Use of Web-Based Surveys to Collect Long-Term Pediatric Outcomes in Patients With Twin-Twin Transfusion Syndrome Treated With Fetoscopic Laser Photocoagulation: Observational Study.

Background: In the United States, patients with monochorionic diamniotic twins who undergo in utero fetoscopic laser photocoagulation (FLP) for twin-twin transfusion syndrome (TTTS) may travel great distances for care. After delivery, many parents cannot return to study sites for formal pediatric evaluation due to geographic location and cost. Objective: The aim of this study was to collect long-term pediatric outcomes in patients who underwent FLP for TTTS. Methods: We assessed the feasibility of using a web-based survey designed in REDCap (Research Electronic Data Capture; Vanderbilt University) to collect parent-reported outcomes in children treated for TTTS at a single center during 2011-2019. Patients with ≥1 neonatal survivor were invited via email to complete 5 possible questionnaires: the child status questionnaire (CSQ); fetal center questionnaire (FCQ); Ages & Stages Questionnaires, Third Edition (ASQ-3); Modified Checklist for Autism in Toddlers, Revised With Follow-Up (M-CHAT-R/F); and thank you questionnaire (TYQ). The R programming language (R Foundation for Statistical Computing) was used to automate survey distribution, scoring, and creation of customized reports. The survey was performed in 2019 and repeated after 12 months in the same study population in 2020. Results: A total of 389 patients in 26 different states and 2 international locations had an email address on file and received an invitation in 2019 to complete the survey (median pediatric age 48.9, IQR 1.0-93.6 months). Among surveyed mothers in 2019, the overall response rate was 37.3% (145/389), and the questionnaire completion rate was 98% (145/148), 87.8% (130/148), 71.1% (81/100), 86.4% (19/22), and 74.3% (110/148) for the CSQ, FCQ, ASQ-3, M-CHAT-R/F, and TYQ, respectively. In 2020, the overall response rate was 57.8% (56/97), and the questionnaire completion rate was 96.4% (54/56), 91.1% (51/56), 86.1% (31/36), 91.7% (11/12), and 80.4% (45/56) for the CSQ, FCQ, ASQ-3, M-CHAT-R/F, and TYQ, respectively. Conclusions: This is the first study to use both REDCap and computer automation to aid in the dissemination, collection, and reporting of surveys to collect long-term pediatric outcomes in the field of fetal medicine.

Participation Rates in 11 National Dental Practice-Based Research Network Surveys 2014-2022.

Surveys of health professionals typically have low response rates, which have decreased in recent years. We report on the methods used, participation rates, and study time for 11 national questionnaire studies of dentists conducted from 2014-2022. Participation rates decreased (87%-25%). Concurrent with this decrease was a decrease in the intensity with which the practitioners were recruited. Participation rates were higher when postal mail invitation and paper options were used (84% vs. 58%, p < .001). Completion rates were nearly twice as high in studies that recruited in waves than those that did not (61% vs. 35%, p = .003). Study time varied from 2.6 to 28.4 weeks. Study time was longest when postal mail and completion on paper were used (26.0 vs. 11.3 weeks, p = .01). Among studies using only online methods, study time was longer when invitations were staggered than when all invitations went out in one bolus (means 12.0 and 5.2, p = .04). Study time was positively correlated with participation rates (Spearman r = .80, p = .005). General dentists participated at an average of 12% higher rates than specialists. Recruitment methodology, such as recruiting in waves or stages, should be considered when designing surveys.

Association of disease duration and PASI response rates at week 12 in patients with moderate-to-severe plaque psoriasis receiving biologics in the real-world psoriasis study of health outcomes (PSoHO).

