So much more than a “pair of brown shoes”: Triumphs of patient and other stakeholder engagement in patient-centered outcomes research

This piece illustrates the “real world” experiences of patients and other stakeholder partners in research to help inform and inspire future patient-centered outcomes research (PCOR) efforts. The Patient-Centered Outcomes Research Institute (PCORI) was created in 2010 to fund research that helps patients, clinicians, and other healthcare stakeholders make informed health decisions. The first 50 funded PCORI Pilot Projects engaged patients, caregivers, parents, patient advocates, clinicians, and other non-traditional research stakeholders to serve in advisory and leadership positions on their research teams, many for the first time. In interviews with seven patients and other stakeholders, several lessons learned emerged, including how to build confidence over the course of a research project; how to offer translation and interpretation insights reflective of practical experience; how to understand the benefits and limitations to stakeholder participation; and how to positively influence the research process and study outcomes. By the completion of their Pilot Projects, the stakeholder partners profiled here considered themselves “empowered” research contributors. The authors are hopeful these stories will encourage more patients and other stakeholders to contribute their time and experiential learnings to improve the process, and results of, PCOR

The Children’s Hospital of Philadelphia Family Partners Program: Promoting child and family-centered care in pediatrics

Involving Family Advisory Councils in decisions that impact pediatric healthcare demonstrates hospitals’ commitment to child- and family-centered care. Yet, reliance on advisors as the sole source of family input has several limitations: infrequent meetings impedes the council’s capacity to address emerging concerns in a timely manner; feedback obtained from a small number of highly-engaged family members may not represent the perspectives of “typical” patients and families; advisors provide feedback in a reactive manner and are generally not equitably involved in problem identification or the initial development of solutions. In recognition of the need to strengthen and advance family partnerships, the Children’s Hospital of Philadelphia made significant structural and operational changes to advance and expand the family centered care model. The Family Partners Program, a component of the broader family centered care portfolio, is a centralized institution-wide resource that enables practitioners, researchers, and healthcare leaders to collaborate with trained family representatives. This paper describes the Family Partner Program infrastructure; approaches to recruiting, onboarding, training, and mentoring parents and caregivers; and the many ways that Family Partners promote the core principles of child- and family-centeredness in clinical care, quality improve, and research contexts

The design, implementation, and formative evaluation of a program for waitlisted caregivers of individuals with developmental disabilities

This dissertation focused on the process of utilizing Maher's (2000) program planning and evaluation framework to design, implement, and conduct a formative evaluation of a waiting list program for caregivers waiting to receive parent-training services. The literature on waiting lists suggests that waitlisted individuals are generally provided with limited or no support while waiting for services. A waiting list program was designed in an attempt to provide support to families who had been waiting for parent training services for more than one-and-a-half years. Participants of the waiting list program received one home-based session, as well as monthly educational worksheets about topics related to caring for an individual with a developmental disability. The program was implemented for five months, followed by a formative program evaluation. Based on results from the formative evaluation, it was determined that the waiting list program was implemented according to design. In addition, the formative evaluation results suggest that the information contained in the worksheets provided caregivers with an opportunity to review previously learned information and be exposed to new information. Participants varied in regard to their satisfaction with the waiting list program, from being dissatisfied to highly satisfied. Caution should be used when interpreting these results due to the small sample size and low response rate of evaluation materials. Based on the evaluation of the program evaluation, it was determined that the evaluation plan was successfully implemented. In addition, the client reported that the information will be useful for future program planning purposes. Recommendations regarding potential adjustments to the design, implementation, and evaluation of the program are provided.Psy.DIncludes bibliographical references (p. 123-128)by Amy Rachel KratchmanIncludes abstrac

Comparative effectiveness of paediatric kidney stone surgery (the PKIDS trial): study protocol for a patient-centred pragmatic clinical trial.

