Nicole Evangelidis

Establishing Core Outcome Domains in Hemodialysis: Report of the Standardized Outcomes in Nephrology–Hemodialysis (SONG-HD) Consensus Workshop

Evidence-informed decision making in clinical care and policy in nephrology is undermined by trials that selectively report a large number of heterogeneous outcomes, many of which are not patient centered. The Standardized Outcomes in Nephrology-Hemodialysis (SONG-HD) Initiative convened an international consensus workshop on November 7, 2015, to discuss the identification and implementation of a potential core outcome set for all trials in hemodialysis. The purpose of this article is to report qualitative analyses of the workshop discussions, describing the key aspects to consider when establishing core outcomes in trials involving patients on hemodialysis therapy. Key stakeholders including 8 patients/caregivers and 47 health professionals (nephrologists, policymakers, industry, and researchers) attended the workshop. Attendees suggested that identifying core outcomes required equitable stakeholder engagement to ensure relevance across patient populations, flexibility to consider evolving priorities over time, deconstruction of language and meaning for conceptual consistency and clarity, understanding of potential overlap and associations between outcomes, and an assessment of applicability to the range of interventions in hemodialysis. For implementation, they proposed that core outcomes must have simple, inexpensive, and validated outcome measures that could be used in clinical care (quality indicators) and trials (including pragmatic trials) and endorsement by regulatory agencies. Integrating these recommendations may foster acceptance and optimize the uptake and translation of core outcomes in hemodialysis, leading to more informative research, for better treatment and improved patient outcomes.

Standardised Outcomes in Nephrology-Children and Adolescents (SONG-Kids) : a protocol for establishing a core outcome set for children with chronic kidney disease

BackgroundChildren with chronic kidney disease (CKD), requiring dialysis or kidney transplantation, have a mortality rate of up to 30-fold higher than the general aged-matched population, and severely impaired quality of life. Symptoms such as fatigue and pain are prevalent and debilitating. Children with CKD are at risk of cognitive impairment, and poorer educational, vocational, and psychosocial outcomes compared with their well peers, which have consequences through to adulthood. Treatment regimens for children with CKD are long-term, complex, and highly intrusive. While many trials have been conducted to improve outcomes in children with CKD, the outcomes measured and reported are often not relevant to patients and clinicians, and are highly variable. These problems can diminish the value of trials as a means to improve the lives of children with CKD. The Standardised Outcomes in Nephrology—Children and Adolescents (SONG-Kids) study aims to develop a core outcome set for trials in children and adolescents with any stage of CKD that is based on the shared priorities of all stakeholders.Methods/DesignSONG-Kids involves five phases: a systematic review to identify outcomes (both domains and measures) that have been reported in randomised controlled trials involving children aged up to 21 years with CKD; focus groups (using nominal group technique) with adolescent patients and caregivers of paediatric patients (all ages) to identify outcomes that are relevant and important to patients and their family and the reasons for their choices; semistructured key informant interviews with health professionals involved in the care of children with CKD to ascertain their views on establishing core outcomes in paediatric nephrology; an international three-round online Delphi survey with patients, caregivers, clinicians, researchers, policy-makers, and members from industry to develop consensus on important outcome domains; and a stakeholder workshop to review and finalise the set of core outcome domains for trials in children with CKD (including nondialysis-dependent, dialysis, and kidney transplantation).DiscussionEstablishing a core outcome set to be reported in all trials conducted in children with any stage of CKD will enhance the relevance, transparency, and impact of research to improve the lives of children and adolescents with CKD.

Standardized Outcomes in Nephrology-Transplantation: A Global Initiative to Develop a Core Outcome Set for Trials in Kidney Transplantation.