PURPOSE: Currently, in the treatment of moderate-to-severe psoriasis (PsO) there is a lack of evidence demonstrating optimal biologic treatment response with respect to disease duration. The aim of this post-hoc analysis, using real world data from the Psoriasis Study of Health Outcomes (PSoHO), is to provide evidence if early intervention with biologics is associated with better treatment outcomes and if there is any difference among drug classes or individual biologics. MATERIALS AND METHODS: For this post-hoc analysis patients were categorised into two subgroups according to shorter (≤2 years) or longer (>2 years) disease duration. Analysis was performed on anti-interleukin (IL)-17A cohort vs other biologics cohort, anti-IL-17A vs other drug classes, and pairwise comparisons of ixekizumab vs individual biologics, provided that the statistical models converged. Analysis investigated the association of disease duration with the proportion of patients achieving 100% improvement in Psoriasis Area Severity Index score (PASI 100) at week 12. Adjusted comparative analyses, reported as odds ratio (OR), were performed using Frequentist Model Averaging (FMA) for each cohort or treatments within each subcategory of the subgroups. RESULTS: At week 12, anti-IL-17A and other biologics cohorts displayed minimal differences in numerical response rate for PASI 100 with respect to disease duration. The anti-IL-17A cohort showed a higher numerical PASI 100 response rate compared to the other biologic cohort irrespective of disease duration (≤2 years: 36.7% vs 21.8%; >2 years: 35.8% vs 21.9%). CONCLUSION: Overall, the results do not clearly indicate that treating patients early is critical in achieving optimal patient outcomes. Furthermore, patients treated with ixekizumab show numerically higher response rates relative to other individual biologics irrespective of disease duration.

Absolute and relative outcomes of psychotherapies for eight mental disorders: a systematic review and meta-analysis.

Psychotherapies are first-line treatments for most mental disorders, but their absolute outcomes (i.e., response and remission rates) are not well studied, despite the relevance of such information for health care users, providers and policy makers. We aimed to examine absolute and relative outcomes of psychotherapies across eight mental disorders: major depressive disorder (MDD), social anxiety disorder, panic disorder, generalized anxiety disorder (GAD), specific phobia, post-traumatic stress disorder (PTSD), obsessive-compulsive disorder (OCD), and borderline personality disorder (BPD). We used a series of living systematic reviews included in the Metapsy initiative (www.metapsy.org), with a common strategy for literature search, inclusion of studies and extraction of data, and a common format for the analyses. Literature search was conducted in major bibliographical databases (PubMed, PsycINFO, Embase, and the Cochrane Register of Controlled Trials) up to January 1, 2023. We included randomized controlled trials comparing psychotherapies for any of the eight mental disorders, established by a diagnostic interview, with a control group (waitlist, care-as-usual, or pill placebo). We conducted random-effects model pairwise meta-analyses. The main outcome was the absolute rate of response (at least 50% symptom reduction between baseline and post-test) in the treatment and control conditions. Secondary outcomes included the relative risk (RR) of response, and the number needed to treat (NNT). Random-effects meta-analyses of the included 441 trials (33,881 patients) indicated modest response rates for psychotherapies: 0.42 (95% CI: 0.39-0.45) for MDD; 0.38 (95% CI: 0.33-0.43) for PTSD; 0.38 (95% CI: 0.30-0.47) for OCD; 0.38 (95% CI: 0.33-0.43) for panic disorder; 0.36 (95% CI: 0.30-0.42) for GAD; 0.32 (95% CI: 0.29-0.37) for social anxiety disorder; 0.32 (95% CI: 0.23-0.42) for specific phobia; and 0.24 (95% CI: 0.15-0.36) for BPD. Most sensitivity analyses broadly supported these findings. The RRs were significant for all disorders, except BPD. Our conclusion is that most psychotherapies for the eight mental disorders are effective compared with control conditions, but absolute response rates are modest. More effective treatments and interventions for those not responding to a first-line treatment are needed.

Tyrosine kinase inhibitors in patients with advanced anaplastic thyroid cancer: an effective analysis based on real-world retrospective studies.

