CP Research and Research Priorities Edward A. Hurvitz, MD Executive committee, CPRN

NIH research Priorities for CP NIH Workshop on CP in November 2014 Key Takeaways Start a national CP registry Pursue Comparative Effectiveness Research question Increase the study of adults Bring more young clinician scientists into the field Advance basic and translational science for CP

NIH Research Priorities for CPRN From the NIH strategic plan for CP research Early diagnosis for early intervention Examine interventions across the clinical spectrum Examine combinations of therapies Examine new study designs Increase quality and availability of PRO’s

Conduct a series of webinars to educate community and clinicians Build and distribute a priorities survey for the patient and providers Convene a workshop to set a research agenda Publish a whitepaper outlining the community’s agenda for CP

From Webinars to the Workshop Webinars provided context and capability Codigital enabled unprecedented engagement in research dialogue Resulted in 390 research ideas Brought top 20 ideas to the workshop in Chicago in June 2017 24 patients/caregivers and 23 clinician / researchers 1.5 day workshop winnowed result to top 16 ideas Other learnings: importance of longitudinal studies, participation as a key outcome measure and the importance of patient engagement

Top Ideas Fit into Three Themes Comparative Effectiveness of Interventions: Which interventions [surgeries, injections, medications and therapies (orthotics, equipment, training)] are associated with better functional outcomes (important to child/ family) controlling for GMFCS level, age and co-morbidities? Exercise, Physical Activity and Chronic Disease Risk: What are the best long term exercise/strength training strategies to improve activity, participation and health, minimize pain, and maximize function in each GMFCS category across the lifespan? Understanding Aging: Research the issues around aging with CP, to understand not only how to treat adults now, but also to update our treatments & therapies with children who have CP to prevent some of the secondary impairments such as pain, fatigue, and functional loss.

Other Research Items, e.g. Increasing age is related to pain and fatigue in people with CP, regardless of GMFCS level. What variables are important to monitor / treat early on in life to prevent the development of pain and fatigue later on in life? How can we best leverage the brain's neuroplasticity to retrain neural pathways for improved motor function, speech function and mobility? Study the outcomes of complementary and nontraditional therapies, i.e. hyperbaric oxygen, hippo, swimming/aquatic, Feldenkrais, massage, yoga, Tai Chi, music, recreational, acupuncture, etc. Reviewing efficacy, costs, insurance support probabilities.

Key Takeaways Importance for researchers to stay engaged w/ extended community Need for longitudinal studies to drive care and research Desire to keep momentum from Research CP initiative Outcomes need to include participation Importance of adult perspective Our next steps in research must be balanced to be inclusive (not just one group [GMFCS] or age)

CP Research Priorities Compartive Effectiveness/Interventions Physical Activity Comparative Effectiveness CPRN Registry SDR vs. Botox Best Practices/QI ITB QI Orthopedic Indications Surgical Disparities Supported Mobility Epilepsy Innovations No Entry Levels of Physical Activty No Entry Exercise Strategies Chronic Disease Risk Affect on Outcomes (e.g. pain)

CP Research Priorities Aging/Longitudinal Pain/Fatigue Understanding Aging Process No Entry Adult Outcomes/Issues CPRN Registry Adult Social/Emotional/Function Adult Pain Longitudinal Outcomes CP PRO Prevention Adult Pain Treatment Trials No Entry

CP Research Priorities CAM Neuroplasticity Comparative Effectiveness No Entry Cost and Coverage Understanding Mechanism No Entry Trials

NIH WORKSHOP PRIORITIES Workshop Priority CPRN Addressing? Start a National Registry Yes Comparative Effectiveness Starting-SDR vs. Botox Studies of Adults Starting-Adult Pain, Adult Outcomes Bring in New Researchers Starting Basic and Translational Not a goal for CPRN

NIH/NCMRR PRIORITIES FOR CP NIH/NCMRR PRIORITY CPRN ADDRESSING? Early Diagnosis and Intervention Starting-Genetics Examine Interventions Across Spectrum Yes-several studies Examine Combinations of Therapies No Examine New Study Designs Yes-PBE Increase PRO’s Yes-CP PRO, Adult studies

Call to Action Physical Activity studies using CPRN More studies in highest priority areas Interventions, especially CER Aging Longitudinal design Pain Early Diagnosis and Treatment Think about: Where does CAM and Plasticity fit for CPRN Other areas of study