The European Kidney Health Alliance (EKHA) and the Decade of the KidneyTM.

The European Kidney Health Alliance (EKHA) is an advocacy organization that defends the case of the kidney patients and the nephrological community at the level of the European Union (EU), and from there, top to bottom, also at the national level of the EU member states and the EU-associated countries. The Decade of the KidneyTM is a global initiative launched by the American Association of Kidney Patients (AAKP) to create greater awareness and organize patient demands for long overdue innovation in kidney care. This article describes the medical and patient burden of kidney disease, the history of EKHA, its major activities and tools for policy action, and the need for innovation of kidney care. We then describe the Decade of the KidneyTM initiative, the rationale behind why EKHA joined this activity to emanate parallel action at the European side, the novel professionalized structure of EKHA, and its immediate targets. The final aim is to align all major stakeholders for an action plan on kidney disease comparable to Europe's successful Beating Cancer Plan, with the additional intent that the EKHA model is applied also by the respective national kidney-related societies to create a broad mobilization at all levels. The ultimate aims are that the EU considers chronic kidney disease (CKD) as a major health and health-economic problem, to consequently have CKD included as a key health research target by the European Commission, and to improve quality of life and outcomes for all kidney patients.

Care of suspected long bone fractures in the emergency department: Families' perspectives and priorities.

OBJECTIVE: Despite growing interests in patient-reported outcomes, youth and families are rarely involved in designing quality improvement measures. Few quality indicators exist for the care of children with injuries in the Emergency Department (ED) and extremity fractures are among the most common injuries in children. This study's aim was to identify both parents' and youth's perspectives about ED care in the context of a suspected long-bone fracture. METHODS: Youth (10-18 years old) and their parents were surveyed prospectively during their ED visit. Participants were asked: 1) to identify their main concerns, 2) to identify quality measures that were most important to them, and 3) to evaluate the ED care they received. Descriptive analyses present participants' responses. Continuous data was analyzed using a Student t-test and categorical data using a Chi-square test. RESULTS: Over 15 months, 350 families met eligibility criteria and were approached to participate, of which 300 participants consented and 249 surveys were completed (71% response rate): 148 parents and 101 youth (median age: 12) completed their respective surveys. Participants placed a high importance on several themes: pain management, short length of stay, and quality interactions with ED clinicians. Youth as a group prioritized their overall wellbeing and the ED environment (e.g., waiting room comfort, signage), while parents focused on accurate diagnoses and treatments. The following items were less prioritized: that radiology be close to the ED, to see the radiograph, to have access to a wheelchair, to know the identities of clinicians on the team, and to have access to entertainment. Parents and youth within the same family often did not share the same priorities. Ninety-two percent of parents reported their child's pain was treated, while 81% and 63% of youth reported their pain was treated sufficiently and quickly, respectively. CONCLUSIONS: Parents and youth can identify their priorities for ED care and should be engaged in efforts to improve and report on the quality of care in the ED. Youths' and parents' perspectives are complimentary and may not align, even within families. The priorities identified in this study can help inform quality improvement initiatives and personalized patient care.

Pro: Can we influence postoperative outcomes of frail patients after cardiac surgery?

A significant proportion of cardiac surgery patients are frail. The first step to improve outcomes is recognizing that despite reduced postoperative mortality in cardiac surgery, postoperative morbidity remains high, most likely due to more frail patients undergoing surgeries. Moving from a traditional to a patient-oriented outcome paradigm could improve decision-making and help achieve treatment goals relevant to frail patients, such as quicker recovery, symptom control, and strategies to reduce postoperative complications. Frailty recognition can prompt preoperative and postoperative interventions. Exercise programs and nutrition optimization support frail patients in the perioperative period and positively alter outcomes.

Con: Can We Influence Postoperative Outcomes of Frail Patients After Cardiac Surgery?

Frail patients are scheduled routinely for elective cardiovascular surgery. Despite the proven association between frailty and postoperative outcomes, currently, there is no consistent evidence that the preoperative optimization of frail patients with preoperative nutrition support and exercise programs can alter postoperative mortality. Established frailty diagnostic tools are not sufficient to grasp the multidimensional nature of frailty in cardiovascular patients. Prehabilitation programs and preoperative nutritional support are time- and resource-consuming and, therefore, are hard to implement in routine clinical practice.

Association between frailty and patient outcomes after cancer surgery: a population-based cohort study.