Background: Tyrosine kinase inhibitors (TKIs) contribute to the treatment of patients with anaplastic thyroid cancer (ATC). Although prospective clinical studies of TKIs exhibit limited efficacy, whether ATC patients benefit from TKI treatment in real-world clinical practice may enlighten future explorations. Therefore, we conducted this effective analysis based on real-world retrospective studies to illustrate the efficacy of TKI treatment in ATC patients. Methods: We systematically searched the online databases on September 03, 2023. Survival curves were collected and reconstructed to summarize the pooled curves. Responses were analyzed by using the "meta" package. The primary endpoints were progression-free survival (PFS), overall survival (OS), objective response rate (ORR), and disease control rate (DCR). Results: 12 studies involving 227 patients were enrolled in the study. Therapeutic strategies included: anlotinib, lenvatinib, dabrafenib plus trametinib, vemurafenib, pembrolizumab plus dabrafenib and trametinib, pembrolizumab plus lenvatinib, pembrolizumab plus trametinib, and sorafenib. The pooled median OS and PFS were 6.37 months (95% CI 4.19-10.33) and 5.50 months (95% CI 2.17-12.03). The integrated ORR and DCR were 32% (95% CI 23%-41%) and 40% (95% CI 12%-74%). Conclusion: In real-world clinical practice, ATC patients could benefit from TKI therapy. In future studies, more basic experiments and clinical explorations are needed to enhance the effects of TKIs in the treatment of patients with ATC.

Survey response over 15 years of follow-up in the Millennium Cohort Study.

BACKGROUND: Patterns of survey response and the characteristics associated with response over time in longitudinal studies are important to discern for the development of tailored retention efforts aimed at minimizing response bias. The Millennium Cohort Study, the largest and longest running cohort study of military personnel and veterans, is designed to examine the long-term health effects of military service and experiences and thus relies on continued participant survey responses over time. Here, we describe the response rates for follow-up survey data collected over 15 years and identify characteristics associated with follow-up survey response and mode of response (paper vs. web). METHOD: Patterns of follow-up survey response and response mode (web, paper, none) were examined among eligible participants (n=198,833), who were initially recruited in four panels from 2001 to 2013 in the Millennium Cohort Study, for a follow-up period of 3-15 years (2004-2016). Military and sociodemographic factors (i.e., enrollment panel, sex, birth year, race and ethnicity, educational attainment, marital status, service component, service branch, pay grade, military occupation, length of service, and time deployed), life experiences and health-related factors (i.e., military deployment/combat experience, life stressors, mental health, physical health, and unhealthy behaviors) were used to examine follow-up response and survey mode over time in multivariable generalized estimating equation models. RESULTS: Overall, an average response rate of 60% was observed across all follow-up waves. Factors associated with follow-up survey response over time included increased educational attainment, married status, female sex, older age, military deployment (regardless of combat experience), and higher number of life stressors, mental health issues, and physical health diagnoses. CONCLUSION: Despite the challenges associated with collecting multiple waves of follow-up survey data from members of the U.S. military during and after service, the Millennium Cohort Study has maintained a relatively robust response rate over time. The incorporation of tailored messages and outreach to those groups least likely to respond over time may improve retention and thereby increase the representativeness and generalizability of collected survey data.

Response rates in clinical quality registries and databases that collect patient reported outcome measures: a scoping review.

BACKGROUND: Patient Reported Outcome Measures (PROMs) are being increasingly introduced in clinical registries, providing a personal perspective on the expectations and impact of treatment. The aim of this study was to describe response rates (RR) to PROMs in clinical registries and databases and to examine the trends over time, and how they change with the registry type, region and disease or condition captured. METHODS: We conducted a scoping literature review of MEDLINE and EMBASE databases, in addition to Google Scholar and grey literature. All English studies on clinical registries capturing PROMs at one or more time points were included. Follow up time points were defined as follows: baseline (if available), < 1 year, 1 to < 2 years, 2 to < 5 years, 5 to < 10 years and 10 + years. Registries were grouped according to regions of the world and health conditions. Subgroup analyses were conducted to identify trends in RRs over time. These included calculating average RRs, standard deviation and change in RRs according to total follow up time. RESULTS: The search strategy yielded 1,767 publications. Combined with 20 reports and four websites, a total of 141 sources were used in the data extraction and analysis process. Following the data extraction, 121 registries capturing PROMs were identified. The overall average RR at baseline started at 71% and decreased to 56% at 10 + year at follow up. The highest average baseline RR of 99% was observed in Asian registries and in registries capturing data on chronic conditions (85%). Overall, the average RR declined as follow up time increased. CONCLUSION: A large variation and downward trend in PROMs RRs was observed in most of the registries identified in our review. Formal recommendations are required for consistent collection, follow up and reporting of PROMs data in a registry setting to improve patient care and clinical practice. Further research studies are needed to determine acceptable RRs for PROMs captured in clinical registries.

