Completion Date Research Articles

Effects of the Strong Hearts program at two years post program completion.

This is a follow-up to the original published article, Effects of the Strong Hearts Program after a Major Cardiovascular Event in Patients with Cardiovascular Disease. This study evaluated the long-term efficacy of the Strong Hearts program up to 2 years after program completion. All study participants who initially completed the Strong Hearts program between 2020 and 2021 (n=128) were contacted at 12months and 24months following the date of program completion. A phone survey was conducted to see if any significant post-cardiovascular events or readmissions to the hospital occurred, and self-reported dates of any occurrences were recorded. Hospital readmissions and cardiac-related procedures were cross-referenced with the hospital's electronic medical record. A chi-square goodness-of-fit analysis was utilized to compare the observed rates of categorical outcomes vs. expected rates yielded from the empirical literature. The rate of all-cause readmission at 6months post-program completion was 2/120 (1.7 %), compared to the expected rate of 50 %, χ2(1) = 112.13, p<0.001. The readmission rate at 1 year post-program completion was 17/120 (14.2 %), vs. the expected rate of 45 %, χ2(1) = 46.09, p<0.001, and at 2 years post-program completion, the readmission rate was 24/120 (20.0 %) compared to the expected rate of 53.8 %, χ2(1) = 56.43, p<0.001. Ten participants (8.3 %) had a subsequent cardiac procedure within 2 years of completing the program, including two requiring percutaneous coronary intervention (1.7 %) and eight requiring coronary artery bypass grafting (CABG, 6.7 %), compared to the expected rates of 13.4 and 57.74 %, χ2(1)=153.08, p<0.001, respectively. Mortality at 2 years post-program completion was 2/128 (1.6 %), compared to 23.4 %, χ2(1)=34.13, p<0.001. Efficacy of the Strong Hearts program continued at 6months, 1 year, and 2 years post-program completion in terms of all-cause readmission, subsequent cardiac event, and all-cause mortality.

The state of individual participant data sharing for the highest-revenue medicines.

Amid growing emphasis from pharmaceutical companies, advocacy groups, and regulatory bodies for sharing of individual participant data, recent audits reveal limited sharing, particularly for high-revenue medicines. Therefore, this study aimed to assess the individual participant data-sharing eligibility of clinical trials supporting the Food and Drug Administration approval of the top 30 highest-revenue medicines for 2021. A cross-sectional analysis was conducted on 316 clinical trials supporting approval of the top 30 revenue-generating medicines of 2021. The study assessed whether these trials were eligible for individual participant data sharing, defined as being publicly listed on a data-sharing platform or confirmed by the trial sponsors as in scope for independent researcher individual participant data investigations. Information was gathered from various sources including ClinicalTrials.gov, the European Union Clinical Trials Register, and PubMed. Key factors such as the trial phase, completion dates, and the nature of the data-sharing process were also examined. Of the 316 trials, 201 (64%) were confirmed eligible for sharing, meaning they were either publicly listed on a data-sharing platform or confirmed by the trial sponsors as in scope for independent researcher individual participant data investigations. A total of 102 (32%) were confirmed ineligible, and for 13 (4%), the sponsor indicated that a full research proposal would be required to determine eligibility. The analysis also revealed a higher rate of individual participant data sharing among companies that utilized independent platforms, such as Vivli, for managing their individual participant data-sharing process. Trials not marked as completed had significantly lower eligibility for individual participant data sharing. This study highlights that a substantial portion of trials for top revenue-generating medicines are eligible for individual participant data sharing. However, challenges persist, particularly for trials that are marked as ongoing and for trials where the sharing processes are managed internally by pharmaceutical companies. Data-sharing rates could be improved by adopting open-access individual participant data-sharing models or using independent platforms. Standardizing policies to facilitate immediate individual participant data availability for approved medicines is necessary.

Development and Evaluation of the Telehealth in Motor Neuron Disease System: The TIME Study Protocol.

