Immunosuppressive therapy for IgA nephropathy in children.

BACKGROUND: IgA nephropathy (IgAN) is the most common cause of primary glomerulonephritis. It is a heterogeneous disease with different presentations and high morbidity. Thirty per cent of adults and 20% of children (followed into adulthood) will have a 50% decline in kidney function or develop kidney failure after 10 years. OBJECTIVES: To determine the benefits and harms of immunosuppressive therapy for the treatment of IgAN in children. SEARCH METHODS: We contacted the Information Specialist and searched the Cochrane Kidney and Transplant Register of Studies up to 03 October 2023 using search terms relevant to this review. Studies in the Register are identified through searches of CENTRAL, MEDLINE, and EMBASE, conference proceedings, the International Clinical Trials Registry Platform (ICTRP) Search Portal, and ClinicalTrials.gov. SELECTION CRITERIA: We included randomised controlled trials (RCTs) and non-randomised studies of interventions (NRSIs) investigating the treatment of IgAN in children with immunosuppressive therapies compared to placebo, no treatment, supportive care, standard therapy (Japanese protocol), other immunosuppressive therapies or non-immunosuppressive therapies. DATA COLLECTION AND ANALYSIS: Two authors independently extracted data and assessed the risk of bias. Random effects meta-analyses were used to summarise estimates of treatment effects. Treatment effects were expressed as risk ratios (RR) and 95% confidence intervals (CI) for dichotomous outcomes, and the mean difference (MD) and 95% CI for continuous outcomes. The risk of bias was assessed using the Cochrane risk of bias tool for RCTs and the ROBIN-I tool for NRSIs. The certainty of the evidence was assessed using Grading of Recommendations, Assessment, Development, and Evaluations (GRADE). MAIN RESULTS: This review included 13 studies with 686 participants. Ten RCTs included 334 children and 191 adults, and three NRSIs included 151 participants, all children. Most participants had mild kidney disease. The risk of bias was unclear for most of the domains relating to allocation concealment, blinding of participants, personnel, and outcome assessment. In children with IgAN, it is uncertain if corticosteroid (steroid) therapy, compared to placebo reduces proteinuria (1 study, 64 children and young adults: RR 0.47, 95% CI 0.13 to 1.72; low certainty evidence) or the decline in estimated glomerular filtration rate (eGFR) (1 study, 64 children and young adults: RR 0.47, 95% CI 0.09 to 2.39; low certainty evidence). It is uncertain if steroids reduce proteinuria compared to supportive care (2 studies, 61 children: RR 0.04, 95% CI -0.83 to 0.72; low certainty evidence). Adverse events associated with steroid therapy were not assessed due to heterogeneity in steroid protocols, including dose and duration, and lack of systematic assessment for adverse events in the included studies. Azathioprine, mycophenolate mofetil, mizoribine, or cyclophosphamide alone or in combination with steroid therapy had uncertain effects on improving proteinuria or preventing eGFR decline in children with IgAN. Fish oil, vitamin E and tonsillectomy had uncertain effects on improving proteinuria or preventing eGFR decline. Effects of other immunosuppressive therapies, secondary outcomes and adverse events were not assessed due to insufficient data. AUTHORS' CONCLUSIONS: There is a lack of high-quality evidence to guide the management of IgAN in children. There is no evidence to indicate that steroids, other immunosuppressive therapies, or tonsillectomy, when added to optimal supportive care, prevent a decline in eGFR or proteinuria in children with IgAN. Available studies were few, with small numbers, low-quality evidence, high or uncertain risk of bias, did not systematically assess harms associated with treatment, or report net benefits or harms. Severe cases and atypical presentations of IgAN were not included in the reviewed studies, and our findings cannot be generalised to these situations.

Role of primary care in enhancing continuity of care for adolescents and young adults with chronic kidney disease undergoing transition to adult health services.

The transition from pediatric to adult health care is a vulnerable period for adolescents and young adults (AYA) with chronic conditions as it involves a multitude of changes and challenges while they enter adulthood. The transition to adult care can be particularly challenging for AYA living with chronic kidney disease (CKD) due to the complex care needed for treatment. Continuity of care is crucial for a successful transition to adult health care. The aim of this educational review is to discuss the potential role of primary care providers in the transition from pediatric to adult health services for AYA with CKD and kidney failure treated with dialysis and/or transplant. We address the significance of the medical home model and how it can provide continuity of care for AYA with CKD. Primary care providers can enhance care for AYA with chronic conditions by providing continuity of care, reducing exacerbation of chronic health conditions, providing holistic care, and fostering collaboration with specialists. Despite their vital role, primary care providers face barriers in maintaining this continuity, necessitating further attention and support in this area. By addressing these barriers and encouraging primary care providers to work alongside pediatric and adult nephrologists during the transition to adult health care, there are significant opportunities to improve the care and health outcomes of AYA with CKD.

The extrafollicular B cell response is a hallmark of childhood idiopathic nephrotic syndrome.

The efficacy of the B cell-targeting drug rituximab (RTX) in childhood idiopathic nephrotic syndrome (INS) suggests that B cells may be implicated in disease pathogenesis. However, B cell characterization in children with INS remains limited. Here, using single-cell RNA sequencing, we demonstrate that a B cell transcriptional program poised for effector functions represents the major immune perturbation in blood samples from children with active INS. This transcriptional profile was associated with an extrafollicular B cell response marked by the expansion of atypical B cells (atBCs), marginal zone-like B cells, and antibody-secreting cells (ASCs). Flow cytometry of blood from 13 children with active INS and 24 healthy donors confirmed the presence of an extrafollicular B cell response denoted by the expansion of proliferating RTX-sensitive extrafollicular (CXCR5-) CD21low T-bet+ CD11c+ atBCs and short-lived T-bet+ ASCs in INS. Together, our study provides evidence for an extrafollicular origin for humoral immunity in active INS.

Development of the Strengths, Skills, and Goals Matrix: a tool for facilitating strengths-based adolescent and young adult engagement in research.