Does the feedback of blood results in observational studies influence response and consent? A randomised study of the Understanding Society Innovation Panel.

BACKGROUND: While medical studies generally provide health feedback to participants, in observational studies this is not always the case due to logistical and financial difficulties, or concerns about changing observed behaviours. However, evidence suggests that lack of feedback may deter participants from providing biological samples. This paper investigates the effect of offering feedback of blood results on participation in biomeasure sample collection. METHODS: Participants aged 16 and over from a longitudinal study - the Understanding Society Innovation Panel-were randomised to three arms - nurse interviewer, interviewer, web survey - and invited to participate in biomeasures data collection. Within each arm they were randomised to receive feedback of their blood results or not. For those interviewed by a nurse both venous and dried blood samples (DBS) were taken in the interview. For the other two arms, they were asked if they would be willing to take a sample, and if they agreed a DBS kit was left or sent to them so the participant could take their own sample and return it. Blood samples were analysed and, if in the feedback arms, participants were sent their total cholesterol and HbA1c results. Response rates for feedback and non-feedback groups were compared: overall; in each arm of the study; by socio-demographic and health characteristics; and by previous study participation. Logistic regression models of providing a blood sample by feedback group and data collection approach controlling for confounders were calculated. RESULTS: Overall 2162 (80.3% of individuals in responding households) took part in the survey; of those 1053 (48.7%) consented to provide a blood sample. Being offered feedback had little effect on overall participation but did increase consent to provide a blood sample (unadjusted OR 1.38; CI: 1.16-1.64). Controlling for participant characteristics, the effect of feedback was highest among web participants (1.55; 1.11-2.17), followed by interview participants (1.35; 0.99 -1.84) and then nurse interview participants (1.30; 0.89-1.92). CONCLUSIONS: Offering feedback of blood results increased willingness to give samples, especially for those taking part in a web survey.

Differential organ-specific tumor response to first-line immune checkpoint inhibitor therapy in non-small cell lung cancer-a retrospective cohort study.

Background: Immune checkpoint inhibitors (ICIs) possess remarkable clinical effectiveness in non-small cell lung cancer (NSCLC). Different immune profiles of tumors may play a key role in the efficacy of treatment with ICIs. This article aimed to determine the differential organ responses to ICI in individuals with metastatic NSCLC. Methods: This research analyzed data of advanced NSCLC patients receiving first-line treatment with ICIs. Major organs such as the liver, lung, adrenal glands, lymph nodes and brain were assessed using the Response Evaluation Criteria in Solid Tumors (RECIST) 1.1 and RECIST-improved organ-specific response criteria. Results: A retrospective analysis was conducted on a total of 105 individuals with advanced NSCLC with programmed death ligand-1 (PD-L1) expression ≥50% who received single agent anti-programmed cell death protein 1 (PD-1)/PD-L1 monoclonal antibodies as first-line therapy. Overall, 105 (100%), 17 (16.2%), 15 (14.3%), 13 (12.4%), and 45 (42.8%) individuals showed measurable lung tumors and liver, brain, adrenal, and other lymph node metastases at baseline. The median size of the lung, liver, brain, adrenal gland, and lymph nodes were 3.4, 3.1, 2.8, 1.9, and 1.8 cm, respectively. The results recorded mean response times of 2.1, 3.4, 2.5, 3.1, and 2.3 months, respectively. Organ-specific overall response rates (ORRs) were 67%, 30.6%, 34%, 39%, and 59.1%, respectively, with the liver having the lowest remission rate and lung lesions having the highest remission rate. There were 17 NSCLC patients with liver metastasis at baseline, and 6 had different responses to ICI treatment, with remission in the primary lung site and progressive disease (PD) in the metastatic liver site. At baseline, the mean progression-free survival (PFS) of the 17 patients with liver metastasis and 88 patients without liver metastasis was 4.3 and 7 months, respectively (P=0.02, 95% CI: 0.691 to 3.033). Conclusions: The liver metastases of NSCLC may be less responsive to ICIs than other organs. The lymph nodes respond most favorably to ICIs. Further strategies may include additional local treatment in case of oligoprogression in these organs in patients with otherwise sustained treatment benefit.

