1. Study Group Leader: Paul Gross
SDR v. Serial Botox
Study Group Leader: Paul Gross
Participants: Kristie Bjornson (Seattle), Joline Bradenburg (Mayo), Darcy Fehlings (SickKids), Jacob Kean (Utah), Jeff Leonard (Nationwide), Unni Narayanan (SickKids), Dody Robinson (Hopkins)
CPRN Houston Investigators Meeting May 31-June 1, 2018

2. AIMS & Hypotheses (History)
Borne out of the NIH Workshop focus on needing more comparative effectiveness research
Originally: Determine what patient characteristics and interventions lead to the best outcomes (PCORI 1)
Revised: What patient characteristics and interventions lead to the best outcomes in spastic diplegics (PCORI 2)
Current: Which patient characteristics and spasticity interventions lead to the best outcomes in diplegics (Study 1R3)
Sub topic: Characterize the practice variation in spasticity management for diplegics in the U.S.

3. Methods – Document Practice Variation
Qualitative interview of medical discipline leaders in spasticity management (clinics where existing) fpr diplegics
Open ended interview questions on neurotoxins, selective dorsal rhizotomy, intrathecal baclofen pumps and orthopedic interventions
Vignettes to capture “spasticity management plan”
Recorded interviews
Will be transcribed, coded and analyzed
May follow-up with a close ended survey
10-15 Centers across U.S.

4. Methods – Dynamic Treatment Regimes
Analysis for treatments that are tailored to a person’s dynamic state
If, then, otherwise
Personalized medicine (rehabilitation medicine)
Botox + PT
SDR + Intensive PT PT

5. Methods – Dynamic Treatment Regimes
Randomized = Sequential, Multiple Assignment, Randomized Trials (SMART)
Non-randomized
Analyses structured to emulate a randomized study
Control of time-varying and time-invariant covariates that could bias outcomes
A note on causal inference from non-randomized studies
When compared, randomized and nonrandomized studies are largely concordant
When discrepant, newer methods address discrepancies
An RCT-only mindset is easy but incorrect; the literature says otherwise

6. Population, Sample Size, Importance
Spastic digplegics at point of indication for surgical intervention
Most likely inclusion at 4-6 years of age
Sample size TBD on outcome measure
Addresses a core question in Research CP – R2: Which interventions lead to the best outcomes.
Target funding mechanism: New NIH Funding Opportunity Announcement for observational trials

7. Call to action
Help Paul schedule interviews with spasticity management clinic leaders in PM&R, neurosurgery, and orthopedics in your institution!