For more responsive care provision for motor neuron disease and caregivers, a digital system called Telehealth in MND-Care (TiM-C) was created. TiM-C sends regular symptom questionnaires to users; their responses are sent to health care professionals (HCPs). To enable people with motor neuron disease to participate in research studies more easily, a parallel platform was developed from TiM-C, called Telehealth in MND-Research (TiM-R). TiM-R can advertise studies, collect data, and make them available to MND researchers. This study has 4 work packages (WPs) to facilitate service approval, codevelop the TiM systems, and evaluate the service. Each WP aims to understand (1) what helps and hinders the approval of the TiM-C system as a National Health Service; (2) what aspects of MND care and research are currently unmet and can be addressed through the TiM-C and TiM-R systems; (3) how TiM-C influences MND care, from the perspective of people with motor neuron disease, their caregivers, and HCPs; and (4) the costs and benefits associated with TiM-C. WP1 will use semistructured interviews with 10-15 people involved in the approval of TiM-C to understand the barriers and facilitators to governance processes. WP2 will use individual and group interviews with 25-35 users (people with motor neuron disease, caregivers, HCPs, MND researchers, and industry) of TiM-C and TiM-R to understand the current unmet needs of these user groups and how TiM services can be developed to meet these needs. WP3 will use a process evaluation involving 5 elements; local context, engagement, user experiences, service impact, and mechanisms of action. A range of methods, including audits, analysis of routine data, questionnaires, interviews, and observations will be used with people with motor neuron disease, caregivers, and HCPs, both those using the system and those who declined the service when invited. WP4 will use data collected through the process evaluation and known costs to conduct a cost-consequence and budget impact analysis to explore the cost-benefit of the TiM-C service. Most data collected will be qualitative, with thematic and framework analysis used to develop themes from transcripts and observations. Descriptive statistics or t tests and chi-square tests will be used to describe and analyze quantitative data. This study has received ethical approval and has begun recruitment in 1 site. Further, 13 specialist MND centers will adopt TiM-C and the TIME study, beginning in July 2024. The study will conclude in November 2026 and a final report will be produced 3 months after the completion date. This study will facilitate the implementation and development of TiM-C and TiM-R and fully evaluate the TiM-C service, enabling informed decision-making among health care providers regarding continued involvement and contribute to the wider literature relating to how technology-enabled care services can affect clinical care. DERR1-10.2196/57685.

PERBANDINGAN FUZZY TIME SERIES MARKOV CHAIN DAN FUZZY TIME SERIES CHENG

Investing is a hot and fast-growing topic right now. Stock.investing.is one of the most popular investments by the public. The JCI is an index that measures the performance of all stocks listed on the IDX. The closing price of the stock is published daily and can be used by investors as an investment benchmark. The occurrence of stock price fluctuations entails a high risk of loss. Predicting stock prices is one way to avoid this risk. Fuzzy time series is a time series model that can be used to minimize the risk of future occurrences. Time parameter data is not stationary, so this research uses fuzzy time series with Markov chains and Cheng's method to determine the JCI completion dates from January 2018 to June 2022. Calculating the accuracy level of the two method approaches using symmetric Mean Absolute Percentage Error (sMAPE), the sMAPE value obtained in the Fuzzy Time Series Markov Chain method is 2.11% and the Fuzzy Time Series Cheng method is 2.98%. The conclusion is that of the two methods the Markov Chain has a smaller value and can be said to be a better method compared to the Cheng method.

GOG-3097/ENGOT-ov81/GTG-UK/RAMP 301: a phase 3, randomized trial evaluating avutometinib plus defactinib compared with investigator’s choice of treatment in patients with recurrent low grade serous ovarian cancer

BackgroundThere are no approved treatments specifically for low grade serous ovarian cancer; current standard of care treatment options are limited in efficacy and tolerability. The combination of avutometinib with defactinib...