The effect of sampling mode on response rate and bias in elite surveys.

The literature frequently recommends purposive sampling of elites based on the assumptions that random sampling negatively affects the response rate and that it induces bias. I test these assumptions drawing on metadata from 282 samples of political, economic, and social elites, and on microdata from 2,658 elites. First I use permutations to calculate confidence intervals for the expected response rate following each sampling method. Second, I estimate the effect of random sampling on the final response rate using a range of regression models. Finally, I compare the distributions of the estimators for the average age, the share of male elites, and elites' ideology by simulating repeated random and purposive samples. Results indicate that both random and purposive sampling of elites generate sufficiently large samples, as well as consistent and unbiased estimators of population parameters. Contradicting methodological guidelines in the field, the conclusion is that random sampling of elites is efficient.

Lisocabtagene maraleucel for relapsed or refractory large B-cell non-Hodgkin lymphoma.

INTRODUCTION: Chimeric antigen receptor T (CAR T) cell therapy epitomizes the success of T cell engineering. Today, it is an integral component of the treatment algorithm for various types of B-cell non-Hodgkin lymphoma (NHL). Large B-cell lymphoma (LBCL) is the most common subtype of NHL accounting for 30-35% of cases. A lack of response to second-line therapy portends a poor prognosis as only 7-15% of patients attain complete remission (CR) with subsequent conventional chemoimmunotherapy. AREAS COVERED: Lisocabtagene maraleucel (liso-cel) is an autologous CD-19 directed CAR T-cell product with a 4-1BB co-stimulatory domain administered as a sequential infusion of 2 separately manufactured components: CD8+ and CD4+ CAR T-cells in equal doses. Liso-cel showed an impressive objective response rate of 73% (CR = 53%) in patients who had received a median of 3 prior therapies. Median time-to-first CR or partial response (PR) was 1 month. EXPERT OPINION: When evaluated in the second line setting in LBCL, liso-cel demonstrated superior event-free survival (EFS) versus standard of care. While acknowledging that choice of a particular CAR T-cell is based chiefly on familiarity of the treating physician with a specific product, liso-cel definitely represents an important addition to the treatment armamentarium of R/R LBCL whether in the second-line setting or beyond.

Optimising response rates in a national postal survey evaluating community mental health care: four interventions trialled.

BACKGROUND: The Community Mental Health Survey (CMHS) is a valuable resource of information on experiences of mental health care in England; however, response rates are declining. AIM: To increase the overall response rate and response rate of young adult service users. METHODS: Four interventions were trialled in a randomised controlled study design alongside the 2017 CMHS. The questionnaire and information letters were modified based on an established framework for influencing behaviour. The modified materials plus a pre-notification card were tested to increase the overall response rate, identified by one-sided z-tests between the intervention and control groups. An information flyer was modified to target service users age 18 to 35, tested using multilevel logistic regression. RESULTS: The overall response rate significantly increased with the modified information letters compared to the control (29.1% vs. 25.1%; p = 0.007). The targeted information flyer did not increase responses from younger service users; though the combination of modified information letters and questionnaire did (24.6% vs. 15.8%; p = 0.01). CONCLUSIONS: Modifying information letters based on the easy, attractive, social and timely (EAST) framework can increase response rate in postal surveys evaluating community mental health care. Modified letters combined with a modified questionnaire can increase the response from younger service users.

