Participant Perceptions in a Long-term Clinical Trial of Autosomal Dominant Polycystic Kidney Disease.

Rationale & Objective: The development of new therapies for autosomal dominant polycystic kidney disease requires clinical trials to be conducted efficiently. In this study, the factors affecting the recruitment and retention of participants enrolled in a 3-year randomized controlled trial in autosomal dominant polycystic kidney disease were investigated. Study Design: Qualitative study. Setting & Participants: All participants (N=187) were invited to complete a 16-item questionnaire at the final study visit of the primary trial. Participants were recruited to complete a semistructured interview using purposeful sampling according to age, self-reported gender, and randomization group. Analytical Approach: Descriptive statistics were used for demographic data and questionnaires. The interview transcripts underwent inductive thematic coding. Results: One hundred and forty-six of the 187 randomized participants (79%) completed the post-trial questionnaire, and 31 of the 187 participants (21%) completed the interview. Most participants (94%) rated their global satisfaction with the trial as high (a score of 8 or more out of 10). Altruism, knowledge gain, and access to new treatments were the main motivators for recruitment. The main reasons for considering leaving the study were concerns about the risk of intervention and family or work issues. Strategies that favored retention included flexibility in attending different study sites, schedule flexibility, staff interactions, and practical support with parking and reminders. The main burden was time away from work with lost wages, and burden associated with magnetic resonance imaging scans and 24-hour urine output collections. Limitations: The study population was restricted to participants in a single nondrug clinical trial, and the results could be influenced by selection and possible social desirability bias. Conclusions: Participants reported high levels of satisfaction that occurred as a function of the trial meeting participants' expectations. Furthermore, retention was a balance between the perceived benefits and burden of participation. Consideration of these perspectives in the design of future clinical trials will improve their efficiency and conduct. Plain-Language Summary: Advances in the clinical practice of autosomal dominant polycystic kidney disease (ADPKD) require affected individuals to voluntarily participate in long-term multicenter randomized controlled trials (RCTs). In this qualitative post hoc study of a 3-year RCT of increased water intake in ADPKD, altruism, knowledge gain, and access to a nondrug treatment positively influenced the decision to volunteer. Ongoing participation was enabled by building flexibility into the study protocol and staff prioritizing a participant's needs during study visits. Although participants completed the required tests, most were considered burdensome. This study highlights the importance of incorporating protocol flexibility into trial design; the preference for interventions with a low risk of adverse effects; and the urgent requirement for robust surrogate noninvasive biomarkers to enable shorter RCTs in ADPKD.

Helping Patients Understand Pancreatic Cancer Using Animated Pancreas Patient Education With Visual Formats of Learning.

OBJECTIVES: Patient education and resources that address barriers to health literacy to improve understanding in pancreatic cancer are limited. We evaluated the impact and outcomes benefits of Animated Pancreas Patient (APP) cancer educational modules (APP website and YouTube). METHODS: A retrospective study of APP metrics and utilization data from September 2013 to February 2021 was conducted. We evaluated audience reach and calculated top views by media type (animation/expert video/patient video/slideshow) and top retention videos from the modules. RESULTS: During the study period, APP had 4,551,079 views worldwide of which 2,757,064 unique visitors or 60% were from the United States. Of these, 54% were patients, 17% were family members or caregivers, 16% were health care providers, and 13% were other. The most popular topic viewed among the animations was "Understanding Clinical Trials" (n = 182,217), and the most common expert video viewed was "What are the different stages of pancreatic cancer?" (n = 15,357). CONCLUSIONS: Pancreatic cancer patient education using APP's visual formats of learning demonstrated a wide reach and had a significant impact on improved understanding among patients, families, and caregivers. Continued efforts should be made to provide patient resources that address health literacy, better quality of life and improved health outcomes in pancreatic cancer.

Retrospective study on the epidemiology of antineutrophil cytoplasmic autoantibodies-associated vasculitis in two Australian health districts.