BACKGROUND: Frailty is associated with poor postoperative outcomes, but existing data do not describe frailty's interaction with tumour characteristics at the time of cancer surgery. Our objective was to estimate the association between frailty and long-term survival, and to explore any interaction with tumour stage and grade. METHODS: This was a population-based cohort study conducted using linked provincial health administrative data in Ontario, Canada (2009-20). Using a cancer registry, we identified adults having elective cancer surgery. Frailty was measured using a validated index (range 0-1; higher score=greater frailty). Associations between frailty and long-term postoperative survival (primary outcome) were estimated using proportional hazards regression. Secondary outcomes were length of stay, discharge destination, days alive at home, and healthcare costs. RESULTS: We identified and included 52 012 patients. Mean frailty score was 0.13 (standard deviation 0.07). During follow-up, 19 378 (37.3%) patients died. After adjustment for risk factors, each 10% increase in frailty was associated with a 1.60-fold relative decrease in survival (95% confidence interval: 1.56-1.64). The frailty-survival association was strongest for patients with lower stage and grade cancers. Increased frailty was associated with longer hospital stays (3 days), fewer days alive and at home (42 days yr-1), more frequent discharge to a nursing facility (2.38-fold), and increased healthcare costs ($6048). CONCLUSIONS: Patient frailty is associated with decreased long-term survival after cancer surgery. The association is stronger for early-stage and -grade cancers, which would otherwise have a better survival prognosis.

Early Sport Specialization in College Athletes and the Impact on Health-Related Quality of Life: A Critically Appraised Topic.

CLINICAL SCENARIO: Youth athletes may specialize in a sport of their choosing, or based on external pressures, to pursue elite status in that sport. Current evidence shows an association between highly specialized athletes and an increase in injuries as well as a connection between injury and lower health-related quality of life (HRQOL). CLINICAL QUESTION: In college athletes, do early sport specialization characteristics (ie, age at specialization and degree of specialization) impact current HRQOL? SUMMARY OF KEY FINDINGS: The literature was searched for studies that investigated the age of specialization (early vs late) or degree of specialization (high, moderate, and low) and the impact on HRQOL. (1) The search returned 6 possible studies related to the clinical question. Three of the studies met the inclusion criteria and were used for this appraisal. (2) Two of the 3 included studies reported that highly specialized athletes noted lower HRQOL. (3) One study found there to be no significant difference in HRQOL between athletes who specialized early versus late but did find those who specialized early to have a greater incidence of injuries that required surgery. CLINICAL BOTTOM LINE: There is moderate evidence that early sport specialization is associated with lower HRQOL compared with late sport specialization. It is important to educate athletes, parents, and coaches on the potential detriments that are associated with early sport specialization to allow stakeholders to make informed decisions regarding participation. STRENGTH OF RECOMMENDATION: Grade B evidence exists to support the idea that early, intensive sport specialization may be associated with decreased HRQOL in current college athletes.

Frailty status among older critically ill patients with severe acute kidney injury.

BACKGROUND: Frailty status among critically ill patients with acute kidney injury (AKI) is not well described despite its importance for prognostication and informed decision-making on life-sustaining therapies. In this study, we aim to describe the epidemiology of frailty in a cohort of older critically ill patients with severe AKI, the outcomes of patients with pre-existing frailty before AKI and the factors associated with a worsening frailty status among survivors. METHODS: This was a secondary analysis of a prospective multicentre observational study that enrolled older (age > 65 years) critically ill patients with AKI. The clinical frailty scale (CFS) score was captured at baseline, at 6 months and at 12 months among survivors. Frailty was defined as a CFS score of ≥ 5. Demographic, clinical and physiological variables associated with frailty as baseline were described. Multivariable Cox proportional hazard models were constructed to describe the association between frailty and 90-day mortality. Demographic and clinical factors associated with worsening frailty status at 6 months and 12 months were described using multivariable logistic regression analysis and multistate models. RESULTS: Among the 462 patients in our cohort, median (IQR) baseline CFS score was 4 (3-5), with 141 (31%) patients considered frail. Pre-existing frailty was associated with greater hazard of 90-day mortality (59% (n = 83) for frail vs. 31% (n = 100) for non-frail; adjusted hazards ratio [HR] 1.49; 95% CI 1.11-2.01, p = 0.008). At 6 months, 68 patients (28% of survivors) were frail. Of these, 57% (n = 39) were not classified as frail at baseline. Between 6 and 12 months of follow-up, 9 (4% of survivors) patients transitioned from a frail to a not frail status while 10 (4% of survivors) patients became frail and 11 (5% of survivors) patients died. In multivariable analysis, age was independently associated with worsening CFS score from baseline to 6 months (adjusted odds ratio [OR] 1.08; 95% CI 1.03-1.13, p = 0.003). CONCLUSIONS: Pre-existing frailty is an independent risk factor for mortality among older critically ill patients with severe AKI. A substantial proportion of survivors experience declining function and worsened frailty status within one year.