Background Although advances in treatment have dramatically improved short-term graft survival and acute rejection in kidney transplant recipients, long-term graft outcomes have not substantially improved. Transplant recipients also have a considerably increased risk of cancer, cardiovascular disease, diabetes, and infection, which all contribute to appreciable morbidity and premature mortality. Many trials in kidney transplantation are short-term, frequently use unvalidated surrogate endpoints, outcomes of uncertain relevance to patients and clinicians, and do not consistently measure and report key outcomes like death, graft loss, graft function, and adverse effects of therapy. This diminishes the value of trials in supporting treatment decisions that require individual-level multiple tradeoffs between graft survival and the risk of side effects, adverse events, and mortality. The Standardized Outcomes in Nephrology-Transplantation initiative aims to develop a core outcome set for trials in kidney transplantation that is based on the shared priorities of all stakeholders. Methods This will include a systematic review to identify outcomes reported in randomized trials, a Delphi survey with an international multistakeholder panel (patients, caregivers, clinicians, researchers, policy makers, members from industry) to develop a consensus-based prioritized list of outcome domains and a consensus workshop to review and finalize the core outcome set for trials in kidney transplantation. Conclusions Developing and implementing a core outcome set to be reported, at a minimum, in all kidney transplantation trials will improve the transparency, quality, and relevance of research; to enable kidney transplant recipients and their clinicians to make better-informed treatment decisions for improved patient outcomes.

Toward Establishing Core Outcome Domains For Trials in Kidney Transplantation: Report of the Standardized Outcomes in Nephrology-Kidney Transplantation Consensus Workshops

Background Treatment decisions in kidney transplantation requires patients and clinicians to weigh the benefits and harms of a broad range of medical and surgical interventions, but the heterogeneity and lack of patient-relevant outcomes across trials in transplantation makes these trade-offs uncertain, thus, the need for a core outcome set that reflects stakeholder priorities. Methods We convened 2 international Standardized Outcomes in Nephrology-Kidney Transplantation stakeholder consensus workshops in Boston (17 patients/caregivers; 52 health professionals) and Hong Kong (10 patients/caregivers; 45 health professionals). In facilitated breakout groups, participants discussed the development and implementation of core outcome domains for trials in kidney transplantation. Results Seven themes were identified. Reinforcing the paramount importance of graft outcomes encompassed the prevailing dread of dialysis, distilling the meaning of graft function, and acknowledging the terrifying and ambiguous terminology of rejection. Reflecting critical trade-offs between graft health and medical comorbidities was fundamental. Contextualizing mortality explained discrepancies in the prioritization of death among stakeholders—inevitability of death (patients), preventing premature death (clinicians), and ensuring safety (regulators). Imperative to capture patient-reported outcomes was driven by making explicit patient priorities, fulfilling regulatory requirements, and addressing life participation. Specificity to transplant; feasibility and pragmatism (long-term impacts and responsiveness to interventions); and recognizing gradients of severity within outcome domains were raised as considerations. Conclusions Stakeholders support the inclusion of graft health, mortality, cardiovascular disease, infection, cancer, and patient-reported outcomes (ie, life participation) in a core outcomes set. Addressing ambiguous terminology and feasibility is needed in establishing these core outcome domains for trials in kidney transplantation.

Standardized Outcomes in Nephrology—Peritoneal Dialysis (SONG-PD): Study Protocol for Establishing a Core Outcome Set in PD

Background: Worldwide, approximately 11% of patients on dialysis receive peritoneal dialysis (PD). Whilst PD may offer more autonomy to patients compared with hemodialysis, patient and caregiver burnout, technique failure, and peritonitis remain major challenges to the success of PD. Improvements in care and outcomes are likely to be mediated by randomized trials of innovative therapies, but will be limited if the outcomes measured and reported are not important for patients and clinicians. The aim of the Standardised Outcomes in Nephrology-Peritoneal Dialysis (SONG-PD) study is to establish a set of core outcomes for trials in patients on PD based on the shared priorities of all stakeholders, so that outcomes of most relevance for decision-making can be evaluated, and that interventions can be compared reliably. Methods: The 5 phases in the SONG-PD project are: a systematic review to identify outcomes and outcome measures that have been reported in randomized trials involving patients on PD; focus groups using nominal group technique with patients and caregivers to identify, rank, and describe reasons for their choice of outcomes; semi-structured key informant interviews with health professionals; a 3-round international Delphi survey involving a multi-stakeholder panel; and a consensus workshop to review and endorse the proposed set of core outcome domains for PD trials. Discussion: The establishment of 3 to 5 high-priority core outcomes, to be measured and reported consistently in all trials in PD, will enable patients and clinicians to make informed decisions about the relative effectiveness of interventions, based upon outcomes of common importance.