IntroductionThe strength of the evidence base for the comparative effectiveness of three common surgical modalities for paediatric nephrolithiasis (ureteroscopy, shockwave lithotripsy and percutaneous nephrolithotomy) and its relevance to patients and caregivers are insufficient. We describe the methods and rationale for the Pediatric KIDney Stone (PKIDS) Care Improvement Network Trial with the aim to compare effectiveness of surgical modalities in paediatric nephrolithiasis based on stone clearance and lived patient experiences. This protocol serves as a patient-centred alternative to randomised controlled trials for interventions where clinical equipoise is lacking.Methods and analysisThe PKIDS is a collaborative learning organisation composed of 26 hospitals that is conducting a prospective pragmatic clinical trial comparing the effectiveness of ureteroscopy, shockwave lithotripsy and percutaneous nephrolithotomy for youth aged 8-21 years with kidney and/or ureteral stones. Embedded within clinical care, the PKIDS trial will collect granular patient-level, surgeon-level and institution-level data, with a goal enrolment of 1290 participants over a 21-month period. The primary study outcome is stone clearance, defined as absence of a residual calculus of >4 mm on postoperative ultrasound. Secondary outcomes include patient-reported physical, emotional and social health outcomes (primarily using the Patient-Reported Outcome Measurement Information System), analgesic use and healthcare resource use. Timing and content of secondary outcomes assessments were set based on feedback from patient partners. Heterogeneity of treatment effect for stone clearance and patient-reported outcomes by participant and stone characteristics will be assessed.Ethics and disseminationThis study is approved by the central institutional review board with reliance across participating sites. Participating stakeholders will review results and contribute to development dissemination at regional, national and international meetings.Trial registration numberNCT04285658; Pre-results

The Preserving Kidney Function in Children With CKD (PRESERVE) Study: Rationale, Design, and Methods

PRESERVE seeks to provide new knowledge to inform shared decision-making regarding blood pressure (BP) management for pediatric chronic kidney disease (CKD). PRESERVE will compare the effectiveness of alternative strategies for monitoring and treating hypertension on preserving kidney function; expand the Patient-Centered Clinical Research Network (PCORnet) common data model by adding pediatric- and kidney-specific variables and linking electronic health record data to other kidney disease databases; and assess the lived experiences of patients related to BP management. Multicenter retrospective cohort study (clinical outcomes) and cross-sectional study (patient-reported outcomes [PROs]). PRESERVE will include approximately 20,000 children between January 2009-December 2022 with mild-moderate CKD from 15 health care institutions that participate in 6 PCORnet Clinical Research Networks (PEDSnet, STAR, GPC, PaTH, CAPRiCORN, and OneFlorida+). The inclusion criteria were≥1 nephrologist visit and≥2 estimated glomerular filtration rate (eGFR) values in the range of 30 to<90mL/min/1.73m2 separated by≥90 days without an intervening value≥90mL/min/1.73m2 and no prior dialysis or kidney transplant. BP measurements (clinic-based and 24-hour ambulatory BP); urine protein; and antihypertensive treatment by therapeutic class. The primary outcome is a composite event of a 50% reduction in eGFR, eGFR of<15mL/min/1.73m2, long-term dialysis or kidney transplant. Secondary outcomes include change in eGFR, adverse events, and PROs. Longitudinal models for dichotomous (proportional hazards or accelerated failure time) and continuous (generalized linear mixed models) clinical outcomes; multivariable linear regression for PROs. We will evaluate heterogeneity of treatment effect by CKD etiology and degree of proteinuria and will examine variation in hypertension management and outcomes based on socio-demographics. Causal inference limited by observational analyses. PRESERVE will leverage the PCORnet infrastructure to conduct large-scale observational studies that address BP management knowledge gaps for pediatric CKD, focusing on outcomes that are meaningful to patients. Hypertension is a major modifiable contributor to loss of kidney function in chronic kidney disease (CKD). The purpose of PRESERVE is to provide evidence to inform shared decision-making regarding blood pressure management for children with CKD. PRESERVE is a consortium of 16 health care institutions in Patient-Centered Clinical Research Network (PCORnet), the National Patient-Centered Clinical Research Network, and includes electronic health record data for>19,000 children with CKD. PRESERVE will (1) expand the PCORnet infrastructure for research in pediatric CKD by adding kidney-specific variables and linking electronic health record data to other kidney disease databases; (2) compare the effectiveness of alternative strategies for monitoring and treating hypertension on preserving kidney function; and (3) assess the lived experiences of patients and caregivers related to blood pressure management