Clinical outcomes after magnetic resonance angiography (MRA) versus computed tomographic angiography (CTA) for pulmonary embolism evaluation.

PURPOSE: To compare patient outcomes following magnetic resonance angiography (MRA) versus computed tomographic angiography (CTA) ordered for suspected pulmonary embolism (PE). METHODS: In this IRB-approved, single-center, retrospective, case-control study, we reviewed the medical records of all patients evaluated for PE with MRA during a 5-year period along with age- and sex-matched controls evaluated with CTA. Only the first instance of PE evaluation during the study period was included. After application of our exclusion criteria to both study arms, the analysis included 1173 subjects. The primary endpoint was major adverse PE-related event (MAPE), which we defined as major bleeding, venous thromboembolism, or death during the 6 months following the index imaging test (MRA or CTA), obtained through medical record review. Logistic regression, chi-square test for independence, and Fisher's exact test were used with a p < 0.05 threshold. RESULTS: The overall 6-month MAPE rate following MRA (5.4%) was lower than following CTA (13.6%, p < 0.01). Amongst outpatients, the MAPE rate was lower for MRA (3.7%) than for CTA (8.0%, p = 0.01). Accounting for age, sex, referral source, BMI, and Wells' score, patients were less likely to suffer MAPE than those who underwent CTA, with an odds ratio of 0.44 [0.24, 0.80]. Technical success rate did not differ significantly between MRA (92.6%) and CTA (90.5%) groups (p = 0.41). CONCLUSION: Within the inherent limitations of a retrospective case-controlled analysis, we observed that the rate of MAPE was lower (more favorable) for patients following pulmonary MRA for the primary evaluation of suspected PE than following CTA.

Primary Total Knee Arthroplasty: How Does Residual Patellar Thickness Affect Patient-Oriented Outcomes?

BACKGROUND: Maintenance of the native patellar thickness has been deemed important for proper clinical outcomes after total knee arthroplasty (TKA). Our objective was to study the effects of the change in patellar thickness on patient-perceived outcomes (PPOs) after TKA. We hypothesized that reestablishing native patellar thickness after TKA results in better PPOs. METHODS: 819 consecutive patients undergoing primary TKA were studied. Patients were classified according to their postoperative patellar thickness into: (1) less than native patella thickness; (2) equal to native patella thickness; and (3) greater than native patella thickness. Difference in postoperative range of motion (ROM), PPOs and clinical scores, and delta-Δ-change were assessed. MANCOVA was used to assess for differences. RESULTS: No significant differences found based on postoperative patellar thickness (mean follow-up: 4.9 ± 2.1 years) for each ROM assessments. Those who reported more stiffness (Western Ontario and McMaster Universities Arthritis Index stiffness; P = .011) and lower knee active flexion (P = .046) preoperatively had "greater than native patella thickness" after surgery. Postoperatively, the "equal to native patella thickness" group reported significantly better quality of life (quality of well-being scale 7 total; P = .008) as well as better physical score (Short Form-36 role physical score; P = .03). The amount of improvement (delta-Δ-change), when restoring patellar thickness equal to the native demonstrated greatest improvements in quality of life (quality of well-being scale 7 total; P = .016) physical measures (Short Form-36 role physical [P = .025], and Western Ontario and McMaster Universities Arthritis Index stiffness scores [P = .006]). CONCLUSION: When compared with the native patellar thickness, a final postoperative difference (delta thickness) that ranges from -1.06 to 2.58 mm provides satisfactory results and does not seem to affect ROM after surgery.

The Impact of Injury on Health-Related Quality of Life in College Athletes.