Effect of Delay on Profitability of Building Construction Projects

In construction, delay could be defined as the time overrun either beyond completion date specified in a contract, or beyond the date that the parties agreed upon for delivery of a project. It is a project slipping over its planned schedule and is considered as common problem in construction projects. The Indian construction sector has acted as an engine of growth for the Indian economy for over the past five-decades and becoming a basic input for the socio-economic development of the country. Construction is the second largest economic activity after agriculture, and has contributed around 6 to 9% of India's GDP over the past five years while registering 8 to 10% growth per annum. This paper refers to study effect of delay in construction industry particularly in profitability or economic factors. Keywords: Construction, Delay, Profitability, GDP, Economic factor

Poor registration and publication practices in clinical trials of targeted therapeutics for endocrine and metabolic diseases: an observational study

ObjectiveTo assess the completeness and concordance of reporting in registries and corresponding publications of interventional trials on targeted therapeutics for endocrine and metabolic disorders. Study Design and SettingWe searched clinical trial registries in September 2022 for completed interventional trials of target therapeutics for endocrine and metabolic disorders registered from 2005 onwards. We used ClinicalTrials.gov and the World Health Organization International Clinical Trial Registration Platform (WHO ICTRP) registration requirements to extract data and assess the completeness of initial entry and final updates to trial registration and concordance with their published journal articles. ResultsAmong 149 clinical trials included, 121 (81%) had corresponding publications. Missing mandatory registration data items were identified in 88 (67%) trials at the initial registration, 17 (13%) at the last registration, and in 85 (77%) corresponding publications. All trials showed changes between initial registration and final registration update, and 98% showed changes from the initial registration to publication. Changes between initial registration and final update were most common in the categories ‘Completion date’ (92%), ‘Key secondary outcomes’ (82%), and ‘Date of first enrolment’ (70%). Changes between initial registration and publication were most common in categories ‘Sample size’ (91%), ‘Key inclusion and exclusion criteria’ (81%), ‘Key secondary outcomes’ (84%), and ‘Completion date’ (83%). ConclusionDespite the legal and journal registration requirements, the completeness and consistency of reporting mandatory data items in registries and corresponding publications regarding targeted therapeutics for endocrine and metabolic disorders are inadequate. Our findings raise questions about the integrity and reliability of clinical trials focusing on targeted therapeutics.

Enabling efficient patient discharge with a mindset, toolset, and skillset framework.

33 Background: Timely access to care for patients coming through the emergency center can be achieved by efficiently discharging patients. When continued bed growth is not sustainable, improvements in team-based approaches to discharges are necessary and require a multi-pronged approach. Our institution embarked on a process improvement effort anchored on completing 1 out of 2 patients discharged by 12PM. We hypothesize that a discharge before noon will assist with getting our next patient to a bed by 2PM. We conducted a pilot on our Stem Cell Transplant units to test and enable our case for change allowing for scale and spread to other units. Methods: Our multi-pronged framework was focused on mindset, toolset, and skillset. As our first step, executive leadership set direction to achieve a shift in mindset by socializing the case for change using a hospital throughput framework including a 9-12-2 branding. A discharge launchpad [toolset] was established to define expectations of discharge tasks including accountability within a 2-day window of medical readiness for discharge. This served as the tool aligning various teams to a date for task completion. Lastly, we evaluated skillset and defined best practices for the unit RN role and APP, along with others, which included empowering various roles to discharge patients. Results: For discharges during the baseline period of 12/1/2022 - 11/30/2023 from the Stem Cell Transplant service, the percent of discharge orders in by 9AM was 10.3% and discharges completed by 12PM was 12.2%. The pilot period month of April 2024 resulted in a month close performance of 52.5% for orders in by 9AM and 55.4% of patients discharged by 12PM. The average time spent by Stem Cell patients boarding in our emergency center during the baseline period was 6 hours and 34 minutes with a reduction to 2 hours and 23 minutes in April 2024. There were no Stem Cell beds added during the pilot period compared to baseline. The period of 12/1/2023 – 3/31/2024 was excluded to limit any improving data from the Hawthorne effect since throughput socialization began during those months. Conclusions: We met the goal within one month with plans underway to assess sustainability for implementation readiness for scale and spread to other units. Early efforts made to scale this model within the institution confirm the multi-pronged model approach is necessary. Additionally, we believe that a frontline leadership model would contribute best to implementation success. Missing any component of the multi-pronged approach or team member, such as the APP role without the RN role, will not yield the ultimate result of discharging patients by noon. This change requires a coordinated, team-based approach. The new institutional governance structure for hospital throughput, including academic and operational departments, and a logistics center in the future may contribute to building trust in leadership’s commitment to improving patient care.