Objective response rate of placebo in randomized controlled trials of anticancer medicines.

Background: Spontaneous regression of advanced solid tumors is infrequent but may occur. Quantifying response rates from placebo in cancer drug trials may provide important information for physicians, patients, and regulators. We aimed to provide a pooled placebo response rate from drug trials in advanced solid tumors. Methods: We pooled the overall response rate (ORR), complete response rate (CR) and partial response rates (PR) in the placebo arm of placebo-controlled randomized controlled trials (RCTs) of cancer drugs for advanced solid tumors published during 2015-2021 using random-effects model. Findings: 45 phase 3 RCTs including 5684 patients on placebo met our inclusion criteria and formed the study cohort. The pooled overall ORR, CR and PR rates in the placebo arm were 1% (95% CI, 0%-2%), 0% (95% CI, 0%-0%), and 1% (95% CI, 0%-2%) respectively. Higher placebo responses were observed in prostate cancer and sarcoma trials. Interpretation: Overall, 1% patients with advanced solid tumors can expect to achieve some response even in absence of treatment. However, complete regression without treatment is extremely rare, almost zero percent. This information will be helpful to patients in their decisions, as well as regulators in evaluating cancer drugs' efficacy based on response rates alone. Funding: None.

Any role for transarterial radioembolization in unresectable intrahepatic cholangiocarcinoma in the era of advanced systemic therapies?

Intrahepatic cholangiocarcinoma (iCCA) is recognized as the second most frequently diagnosed liver malignancy, following closely after hepatocellular carcinoma. Its incidence has seen a global upsurge in the past several years. Unfortunately, due to the lack of well-defined risk factors and limited diagnostic tools, iCCA is often diagnosed at an advanced stage, resulting in a poor prognosis. While surgery is the only potentially curative option, it is rarely feasible. Currently, there are ongoing investigations into various treatment approaches for unresectable iCCA, including conventional chemotherapies, targeted therapies, immunotherapies, and locoregional treatments. This study aims to explore the role of transarterial radioembolization (TARE) in the treatment of unresectable iCCA and provide a comprehensive review. The findings suggest that TARE is a safe and effective treatment option for unresectable iCCA, with a median overall survival (OS) of 14.9 months in the study cohort. Studies on TARE for unresectable iCCA, both as a first-line treatment (as a neo-adjuvant down-staging strategy) and as adjuvant therapy, have reported varying median response rates (ranging from 34% to 86%) and median OS (12-16 mo). These differences can be attributed to the heterogeneity of the patient population and the limited number of participants in the studies. Most studies have identified tumor burden, portal vein involvement, and the patient's performance status as key prognostic factors. Furthermore, a phase 2 trial evaluated the combination of TARE and chemotherapy (cisplatin-gemcitabine) as a first-line therapy for locally advanced unresectable iCCA. The results showed promising outcomes, including a median OS of 22 mo and a 22% achievement in down-staging the tumor. In conclusion, TARE represents a viable treatment option for unresectable iCCA, and its combination with systemic chemotherapy has shown promising results. However, it is important to consider treatment-independent factors that can influence prognosis. Further research is necessary to identify optimal treatment combinations and predictive factors for a favorable response in iCCA patients.

Women's Perspectives on Factors Influencing Florida Pregnancy Risk Assessment Monitoring System (PRAMS) Response.