CONTEXT: Health-related quality of life (HRQOL) is a broad term for the impact of injury or illness on physical, psychological, and social health dimensions. Injury has been associated with decreased HRQOL in athletes. However, the influence of injury history, participation status, time since last injury, and injury severity on HRQOL remains unclear. OBJECTIVE: To compare HRQOL in collegiate athletes based on injury history, participation status, time since last injury, and injury severity and to examine relationships between HRQOL outcomes. DESIGN: Cross-sectional. SETTING: 3 National Collegiate Athletic Association (NCAA) institutions. PARTICIPANTS: 467 collegiate athletes (199 males, 268 females; 19.5 ± 1.3 y, 173.9 ± 10.5 cm, 71.9 ± 13.6 kg) were recruited from NCAA Division I (n = 299) and Division III (n = 168) institutions. Athletes were included regardless of participation status, which created a diverse sample of current and past injury histories. MAIN OUTCOME MEASURES: During a single session, participants completed an injury history form, the Disablement in the Physically Active Scale (DPA), and the Fear-Avoidance Beliefs Questionnaire (FABQ). Dependent variables included DPA-Physical Summary Component (DPA-PSC), DPA-Mental Summary Component (DPA-MSC), and FABQ Scores. RESULTS: HRQOL differences were detected between groups based on injury history, participation status, and time since last injury. No differences were detected for injury severity. A moderate correlation was identified between the DPA-PSC and FABQ (rs = 0.503, P < .001) and a weak relationship was identified between the DPA-MSC and FABQ (rs = 0.266, P < .001). CONCLUSIONS: Injury negatively influenced HRQOL in athletes with a current injury. While those individuals participating injured reported better HRQOL than the athletes sidelined due to injury, deficits were still present and should be monitored to ensure a complete recovery. Identifying the patient's perception of impairment will help facilitate evidencebased treatment and rehabilitation strategies that target the physical and psychosocial aspects of health.

Nurses' Perceptions of Interventions for the Management of Patient-Oriented Outcomes: A Key Factor for Evidence-Based Practice.

BACKGROUND: Various barriers and facilitators to implementing evidence-based interventions in practice have been acknowledged. Nurses' perspectives on these interventions were overlooked as potential factors that influence their uptake in practice. The purpose of this study was to explore nurses' perception of evidence-based interventions targeting patient-oriented outcomes. METHODS: A mixed method design involving concurrent application of quantitative and qualitative approaches was used. Nurses (n = 56) working in acute and rehabilitation care settings completed the Intervention Acceptability scale and responded to open-ended questions. The scale presented information on the components, activities, dose, and mode of delivering evidence-based interventions targeting each patient-oriented outcome (fatigue, nausea and vomiting, dyspnea, pain, physical function, self-care) and items to rate the interventions on five attributes (relevance, applicability, frequency of use, likelihood, and comfort in implementation). The open-ended questions inquired about the appropriateness and resources needed to use the interventions in practice. RESULTS: The quantitative results indicated favorable perceptions of most interventions. Nurses rated acupressure, guided imagery, massage, and relaxation as having limited appropriateness and they reported low levels of comfort in applying them. The qualitative themes clarified the reasons underlying nurses' ratings. LINKING EVIDENCE TO ACTION: This study's findings highlight the importance of examining nurses' perceptions of evidence-based interventions as an initial step toward promoting the adoption of interventions in practice. Effective interventions that are considered of limited relevance are unlikely to be implemented in practice. Nurses' perceptions can guide the design or selection of dissemination strategies to clarify any misconception about the effectiveness and risk of evidence-based interventions.

A prospective study of changes in oral health-related quality of life during immediate function implant procedures for edentulous individuals.

OBJECTIVE: The aim of this prospective clinical study was to evaluate longitudinal changes in oral health-related quality of life (OHRQoL) attributable to fixed dental prostheses during All-on-4(®) treatment in one or both jaws. MATERIALS AND METHODS: Ten patients underwent placement of four or six endosteal dental implants on the basis of the All-on-4(®) treatment concept in the edentulous maxilla or both jaws and immediate loading with acrylic interim prostheses. The prostheses were replaced after 3-6 months, and definitive prostheses with titanium framework and reinforced resin facing were fixed after another 5 months or more. The subjects completed the shortened Japanese version of the Oral Health Impact Profile (OHIP-J14) before the surgery (T0), 1 week after the initial (T1) and secondary (T2) interim prostheses were placed, and 3 months after definitive prosthesis placement (T3). Complete data of nine subjects were analyzed with the Wilcoxon signed-rank test. RESULTS: The total OHIP-J14 score significantly reduced only at T3 (P < 0.05). "Functional limitation," "physical pain," "physical disability," and "psychological disability" scores significantly decreased at T3, and "psychological discomfort" scores also significantly dropped at T2. "Social disability" and "handicap" scores remained unchanged throughout. CONCLUSION: Fixed definitive prostheses with metal framework are more effective than fixed all-acrylic prostheses in improving OHRQoL during All-on-4(®) treatment.

A randomized controlled trial of patient-reported outcomes with tai chi exercise in Parkinson's disease.