A Digital Intervention for Capturing the Real-Time Health Data Needed for Epilepsy Seizure Forecasting: Protocol for a Formative Co-Design and Usability Study (The ATMOSPHERE Study).

Epilepsy is a chronic neurological disorder affecting individuals globally, marked by recurrent and apparently unpredictable seizures that pose significant challenges, including increased mortality, injuries, and diminished quality of life. Despite advancements in treatments, a significant proportion of people with epilepsy continue to experience uncontrolled seizures. The apparent unpredictability of these events has been identified as a major concern for people with epilepsy, highlighting the need for innovative seizure forecasting technologies. The ATMOSPHERE study aimed to develop and evaluate a digital intervention, using wearable technology and data science, that provides real-time, individualized seizure forecasting for individuals living with epilepsy. This paper reports the protocol for one of the workstreams focusing on the design and testing of a prototype to capture real-time input data needed for predictive modeling. The first aim was to collaboratively design the prototype (work completed). The second aim is to conduct an "in-the-wild" study to assess usability and refine the prototype (planned research). This study uses a person-based approach to design and test the usability of a prototype for real-time seizure precipitant data capture. Phase 1 (work completed) involved co-design with individuals living with epilepsy and health care professionals. Sessions explored users' requirements for the prototype, followed by iterative design of low-fidelity, static prototypes. Phase 2 (planned research) will be an "in-the-wild" usability study involving the deployment of a mid-fidelity, functional prototype for 4 weeks, with the collection of mixed methods usability data to assess the prototype's real-world application, feasibility, acceptability, and engagement. This phase involves primary participants (adults diagnosed with epilepsy) and, optionally, their nominated significant other. The usability study will run in 3 rounds of deployment and data collection, aiming to recruit 5 participants per round, with prototype refinement between rounds. The phase-1 co-design study engaged 22 individuals, resulting in the development of a mid-fidelity, functional prototype based on identified requirements, including the tracking of evidence-based and personalized seizure precipitants. The upcoming phase-2 usability study is expected to provide insights into the prototype's real-world usability, identify areas for improvement, and refine the technology for future development. The estimated completion date of phase 2 is the last quarter of 2024. The ATMOSPHERE study aims to make a significant step forward in epilepsy management, focusing on the development of a user-centered, noninvasive wearable device for seizure forecasting. Through a collaborative design process and comprehensive usability testing, this research aims to address the critical need for predictive seizure forecasting technologies, offering a promising approach to improving the lives of individuals with epilepsy. By leveraging predictive analytics and personalized machine learning models, this technology seeks to offer a novel approach to managing epilepsy, potentially improving clinical outcomes, including quality of life, through increased predictability and seizure management. DERR1-10.2196/60129.

Order Management in the Era of Customization

The economic growth and development of the country largely depends on success in the main industries, where mechanical engineering plays a key role. The economies of different countries rely on industrial enterprises that satisfy people’s needs through the production of goods and services. To ensure competitiveness in a dynamically changing market, continuous improvement and development of enterprises is required. In the current conditions, mechanical engineering enterprises increasingly need to adapt to each individual customer, which in turn forces them to rebuild enterprises to the parameters typical for single or small-scale production. The production process is the joint work of tools, equipment and personnel through which products are produced. The complexity of the process of releasing new products is due to the involvement of a large number of departments involved in this process. Order execution is based primarily on the management of production systems. One of the most important processes in enterprise management is the order management process.The purpose of this study is to determine the methods of optimizing the order management process and reducing the influence of the internal cooperation factor on the increase of the period of its implementation at machine-building enterprises. As a methodological basis such general scientific methods as analysis, synthesis, comparison and contrast were used. As the information base, the article analyzes the works of modern authors who consider the issues of order management and such an element of the organizational structure as internal cooperation between departments, their influence on the internal processes of the enterprise and the timing of order execution. The features of the planning process for a single type of production are considered. In the course of the work, the main reasons for the shift in previously determined order completion dates, the most important indicators for order fulfillment, the peculiarities of the planning process at the unit type of production were considered, as well as the most frequently encountered problems during project implementation were identified. In connection with the trend towards changes and development of enterprises, in terms of changes in order management processes for customized products, an increase in the number of created enterprises and an increase in the share of customized products, a conclusion was made about the relevance of studying the order management process in a single type of production at machine-building enterprises. Conclusions are drawn about the need for further study of the issues under consideration. The materials can be used in companies in the real sector of the economy, in administrative structures and in the educational process.