BACKGROUND: The involvement of adolescents and young adults (AYAs) with lived experience of health and mental health conditions as partners in research is increasing given the prominence of participatory approaches to research, including patient-oriented research (POR). Much of the relevant research is conducted by graduate students. While guiding AYA engagement frameworks and models exist, the processes of partnering with AYAs in patient-oriented graduate-level research projects have not been well established. Co-developed tools and practices are required to support strengths-based, developmentally appropriate AYA-graduate student partnerships. OBJECTIVES: The objectives of this commentary are: (1) to share the processes of partnership between a graduate student and five Young Adult Research Partners (YARP), (2) to describe the co-design and implementation of the Strengths, Skills, and Goals Matrix (SSGM), a tool for facilitating strengths-based AYA engagement in research, and (3) to outline considerations for applying this tool across a variety of research contexts with patient partners. MAIN BODY: Within the YARP-graduate student partnership, the SSGM offered extensive benefits, including tangible skill development, peer mentorship, and rapport building among all members. This tool offers strategies for strengths-based engagement practices which emphasize AYAs' preferences and goals throughout POR projects. Practical recommendations and considerations for applying the SSGM within graduate-level research and beyond are described, including the importance of connecting AYAs' current (and desired) skills to specific tasks within the research project and resulting outputs. CONCLUSIONS: The SSGM has possible relevance in a variety of settings given its broadly applicable structure. Future research could explore the adaptation, application, and evaluation of the SSGM across research contexts to determine its feasibility and ease of implementation. PATIENT OR PUBLIC CONTRIBUTION: This article was conceived of and co-authored by five young adult research partners. The YARP co-designed the SSGM presented in this article, the figures, and substantially contributed to the preparation of the article.

The involvement of adolescents and young adults (AYAs) with lived experience of health and mental health conditions as partners in research is becoming more common in student research projects. Though guidelines for engaging AYAs as partners in research exist, the steps for developing meaningful AYA-student partnerships are not clearly defined. These partnerships require tools and practices that are co-developed by AYAs and students to be successful. In this commentary, we share the details of a partnership between a graduate student and five AYA research partners. Next, we describe how we developed the Strengths, Skills, and Goals Matrix (SSGM), a tool for supporting AYA engagement in research which emphasizes the capabilities and goals of each AYA partner. Finally, we outline suggestions for patient partners and research teams interested in using this tool in different settings. Within our research partnership, the SSGM helped all members build relationships, develop skills, and share their skills with one another. The SSGM focuses on the strengths of each person and allows patient partners to determine their own goals for engaging in research. Future research should explore how the SSGM works for different types of research teams or projects.

Transition readiness of youth with co-occurring chronic health and mental health conditions: A mixed methods study.

BACKGROUND: A large proportion of youth with chronic conditions have mental health comorbidities. However, the effect of these comorbidities on paediatric-adult transition readiness, and the relevance of widely used tools for measuring transition readiness, are unknown. OBJECTIVE: The objectives of this study were to describe and explore the transition readiness of youth with co-occurring chronic health and mental health conditions using a combination of quantitative data obtained from participants completing the Transition Readiness Assessment Questionnaire (TRAQ) and qualitative data. DESIGN AND PARTICIPANTS: A three-phase sequential explanatory mixed methods design was employed, with the qualitative strand taking priority. First, the TRAQ scores (range 1-5) of youth with co-occurring conditions (n = 61) enroled in a multisite randomized controlled trial were measured, followed by qualitative interviews with a sample of youth (n = 9) to explain the quantitative results. Results from both strands were then integrated, yielding comprehensive insights. RESULTS: Median TRAQ scores ranged from 2.86 on the appointment keeping subscale to 5.00 on the talking with providers subscale. The qualitative results uncovered the complexities faced by this group concerning the impact of a mental health comorbidity on transition readiness and self-management skills across TRAQ domains. The integrated findings identified a diverse and highly individualized set of strengths and challenges amongst this group that did not align with overarching patterns as measured by the TRAQ. CONCLUSIONS: This mixed methods study generated novel understandings about how youth with co-occurring conditions develop competencies related to self-care, self-advocacy and self-management in preparation for paediatric-adult service transitions. Results demonstrated the assessment of transition readiness using a generic scale does not address the nuanced and complex needs of youth with co-occurring chronic health and mental health conditions. Our findings suggest tailoring transition readiness practices for this group based on youths' own goals, symptoms, coping mechanisms and resources. PATIENT OR PUBLIC INVOLVEMENT: This study was conducted in collaboration with five young adult research partners (YARP) with lived experience transitioning from paediatric to adult health/mental health services. The YARP's contributions across study phases ensured the perspectives of young people were centred throughout data collection, analysis, interpretation and presentation of findings. All five YARP co-authored this manuscript.

Perspectives of Pediatric Nephrologists, Intensivists and Nurses Regarding AKI Management and Expected Outcomes.

Background: Acute kidney injury (AKI) in critically ill children is associated with increased risk for short- and long-term adverse outcomes. Currently, there is no systematic follow-up for children who develop AKI in intensive care unit (ICU). Objective: This study aimed to assess variation regarding management, perceived importance, and follow-up of AKI in the ICU setting within and between healthcare professional (HCP) groups. Design: Anonymous, cross-sectional, web-based surveys were administered nationally to Canadian pediatric nephrologists, pediatric intensive care unit (PICU) physicians, and PICU nurses, via professional listservs. Setting: All Canadian pediatric nephrologists, PICU physicians, and nurses treating children in the ICU were eligible for the survey. Patients: N/A. Measurements: Surveys included multiple choice and Likert scale questions on current practice related to AKI management and long-term follow-up, including institutional and personal practice approaches, and perceived importance of AKI severity with different outcomes. Methods: Descriptive statistics were performed. Categorical responses were compared using Chi-square or Fisher's exact tests; Likert scale results were compared using Mann-Whitney and Kruskal-Wallis tests. Results: Surveys were completed by 34/64 (53%) pediatric nephrologists, 46/113 (41%) PICU physicians, and 82 PICU nurses (response rate unknown). Over 65% of providers reported hemodialysis to be prescribed by nephrology; a mix of nephrology, ICU, or a shared nephrology-ICU model was reported responsible for peritoneal dialysis and continuous renal replacement therapy (CRRT). Severe hyperkalemia was the most important renal replacement therapy (RRT) indication for both nephrologists and PICU physicians (Likert scale from 0 [not important] to 10 [most important]; median = 10, 10, respectively). Nephrologists reported a lower threshold of AKI for increased mortality risk; 38% believed stage 2 AKI was the minimum compared to 17% of PICU physicians and 14% of nurses. Nephrologists were more likely than PICU physicians and nurses to recommend long-term follow-up for patients who develop any AKI during ICU stay (Likert scale from 0 [none] to 10 [all patients]; mean=6.0, 3.8, 3.7, respectively) (P < .05). Limitations: Responses from all eligible HCPs in the country could not obtained. There may be differences in opinions between HCPs that completed the survey compared to those that did not. Additionally, the cross-sectional design of our study may not adequately reflect changes in guidelines and knowledge since survey completion, although no specific guidelines have been released in Canada since survey dissemination. Conclusions: Canadian HCP groups have variable perspectives on pediatric AKI management and follow-up. Understanding practice patterns and perspectives will help optimize pediatric AKI follow-up guideline implementation.