A previous randomized, controlled trial of tai chi showed improvements in objectively measured balance and other motor-related outcomes in patients with Parkinson's disease. This study evaluated whether patient-reported outcomes could be improved through exercise interventions and whether improvements were associated with clinical outcomes and exercise adherence. In a secondary analysis of the tai chi trial, patient-reported and clinical outcomes and exercise adherence measures were compared between tai chi and resistance training and between tai chi and stretching exercise. Patient-reported outcome measures were perceptions of health-related benefits resulting from participation, assessed by the Parkinson's Disease Questionnaire (PDQ-8) and Vitality Plus Scale (VPS). Clinical outcome measures included motor symptoms, assessed by a modified Unified Parkinson's Disease Rating Scale-Motor Examination (UPDRS-ME) and a 50-foot speed walk. Information on continuing exercise after the structured interventions were terminated was obtained at a 3-month postintervention follow-up. Tai chi participants reported significantly better improvement in the PDQ-8 (-5.77 points, P = 0.014) than did resistance training participants and in PDQ-8 (-9.56 points, P < 0.001) and VPS (2.80 points, P = 0.003) than did stretching participants. For tai chi, patient-reported improvement in the PDQ-8 and VPS was significantly correlated with their clinical outcomes of UPDRS-ME and a 50-foot walk, but these correlations were not statistically different from those shown for resistance training or stretching. However, patient-reported outcomes from tai chi training were associated with greater probability of continued exercise behavior than were either clinical outcomes or patient-reported outcomes from resistance training or stretching. Tai chi improved patient-reported perceptions of health-related benefits, which were found to be associated with a greater probability of exercise adherence. The findings indicate the potential of patient perceptions to drive exercise behavior after structured exercise programs are completed and the value of strengthening such perceptions in any behavioral intervention.

Effects of improved patient participation in primary care on health-related outcomes: a systematic review.

BACKGROUND: In primary care, many consultations address symptom-based complaints. Recovery from these complaints seldom exceeds placebo effects. Patient participation, because of its supposed effects on trust and patient expectancies, is assumed to benefit patients' recovery. While the idea is theoretically promising, it is still unclear what the effects of increased patient participation are on patient outcomes. Aim. To review the effects of controlled intervention studies aiming to improve patient participation in face-to-face primary care consultations on patient-oriented and/or disease-oriented outcomes. METHODS: This study is a systematic review. A systematic search was undertaken for randomized controlled trials designed to measure the effects of interventions that aimed to improve adult patients' participation in primary care visits. The CINAHL, Cochrane, EMBASE, PsycINFO and PubMed databases were searched. RESULTS: Seven different trials fulfilled the inclusion criteria. Three of the studies were related to symptom-based complaints. Five studies measured patient-oriented outcomes, the primary outcome of interest for this review. All studies suffered from substantial bias. Studies varied widely in their aims, types of complaints/diseases, strength of the interventions and their outcomes. The effects on patient-oriented outcomes and disease-oriented outcomes were ambiguous. CONCLUSION: Little research has been performed on health outcomes of interventions aiming to increase patient participation in general practice visits among patients suffering from symptom-based complaints. The results still are non-conclusive. The quality of the trials has been weak, possibly due to the complexity of the concept. This weak quality may explain the lack of conclusive results. Proposals for future research designs are offered.

How to Improve the Care of Women after Childbirth in the Rooming-in Unit: A Prospective Observational Study.

Rooming-in is the WHO-recommended care system for mothers in the puerperium and their babies. This system allows the newborn to stay with the mother in the same room, 24 h a day. We aimed to investigate the need to entrust a newborn (NEN) in the care of maternity rooming-in staff during the COVID-19 pandemic, and its relationship to pain, anxiety, and blood loss after delivery. A prospective study of 200 adult women in the maternity ward operating in the rooming-in system focussed on NEN in the care of maternity rooming-in staff on the first (T1) and the second day of puerperium (T2). Women who declared having NEN were compared with women without NEN for anxiety, pain, and a drop in haemoglobin in the blood after delivery. In T1, 34% and in T2, 27% of women felt NEN in the care of maternity rooming-in staff. The NEN of women after a cesarean section was higher on both days than the NEN of women after vaginal delivery. Women with NEN had higher levels of pain, state anxiety, and higher levels of postpartum anxiety than women without NEN. Further research should be warranted to investigate whether women who give birth in hospitals that satisfy the NEN in the care of maternity rooming-in staff in their rooming-in units experience less pain and anxiety in comparison to those who give birth in hospital units without such a possibility and whether this factor is an important element in reducing anxiety and pain during puerperium.

Validation of the Numerical Anxiety Rating Scale in postpartum females: a prospective observational study.