The Long Haul to Surgery: Long COVID Has Minimal Burden on Surgical Departments.

Many patients infected with the SARS-CoV-2 virus (COVID-19) continue to experience symptoms for weeks to years as sequelae of the initial infection, referred to as "Long COVID". Although many studies have described the incidence and symptomatology of Long COVID, there are little data reporting the potential burden of Long COVID on surgical departments. A previously constructed database of survey respondents who tested positive for COVID-19 was queried, identifying patients reporting experiencing symptoms consistent with Long COVID. Additional chart review determined whether respondents had a surgical or non-routine invasive procedure on or following the date of survey completion. Outcomes from surgeries on patients reporting Long COVID symptoms were compared to those from asymptomatic patients. A total of 17.4% of respondents had surgery or a non-routine invasive procedure in the study period. A total of 48.8% of these patients reported experiencing symptoms consistent with Long COVID. No statistically significant differences in surgical outcomes were found between groups. The results of this analysis demonstrate that Long COVID does not appear to have created a significant burden of surgical disease processes on the healthcare system despite the wide range of chronic symptoms and increased healthcare utilization by this population. This knowledge can help guide surgical operational resource allocation as a result of the pandemic and its longer-term sequelae.

Adverse Effects Associated With Multimodal Analgesic Regimens in Critically Ill, Nonintubated Patients: A Systematic Review and Meta-Analysis

Objective: To evaluate the safety of utilizing multimodal analgesic regimens in critically ill, nonintubated patients. Data Sources: A systematic review was conducted using Embase, MEDLINE, Cochrane, SciELO, Web of Science, and the Korean Journal Index. Clinical trials of critically ill, nonintubated patients that contained complete safety outcomes date, including the incidence of specific adverse drug effects (ADE) associated with a multimodal analgesic medication or regimen, were included. Study Selection and Data Extraction: The primary outcome was the incidence of adverse drug effects associated with multimodal analgesics in comparison to standard-of-care analgesic strategies in our patient population. The secondary outcome was a subgroup analysis of adverse drug effect by type and by medication. Data Synthesis: 10 trials were included in this systematic review, of which 6 were randomized control trials that were evaluated in the meta-analysis. There was no statistically significant difference with respect to the primary outcome (mean difference = −0.11, P = 0.31, 95% CI = [−0.35, 0.13]). The subgroup analysis, which was conducted on randomized clinical control trials that documented a single adjunctive analgesic rather than a multimodal regimen, was stratified by the type of adverse effect encountered and the medication in question. There were no statistically significant findings regarding the incidence of specific ADE. Nonrandomized data included in this study support these findings. Conclusions: While concerns of the additive deleterious adverse effects commonly encountered in polypharmacy are valid, our findings support the use of multimodal analgesic regimens in the provision of analgesic in critically ill, nonintubated patients.

