Coller Ryan J, Singh-Verdeflor Kristina, Eickhoff Jens, Chung Paul J, Kloster Heidi M, Cushing Christopher C, Gerber Danielle M, Katz Barbara J, Ia Siem, Wagner Teresa, Delgado-Martinez Roxana, Warner Gemma, Porras-Javier Lorena, Klitzner Thomas S, Lerner Carlos F
Department of Pediatrics, University of Wisconsin School of Medicine and Public Health, Madison, Wisconsin, United States of America.
Department of Biostatistics and Medical Informatics, University of Wisconsin School of Medicine and Public Health, Madison, Wisconsin, United States of America.
PLoS One. 2025 Jun 16;20(6):e0325970. doi: 10.1371/journal.pone.0325970. eCollection 2025.
Respiratory 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.
This 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.
Sixty 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.
RE-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.
呼吸系统疾病一直是重度脑瘫（CP）患者死亡和住院的主要原因。呼吸加重-行动计划与护理过渡（RE-PACT）是一种及时的适应性干预措施，旨在预防重度脑瘫患者的呼吸系统疾病。RE-PACT将早期疾病检测与快速临床反应相结合，以尽早应对呼吸系统疾病的各种病因，从而改变疾病轨迹。本研究的目的是确定RE-PACT的可行性、可接受性、保真度和估计效应大小。
这项双中心随机对照试验于2022年4月至2024年2月在人口统计学和地理位置不同的地点进行。从复杂护理项目中招募照顾者-儿童对，儿童的粗大运动功能分类系统水平为4-5级脑瘫，并且接受肺科医生护理或每日呼吸治疗。儿童被随机分为接受常规护理或RE-PACT，为期6个月。主要结局是具有先验成功定义的可行性、可接受性和保真度指标。主要临床结局是严重呼吸系统疾病（SRI）事件发生率，定义为因呼吸系统诊断而住院的情况。Clinicaltrials.gov注册号为NCT05292365。
共纳入60名儿童，其中26名被随机分配到RE-PACT组。各项指标证实了RE-PACT的可行性、可接受性和保真度，例如短信回复率为97.5%，且未错过任何行动计划或临床反应活动。系统可用性量表得分“良好至优秀”（均值[标准差]，79.5[11.7]）。RE-PACT组的SRI事件发生率（95%置信区间，CI）为每人年0.71（0.36-1.14），而常规护理组的事件发生率为每人年1.08（0.61-1.91），风险比为0.66（0.28-1.56）。次要结局和定性数据强化了RE-PACT的积极影响。
RE-PACT是一种可行、可接受的干预措施，可以高保真地提供给照顾重度脑瘫儿童的不同家庭。这些数据为RE-PACT预防严重呼吸系统疾病能力的疗效测试所需的样本和设计特征提供了信息。