OBJECTIVES: Perinatal anxiety is important for the quality of life of mothers and their offspring. The Numerical Anxiety Rating Scale (NRS-A) allows the level of anxiety in patients to be quickly assessed. Until now, the NRS-A has not been validated in the postpartum female population. The aim of this study was to assess the accuracy and reliability of the NRS-A when compared with the reference methods for measuring anxiety. MATERIAL AND METHODS: The observational prospective study included a group of 200 adult postpartum females of a hospital maternity ward. The validity between the Numerical Rating Scale for Anxiety (NRS-A) and the State and Trait Anxiety Inventory (STAI), and between the NRS-A and the Hospital Anxiety and Depression Scale (HADS-A), was determined. The detection thresholds for high anxiety were examined. RESULTS: Both measurements showed a positive high correlation between the NRS and STAI-S (in T1 rho = 0.807, in T2 rho = 0.778; p < 0.001), and a comparable relationship of both scales (STAI-S and NRS-A) with the STAI-T and HADS-A. The analysis of the ROC curve indicated that the value of the NRS-A equal to 3.5/10 can be considered the threshold that allows for a differentiation of patients with high anxiety from those without high anxiety in the studied population. CONCLUSIONS: The NRS-A is an accurate tool for measuring anxiety in Polish postpartum females. Routine anxiety measurements using the NRS-A can be used to identify people with high anxiety in order to provide emotional support to patients in the early postpartum period.

Mental Health Care for Adults Treated With Dialysis in Canada: A Scoping Review.

Purpose of review: Mental illnesses, especially depression and anxiety, are common conditions frequently underrecognized and untreated among individuals with end-stage kidney disease. Existing tools/interventions, approaches to care, and resources to support mental health for people treated with dialysis in Canada are not well known. The aim of this scoping review was to systematically describe how mental health care is provided to adults treated with dialysis in Canada. The research questions we sought to answer were the following: (1) What mental health assessment tools and interventions for adults treated with dialysis have been investigated and utilized in Canada? (2) what is the structure and process of mental health care delivered by kidney care to adults treated with dialysis in Canada? and (3) what is the availability of mental health care resources developed for adults treated with dialysis in Canada? Sources of information: Electronic databases included Medline, Embase, CINAHL, PsycINFO, Cochrane Library, Scopus, and Web of Science for peer-reviewed literature, and Google search engine for gray literature. Methods: Systematic searches were conducted to identify peer-reviewed and gray literature that focused on mental health care or support offered to adults receiving any form of dialysis in Canadian kidney care settings and/or community organizations. Mental health care and support was defined to include mental health assessment tools, interventions, resources, guidelines, and/or pathways used in dialysis in Canada. Title, abstracts, and full texts were screened independently by 2 reviewers with discrepancies resolved by additional team members. Web sites were screened by individual members. Data from included studies and Web sites were extracted based on the abovementioned research questions. Key findings: We identified 8 peer-reviewed articles from electronic databases, and 28 separate Web site addresses. Of the 8 articles, 4 investigated mental health assessment tools and 4 examined mental health interventions for individuals treated with dialysis in Canada. The mental health assessment tools that have been used are Beck Depression Inventory-II (BDI-II), Edmonton System Assessment Scale (ESAS), Edmonton System Assessment Scale revised (ESAS-r): Renal, Hospital Anxiety and Depression Scale (HADS), and Distress Assessment and Response Tool (DART), and the nonpharmacological interventions that have been investigated are intradialytic exercise, tai chi exercise program, medical music therapy, and brief mindfulness meditation. Of the 28 Web site addresses, 2 contained clinical tools for kidney care providers for the management of depression and anxiety in individuals treated with dialysis. The 26 remaining Web sites contained mental health resources for individuals with kidney disease, which presented different types of resources, including psychoeducation, peer support, and linkage to other services. Conclusion: In conclusion, we found only a limited number of studies investigating mental health assessment tools and interventions in Canada; there was a paucity of randomized controlled trials. Clinical pathways for the assessment and management of mental illness or symptoms in individuals treated with dialysis in Canada are also limited, and no clinical practice guidelines exist. Several mental health resources for people living with dialysis are available, predominantly focused on psychoeducation and peer support. Despite increasing prevalence of mental health concerns by people treated with dialysis, mental health care remains underaddressed. Limitations: It is plausible that additional literature related to mental health assessment tools, interventions, resources, guidelines, and pathways exists that we did not find during our systematic search, especially in gray literature that was limited to one search engine. In addition, health care agencies or organizations may have developed other mental health resources, which may not be available on the Internet.

