Analysis of baseline parameters in the HALT polycystic kidney disease trials

HALT PKD consists of two ongoing randomized trials with the largest cohort of systematically studied patients with autosomal dominant polycystic kidney disease to date. Study A will compare combined treatment with an angiotensin-converting inhibitor and receptor blocker to inhibitor alone and standard compared with low blood pressure targets in 558 early-stage disease patients with an eGFR over 60ml/min per 1.73m2. Study B will compare inhibitor-blocker treatment to the inhibitor alone in 486 late-stage patients with eGFR 25–60ml/min per 1.73m2. We used correlation and multiple regression cross-sectional analyses to determine associations of baseline parameters with total kidney, liver, or liver cyst volumes measured by MRI in Study A and eGFR in both studies. Lower eGFR and higher natural log-transformed urine albumin excretion were independently associated with a larger natural log–transformed total kidney volume adjusted for height (ln(HtTKV)). Higher body surface area was independently associated with a higher ln(HtTKV) and lower eGFR. Men had larger height-adjusted total kidney volume and smaller liver cyst volumes than women. A weak correlation was found between the ln(HtTKV) and natural log–transformed total liver volume adjusted for height or natural log liver cyst volume in women only. Women had higher urine aldosterone excretion and lower plasma potassium. Thus, our analysis (1) confirms a strong association between renal volume and functional parameters, (2) shows that gender and other factors differentially affect the development of polycystic disease in the kidney and liver, and (3) suggests an association between anthropomorphic measures reflecting prenatal and/or postnatal growth and disease severity

Macrocytosis may be associated with mortality in chronic hemodialysis patients: a prospective study

<p>Abstract</p> <p>Background</p> <p>Macrocytosis occurs in chronic hemodialysis (CHD) patients; however, its significance is unknown. The purpose of this study was to establish the prevalence and distribution of macrocytosis, to identify its clinical associations and to determine if macrocytosis is associated with mortality in stable, chronic hemodialysis patients.</p> <p>Methods</p> <p>We conducted a single-centre prospective cohort study of 150 stable, adult CHD patients followed for nine months. Macrocytosis was defined as a mean corpuscular volume (MCV) > 97 fl. We analyzed MCV as a continuous variable, in tertiles and using a cutoff point of 102 fl.</p> <p>Results</p> <p>The mean MCV was 99.1 ± 6.4 fl, (range 66-120 fl). MCV was normally distributed. 92 (61%) of patients had an MCV > 97 fl and 45 (30%) > 102 fl. Patients were not B12 or folate deficient in those with available data and three patients with an MCV > 102 fl had hypothyroidism. In a logistic regression analysis, an MCV > 102 fl was associated with a higher Charlson-Age Comorbidity Index (CACI) and higher ratios of darbepoetin alfa to hemoglobin (Hb), [(weekly darbepoetin alfa dose in micrograms per kg body weight / Hb in g/L)*1000]. There were 23 deaths at nine months in this study. Unadjusted MCV > 102 fl was associated with mortality (HR 3.24, 95% CI 1.42-7.39, P = 0.005). Adjusting for the CACI, an MCV > 102 fl was still associated with mortality (HR 2.47, 95% CI 1.07-5.71, P = 0.035).</p> <p>Conclusions</p> <p>Macrocytosis may be associated with mortality in stable, chronic hemodialysis patients. Future studies will need to be conducted to confirm this finding.</p

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

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

Core outcome domains for trials in autosomal dominant polycystic kidney disease: An international Delphi survey

Rationale & Objective Outcomes reported in trials involving patients with autosomal dominant polycystic kidney disease (ADPKD) are heterogeneous and rarely include patient-reported outcomes. We aimed to identify critically important consensus-based core outcome domains to be reported in trials in ADPKD. Study Design An international 2-round online Delphi survey was conducted in English, French, and Korean languages. Setting & Participants Patients/caregivers and health professionals completed a 9-point Likert scale (7-9 indicating critical importance) and a Best-Worst Scale. Analytical Approach The absolute and relative importance of outcomes were assessed. Comments were analyzed thematically. Results 1,014 participants (603 [60%] patients/caregivers, 411 [40%] health professionals) from 56 countries completed round 1, and 713 (70%) completed round 2. The prioritized outcomes were kidney function (importance score, 8.6), end-stage kidney disease (8.6), death (7.9), blood pressure (7.9), kidney cyst size/growth (7.8), and cerebral aneurysm (7.7). Kidney cyst–related pain was the highest rated patient-reported outcome by both stakeholder groups. Seven themes explained the prioritization of outcomes: protecting life and health, directly encountering life-threatening and debilitating consequences, specificity to ADPKD, optimizing and extending quality of life, hidden suffering, destroying self-confidence, and lost opportunities. Limitations Study design precluded involvement from those without access to internet or limited computer literacy. Conclusions Kidney function, end-stage kidney disease, and death were the most important outcomes to patients, caregivers, and health professionals. Kidney cyst–related pain was the highest rated patient-reported outcome. Consistent reporting of these top prioritized outcomes may strengthen the value of trials in ADPKD for decision making