Clinical trials operated in Egypt : big data analysis study

Abstract: Clinical trials are one of the most direction, approached by physicians/researchers in the medical sciences field. Clinicaltrials.gov is one of the most comprehensive databases around the world, in this field. It is including clinical trials going in United States, and outside United States. The website is mentioning that the database or US government is not scientifically responsible for these trials. “The U.S. government does not review or approve the safety and science of all studies listed on this website” Egypt is one of the most important countries in the Middle East, with a long history in medical sciences as well. Provided in clinicaltrials.gov around 10 thousand trails going in Egypt. Goal of this study: In this study the author analyzed the whole trials going in Egypt, to reflect the patterns and elements of these medical trials. Research method: The method I used, is the scientometric analysis approach, analyzing the whole big data with the number of 10530 medical clinical trials, in Egypt. Conclusions: Findings varies between several elements, such as: The most rated subject is “Postoperative Pain” as this is the most condition/subject studied in clinical trials ran in Egypt. The majority of clinical trials in Egypt, started 1/1/2019. Number of these trials were 129 clinical trials. The majority of “primary completed date” was December the first, 2023. Number of trials completed at that date were 64 clinical trials in Egypt. The majority of “completion date” was December, 2023. Number of trials completed at that date were 72 clinical trials in Egypt. The majority of “first posted date” was May, 2023. Keywords: Big data, Medical research, Medical information, Clinical trials, Egypt, Egyptian clinical trials, Scientometric analysis.

The Spherical Tokamak for Energy Production (STEP) in context: UK public sector approach to fusion energy.

The UK's fusion energy approach has developed over the past 5 years to include government policy initiatives and a range of public sector investments designed to be delivered in partnership with the private sector. These have aimed to create an environment that stimulates innovation and investment to deliver economic as well as scientific and environmental benefits throughout the lifetime of the public sector fusion energy programme. The Spherical Tokamak for Energy Production acts as a focus and anchor for both public and private sector efforts to develop fusion energy, developing the supply chain and potential for Intellectual Property development and export opportunities well ahead of the anticipated STEP completion date of 2040. This is maximized by the UK's approach to a holistic research and innovation programme backed up by a regulatory and skills programme. This article is part of the theme issue 'Delivering Fusion Energy - The Spherical Tokamak for Energy Production (STEP)'.

The progress of tumor vaccines clinical trials in non-small cell lung cancer.

Non-small cell lung cancer (NSCLC) remains a significant global health challenge, with high mortality rates and limited treatment options. Tumor vaccines have emerged as a potential therapeutic approach, aiming to stimulate the immune system to specifically target tumor cells. This study screened 283 clinical trials registered on ClinicalTrials.gov through July 31, 2023. After excluding data that did not meet the inclusion criteria, a total of 108 trials were assessed. Data on registered number, study title, study status, vaccine types, study results, conditions, interventions, outcome measures, sponsor, collaborators, drug target, phases, enrollment, start date, completion date and locations were extracted and analyzed. The number of vaccines clinical trials for NSCLC has continued to increase in recent years, the majority of which were conducted in the United States. Most of the clinical trials were at stages ranging from Phase I to Phase II. Peptide-based vaccines accounted for the largest proportion. Others include tumor cell vaccines, DNA/RNA vaccines, viral vector vaccines, and DC vaccines. Several promising tumor vaccine candidates have shown encouraging results in early-phase clinical trials. However, challenges such as heterogeneity of tumor antigens and immune escape mechanisms still need to be addressed. Tumor vaccines represent a promising avenue in the treatment of NSCLC. Ongoing clinical trials are crucial for optimizing vaccine strategies and identifying the most effective combinations. Further research is needed to overcome existing limitations and translate these promising findings into clinical practice, offering new hope for NSCLC patients.

Evaluating the Efficacy of a Digital Therapeutic (CT-152) as an Adjunct to Antidepressant Treatment in Adults With Major Depressive Disorder: Protocol for the MIRAI Remote Study.