Justification: Les troubles de santé mentale, la dépression et l'anxiété particulièrement, sont des affections courantes, mais souvent non reconnues et non traitées chez les personnes atteintes d'insuffisance rénale terminale. Les outils, interventions, approches de soins et ressources qui existent pour soutenir la santé mentale des personnes traitées par dialyse au Canada sont eux aussi mal connus. Cette revue de littérature vise à décrire systématiquement la façon dont les soins de santé mentale sont prodigués aux adultes dialysés au Canada. Pour ce faire, nous avons tenté de répondre aux questions de recherche suivantes: (1) Quels outils d'évaluation de la santé mentale et interventions auprès des adultes dialysés ont été étudiés et sont utilisés au Canada? (2) Quelle est la structure des soins de santé mentale dispensés par l'équipe des soins rénaux aux adultes dialysés au Canada, et quel en est le processus? (3) Les ressources en santé mentale mises au point pour les adultes dialysés au Canada sont-elles disponibles? Sources: Les bases de données électroniques Medline, Embase, CINAHL, PsycINFO, Cochrane Library, Scopus et Web of Science ont été consultées à la recherche de littérature évaluée par des pairs. Le moteur de recherche Google a été utilisé pour la littérature grise. Méthodologie: Des recherches systématiques ont été effectuées pour répertorier la littérature grise et la littérature évaluée par les pairs portant sur le soutien ou les soins de santé mentale offerts aux adultes recevant une forme quelconque de dialyse dans les établissements de santé rénale et/ou organismes communautaires canadiens. Le soutien et les soins en santé mentale ont été définis de façon à inclure les outils d'évaluation, interventions, ressources, lignes directrices ou schémas utilisés en contexte de dialyse au Canada. Les titres, résumés et articles complets ont été examinés de façon indépendante par deux auteurs; les désaccords ayant été résolus par d'autres membres de l'équipe. Les sites Web ont été examinés par des membres individuels. Les questions susmentionnées ont servi à l'extraction des données d'intérêt des études et sites inclus. Principaux résultats: Les bases de données électroniques ont permis de répertorier huit articles évalués par des pairs et 28 adresses de sites Web différentes. Des huit articles inclus, quatre avaient examiné des outils d'évaluation et quatre avaient examiné des interventions en santé mentale pour les personnes dialysées au Canada. Les outils d'évaluation de la santé mentale utilisés étaient BDI-II, ESAS, ESAS-r: Renal, HADS et DART. Les interventions non pharmacologiques incluaient des exercices intradialytiques, un programme de tai-chi, la musicothérapie médicale et une courte séance de méditation de pleine conscience. Sur les 28 adresses de site Web retenues, deux contenaient des outils cliniques destinés aux professionnels de la santé rénale pour la prise en charge de la dépression et de l'anxiété chez les personnes dialysées. Les 26 autres contenaient des ressources en santé mentale destinées aux personnes atteintes d'une néphropathie, soit différents types de ressources incluant notamment de la psychoéducation, du soutien par les pairs et des liens vers d'autres services.Pour résumer, nous n'avons trouvé qu'un nombre limité d'études portant sur les outils d'évaluation et les interventions pour la santé mentale au Canada, dont aucun essai randomisé contrôlé. Les cheminements cliniques pour évaluer et prendre en charge les troubles de santé mentale et leurs symptômes chez les personnes traitées par dialyse au Canada sont également limités, et il n'existe aucune ligne directrice en matière de pratique clinique. Plusieurs ressources en santé mentale sont disponibles pour cette population, elles sont principalement axées sur la psychoéducation et le soutien par les pairs. Malgré la prévalence croissante des troubles de santé mentale chez les personnes dialysées, les soins demeurent rarement abordés. Limites: Il est possible que d'autres documents existent sur les outils d'évaluation de la santé mentale, les interventions, les ressources, les lignes directrices ou les voies d'accès, et que nous les ayons manqués lors de la recherche systématique, particulièrement dans la littérature grise puisque celle-ci était limitée à un seul moteur de recherche. Il est également possible que des agences ou organismes de soins de santé aient mis au point d'autres ressources en santé mentale qui ne sont pas disponibles sur Internet.

Youth-oriented outcomes of education, employment and training interventions for upcoming youth: Protocol for a discrete choice experiment.

AIM: The issue of youth who are not engaged in education, employment or training has been a focus of policymakers for decades. Although interventions exist for these youth, they often measure success in ways that fail to capture what youth seek to gain. The project aims to address this gap by assessing youth-oriented outcomes for interventions targeting upcoming youth. Acknowledging the stigma attached to the deficit-based notion of not engaged in education, employment or training, hereafter we refer to 'upcoming youth', a term coined by youth partners on the project. This study asks what youth want to achieve by participating in an intervention for upcoming youth, with a view to guiding service and research design. METHODS: A mixed-methods discrete choice experiment will be conducted with youth engaged as partners. A qualitative (focus group) stage will be conducted to design discrete-choice experiment attributes and levels. The experiment will be piloted and administered online to approx. 500 youth (aged 14-29) across Canada to identify the outcomes that youth prioritize for interventions. Latent class analyses will then be conducted to explore clusters of outcomes that different groups of youth prioritize. CONCLUSIONS: From a strengths-based recovery-oriented framework, hearing the voices of the target population is important in designing and evaluating services. This youth-oriented research project will identify the intervention outcomes that are the highest priority for upcoming youth. Findings will inform the development, implementation and testing of interventions targeting relevant outcomes for youth who are not engaged in education, employment or training.

