on respondent-driven sharing, with initial posts on patient-run Facebook pages.

Main Results:

• 671 Fontan patients completed the survey, including 76 with PLE and 46 with PB.
• Social media platforms, particularly Facebook, were the primary referral source, accounting for 84% of responses.
• Recruitment surges correlated with reaching new online patient populations, indicating effective virtual clustering.

Conclusions:

• Social media is a highly effective modality for recruiting large cohorts of rare disease patients, including those with PLE and PB.
• This online research methodology can be adapted for other rare pediatric conditions.
• Internet-based recruitment overcomes limitations of small, geographically dispersed patient populations.