Contexte: L'insuffisance rénale aiguë (IRA) chez les enfants gravement malades est associée à un risque accru d'issues défavorables à court et à long terme. En ce moment, il n'existe aucun suivi systématique pour les enfants qui développent une IRA pendant un séjour à l'unité des soins intensifs (USI). Objectif: Cette étude visait à évaluer les variations dans la prise en charge de l'IRA, de son importance perçue et de son suivi, tant au sein des groupes de professionnels de la santé (PS) qu'entre les différents groupes de PS. Conception: Des sondages transversaux à remplir de façon anonyme en ligne ont été menés à l'échelle nationale auprès de néphrologues pédiatriques canadiens, de médecins des unités de soins intensifs pédiatriques (USIP) et de membres du personnel infirmier des USIP ayant été répertoriés à partir de listes professionnelles. Cadre: Tous les néphrologues pédiatriques canadiens, médecins et membres du personnel infirmier qui traitent des enfants en USI étaient admissibles à répondre au sondage. Patients: S/O. Mesures: Les sondages comportaient des questions à choix multiples et des questions de type échelle de Likert qui portaient sur les pratiques actuelles de la gestion et de suivi à long terme de l'IRA, notamment sur les approches institutionnelles et personnelles de pratique et sur l'importance perçue de la gravité de l'IRA avec différents résultats. Méthodologie: Des statistiques descriptives ont été réalisées. Les réponses catégorielles ont été comparées à l'aide du chi-carré ou de tests exacts de probabilité de Fisher; les résultats des échelles de Likert ont été comparés à l'aide de tests de Mann-Whitney et de Kruskal-Wallis. Résultats: Les sondages ont été complétés par 53 % des néphrologues pédiatriques (34/64), 41 % des médecins d'USIP (46/113) et par 82 membres du personnel infirmier d'USIP (taux de réponse inconnu). Plus de 65 % des prestataires de soins ont déclaré que l'hémodialyse était prescrite par le service de néphrologie, alors que la dialyze péritonéale et la thérapie de remplacement rénal continu (TRRC) étaient confiées à la fois à la néphrologie, à l'USI ou à un modèle partagé néphrologie-USI. L'hyperkaliémie grave était l'indication la plus importante de la TRR pour les néphrologues et les médecins en USIP (échelle de Likert de 0 [pas important] à 10 [le plus important]; médiane = 10, 10, respectivement). Les néphrologues ont signalé un seuil inférieur d'IRA pour l'augmentation du risque de mortalité; 38 % d'entre eux estimaient que l'IRA de stade 2 était le seuil minimum, contre 17 % des médecins en USI et 14 % du personnel infirmier. Les néphrologues étaient plus susceptibles que les médecins et le personnel infirmier des USIP de recommander un suivi à long terme pour les patients qui développent une IRA pendant leur séjour en USI (échelle Likert de 0 [aucun] à 10 [tous les patients]; moyennes respectives = 6,0; 3,8 et 3,7 [p < 0,05]). Limites: Il n'a pas été possible d'obtenir les réponses de tous les PS admissibles au pays. Des différences d'opinions sont possibles entre les PS qui ont répondu au sondage et ceux qui ne l'ont pas fait. De plus, la conception transversale de notre étude pourrait ne pas refléter adéquatement les changements apportés aux lignes directrices et aux connaissances depuis la fin de cette enquête, bien qu'aucune ligne directrice particulière n'ait été publiée au Canada depuis la diffusion du sondage. Conclusion: Les divers groupes de professionnels de la santé canadiens ont des points de vue différents en ce qui concerne la prise en charge et le suivi de l'IRA chez les enfants. La compréhension des modèles de pratique et des perspectives permettra d'optimiser la mise en œuvre de directives de suivi de l'IRA pédiatrique.

"They go hand in hand": a patient-oriented, qualitative descriptive study on the interconnectedness between chronic health and mental health conditions in transition-age youth.

BACKGROUND: Transition-age youth (TAY) with chronic health conditions frequently experience co-occurring mental health conditions. However, little is known about the perspectives of TAY with co-occurring diagnoses preparing to exit pediatric health and mental health services. Research is needed to understand the impact of a mental health condition on transition readiness and self-management in TAY with chronic health conditions. METHODS: TAY (aged 16-20 years) with co-occurring chronic health and mental health conditions were recruited in Alberta, Canada. Nine semi-structured individual interviews were completed by phone or videoconference, and transcribed verbatim. Guided by qualitative description, we analyzed the data using thematic analysis in partnership with five young adults with lived experience in the health/mental health systems. RESULTS: Participants shared their experiences living with simultaneous physical and mental health concerns and preparing for transition to adult care. Our analysis revealed three overarching themes: 1) "they're intertwined": connections between chronic health and mental health conditions in TAY, 2) impact of mental health on transition readiness and self-management, and 3) recommendations for service provision from the perspectives of TAY. CONCLUSIONS: Our findings highlighted the myriad ways in which physical and mental health are connected as TAY prepare for service transitions using specific examples and powerful metaphors. TAY endorsed the importance of providers discussing these connections in routine clinical care. Future research should involve co-designing and evaluating educational material addressing this topic with diverse TAY, caregivers, and service providers.

"Walking on both sides of the fence": A qualitative exploration of the challenges and opportunities facing emergent clinician-scientists in child health.