BACKGROUND: Antineutrophil cytoplasmic autoantibodies (ANCA)-associated vasculitis (AAV) is more prevalent in rural Australia compared with metropolitan areas, suggesting a role of environment in disease pathogenesis. However, the prevalence of environmental risk factors in Australian AAV patients has not been described. AIMS: To compare the incidence of AAV between two health districts (Illawarra Shoalhaven Local Health District (ISLHD), a mixed rural/metropolitan region, and South Eastern Sydney Local Health District (SESLHD), a metropolitan region) in Australia and its relationship to environmental exposures. METHODS: Cases of AAV from 2002 to 2017 were retrospectively identified from ISLHD and SESLHD using electronic medical records. Eligible participants were invited to complete a standardised questionnaire examining their exposure to silica, solvents, metal, dust, farming, gardening and sunlight. RESULTS: One hundred and fifty-six cases of AAV were identified from 2002 to 2017. A higher cumulative incidence of AAV was observed in the ISLHD (184.2 (95% confidence interval (CI) 143.6-232.7) per million) compared with SESLHD (102.6 (95% CI 82.1-126.8) per million). Over 50% of the cohort had high levels of silica and solvents exposure, based on self-reported questionnaires. There was no significant relationship between region and exposure to silica (P = 0.96), solvents (P = 0.44), metal (P = 0.33), dust (P = 0.25), farming (P = 0.90), gardening (P = 0.93) or sunlight (P = 0.55). CONCLUSIONS: We found a higher incidence of AAV in ISLHD compared with SESLHD with high levels of exposure to silica and solvents in both regions based on self-reported questionnaires. Prospective systematic collection of data, such as a registry of AAV, is warranted to further explore the relationship between environmental exposures and AAV.

Prescribed Water Intake in Autosomal Dominant Polycystic Kidney Disease.

Prescribed Water Intake in Autosomal Dominant Polycystic Kidney Disease The effect of increased water intake on kidney cyst growth in patients with polycystic kidney disease was compared for two groups randomly assigned to either prescribed or ad libitum water intake. Over 3 years, there was no difference in height-corrected total kidney volume between the groups.

Artificial Intelligence and Early Detection of Pancreatic Cancer: 2020 Summative Review.

ABSTRACT: Despite considerable research efforts, pancreatic cancer is associated with a dire prognosis and a 5-year survival rate of only 10%. Early symptoms of the disease are mostly nonspecific. The premise of improved survival through early detection is that more individuals will benefit from potentially curative treatment. Artificial intelligence (AI) methodology has emerged as a successful tool for risk stratification and identification in general health care. In response to the maturity of AI, Kenner Family Research Fund conducted the 2020 AI and Early Detection of Pancreatic Cancer Virtual Summit (www.pdac-virtualsummit.org) in conjunction with the American Pancreatic Association, with a focus on the potential of AI to advance early detection efforts in this disease. This comprehensive presummit article was prepared based on information provided by each of the interdisciplinary participants on one of the 5 following topics: Progress, Problems, and Prospects for Early Detection; AI and Machine Learning; AI and Pancreatic Cancer-Current Efforts; Collaborative Opportunities; and Moving Forward-Reflections from Government, Industry, and Advocacy. The outcome from the robust Summit conversations, to be presented in a future white paper, indicate that significant progress must be the result of strategic collaboration among investigators and institutions from multidisciplinary backgrounds, supported by committed funders.

Assessment of Dietary Sodium Intake Using the Scored Salt Questionnaire in Autosomal Dominant Polycystic Kidney Disease.

The excess intake of dietary sodium is a key modifiable factor for reducing disease progression in autosomal dominant polycystic kidney disease (ADPKD). The aim of this study was to test the hypothesis that the scored salt questionnaire (SSQ; a frequency questionnaire of nine sodium-rich food types) is a valid instrument to identify high dietary salt intake in ADPKD. The performance of the SSQ was evaluated in adults with ADPKD with an estimated glomerular filtration rate (eGFR) ≥ 30 mL/min/1.73 m2 during the screening visit of the PREVENT-ADPKD trial. High dietary sodium intake (HSI) was defined by a mean 24-h urinary sodium excretion ≥ 100 mmol/day from two collections. The median 24-h urine sodium excretion was 132 mmol/day (IQR: 112-172 mmol/d) (n = 75; mean age: 44.6 ± 11.5 years old; 53% female), and HSI (86.7% of total) was associated with male gender and higher BMI and systolic blood pressure (p < 0.05). The SSQ score (73 ± 23; mean ± SD) was weakly correlated with log10 24-h urine sodium excretion (r = 0.29, p = 0.01). Receiving operating characteristic analysis showed that the optimal cut-off point in predicting HSI was an SSQ score of 74 (area under the curve 0.79; sensitivity 61.5%; specificity 90.0%; p < 0.01). The evaluation of the SSQ in participants with a BMI ≥ 25 (n = 46) improved the sensitivity (100%) and the specificity (100%). Consumers with an SSQ score ≥ 74 (n = 41) had higher relative percentage intake of processed meats/seafood and flavourings added to cooking (p < 0.05). In conclusion, the SSQ is a valid tool for identifying high dietary salt intake in ADPKD but its value proposition (over 24-h urinary sodium measurement) is that it may provide consumers and their healthcare providers with insight into the potential origin of sodium-rich food sources.

