Primary clinical endpoint in the RE-PACT trial.

BackgroundRespiratory illness is consistently the leading cause of death and hospitalization in severe cerebral palsy (CP). Respiratory Exacerbations-Plan for Action and Care Transitions (RE-PACT) is a just-in-time adaptive intervention to prevent respiratory illness in severe CP. RE-PACT combines early illness detection with rapid clinical response to address varying causes of respiratory illness early enough to modify illness trajectory. This study’s objective was to determine RE-PACT’s feasibility, acceptability, fidelity, and estimated effect size.MethodsThis two-site randomized controlled trial occurred from April 2022-February 2024 in demographically and geographically distinct locations. Caregiver-child pairs were recruited from complex care programs, and children had both gross motor function classification system level 4–5 CP and either pulmonologist care or daily respiratory treatments. Children were randomized to usual care or RE-PACT for six months. Primary outcomes were feasibility, acceptability, and fidelity measures having a priori definitions of success. The primary clinical outcome was the severe respiratory illness (SRI) event rate, defined as hospitalizations due to respiratory diagnoses. Clinicaltrials.gov registration is NCT05292365.ResultsSixty children were enrolled, of which 26 were randomized into RE-PACT. Measures confirmed RE-PACT’s feasibility, acceptability, and fidelity, e.g., text message response rates were 97.5%, and no action planning or clinical responder activities were missed. System usability scale scores were “good to excellent” (mean [SD], 79.5 [11.7]). The RE-PACT SRI event rate (95% confidence interval, CI) was 0.71 (0.36–1.14) per person-year compared to the usual care event rate 1.08 (0.61–1.91) per person-year, a risk ratio of 0.66 (0.28–1.56). Secondary outcomes and qualitative data reinforced RE-PACT’s positive impact.ConclusionsRE-PACT is a feasible, acceptable intervention that can be delivered with high fidelity to diverse families caring for children with severe CP. These data inform the sample and design characteristics needed for efficacy testing of RE-PACT’s ability to prevent severe respiratory illness.