The Hydrocephalus Association Patient-Powered Interactive Engagement Registry (HAPPIER): Design and Initial Baseline Report

Abstract

Noriana E Jakopin,1,* Samantha N Lanjewar,1,* Amanda Garzon,1 Paul Gross,2 Richard Holubkov,3 Abhay Moghekar,4 Jason Preston,1 Margaret Romanoski,5 Chevis N Shannon,6 Mandeep S Tamber,7 Tessa Van der Willigen,8 Melissa Sloan,9 Monica J Chau,1 Jenna E Koschnitzky1 1Research Department, Hydrocephalus Association, Bethesda, MD, 20814, USA; 2Cerebral Palsy Research Network, Greenville, SC, 29604, USA; 3Department of Pediatrics, University of Utah, Salt Lake City, UT, 84132, USA; 4Department of Neurology, Johns Hopkins University School of Medicine, Baltimore, MD, 21287, USA; 5Ascension St. John Hospital, Detroit, MI, 48236, USA; 6American Society for Reproductive Medicine, Birmingham, AL, 35216, USA; 7Division of Neurosurgery, University of British Columbia, British Columbia Children’s Hospital, Vancouver, BC, V6H 3V4, Canada; 8Georgetown University, Washington, DC, 20057, USA; 9American University, Washington, DC, 20016, USA*These authors contributed equally to this workCorrespondence: Monica J Chau, Hydrocephalus Association, 6903 Rockledge Drive, Suite 740, North Bethesda, MD, 20817, USA, Email [email protected]: Hydrocephalus is a neurological condition characterized by an accumulation of cerebrospinal fluid (CSF) with no cure and limited treatments. There is a significant gap in hydrocephalus research where patients lack opportunities to voice their perspectives on their condition. The Hydrocephalus Association Patient-Powered Interactive Engagement Registry (HAPPIER) database captures the lived experiences of those affected by hydrocephalus and provides a platform for researchers to access these data or distribute their own surveys, ultimately aiming to improve patient-centered care and outcomes. This publication introduces the registry by highlighting the demographics, etiology, treatments, symptom profiles, and diagnosed comorbidities of the participants.Methods: The Hydrocephalus Association and a 10-member steering committee developed HAPPIER. Other patient registries, existing surveys and assessments, and University of Utah Data Center faculty guided survey development. The Hydrocephalus Association recruited participants using social and traditional media, medical referrals, and advertisements at events.Results: Of the 691 survey participants with hydrocephalus, 451 (65.3%) responded for themselves. The majority of the registry was female (55.0%), white (86.0%), and from the United States and territories (87.7%). Most were diagnosed between 0– 11 months (46.2%), with congenital hydrocephalus as the most reported etiology (43.8%). Participants reported a shunt(s) as the most prevalent treatment (71.2%) and headaches as the most frequent symptom (60.3%), while 69.9% of participants reported being diagnosed with movement impairments and 70.8% with other health conditions.Conclusion: HAPPIER is a novel database that addresses gaps in data on non-clinical outcomes of hydrocephalus, which are critical to clinical care and understanding hydrocephalus. Patient perspectives and outcomes remain historically underrepresented. By directly engaging individuals living with hydrocephalus and their caregivers, HAPPIER incorporates essential patient perspectives through planned longitudinal data collection and patient surveys. These data are open to investigators interested in analyzing the collected data.Plain Language Summary: Hydrocephalus is a lifelong condition where excess fluid builds up in the brain. A significant gap in research is the lack of information on how hydrocephalus affects the daily lives of those with the condition. To bridge this gap, the Hydrocephalus Association created the Hydrocephalus Association Patient-Powered Interactive Engagement Registry (HAPPIER), a patient registry that gathers the real-world experiences from people living with hydrocephalus. This database helps researchers better understand symptoms, treatments, and challenges, ultimately working toward better patient care.Experts contributed to the development of HAPPIER, using guidance from existing patient registries and surveys. Participants joined through media recruitment, medical referrals, and outreach events. The registry includes 691 people with hydrocephalus, most of whom answered the survey themselves. More than half are female, and the majority are from the United States. Many were diagnosed as infants (between 0-11 months), with congenital hydrocephalus being the most common etiology. Participants most frequently reported receiving a shunt(s) as treatment and experiencing headaches as the most common symptom. Many participants also experienced movement difficulties and other health conditions.The HAPPIER registry gathers data on people with hydrocephalus, including their backgrounds, treatments, symptoms, and other health conditions. The goal is to use ongoing surveys to better understand their experiences and find ways to improve quality of life. Researchers can access these data or conduct their own surveys through HAPPIER, giving patients and caregivers a voice in research and ensuring the patient perspective guides advancements in future studies, treatment, support, and clinical care.Keywords: hydrocephalus, database, patient registry

Keywords

• hydrocephalus
• database
• patient registry