RATIONALE, AIMS, AND OBJECTIVES: While paediatric clinician-scientists are ideally positioned to generate clinically relevant research and translate research evidence into practice, they face challenges in this dual role. The authors sought to explore the unique contributions, opportunities, and challenges of paediatric clinician-scientists, including issues related to training and ongoing support needs to ensure their success. METHOD: The authors used a qualitative descriptive approach with thematic analysis to explore the experiences of clinician-scientist stakeholders in child health (n = 39). Semi-structured interviews (60 min) were conducted virtually and recorded. Thematic analysis was conducted according to the phases outlined by Braun and Clarke (2006). RESULTS: The analysis resulted in the creation of three themes: (1) "Walking on both sides of the fence": unique positioning of clinician-scientists for advancing clinical practice and research; (2) the clinician-scientist: a specialized role with significant challenges; and (3) beyond the basics of clinical and research training programmes: essential skill sets and knowledge for future clinician-scientists. CONCLUSIONS: While clinician-scientists can make unique contributions to the advancement of evidence-based practice, they face significant barriers straddling their dual roles including divergent institutional cultures in healthcare and academia and a lack of infrastructure to effectively support clinician-scientist positions. Training programmes can play an important role in mentoring and supporting early-career clinician-scientists.

Child and caregiver perspectives on access to psychosocial and educational support in pediatric chronic kidney disease: a focus group study.

BACKGROUND: Children with chronic kidney disease (CKD) generally have worse educational and psychosocial outcomes compared with their healthy peers. This can impair their ability to manage their treatment, which in turn can have long-term health consequences through to adulthood. We attempted to capture the experiences of children with CKD and to describe the perspectives of their parents and caregivers on access to educational and psychosocial support. METHODS: Children with CKD (n = 34) and their caregivers (n = 62) were sampled via focus groups from pediatric hospitals in Australia, Canada, and the USA. Sixteen focus groups were convened and the transcripts were analyzed thematically. RESULTS: We identified four themes: disruption to self-esteem and identity (emotional turmoil of adolescence, wrestling with the sick self, powerlessness to alleviate child's suffering, balancing normality and protection); disadvantaged by lack of empathy and acceptance (alienated by ignorance, bearing the burden alone); a hidden and inaccessible support system (excluded from formal psychological support, falling behind due to being denied special considerations); and building resilience (finding partners in the journey, moving towards acceptance of the illness, re-establishing childhood). CONCLUSIONS: Children with CKD and their caregivers encountered many barriers in accessing psychosocial and educational support and felt extremely disempowered and isolated as a consequence. Improved availability and access to psychosocial and educational interventions are needed to improve the wellbeing and educational advancement of children with CKD. A higher resolution version of the Graphical abstract is available as Supplementary information.

IPNA clinical practice recommendations for the diagnosis and management of children with steroid-sensitive nephrotic syndrome.

Idiopathic nephrotic syndrome is the most frequent pediatric glomerular disease, affecting from 1.15 to 16.9 per 100,000 children per year globally. It is characterized by massive proteinuria, hypoalbuminemia, and/or concomitant edema. Approximately 85-90% of patients attain complete remission of proteinuria within 4-6 weeks of treatment with glucocorticoids, and therefore, have steroid-sensitive nephrotic syndrome (SSNS). Among those patients who are steroid sensitive, 70-80% will have at least one relapse during follow-up, and up to 50% of these patients will experience frequent relapses or become dependent on glucocorticoids to maintain remission. The dose and duration of steroid treatment to prolong time between relapses remains a subject of much debate, and patients continue to experience a high prevalence of steroid-related morbidity. Various steroid-sparing immunosuppressive drugs have been used in clinical practice; however, there is marked practice variation in the selection of these drugs and timing of their introduction during the course of the disease. Therefore, international evidence-based clinical practice recommendations (CPRs) are needed to guide clinical practice and reduce practice variation. The International Pediatric Nephrology Association (IPNA) convened a team of experts including pediatric nephrologists, an adult nephrologist, and a patient representative to develop comprehensive CPRs on the diagnosis and management of SSNS in children. After performing a systematic literature review on 12 clinically relevant PICO (Patient or Population covered, Intervention, Comparator, Outcome) questions, recommendations were formulated and formally graded at several virtual consensus meetings. New definitions for treatment outcomes to help guide change of therapy and recommendations for important research questions are given.

The Diagnostic Journey of Childhood Idiopathic Nephrotic Syndrome: Perspectives of Children and Their Caregivers.

Background: Childhood nephrotic syndrome is a rare kidney disease characterized by sudden onset of edema, massive proteinuria, and hypoalbuminemia. Rare diseases can have a long and difficult trajectory to diagnosis. Objective: We aimed to explore the experiences of children with nephrotic syndrome and their caregivers in their search of a nephrotic syndrome diagnosis. Design: An exploratory, qualitative descriptive study design. Setting: The Alberta Children's Hospital outpatient nephrology program in Calgary, Alberta, Canada. Sample: Children aged 9 to 18 years with steroid-sensitive nephrotic syndrome and their caregivers. Methods: We undertook semi-structured interviews with children (alone or with a caregiver present) and their caregivers using a question guide suitable to their age and role. We used a thematic analysis approach to inductively code the data and characterize themes related to our research question. Results: Participants included 10 children aged 9 to 18 years (6 boys and 4 girls) and 18 caregivers (8 men and 10 women). We characterized 3 themes related to participants' experiences in search of a diagnosis of nephrotic syndrome: (1) unexpected and distressing symptom onset, (2) elusiveness of a diagnosis, and (3) encountering a diagnosis. Children with nephrotic syndrome and their caregivers described experiencing initial anxiety due to their unusual and unexpected symptom onset and lack of awareness about the disease. Perceived diagnostic delays and incorrect diagnosis early in the course of the disease contributed to multiple consultations with a variety of care providers. Overall, participants expressed a desire to move past their diagnosis, learn about nephrotic syndrome, and engage in their treatment plans. Limitations: The views expressed by participants may not reflect those of individuals from other settings. The time elapsed since participants' nephrotic syndrome diagnosis may have influenced their recall of events and reactions to this diagnosis. Conclusions: In characterizing the diagnostic experiences of children and their caregivers, our study provides insight into how patients with nephrotic syndrome and their caregivers can be supported by the healthcare team along this journey. Focused strategies to increase awareness and understanding of nephrotic syndrome among healthcare providers are needed to improve patients' and families' diagnostic experiences.

