Karine E. Manera

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.

Establishing a Core Outcome Measure for Life Participation: A Standardized Outcomes in Nephrology – Kidney Transplantation (SONG-Tx) Consensus Workshop Report.

SONG-Tx. Background Graft loss, a critically important outcome for transplant recipients, is variably defined and measured and incompletely reported in trials. We convened a consensus workshop on establishing a core outcome measure for graft loss for all trials in kidney transplantation. Methods Twenty-five kidney transplant recipients/caregivers and 33 health professionals from eight countries participated. Transcripts were analyzed thematically. Results Five themes were identified. “Graft loss as a continuum” conceptualizesgraft loss as a process, but requiring an endpoint defined as a discrete event. In “defining an event with precision and accuracy,” loss of graft function requiring chronic dialysis(minimum 90 days)provided an objective and practical definition; re-transplant would capture pre-emptive transplantation; relisting was readily measured but would overestimate graft loss; and allograft nephrectomy was redundant in being preceded by dialysis. However, the thresholds for renal replacement therapy varied. Conservative management was regarded as too ambiguous and complexto use routinely. “Distinguishing death-censored graft loss” would ensure clarity and meaningfulness in interpreting results. “Consistent reporting for decision-making” by specifying time points and metrics (i.e. time to event) was suggested. “Ease of ascertainment and data collection” of the outcome from registries could support use of registry data to efficiently extend follow-up of trial participants. Conclusions A practical and meaningful core outcome measure for graft loss may be defined as chronic dialysis or re-transplant, and distinguished from loss due to death. Consistent reporting of graft loss using standardized metrics and time points may improve the contribution of trials to decision-making in kidney transplantation.

Standardised Outcomes in Nephrology-Polycystic Kidney Disease (SONG-PKD): study protocol for establishing a core outcome set in polycystic kidney disease

BackgroundAutosomal dominant polycystic kidney disease (ADPKD) is the most common potentially life threatening inherited kidney disease and is responsible for 5–10% of cases of end-stage kidney disease (ESKD). Cystic kidneys may enlarge up to 20 times the weight of a normal kidney due to the growth of renal cysts, and patients with ADPKD have an increased risk of morbidity, premature mortality, and other life-time complications including renal and hepatic cyst and urinary tract infection, intracranial aneurysm, diverticulosis, and kidney pain which impair quality of life. Despite some therapeutic advances and the growing number of clinical trials in ADPKD, the outcomes that are relevant to patients and clinicians, such as symptoms and quality of life, are infrequently and inconsistently reported. This potentially limits the contribution of trials to inform evidence-based decision-making. The Standardised Outcomes in Nephrology—Polycystic Kidney Disease (SONG-PKD) project aims to establish a consensus-based set of core outcomes for trials in PKD (with an initial focus on ADPKD but inclusive of all stages) that patients and health professionals identify as critically important.MethodsThe five phases of SONG-PKD are: a systematic review to identify outcomes that have been reported in existing PKD trials; focus groups with nominal group technique with patients and caregivers to identify, rank, and describe reasons for their choices; qualitative stakeholder interviews with health professionals to elicit individual values and perspectives on outcomes for trials involving patients with PKD; an international three-round Delphi survey with all stakeholder groups (including patients, caregivers, healthcare providers, policy makers, researchers, and industry) to gain consensus on critically important core outcome domains; and a consensus workshop to review and establish a set of core outcome domains and measures for trials in PKD.DiscussionThe SONG-PKD core outcome set is aimed at improving the consistency and completeness of outcome reporting across ADPKD trials, leading to improvements in the reliability and relevance of trial-based evidence to inform decisions about treatment and ultimately improve the care and outcomes for people with ADPKD.

Expectations and experiences of follow up and self-care after living kidney donation: a focus group study

Background Ensuring donor wellbeing warrants ongoing monitoring after living kidney donation. However, there is considerable variability in donor follow-up processes, including information provided to donors regarding self-care. Loss to follow-up is common, suggesting that the aims and benefits of monitoring and follow-up may not be apparent. We aimed to describe the experiences and expectations of living kidney donors regarding follow-up and self-care after donation. Methods Participants from 3 transplant centers in Australia and Canada participated in 14 focus groups (n = 123). Transcripts were analyzed thematically. Results We identified 4 themes: lacking identification as a patient (invincibility and confidence in health, immediate return to normality, avoid burdening specialty services, redundancy of specialist attention, unnecessary travel), empowerment for health (self-preservation for devastating consequences, self-advocacy and education, needing lifestyle advice, tracking own results), safety net and reassurance (availability of psychosocial support, confidence in kidney-focused care, continuity and rapport, and access to waitlist priority), and neglect and inattention (unrecognized ongoing debilitations, primary focus on recipient, hospital abandonment, overlooking individual priorities, disconnected from system, coping with dual roles, and lacking support for financial consequences). Conclusions Living kidney donors who felt well and confident about their health regarded specialist follow-up as largely unnecessary. However, some felt they did not receive adequate medical attention, were prematurely detached from the health system, or held unresolved anxieties about the consequences of their decision to donate. Ongoing access to healthcare, psychosocial support, and education may reassure donors that any risks to their health are minimized.