OBJECTIVES: To inform updates to the Pregnancy Risk Assessment Monitoring System (PRAMS) design and processes, African American/Black and Hispanic/Latina women in Florida provided feedback on their awareness and perceptions of the PRAMS survey, and preferences for survey distribution, completion, design and content. METHODS: Focus groups were conducted in English and Spanish with 29 women in two large metropolitan counties. Participants completed a brief survey, reviewed the PRAMS questionnaire and recruitment materials, engaged in discussion, and gave feedback directly onto cover design posters. RESULTS: Participants reported limited awareness of PRAMS. Preferences for survey distribution and completion varied by participant lifestyle. Interest in topics covered by PRAMS was as a motivator for completion, while distrust and confidentiality concerns were deterrents. Participants were least comfortable answering questions about income, illegal drug use, and pregnancy loss/infant death. Changes to the length of the survey, distribution methods, and incentives/rewards for completion were recommended. CONCLUSIONS FOR PRACTICE: Results highlight the need to increase PRAMS awareness, build trust, and consider the design, length and modality for questionnaire completion as possible avenues to improve PRAMS response rates.

Survey Response Rates to a Self-Initiated Longitudinal Survey Accessed by a Quick Response Code in Six Different Regions of the United States.

Background A quick response (QR) code allows rapid access to an online survey via a smartphone and may improve response rates for web-based surveys. We report the response rates for a QR code-based, self-initiated, longitudinal survey of opioid use and pain scores following hospital discharge in pediatric surgical patients. Methodology All parents of pediatric patients who underwent surgery at one of six pediatric medical facilities were asked to participate in the study from October 5, 2020, until July 15, 2021. Those who chose to participate accessed the initial enrollment survey using a QR code on a handout provided. The next day they received an emailed link to a daily survey until their child was not requiring opioids and had pain scores of less than 4 for the previous 48 hours. Results A total of 1,759 families were asked to participate in the study. The parents of 44 patients completed the initial enrollment survey by accessing the QR code (response rate of 2.5%). Of those who completed the initial survey, 67% were lost to follow-up during the survey series. Conclusions We found an extremely low response rate for a self-initiated survey accessed by QR code. Additionally, we found a drop in the response rate with each successive daily email-based survey. At the end of the survey series, the majority of the initial participants had dropped out. We recommend using alternative modalities (informed consent, telephone call, weekly surveys) for initiating and delivering surveys to improve response rates for similarly designed studies.

Immune checkpoint inhibitor-based therapy for advanced clear cell renal cell carcinoma: A narrative review.

The prognosis for advanced clear cell renal cell carcinoma (ccRCC) is not satisfactory, even though its treatment has evolved rapidly over the past 20 years. Systemic ccRCC treatment options mainly involve antiangiogenic therapy, immune checkpoint blockade, or a combination of these therapies, and as more clinical evidence becomes available, immune checkpoint inhibitors (ICIs) are increasingly dominant. Conventional ICIs lead to the restoration of T-cell activation and a reduction in T-cell depletion by specifically blocking programmed cell death 1 (PD-1), programmed cell death 1 ligand 1 (PD-L1) or cytotoxic T lymphocyte antigen 4 (CTLA-4), ultimately enhancing the antitumor immune response. There is no doubt that these therapies have achieved some clinical efficacy in the overall ccRCC population, but response rates and durability remain a great challenge. Therefore, novel immune checkpoints or new combination therapeutic strategies based on ICIs continue to be sought and developed. This review will provide a comprehensive overview of ICI-based therapeutic strategies in advanced ccRCC, including their mechanisms of action and the latest clinical evidence.

The MOBIS dataset: a large GPS dataset of mobility behaviour in Switzerland.