Contexte: Le syndrome néphrotique infantile est une néphropathie rare caractérisée par l'apparition soudaine d'un œdème, d'une importante protéinurie et d'une hypoalbuminémie. La trajectoire jusqu'au diagnostic d'une maladie rare peut être longue et difficile. Objectif: Nous voulions sonder l'expérience des enfants atteints du syndrome néphrotique et celles de leurs soignants pendant le processus d'un diagnostic de syndrome néphrotique. Conception: Étude descriptive exploratoire et qualitative. Cadre: Le program de néphrologie ambulatoire du Alberta Children's Hospital de Calgary (Alberta) au Canada. Participants: Des enfants (9 à 18 ans) atteints du syndrome néphrotique sensible aux stéroïdes, et leurs soignants. Méthodologie: Un questionnaire adapté selon l'âge et le rôle a servi de guide pour les entrevues semi-structurées menées avec les enfants (seuls ou en présence d'un soignant) et leurs soignants. Nous avons utilisé une approche d'analyze thématique pour coder les données de façon inductive et caractériser les thèmes liés à notre question de recherche. Résultats: L'étude a inclus 10 enfants âgés de 9 à 18 ans (6 garçons, 4 filles) et 18 soignants (8 hommes, 10 femmes). Trois thèmes liés à l'expérience des participants dans le processus de recherche d'un diagnostic de syndrome néphrotique ont été caractérisés: (a) l'apparition de symptômes inattendus et affligeants (b) le caractère insaisissable du diagnostic, et (c) l'annonce du diagnostic. Les patients et les soignants ont mentionné avoir d'abord ressenti de l'anxiété en raison de l'apparition soudaine de symptômes inhabituels et du manque de sensibilisation à la maladie. Des retards perçus dans le diagnostic et les erreurs de diagnostic dans les stades précoces ont entraîné de nombreuses consultations auprès de divers fournisseurs de soins. Dans l'ensemble, les participants ont exprimé leur désir de dépasser le diagnostic, d'en apprendre davantage sur le syndrome néphrotique et de s'engager dans le plan de traitement. Limites: Les avis exprimés par les participants pourraient ne pas refléter l'expérience d'autres individus dans d'autres contextes. Le temps écoulé depuis le diagnostic du syndrome néphrotique chez les participants peut avoir influencé leur rappel des événements et les réactions mentionnées face à ce diagnostic. Conclusion: En caractérisant les expériences de diagnostic des enfants et de leurs soignants, notre étude fournit un aperçu de la façon dont les patients atteints du syndrome néphrotique et leurs soignants pourraient être soutenus par l'équipe soignante tout au long de leur parcours. Des stratégies ciblées visant à accroître la sensibilisation au syndrome néphrotique et sa compréhension chez les prestataires de soins de santé sont nécessaires pour améliorer l'expérience des patients et des familles face au diagnostic.

Involving Patient Partners in the KRESCENT Peer Review: Intent, Process, Challenges, and Opportunities.

Purpose of review: The Kidney Research Scientist Core Education and National Training (KRESCENT) is a national Canadian training program for kidney scientists, funded by the Kidney Foundation of Canada (KFOC), the Canadian Institutes of Health Research (CIHR), and the Canadian Society of Nephrology (CSN). We describe our first year of incorporating patient partners into a scientific peer-review committee, the 2017 committee to select senior research trainees and early-career kidney researchers for funding and training, in the hope that it will be helpful to others who wish to integrate the perspective of people with lived experience into the peer-review process. Sources of information: Other peer-review committees, websites, journal articles, patient partners, Kidney Foundation of Canada Research Council, Canadians Seeking Solutions and Innovations to Overcome Chronic Kidney Disease (Can-SOLVE CKD) Patient Council, participants in the 2017 Kidney Foundation of Canada KRESCENT peer-review panel. Methods: We describe our motivation, rationale, guiding principles, plans, feedback, implementation, and response. Key findings: We disseminated a "call for patient partners" 8 weeks before the meeting, seeking patients or their care givers to partner with the KRESCENT peer-review panel; we defined these people with lived experience of kidney disease as patient partners. Eight patient partners came forward and all participated as reviewers. Patient partners first participated in a webinar to learn about the function, structure, and processes of a peer-review committee. They practiced reviewing plain language summaries and giving feedback. In a subsequent teleconference, they shared and discussed their reviews. Plain language summaries were scored, overall, on the same 0-5 quality scale used by scientific reviewers. Three patient reviewers participated in some or all of the 6-hour meeting, which was conducted as usual, for this panel, by teleconference (initially audio only; from 2020 onwards by videoconference). In the meeting, the 2 assigned scientific reviewers first gave their scores, followed by the patient reviewers giving their scores, and discussion (mostly scientific, and conducted in usual scientific language). Scientific reviewers then negotiated a consensus score based on their initial scores, the discussion, patient reviewers' scores and statements, and the scientific officer's notes. Patient reviewers, scientific reviewers, and the Kidney Foundation of Canada (KFOC) were generally positive about the process. The increased length of the meeting (estimated at 1 hour) was generally thought to be acceptable. Patient reviewers also provided feedback on the methods used to incorporate patients into the research under review. These comments were concrete, insightful, and helpful. The patients did not uniformly recommend that basic scientists involve patients in their work. We did not detect bias against preclinical science, work that did not involve patients, or rarer diseases. Some patients found participation inspiring and enlightening. All participants appreciated the idea of patient partners as community witnesses to a group process committed to fairness and supportiveness. We discussed assigning formal meaningful weight to patient reviewers' assessments. Most, but not all, patients thought that the scientific reviewers were ultimately the best judges of the allocation of scarce research resources. Limitations: Patient participants tended to be Caucasian, middle class, and well educated. Because of the difficulties of travel for some people living with or supporting those living with kidney disease, our findings may not generalize fully to peer-review meetings that are conducted face to face. This is explicitly a supportive panel, committed to reviewing junior scientists with kindness as well as rigor; our findings may not generalize to panels conducted differently. We did not use formal qualitative methodology. Implications: Inclusion of patient partners as patient reviewers for the KRESCENT program peer-review panel was feasible, added value for scientific and patient reviewers, and for the funding stakeholders (CIHR, KFOC, and CSN). We were glad that we had taken this step and continue to refine the process with each successive competition.