Polyunsaturated fatty acid food frequency questionnaire validation in people with end stage renal disease on dialysis.

AIM: To validate the polyunsaturated food frequency questionnaire (PUFA FFQ) and test for reproducibility in people with end stage renal disease on dialysis treatment. METHODS: Participants (n = 32) completed the PUFA FFQ and three 24-hour recalls. Erythrocyte samples (n = 29) were used for erythrocyte fatty acid analysis. The triangular relationship between the PUFA FFQ, 24-hour recalls and the biomarker was assessed using the method of triads. Agreement between the two dietary methods was also assessed using Bland-Altman plots and classification by quintiles. Reproducibility was tested on a subset of the group (n = 8). RESULTS: The PUFA FFQ was a valid measure of all PUFA except for docosapentaenoic acid (DPA) and arachidonic acid (AA). Strong validity coefficients were found for n-3 long-chain PUFA (LCPUFA), eicosapentaenoic acid (EPA) and docosahexaenoic acid (DHA) of 0.914 (95% CI: 0.665, 0.997) and 0.889 (95% CI: 0.706, 0.994), respectively. In the Bland-Altman plots 91-100% of observations fell between the limits of agreement for all PUFA. There were significant correlations between the initial FFQ and the repeat FFQ for all PUFA except DPA and AA. CONCLUSIONS: The PUFA FFQ is a valid tool for assessing PUFA intake in people with end stage renal disease.

Understanding Pancreatic Diseases Using Animated Pancreas Patient: Informing Patients for Better Health Outcomes With Visual Formats of Learning.

OBJECTIVES: The aim of this study was to evaluate the impact of Animated Pancreas Patient (APP) educational modules (APP website and YouTube) on pancreas education, awareness, and health outcomes. METHODS: This was a retrospective study of APP metrics data from September 2013 to October 2017. We evaluated audience reach (number of visit sessions, unique visitors, page views) and calculated top views by media type (animation, expert video, patient video, and slide show) and top retention videos from the modules. We also assessed the educational impact through learner feedback survey. RESULTS: The APP had 1,475,252 views (547,693 unique visitors, 63.1% in United States) during the study period. Most popular topic viewed among the animations was "Role and Anatomy of the Pancreas" (n = 361,116), and most common expert video viewed was "Chronic Pancreatitis: What Foods and Beverages Should I Avoid?" (n = 31,667). Participants who completed the online feedback survey reported knowledge gains and commitments to change. CONCLUSIONS: Pancreas education in visual formats of learning provided by APP demonstrated wide reach and has substantial potential to inform and impact behaviors of patients and caregivers. Continued efforts should be made to provide patient resources that address health literacy and patient education and respond to patient needs for better quality of life and improved health outcomes in pancreatic diseases.

Diet quality in patients with end-stage kidney disease undergoing dialysis.