Auxiliar: A Case-based System To Assist Online Courses

This paper presents the REASONING, the third module of a computerized system (AUXILIAR), designed to help human-tutors redefine information for those students who have difficulties in grasping deployed educational topics. To this end, there is an intelligent mechanism that facilitates the assessment process and searches for similar cases produced by other students in past situations. This search enables the system to redefine the information content to be rendered to the student in order to eliminate deficiencies detected during a formative assessment without considerable intervention by the tutor. To reach these goals, concepts of Artificial Intelligence (Case-based Reasoning, more specifically) and man-machine interaction are exploited. Thereby, there are presented the format of a Case, the structure of the Case Library, as well as the intelligent retrieval mechanism that was developed.1197201Anderson, J.R., Corbett, A.T., Koedinger, K., Pelletier, R., Cognitive tutors: Lessons learned (1995) The Journal of the Learning Sciences, 4 (2), pp. 167-207Bauer, M., A dempster-shafer approach to modeling agent references for plan recognition (1996) User Modelling and User-adapted Interaction, 5, pp. 317-348Daugherty, M., Funke, B.L., University faculty and student perceptions of web-based instruction (1998) Journal of Distance Education, 13 (1), pp. 21-39Grace, L.J., Smith, P.J., Flexible delivery in the Australian vocational education and training sector: Barriers to success identified in case studies of four adult learners (2001) Distance Education, 22 (2), pp. 196-211Hare, C., McCartan, A., Maximising resources in search of quality: Identifying factors to enable thee integrative uso of IT in teaching and learning (1996) Innovations in Education and Training International, 33 (4), pp. 178-184Hawkes, L.W., Deny, S.J., Rundensteiner, E.A., Individualized tutoring using an intelligent fuzzy temporal relational database (1990) International Journal of Man-machine Studies, 33, pp. 409-429James, R., Beattie, K., Postgraduate coursework beyond the classroom: Issues in implementing flexible delivery (1996) Distance Education, 17 (2), pp. 355-368Jameson, A., Numerical uncertainty management in user and student modeling: An overview of systems and issues (1996) User Modelling and User Adapter Interaction, 5, pp. 193-251Kolodner, J., (1993) Case-based Reasoning, p. 612. , Morgan Kaufman PublishersMetcalf, T., Distance education: The issue of faculty time (1997) 5th Annual Distance Education Conference: 1997 Conference Proceedings, , Texas A&M, Center for Distance Education ResearchPajo, K., Wallace, C., Barriers to the uptake of web-based technology by university teachers (1999) Journal of Distance Education, 16 (1), pp. 70-84Petrushin, V.A., Sinista, K.M., Using probabilistic reasoning techniques for leaner modeling (1993) World Conference on AI in Education, pp. 418-425. , Edinburgh, ScotlandPiva, D., Freitas, R.L., AUXILIAR: Uma aplicação de inteligência artificial que possibilita a potencialização da aprendizagem (2002) Workshop de Informática na Educação, Congresso da Sociedade Brasileira de Computação, pp. 217-221. , FlorianopólisPiva Jr., D., Miskulin, M.S., Gunawardena, C.N., Gonçalves Jr., G., Miskulin, R.S., An artificial intelligence-based application for facilitating interaction and learning assessment in on-line engineering courses (2002) ASEE 2002 - Annual Conference and Exposition, pp. 16-19. , Section 1496, Montréal, Quebec Canadá, JuneThompson, D.J., Holt, D.M., Tertiary pedagogy encounters the technological imperative (1996) Distance Education, 17 (2), pp. 335-354Villano, M., Probabilistic students models: Bayesian belief networks and knowledge space theory (1992) Second International Conference on Intelligent Tutoring System, pp. 491-498. , Montreal, CanadaWatson, I., (1997) Applying Case-based Reasoning: Techniques for Enterprise Systems, , San Francisco: Morgan KaufmannYong, Y., Wang, S., Faculty perceptions on a new approach to distance learning: Teletchnet (1996) Journal of Instructional Delivery Systems, 10 (2), pp. 3-

An Cbr System To Aid The Assessment In Engineering Online Courses

This work shows a computational system, called AUXILIAR, which is used in the development and implementation of online courses, and explores its potential to conduct the student's formative assessment in the teaching-learning process. It shows how a computational tool can help to reduce the school space-time limitations that usually limit the attainment of educational objectives. The time spent to prepare the educational modules, the possibilities of content adaptation to individual demands, and the adoption of a formative assessment as basis to conduct the learning process could be improved with the system's use.10296299Allai, L., Cardinet, J., Perrenoud, P., (1986) Avaliação Formativa Num Ensino Diferenciado, , Coimbra: Almedina (in Portuguese)Bertagna, R.H., (2003) Progressão Continuada: Limites e Possibilidades, , Tese de Doutorado. Campinas, Faculdade de Educação da Unicamp (in Portuguese)Bloom, B.S., Hastings, J.T., Madaus, G.F., (1971) Handbook on Formative and Summative Evaluation on Student Learning, , New York: McGraw HillBourdieu, P., Passeron, J.C., (1975) A Reprodução, , Rio de Janeiro: Francisco Alves (in Portuguese)Freitas, L.C., (1995) Critica Da Organização do Trabalho Pedagógico e Da Didática, , 6 a. ed., Campinas, Papirus (in Portuguese)Freitas, L.C., Educação hoje: questões em debate. Que escola desejamos? (2001) Estudos Avançados, 15 (42), pp. 48-53. , (in Portuguese)Freitas, L.C., (2003) Ciclos, Seriação e Avaliação: Confronte de Lógicas, , 1 a. ed., São Paulo: Moderna (in Portuguese)Piva Jr., D., Freitas, R.L., AUXILIAR: Uma aplicação de inteligência artificial que possibilita a potencialização da aprendizagem (2002) Workshop de Informática na Educação, Congresso Da Sociedade Brasileira de Computação, Florianópolis, pp. 217-221. , julho de 2002 (in Portuguese