This article presents the MOBIS dataset and underlying survey methods used in its collection. The MOBIS study was a nation-wide randomised controlled trial (RCT) of transport pricing in Switzerland, utilising a combination of postal recruitment, online surveys, and GPS tracking. 21,571 persons completed the first online survey, and 3680 persons completed 8 weeks of GPS tracking. Many continued tracking for over a year after the study was completed. In the field experiment, participants participated through the use of a GPS tracking app, Catch-my-Day, which logged their daily travel on different transport modes and imputed the trip segments and modes. The experiment lasted 8 weeks, bookended by two online surveys. After the first 4-week control phase, participants were split into two different treatment groups and a continued control group. An analysis of the survey participation shows that the technology is capable of supporting such an experiment on both Android and iOS, the two main mobile platforms. Significant differences in the engagement and attrition were observed between iOS and Android participants over the 8-week period. Finally, the attrition rate did not vary between treatment groups. This paper also reports on the wealth of data that are being made available for further research, which includes over 3 million trip stages and activities, labelled with transport mode and purpose respectively. Supplementary Information: The online version contains supplementary material available at 10.1007/s11116-022-10299-4.

Changes in Serum Interferon Gamma and Interleukin-10 in Relation to Direct-Acting Antiviral Therapy of Chronic Hepatitis C Genotype 4: A Pilot Study.

Introduction: This study analyzes the changing levels of circulating inflammatory cytokines Interferon gamma (IFN-γ) and interleukin (IL)-10 (as the main cytokines of T-helper-1 and T-helper-2 immune responses) in patients with chronic hepatitis C virus (HCV) infection undergoing therapy with direct-acting antivirals (DAAs) and to correlate them with laboratory markers. Methods: This Pilot study included 50 HCV monoinfected patients who received DAAs for 12 or 24 weeks. They were followed up monthly during therapy and 3 months after the end of the treatment. Liver disease was determined by transient elastography, in addition to FIB-4 indices. Analysis of IFN-gamma and IL-10 was carried out using an enzyme-linked immunosorbent assay. Results: All patients carried HCV genotype 4. The Sustained virological response was 100% and 92% in cirrhotics and noncirrhotics, respectively. There was no significant difference between groups in baseline IL-10 or IFN-gamma. In noncirrhotics, IL-10 showed a significant reduction at Week 4 after treatment start. In cirrhotics, IL-10 showed a significant reduction at Week 4 after treatment starts and a significant reduction at Week 12 after the end of the treatment. At Week 12 after the end of the treatment, serum IL-10 levels were significantly lower in cirrhotics. IFN-γ showed nonsignificant changes in noncirrhotics. A significant increase of IFN-γ occurred in cirrhotics from Week 4 after treatment starts to 12 weeks after the end of the treatment. IFN-γ was significantly higher in cirrhotics at Week 12 after the end of the treatment. IFN-γ and IL-10 showed different correlations with laboratory markers. Conclusion: Viral eradication induced by DAAs caused a significant change in IL-10 and IFN-gamma.

Long-term efficacy of proton pump inhibitors as a treatment modality for eosinophilic esophagitis.

BACKGROUND: Proton pump inhibitors (PPIs) are a first-line treatment for EoE, but data are limited concerning response durability. We aimed to determine long-term outcomes in EoE patients responsive to PPI-therapy. METHODS: We conducted a retrospective cohort study of newly diagnosed adults with EoE who had initial histologic response (<15 eosinophils per high-power-field) to PPI-only therapy. We extracted data regarding their subsequent clinical course and outcomes. We compared findings between the initial PPI-response endoscopy and the final endoscopy, and assessed factors associated with loss of PPI response. RESULTS: Of 138 EoE patients with initial histologic response to PPI, 50 had long-term endoscopic follow-up, 40 had clinical follow-up, 10 changed treatments, and 38 had no long-term follow-up. Of those with endoscopic follow-up, mean follow-up-time was 3.6 ± 2.9 years; 30 and 32 patients (60%; 64%) maintained histologic and symptom responses, respectively. However, fibrotic endoscopic findings of EoE were unchanged. Younger age (aOR 1.05, 95% CI: 1.01-1.11) and dilation prior to PPI treatment (aOR 0.21, 95% CI: 0.05-0.83) were the only factors associated with long-term loss of PPI response. CONCLUSIONS: Long-term histologic and clinical response rates for PPI therapy were 60% and 64%, respectively. Younger age and dilation at baseline were associated with histologic loss of response. These data can inform long-term EoE treatment selection.