BACKGROUND: People on haemodialysis (HD) are at risk of consuming a poor quality diet. This includes inadequate intake of omega-3 long chain polyunsaturated fatty acids (n-3 LCPUFA). OBJECTIVE: This study aims to investigate diet quality, with a particular focus on n-3 LCPUFA intake, in a population of incentre HD patients. DESIGN: Dietary intake was measured using three 24 hour recalls; the Polyunsaturated food frequency questionnaire (PUFA FFQ) and the Total Diet Score (TDS). Dietary intake was also compared to evidence based practice guidelines (EBPG). Nutritional status was assessed using the Patient Generated Subjective Global Assessment (PG SGA). SUBJECTS: A total of 32 dialysis patients were recruited, from two regional HD centres in New South Wales, Australia. MAIN OUTCOME MEASURE: Diet quality was the main outcome measure. RESULTS: Diet quality of study participants was poor, with the majority not meeting the EBPG for energy, protein and potassium. All participants exceeded the recommended amount of saturated fat. The mean TDS of the dialysis cohort was 10.2, which was significantly higher than the TDS of 9.3 of a healthy disease free cohort (p < 0.05). Positive correlations were found between TDS and LC omega-3 intake (r = 0.392) and TDS and total omega-6 intake (r = 0.363). Only 22% of participants met the suggested dietary target for n-3 LCPUFA intake. CONCLUSION: Dialysis patients in this study had suboptimal diet quality. Improvements are required for better adherence to the EBPG. Increased consumption of n-3 LCPUFA fatty acids may also be of benefit.

Research considerations in the evaluation of minimally invasive pancreatic resection (MIPR).

The IHPBA/AHPBA-sponsored 2016 minimally invasive pancreatic resection (MIPR) conference held on April 20th, 2016 included a session designed to evaluate what would be the most appropriate scientific contribution to help define the increasing role of MIPR internationally. Participants in the conference reviewed the assessment of numerous pertinent scientific designs including randomized controlled trial (RCT), pragmatic international RCT, registry-RCT, non-RCT with propensity matching, and various types of clinical registries including those aiming to create a quality improvement data system or a learning health care system. The strengths and weaknesses of each of these designs, the status of trials which are currently recruiting patients, and pragmatic considerations were evaluated. A recommendation was made to establish a clinical registry to collect data prospectively from around the world to assess current practices and provide a framework for future studies in MIPR.

The effectiveness of foot reflexology in inducing ovulation: a sham-controlled randomized trial.

OBJECTIVE: To determine whether foot reflexology, a complementary therapy, has an effect greater than sham reflexology on induction of ovulation. DESIGN: Sham-controlled randomized trial with patients and statistician blinded. SETTING: Infertility clinic in Plymouth, United Kingdom. PATIENT(S): Forty-eight women attending the clinic with anovulation. INTERVENTION(S): Women were randomized to receive eight sessions of either genuine foot reflexology or sham reflexology with gentle massage over 10 weeks. MAIN OUTCOME MEASURE(S): The primary outcome was ovulation detected by serum progesterone level of >30 nmol/L during the study period. RESULT(S): Twenty-six patients were randomized to genuine reflexology and 22 to sham (one randomized patient was withdrawn). Patients remained blinded throughout the trial. The rate of ovulation during true reflexology was 11 out of 26 (42%), and during sham reflexology it was 10 out of 22 (46%). Pregnancy rates were 4 out of 26 in the true group and 2 out of 22 in the control group. Because of recruitment difficulties, the required sample size of 104 women was not achieved. CONCLUSION(S): Patient blinding of reflexology studies is feasible. Although this study was too small to reach a definitive conclusion on the specific effect of foot reflexology, the results suggest that any effect on ovulation would not be clinically relevant. Sham reflexology may have a beneficial general effect, which this study was not designed to detect.

Physical therapy evaluation of patients with chronic pelvic pain: a controlled study.

OBJECTIVE: The purpose of this study was to determine the relative frequency of positive musculoskeletal exam findings between patients with chronic pelvic pain (CPP) and healthy control subjects. STUDY DESIGN: We conducted a masked, prospective, cross-sectional study of abnormal pelvic, abdominal, and back examination findings in 19 women with CPP vs 20 healthy control subjects. RESULTS: Women with CPP had more frequent abnormal musculoskeletal findings than did control subjects asymmetric iliac crests (61% vs 25%), pubic symphysis heights (50% vs 10%), and positive posterior pelvic provocation testing (37% vs 5%; all P < .05). Patients with pain exhibited more tenderness at several abdominal muscle sites, had higher median total pelvic floor tenderness scores (3/24 vs 0/24; P < .05), and less control of the pelvic floor (unable to maintain 10 seconds of relaxation, 78% vs 20%; P < .001). CONCLUSION: The higher frequency of positive pelvic musculoskeletal findings in CPP suggests that an investigation of somatic pain generators is warranted in these patients.

