NephCure Accelerating Cures Institute: A Multidisciplinary Consortium to Improve Care for Nephrotic Syndrome.

INTRODUCTION: NephCure Accelerating Cures Institute (NACI) is a collaborative organization sponsored by NephCure Kidney International and the University of Michigan. The Institute is composed of 7 cores designed to improve treatment options and outcomes for patients with glomerular disease: Clinical Trials Network, Data Warehouse, Patient-Reported Outcomes (PRO) and Endpoints Consortium, Clinical Trials Consulting Team, Quality Initiatives, Education and Engagement, and Data Coordinating Center. METHODS: The Trials Network includes 22 community- and hospital-based nephrology practices, 14 of which are trial-only sites. Eight sites participate in the NACI Registry, and as of October 2017, 1054 patients are enrolled with diagnoses including but not limited to focal segmental glomerulosclerosis, minimal change disease, membranous nephropathy, IgA nephropathy, and childhood-onset nephrotic syndrome. By using electronic health record data extraction, robust and efficient clinical data are captured while minimizing the burden to site-based network staff. RESULTS: The Data Warehouse includes her-extracted data from registry patients, PRO development data, and data from completed observational studies and clinical trials. The Clinical Trial Consulting Team provides support for trial design in rare diseases leveraging these data. The PRO and Endpoints Consortium develops shorter-term endpoints while capturing the patient-reported significance of interventions under study. The Quality Initiatives and Education/Engagement cores elevate the level of care for patients. The Data Coordinating Center manages the analysis and operations of the Institute. CONCLUSION: By engaging with patients, academia, industry, and patient advocate community representatives, including our Patient Advisory Board, NACI strives for better outcomes and treatments using evidence-based support for clinical trial design.

Provider perspectives on treatment decision-making in nephrotic syndrome.

BACKGROUND: Managing patients with nephrotic syndrome (NS) remains difficult for the practicing nephrologist. This often young patient population is faced with a debilitating, relapsing and remitting disease with non-specific treatment options that are often poorly tolerated. Clinicians managing these complex patients must attempt to apply disease-specific evidence while considering the individual patient's clinical and personal situation. METHODS: We conducted qualitative interviews to ascertain the provider perspectives of NS, treatment options and factors that influence recommendations for disease management, and administered a survey to assess both facilitators and barriers to the implementation of the Kidney Disease: Improving Global Outcomes (KDIGO) guidelines. RESULTS: When making treatment recommendations, providers considered characteristics of various treatments such as efficacy, side effects and evaluation of risk versus benefit, taking into account how the specific treatment fit with the individual patient. Time constraints and the complexity of explaining the intricacies of NS were noted as significant barriers to care. Although the availability of guidelines was deemed a facilitator to care, the value of the KDIGO guidelines was limited by the perception of poor quality of evidence. CONCLUSIONS: The complexity of NS and the scarcity of robust evidence to support treatment recommendations are common challenges reported by nephrologists. Future development and use of shared learning platforms may support the integration of best available evidence, patient/family preferences and exchange of information at a pace that is unconstrained by the outpatient clinic schedule.

Learning to live with nephrotic syndrome: experiences of adult patients and parents of children with nephrotic syndrome.

Background: People living with nephrotic syndrome (NS) need to develop an in-depth understanding of their condition in order to participate in treatment decisions, develop self-management skills and integrate illness into daily life. However, the learning needs of adult patients and parents of children with NS are unknown. We therefore explored patient and parent perspectives on learning needs related to NS as part of a larger study to develop a shared learning tool for NS. Methods: Qualitative data were collected using semistructured focus groups and individual interviews with adult patients (n = 22) and parents of children with NS (n = 25). Results: The complexity of NS and its treatment made decision making challenging, as patients/parents often had to assimilate information about a condition that is poorly understood. Specific informational needs related to understanding the diagnosis and treatment approaches as well as learning to manage NS were identified. Difficulty in getting accurate information often made learning challenging. The importance of learning to monitor their condition, including understanding triggers that might precipitate a relapse, was highlighted, underscoring the need for individualized approaches to ensure unique learning needs are addressed. Conclusions: Our findings reveal some of the unique concerns of people with NS given its uncertain course and the limited information available specific to NS. These results suggest the need for shared communication between the patient/parents and providers to elicit the patient's/parents' understanding of NS and to support them in meeting their unique learning needs.