Motif de la revue: Le KRESCENT (Kidney Research Scientist Core Education and National Training) est un programme national de formation pour les chercheurs en santé rénale financé par la Fondation canadienne du rein (FCR), les Instituts de recherche en santé du Canada (IRSC) et la Société canadienne de néphrologie (SCN). Nous décrivons notre première année d'intégration de partenaires patients dans un comité d'examen scientifique par les pairs, le comité de 2017, visant la sélection de stagiaires de recherche et de chercheurs en santé rénale en début de carrière pour le financement et la formation, dans l'espoir que cela sera utile à ceux qui souhaitent intégrer la perspective des personnes ayant une expérience vécue au processus d'examen par les pairs. Sources: Autres comités d'examen par les pairs, sites Web, articles de revues, partenaires patients, Conseil de recherche de la Fondation canadienne du rein, conseil des patients de Canadians Seeking Solutions and Innovations to Overcome Chronic Kidney Disease (CAN-SOLVE CKD), participants au comité d'examen par les pairs de la Fondation canadienne du rein de 2017. Méthodologie: Nous décrivons ce qui a motivé cette étude, notre raisonnement, nos principes directeurs, nos plans, la rétroaction, la mise en œuvre et les réponses. Principaux résultats: Nous avons diffusé un « appel à des partenaires patients ¼ huit semaines avant la réunion pour trouver des patients ou des soignants prêts à collaborer avec le comité d'examen par les pairs de KRESCENT; nous avons défini comme partenaires patients les personnes ayant une expérience vécue de maladie rénale. Huit partenaires patients ont répondu à l'appel et tous ont participé en tant qu'examinateurs. Les partenaires patients ont d'abord participé à un webinaire pour en apprendre davantage sur la fonction, la structure et les processus d'un comité d'examen par les pairs. Ils se sont ensuite entraînés à examiner des résumés en langage simple et à donner des commentaires. Lors d'une téléconférence ultérieure, ils ont partagé et discuté de leurs examens respectifs. Les résumés en langage clair ont été notés, dans l'ensemble, sur la même échelle de qualité de 0 à 5 utilisée par les examinateurs scientifiques. Trois patients examinateurs ont participé à une partie ou à la totalité de la réunion de 6 heures, qui s'est tenue comme d'habitude, pour ce panel, par téléconférence (initialement en audio seulement; par vidéoconférence à partir de 2020). Au cours de la réunion, les deux examinateurs scientifiques désignés ont d'abord donné leurs notes, puis les patients examinateurs ont donné leurs notes, et une discussion a suivi (principalement scientifique, et menée dans le langage scientifique habituel). Les examinateurs scientifiques ont ensuite négocié pour établir une note consensuelle en fonction de leurs notes initiales, de la discussion, des notes et des commentaires des patients examinateurs et des notes de l'agent scientifique.Les patients examinateurs, les examinateurs scientifiques et la Fondation canadienne du rein étaient généralement positifs à l'égard du processus. La durée accrue de la réunion (estimée à 1 heure) a généralement été jugée acceptable. Les patients examinateurs ont également fourni des commentaires sur les méthodes utilisées pour intégrer les patients à la recherche à l'étude. Ces commentaires étaient concrets, pertinents et utiles. Les patients ne recommandent pas uniformément que les scientifiques en recherche fondamentale impliquent les patients dans leur travail. Nous n'avons pas détecté de biais contre la science préclinique, les études qui n'impliquent pas de patients ou les maladies plus rares. Certains patients ont trouvé la participation inspirante et instructive. Tous les participants ont aimé l'idée des partenaires patients comme témoins communautaires d'un processus de groupe engagé dans l'équité et le soutien.Nous avons discuté de l'attribution d'un poids formel significatif aux évaluations des patients examinateurs. La plupart des patients, mais pas tous, étaient d'avis que les examinateurs scientifiques étaient en fin de compte les meilleurs juges de l'allocation des ressources limitées de la recherche. Limites: Les patients participants étaient pour la plupart de race blanche, de classe moyenne et bien éduqués. En raison des difficultés de déplacement pour certaines personnes vivant avec ou soutenant les personnes vivant avec une maladie rénale, nos résultats peuvent ne pas se généraliser entièrement aux réunions d'examen par les pairs menées en personne. Il s'agit essentiellement d'un groupe de soutien, qui s'est engagé à examiner les jeunes chercheurs avec bienveillance et rigueur; nos conclusions peuvent ne pas se généraliser à des groupes de travail menés différemment. Nous n'avons pas utilisé de méthodologie qualitative officielle. Résultats: L'inclusion de partenaires patients comme examinateurs dans un comité d'examen par les pairs du programme KRESCENT s'est avérée réalisable, et une valeur ajoutée pour les examinateurs scientifiques, les patients examinateurs et les parties responsables du financement (IRSC, FCR et SCN). Nous sommes heureux d'avoir franchi cette étape, nous continuons de raffiner le processus à chaque concours successif.

Equity, diversity and inclusion of pediatric clinician-scientists in Canada: a thematic analysis.

BACKGROUND: Underrepresented voices and perspectives are missing from academic and clinical health sciences. We aimed to define the unique opportunities and challenges of pediatric clinician-scientists related to equity, diversity and inclusion; and to identify key components of training needed to support people from equity-seeking groups as emerging and early-career pediatric clinician-scientists to generate diverse health research leaders in knowledge generation, implementation and translation. METHODS: Using a qualitative descriptive approach, we examined the experiences of clinician stakeholders. Semistructured interviews were conducted with pediatric clinician-scientist stakeholders. Thematic analysis was performed. RESULTS: We interviewed a total of 39 individuals. Our analysis resulted in 4 interrelated themes: the pervasiveness and invisibility of sexism; the invisibility and visibility of racism; proposed individual-level solutions to the sexism and racism; and proposed institutional and system-level changes to address the porous and leaky pipeline. These themes acknowledged that, ultimately, system change is required for addressing equity, diversity and inclusion in clinical and academic training environments. INTERPRETATION: These findings highlight the importance of addressing systemic biases that limit the inclusion of women and racialized individuals in pediatric clinician-scientist careers. Further research is needed to explore the problem of exclusion, which will, in turn, inform education of pediatric clinician-scientists and inform better ways to promote equity, diversity and inclusivity; these steps are needed to foster systemic change in the cultures that perpetuate exclusivity in both academic and clinical communities.