Outcomes in pediatric studies of medium-chain acyl-coA dehydrogenase (MCAD) deficiency and phenylketonuria (PKU): a review.

BACKGROUND: Inherited metabolic diseases (IMDs) are a group of individually rare single-gene diseases. For many IMDs, there is a paucity of high-quality evidence that evaluates the effectiveness of clinical interventions. Clinical effectiveness trials of IMD interventions could be supported through the development of core outcome sets (COSs), a recommended minimum set of standardized, high-quality outcomes and associated outcome measurement instruments to be incorporated by all trials in an area of study. We began the process of establishing pediatric COSs for two IMDs, medium-chain acyl-CoA dehydrogenase (MCAD) deficiency and phenylketonuria (PKU), by reviewing published literature to describe outcomes reported by authors, identify heterogeneity in outcomes across studies, and assemble a candidate list of outcomes. METHODS: We used a comprehensive search strategy to identify primary studies and guidelines relevant to children with MCAD deficiency and PKU, extracting study characteristics and outcome information from eligible studies including outcome measurement instruments for select outcomes. Informed by an established framework and a previously published pediatric COS, outcomes were grouped into five, mutually-exclusive, a priori core areas: growth and development, life impact, pathophysiological manifestations, resource use, and death. RESULTS: For MCAD deficiency, we identified 83 outcomes from 52 articles. The most frequently represented core area was pathophysiological manifestations, with 33 outcomes reported in 29/52 articles (56%). Death was the most frequently reported outcome. One-third of outcomes were reported by a single study. The most diversely measured outcome was cognition and intelligence/IQ for which eight unique measurement instruments were reported among 14 articles. For PKU, we identified 97 outcomes from 343 articles. The most frequently represented core area was pathophysiological manifestations with 31 outcomes reported in 281/343 articles (82%). Phenylalanine concentration was the most frequently reported outcome. Sixteen percent of outcomes were reported by a single study. Similar to MCAD deficiency, the most diversely measured PKU outcome was cognition and intelligence/IQ with 39 different instruments reported among 82 articles. CONCLUSIONS: Heterogeneity of reported outcomes and outcome measurement instruments across published studies for both MCAD deficiency and PKU highlights the need for COSs for these diseases, to promote the use of meaningful outcomes and facilitate comparisons across studies.

Using a meta-narrative literature review and focus groups with key stakeholders to identify perceived challenges and solutions for generating robust evidence on the effectiveness of treatments for rare diseases.

INTRODUCTION: For many rare diseases, strong analytic study designs for evaluating the efficacy and effectiveness of interventions are challenging to implement because of small, geographically dispersed patient populations and underlying clinical heterogeneity. The objective of this study was to integrate perspectives from published literature and key rare disease stakeholders to better understand the perceived challenges and proposed methodological approaches to research on clinical interventions for rare diseases. METHODS: We used a meta-narrative literature review and focus group interviews with key rare disease stakeholders to better understand the perceived challenges in generating and synthesizing treatment effectiveness evidence, and to describe various research methods for mitigating these identified challenges. Data from both components of this study were synthesized narratively according to research paradigms that emerged from our data. RESULTS: Results from our meta-narrative literature review and focus group interviews revealed three fundamental challenges in generating robust treatment effectiveness evidence for rare diseases: i) limitations in recruiting a sufficient sample size to achieve planned statistical power; ii) inability to account for clinical heterogeneity and assess treatment effects across a clinical spectrum; and iii) reliance on short-term, surrogate outcomes whose clinical relevance is often unclear. We mapped these challenges and associated solutions to three interrelated research paradigms: i) explanatory evidence generation; ii) comparative effectiveness/pragmatic evidence generation; and iii) patient-oriented evidence generation. Within each research paradigm, numerous criticisms and potential solutions have been described with respect to overcoming these challenges from a research study design perspective. CONCLUSIONS: Over time, discussions about clinical research for interventions for rare diseases have moved beyond methodological approaches to overcome challenges related to explanatory evidence generation, with increased recognition of the importance of pragmatic and patient-oriented evidence. Future directions for our work include developing a framework to expand current evidence synthesis practices to take into consideration many of the concepts discussed in this paper.