The Power of the Patient Voice: Conducting and Using Qualitative Research to Improve Care and Outcomes in Peritoneal Dialysis

242 The growing emphasis on patient-centered care has seen increased efforts in establishing partnerships and promoting shared decision-making among healthcare providers, patients, and their families (1,2). For patients with end-stage kidney disease, including those requiring peritoneal dialysis (PD), being involved in the decision-making process can often be confronting, confusing, and challenging, because possible management strategies have different and profound impacts on their health and quality of life (3). While trials can provide evidence about the relative effectiveness of interventions, qualitative research can elucidate the meaning of the risks and consequences of treatment, as well as detailed and nuanced understanding about patients’ values, goals, and priorities. Both types of information are necessary for informed decision-making. Qualitative studies are being published more frequently in nephrology journals, though only a few relate to PD (4). In the past 3 years, only 7 qualitative studies have been published in Peritoneal Dialysis International. This article highlights some of the recent contributions of qualitative research in PD, and offers principles to guide the conduct and appraisal of qualitative studies.

Identifying and integrating patient and caregiver perspectives in clinical practice guidelines for percutaneous renal biopsy: Consumer involvement in guidelines

Percutaneous renal biopsy is often essential for providing reliable diagnostic and prognostic information for people with suspected kidney disease, however the procedure can lead to complications and concerns among patients. This study aims to identify and integrate patient priorities and perspectives into the Kidney Health Australia – Caring for Australasians with Renal Impairment clinical practice guidelines for renal biopsy, to ensure patient‐relevance.

Children’s experiences of congenital heart disease: a systematic review of qualitative studies

We aimed to describe the experiences of children and adolescents with congenital heart disease (CHD). Electronic databases were searched until August 2016. Qualitative studies of children’s perspectives on CHD were included. Data was extracted using thematic synthesis. From 44 studies from 12 countries involving 995 children, we identified 6 themes: disrupting normality (denying the diagnosis, oscillating between sickness and health, destabilizing the family dynamic), powerlessness in deteriorating health (preoccupation with impending mortality, vulnerability to catastrophic complications, exhaustion from medical testing), enduring medical ordeals (traumatized by invasive procedures, disappointed by treatment failure, displaced by transition, valuing empathy and continuity in care, overcoming uncertainty with information), warring with the body (losing stamina, distressing inability to participate in sport, distorted body image, testing the limits), hampering potential and goals (feeling disabled, unfair judgment and exclusion, difficulties with academic achievement, limiting attainment and maintenance of life milestones), and establishing one’s own pace (demarcating disease from life, determination to survive, taking limitations in their stride, embracing the positives, finding personal enrichment, relying on social or spiritual support).Conclusion: Children with CHD feel vulnerable and burdened by debilitating physical symptoms, unpredictable complications, and discrimination. Clinicians may support patients by sharing recognition of these profound psychosocial consequences.What is Known:• CHD is associated with difficulties in learning and attention, school absenteeism, decreased endurance, poor body image, and peer socialization• What is lesser known is how young patients cope with the symptoms, prognostic uncertainty, and treatment burdenWhat is New:• We found that children are challenged by lifestyle restrictions, fear of invasive procedures, impaired body image, discrimination, and uncertainty about the future. Feelings of disempowerment are intensified by the unpredictability of disease progression• Thus, strategies to improve outcomes include improved patient education on disease and lifestyle management and partnership with school teachers and counselors for unique psychosocial support

Research priorities for childhood chronic conditions: a workshop report

Background Chronic conditions are the leading cause of mortality, morbidity and disability in children. However, children and caregivers are rarely involved in identifying research priorities, which may limit the value of research in supporting patient-centred practice and policy. Objective To identify priorities of patients, caregivers and health professionals for research in childhood chronic conditions and describe the reason for their choices. Setting An Australian paediatric hospital and health consumer organisations. Methods Recruited participants (n=73) included patients aged 8 to 14 years with a chronic condition (n=3), parents/caregivers of children aged 0 to 18 years with a chronic condition (n=19), representatives from consumer organisations (n=13) and health professionals including clinicians, researches (n=38) identified and discussed research priorities. Transcripts were thematically analysed. Results Seventy-eight research questions were identified. Five themes underpinned participants’ priorities: maintaining a sense of normality (enabling participation in school, supporting social functioning, promoting understanding and acceptance), empowering self-management and partnership in care (overcoming communication barriers, gaining knowledge and skills, motivation for treatment adherence, making informed decisions, access and understanding of complementary and alternative therapies),strengthening ability to cope (learning to have a positive outlook, preparing for home care management, transitioning to adult services), broadening focus to family (supporting sibling well-being, parental resilience and financial loss, alleviating caregiver burden), and improving quality and scope of health and social care (readdressing variability and inequities, preventing disease complications and treatment side effects, identifying risk factors, improving long-term outcomes, harnessing technology, integrating multidisciplinary services). Conclusion Research priorities identified by children, caregivers and health professionals emphasise a focus on life participation, psychosocial well-being, impact on family and quality of care. These priorities may be used by funding and policy organisations in establishing a paediatric research agenda.