Association between transition readiness and mental health comorbidity in youth with chronic health conditions.

PURPOSE: Between 33 and 59% of youth with chronic health conditions experience mental health conditions. Transition readiness, or the acquisition of knowledge and self-management skills, facilitates successful transition to adult care. Transition readiness among youth with co-occurring chronic health and mental health conditions has not been explored. DESIGN AND METHODS: This study used a sample of 201 patients (aged 16-21) with chronic conditions. All patients completed the Transition Readiness Assessment Questionniare (TRAQ) and were grouped into Cohort A: chronic health conditions only (n = 140), and Cohort B: co-occurring chronic health and mental health conditions (n = 61). A quantile regression at the 50th percentile was conducted to examine associations between TRAQ score and mental health comorbidity, age, gender and immigration status. RESULTS: The median TRAQ score for Cohort A was 3.87 (IQR 0.84) versus 4.00 (IQR 0.87) for Cohort B. Our analysis revealed that having a mental health comorbidity (b = 0.402, p = 0.034), being older in age (b = 0.540, p = 0.004) and being female (b = 0.388, p = 0.001) were associated with higher overall TRAQ score. CONCLUSIONS: The presence of a mental health comorbidity was associated with greater transition readiness as measured by the TRAQ in our sample. Future research should explore why youth with co-occurring chronic health and mental health conditions exhibit greater transition readiness. PRACTICE IMPLICATIONS: Youth with co-occurring chronic health and mental health conditions may develop transition readiness as a result of coping with mental health challenges. Practitioners could invite them to reflect on how their physical and mental health are related and affect their level of preparedness for adult care.

Curricula, Teaching Methods, and Success Metrics of Clinician-Scientist Training Programs: A Scoping Review.

PURPOSE: To describe the literature on clinician-scientist training programs to inform the development of contemporary and inclusive training models. METHOD: The authors conducted a scoping review, searching the PubMed/MEDLINE, CINAHL, and Embase databases from database inception until May 25, 2020. Studies presenting primary research that described and evaluated clinician-scientist training programs were identified for data abstraction. On the basis of deductive and inductive methods, information about program characteristics, curricula, teaching strategies, and success metrics was extracted. The extracted variables were analyzed using descriptive statistics. RESULTS: From the initial 7,544 citations retrieved and 4,974 unique abstracts screened, 81 studies were included. Of the 81 included studies, 65 (80.2%) were published between 2011 and 2020, 54 (66.7%) were conducted in the United States, and 64 (79.0%) described programs that provided broad clinician-scientist training. Few programs provided funding or protected research time or specifically addressed needs of trainees from underrepresented minority groups. Curricula emphasized research methods and knowledge dissemination, whereas patient-oriented research competencies were not described. Most programs incorporated aspects of mentorship and used multiple teaching strategies, such as direct and interactive instruction. Extrinsic metrics of success (e.g., research output) were dominant in reported program outcomes compared with markers of intrinsic success (e.g., career fulfillment). CONCLUSIONS: Although programs are providing clinician-scientists with practical skills training, opportunities exist for curricular and pedagogic optimization that may better support this complex career path. Training programs for clinician-scientists can address contemporary issues of wellness and equity by reconsidering metrics of program success and evolving the core tenets of their education models to include equity, diversity, and inclusion principles and patient-oriented research competencies.

Primary care service use during adolescence and young adulthood: Tertiary care cohort affected by chronic health conditions.

OBJECTIVE: To understand use of family physician services and emergency department visits by adolescents and young adults with chronic health conditions. DESIGN: Longitudinal retrospective observational cohort study using administrative health data. SETTING: Chronic care clinics at a tertiary care pediatric hospital in Calgary, Alta. PARTICIPANTS: In total, 1326 adolescents who were between 12 and 15 years old in 2008, who were observed until 2016, and who received medical services for chronic conditions were enrolled in the study. Eligible participants had at least 4 visits to the same chronic disease clinic in any 2-year window before age 18. MAIN OUTCOMES MEASURES: Group-based trajectory modeling was used to identify groups of adolescents with distinct patterns of health care use (for visits to emergency departments and to primary care practices), while 2 tests explored trajectory group differences (eg, sex, location of residence). RESULTS: Median age was 14 years (range 12 to 17 years) at study entry, and 22 years (range 14 to 24 years) at study exit. Half were female and most (85.4%) lived in an urban area. Median observation period was 8.7 person-years (range 1.3 to 9.1 years). Group-based trajectory modeling identified 5 distinct trajectory groups of primary care use and 4 groups of emergency services use. Groups differed by sex and location of residence in each trajectory model. CONCLUSION: Many adolescents increased their use of emergency services between the ages of 12 and 24 years, with distinct patterns of primary care use being observed. Association of additional patient- and system-level factors (eg, disease severity, distance to nearest family physician office) should be explored.

Community based Primary Care for Adolescents and Young Adults Transitioning From Pediatric Specialty Care: Results from a Scoping Review.

BACKGROUND: Ongoing primary care during adolescence is recommended by best practice guidelines for adolescents and young adults (AYAs; ages 12-25) with chronic conditions. A synthesis of the evidence on the roles of Primary Care Physicians (PCPs) and benefits of primary care is needed to support existing guidelines. METHODS: We used Arksey and O'Malley's scoping review framework, and searched databases (MEDLINE, EMBASE, PsychINFO, CINAHL) for studies that (i) were published in English between 2004 and 2019, (ii) focused on AYAs with a chronic condition(s) who had received specialist pediatric services, and (iii) included relevant findings about PCPs. An extraction tool was developed to organize data items across studies (eg, study design, participant demographics, outcomes). RESULTS: Findings from 58 studies were synthesized; 29 (50%) studies focused exclusively on AYAs with chronic health conditions (eg, diabetes, cancer), while 19 (33%) focused exclusively on AYAs with mental health conditions. Roles of PCPs included managing medications, "non-complex" mental health conditions, referrals, and care coordination, etc. Frequency of PCP involvement varied by AYAs; however, female, non-Black, and older AYAs, and those with severe/complex conditions appeared more likely to visit a PCP. Positive outcomes were reported for shared-care models targeting various conditions (eg, cancer, concussion, mental health). CONCLUSION: Our findings drew attention to the importance of effective collaboration among multi-disciplinary specialists, PCPs, and AYAs for overcoming multiple barriers to optimal transitional care. Highlighting the need for further study of the implementation of shared care models to design strategies for care delivery during transitions to adult care.

