Abstract Details

Title Top Ten Research Themes for Dystonia in Cerebral Palsy: A community-driven research agenda

Topic Child Neurology and Developmental Neurology

Presentation(s) P13 - Poster Session 13 (8:00 AM-9:00 AM)

Poster/Presentation
Number 6-004

Background Even though cerebral palsy (CP) is the most common cause of childhood dystonia, the unique needs of people affected by D-CP remain largely uncharacterized and unaddressed in the current body of evidence. The CP community (people with CP and their caregivers) is uniquely equipped to help determine the research questions that best address their needs.

Objective

To develop a community-driven research agenda for dystonia in cerebral palsy (D-CP): a common, debilitating, but understudied condition.

Design/Methods

We developed a community-driven D-CP research agenda using the well-established James Lind Alliance methodology. CP community members, researchers, and clinicians were recruited via multiple advocacy, research, and professional organizations. To ensure shared baseline knowledge, participants watched webinars on D-CP outlining current definitions, management, and research prepared by a Steering Group of field experts (https://cprn.org/research-cp-dystonia-edition). Participants next submitted their remaining uncertainties about D-CP and prioritized them between randomly generated serial pairs. The top uncertainties were consolidated into themes via iterative consensus-building discussions within the Steering Group. The themes were ranked in order of the highest ranked uncertainty that contributed to the theme.

Results

166 webinar viewers generated 113 uncertainties yielding 67 unique uncertainties. 29 uncertainties (17 from community members) were prioritized higher than their randomly matched pairs. These coalesced into the following Top 10 D-CP Themes:

1) Develop new treatments;

2) Assess rehabilitation and psychological management approaches;

3) Compare effectiveness of current treatments;

4) Improve diagnosis and severity assessments;

5) Assess the impact of 'mixed' tone (spasticity and dystonia) in outcomes and approaches;

6) Assess predictors of treatment responsiveness;

7) Determine pathophysiologic mechanisms;

8) Describe the natural history;

9) Develop dystonia pain treatments;

10) Increase family awareness.

Conclusions We have identified areas of research/clinical priority for D-CP. Community members generated the majority of top-prioritized uncertainties, highlighting the important contributions community members can make to research agendas, even beyond D-CP.

Authors/Disclosures
Laura Gilbert, DO, MBA PRESENTER	Dr. Gilbert has nothing to disclose.
	No disclosure on file
	No disclosure on file
Michael Kruer, MD (Sanford Children's Speciality Clinic)	The institution of Dr. Kruer has received research support from NIH NINDs. Dr. Kruer has received research support from Cure CP. Dr. Kruer has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant with NHRSA.
	No disclosure on file
Jonathan W. Mink, MD, PhD, FAAN	The institution of Dr. Mink has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Amicus. The institution of Dr. Mink has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Neurogene. Dr. Mink has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for TEVA. Dr. Mink has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for PTC Therapeutics. Dr. Mink has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Applied Therapeutics. Dr. Mink 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. Mink has received research support from Neurogene. The institution of Dr. Mink has received research support from NIH. Dr. Mink has received publishing royalties from a publication relating to health care. Dr. Mink has received personal compensation in the range of $500-$4,999 for serving as a Member, Study Section with NINDS.
	No disclosure on file
Bhooma R. Aravamuthan, MD, PhD (Washington University in St. Louis)	Dr. Aravamuthan has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Neurocrine Biosciences. An immediate family member of Dr. Aravamuthan has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for SK Life Science, Inc.. Dr. Aravamuthan has received research support from National Institute of Neurological Disorders and Stroke. Dr. Aravamuthan has received publishing royalties from a publication relating to health care.