Measuring protein excretion in pregnancy.

The recognition and detection of proteinuria has been acknowledged as an important clinical marker of renal disease since 1827 when Richard Bright published his landmark medical case reports. In more recent times, the broader community of clinicians has come to share the enthusiasm of nephrologists in recognizing the importance of protein excretion, not only as a marker of current renal disease but also as a predictor of long-term renal and cardiovascular morbidity and mortality. It is important that methods for detecting and measuring proteinuria are accurate, and this is particularly relevant to diseases that are defined by the detection of proteinuria, such as pre-eclampsia. This review will first discuss current knowledge of protein handling by the normal kidney, then the changes in normal and hypertensive pregnancy, and finally, how recent advances in our understanding of proteinuria may affect our future management of hypertensive pregnancies.

Predicting transformation from gestational hypertension to preeclampsia in clinical practice: a possible role for 24 hour ambulatory blood pressure monitoring.

OBJECTIVE: To identify parameters that may assist clinicians in predicting which women will develop preeclampsia (PE) after initially presenting with gestational hypertension (GH). METHODS: 118 women were recruited to the study with GH or PE. They were divided into three groups based on their diagnosis at delivery- (1) GH, (2) PE from the time of presentation, (3) those with an initial diagnosis of GH who progressed to PE. Women underwent 24 hour ambulatory blood pressure monitoring (ABPM) and had serum estrogen, progesterone, beta-HCG, leptin and adiponectin measured as possible predictors of transformation of GH to PE. RESULTS: Women who presented with GH, and progressed to PE, presented four weeks earlier (33 vs 37 weeks, p < 0.001) than those who did not progress. Women with PE, either as their initial diagnosis or after progression from GH, were delivered earlier (p < 0.001) and had more small for gestational age (SGA) babies than women with GH at delivery (p < 0.05). Those who developed PE after presenting with GH generally had higher blood pressures than those who remained as GH, significant for awake and 24 hour systolic blood pressures (p < 0.05). beta-HCG, estrogen, progesterone or leptin values were similar across the groups. Adiponectin was higher in women with established PE at presentation compared to women with GH (p = 0.02) but adiponectin failed to discriminate those women with an initial diagnosis of GH who progressed to PE. CONCLUSION: 24 hr ABPM may provide a non-invasive method of identifying this 'at risk' GH population, particularly in the case of early presentation.

Microscopic hematuria in pregnancy: relevance to pregnancy outcome.

BACKGROUND: The significance of dipstick or microscopic hematuria in pregnancy is uncertain, with some studies suggesting this is associated with a greater risk for preeclampsia. We sought to determine the prevalence and clinical significance of microscopic hematuria during pregnancy. METHODS: This was a prospective case-control study in the antenatal Clinic of St George Hospital, Kogarah, Australia, a teaching hospital without tertiary referral antenatal care, with approximately 2,600 deliveries per year. One thousand pregnant women attending for routine antenatal care were invited to have a routine urinalysis performed and be referred to a nephrology clinic for further investigation if dipstick microscopic hematuria was detected on more than 1 occasion before 32 weeks' gestation. Main outcome measures were the prevalence of dipstick hematuria, prevalence of hematuria confirmed by urine microscopy, and the development of preeclampsia or gestational hypertension or delivery of a small-for-gestational-age baby. RESULTS: One hundred seventy-eight of 902 women (20%) who entered the study had dipstick hematuria on at least 2 occasions in pregnancy; 66 of 126 women (53%) who had hematuria before 32 weeks attended the nephrology clinic, where microscopic hematuria was confirmed in 40 women (61%). Renal imaging results were normal in all except 1 woman, and all women had a serum creatinine level of 0.90 mg/dL or less (< or =80 micromol/L). The development of preeclampsia or gestational hypertension or delivery of a small-for-gestational-age baby were similar in women with and without dipstick hematuria. Microscopic hematuria persisted in half (15 women) of those who attended for follow-up after 3 months postpartum. CONCLUSION: Dipstick hematuria is very common during pregnancy, but rarely signifies a disorder likely to impact on the pregnancy outcome. Postpartum follow-up is recommended to detect women who have persistent hematuria and presumed underlying mild glomerulonephritis.