Rituximab Use for the Treatment of Childhood Nephrotic Syndrome by Canadian Pediatric Nephrologists: A National Survey.

Background: There is known practice variation in the treatment of frequently relapsing, steroid-dependent, and steroid-resistant nephrotic syndrome in children. Rituximab is an emerging therapy for difficult-to-treat nephrotic syndrome; however, there are no clear treatment guidelines. We therefore hypothesized that a wide variety of approaches to this therapy exist. Objective: To evaluate when and how rituximab is used for the treatment of childhood nephrotic syndrome in Canada. Design and setting: An online survey was used. Participants: Canadian pediatric nephrologists. Methods: A cross-sectional survey was distributed across Canada through the Canadian Association of Pediatric Nephrologists (CAPN) to evaluate rituximab treatment practices. Results: Of a total of 20 responses, 19 (95%) use rituximab in the treatment of nephrotic syndrome, usually as a third or fourth agent. For the number of rituximab doses, the majority (68%) uses 2 doses each time they use it. Eighteen respondents (90%) measure B cells when using this medication, mostly monthly (50%) or every 3 months (39%). Respondents were administered additional doses of rituximab prophylactically (74%) or at first relapse (47%). Long-term drug safety and drug funding were identified as the main barriers to rituximab use. Limitations: This survey represents the practice styles of physicians in a single country, and there is a nonresponse bias of 63%. Also, associations were not calculated. Conclusions: Among Canadian pediatric nephrologists, rituximab use for nephrotic syndrome appears to be increasing, but significant practice variations remain, including approaches to B-cell monitoring. It is reserved mostly for second-line and third-line use due to cost, funding issues, and residual uncertainty regarding long-term safety. Understanding these critical areas of practice uncertainty is a first step to optimize treatment of nephrotic syndrome in children.

Contexte: Des variations sont connues dans les pratiques liées au traitement du syndrome néphrotique infantile, dépendant ou résistant aux stéroïdes, à récidives fréquentes. Le rituximab constitue une nouvelle approche thérapeutique pour soigner le syndrome néphrotique difficile à traiter. Il n'existe cependant aucune directive de traitement claire dans ce contexte. Nous avons donc émis l'hypothèse qu'il existait une grande variété d'approches dans l'utilisation de ce médicament. Objectifs: Examiner les pratiques d'utilisation (quand et comment) du rituximab dans le traitement du syndrome néphrotique infantile au Canada. Cadre et type d'étude: Étude menée par sondage en ligne. Participants: Des néphrologues pédiatriques canadiens. Méthodologie: Un sondage transversal a été distribué partout au Canada par l'entremise de l'Association canadienne des néphrologues pédiatriques (ACPN) afin d'examiner les pratiques de traitement au rituximab. Résultats: Parmi les 20 néphrologues ayant répondu au sondage, 19 (95 %) utilisent le rituximab dans le traitement du syndrome néphrotique, généralement comme 3e ou 4e agent, et la majorité d'entre eux (68 %) administre deux doses à chaque utilisation. Dix-huit répondants (90 %) mesurent les lymphocytes B lorsqu'ils emploient ce médicament, principalement tous les mois (50 %) ou tous les trois mois (39 %). Certains répondants avaient administré des doses additionnelles de rituximab à des fins prophylactiques (74 %) ou lors de la première rechute (47 %). Le recours au rituximab serait principalement freiné par des enjeux liés à son innocuité à long terme et au financement des médicaments. Limitations: Ce sondage représente les pratiques des médecins d'un seul pays et comporte un biais de non-réponse de 63 %. De plus, les associations n'ont pas été calculées. Conclusion: Chez les néphrologues pédiatriques canadiens, l'utilisation du rituximab dans le traitement du syndrome néphrotique semble augmenter; bien que des variations significatives demeurent dans la pratique, notamment en ce qui concerne les approches de surveillance des lymphocytes B. Actuellement, le rituximab est surtout utilisé comme thérapie de deuxième et de troisième ligne en raison de son coût, d'enjeux liés au financement et d'une incertitude résiduelle en ce qui concerne son innocuité à long terme. La compréhension de ces zones critiques d'incertitude dans les pratiques est une première étape pour optimiser le traitement du syndrome néphrotique chez les enfants.

Perspectives of Clinicians on Shared Decision Making in Pediatric CKD: A Qualitative Study.

RATIONALE & OBJECTIVE: Clinical decision-making priorities may differ among children, their parents, and their clinicians. This study describes clinicians' perspectives on shared decision making in pediatric chronic kidney disease (CKD) and identifies opportunities to improve shared decision making and care for children with CKD and their families. STUDY DESIGN: Semistructured interviews. SETTING & PARTICIPANTS: Fifty clinicians participated, including pediatric nephrologists, nurses, social workers, surgeons, dietitians, and psychologists involved in providing care to children with CKD. They worked at 18 hospitals and 4 university research departments across 11 countries (United States of America, Canada, Australia, People's Republic of China, United Kingdom, Germany, France, Italy, Lithuania, New Zealand, and Singapore). ANALYTICAL APPROACH: Interview transcripts were analyzed thematically. RESULTS: We identified 4 themes: (1) striving to blend priorities (minimizing treatment burden, emphasizing clinical long-term risks, achieving common goals), (2) focusing on medical responsibilities (carrying decisional burden and pressure of expectations, working within system constraints, ensuring safety is foremost concern), (3) collaborating to achieve better long-term outcomes (individualizing care, creating partnerships, encouraging ownership and participation in shared decision making, sensitive to parental distress), and (4) forming cumulative knowledge (balancing reassurance and realistic expectations, building understanding around treatment, harnessing motivation for long-term goals). LIMITATIONS: Most clinicians were from high-income countries, so the transferability of the findings to other settings is uncertain. CONCLUSIONS: Clinicians reported striving to minimize treatment burden and working with children and their families to manage their expectations and support their decision making. However, they are challenged with system constraints and sometimes felt the pressure of being responsible for the child's long-term outcomes. Further studies are needed to test whether support for shared decision making would promote strategies to establish and improve the quality of care for children with CKD.