Major depressive disorder (MDD) is common worldwide and can be highly disabling. People with MDD face many barriers to treatment and may not experience full symptom relief even when treated. Therefore, new treatment modalities are needed for MDD. Digital therapeutics (DTx) may provide people with MDD an additional treatment option. This study aimed to describe a phase 3 remote, multicenter, randomized, masked, sham-controlled trial evaluating the efficacy of a smartphone app-based DTx (CT-152) in adult participants diagnosed with MDD, used as an adjunct to antidepressant therapy (ADT). Participants aged 22-64 years with a current primary diagnosis of MDD and an inadequate response to ADT were included. Participants were randomized 1:1 to CT-152 or a sham DTx. CT-152 is a smartphone app-based DTx that delivers a cognitive-emotional and behavioral therapeutic intervention. The core components of CT-152 are the Emotional Faces Memory Task exercises, brief lessons to learn and apply key therapeutic skills, and SMS text messaging to reinforce lessons and encourage engagement with the app. The sham DTx is a digital working memory exercise with emotionally neutral stimuli designed to match CT-152 for time and attention. Participants took part in the trial for up to 13 weeks. The trial included a screening period of up to 3 weeks, a treatment period of 6 weeks, and an extension period of 4 weeks to assess the durability of the effect. Sites and participants had the option of an in-person or remote screening visit; the remaining trial visits were remote. Efficacy was evaluated using the Montgomery-Åsberg Depression Rating Scale, the Generalized Anxiety Disorder-7, Clinical Global Impression-Severity scale, the Patient Health Questionnaire-9, and the World Health Organization Disability Assessment Schedule 2.0. The durability of the effect was evaluated with the Montgomery-Åsberg Depression Rating Scale and Generalized Anxiety Disorder-7 scale. Adverse events were also assessed. Satisfaction, measured by the Participant and Healthcare Professional Satisfaction Scales, and health status, measured by the EQ-5D-5L, were summarized using descriptive statistics. This study was initiated in February 2021 and had a primary completion date in October 2022. This represents the methodological design for the first evaluation of CT-152 as an adjunct to ADT. This study protocol is methodologically robust and incorporates many aspects of conventional pivotal pharmaceutical phase 3 trial design, such as randomization and safety end points. Novel considerations included the use of a sham comparator, masking considerations for visible app content, and outcome measures relevant to DTx. The rigor of this methodology will provide a more comprehensive understanding of the effectiveness of CT-152. ClinicalTrials.gov NCT04770285; https://clinicaltrials.gov/study/NCT04770285. RR1-10.2196/56960.

The observational EURACAN prospective clinical registry dedicated to epithelioid hemangioendothelioma: The protocol of an international and collaborative effort on an ultra-rare entity.

Epithelioid hemangioendothelioma (EHE) is an ultra-rare sarcoma, marked by distinctive molecular and pathological features and with a variable clinical behavior. Its natural history is still partially understood, reliable prognostic and predictive factors are lacking and many questions are still open on the optimal management. In the context of EURACAN, a prospective registry specifically dedicated to EHE was developed and launched with the aim of providing, through high-quality prospective data collection, a better understanding of this disease. Registry-based cohort study including only new cases of patients with a pathological and molecularly confirmed diagnosis of EHE. To improve the understanding of EHE natural history, validate and identify new prognostic and predictive factors, clarify the activity and efficacy of currently available treatment options, describe treatment pattern. Settings and participantsIt is an hospital-based registry established in centers with expertise in EHE including adult patients with a new pathological and molecularly confirmed diagnosis of EHE starting from the 1st December 2023. The characteristics of each patient in the facility who meets the above-mentioned inclusion criteria will be collected prospectively and longitudinally with follow-up at cancer progression and / or cancer relapse or patient death. It is a secondary use of data which will be collected from the clinical records. The data collected for the registry will not entail further examinations or admissions to the facility and/or additional appointments to those normally provided for routine patient follow-up. VariablesFull details on patients and disease features, treatment and outcome will be collected, according to common clinical practice guidelines developed and shared with all the contributing centers. In addition, data on potential confounders (e.g. comorbidity; functional status etc.) will also be collected. Statistical methodsThe data analyses will include descriptive statistics and analytical analyses. Multivariable Cox's proportional hazards model and Hazard ratios (HR) for all-cause or cause-specific mortality will be used to determine independent predictors of overall survival, recurrence and progression. The registry has been joined by 21 sarcoma reference centers across EU and UK, covering 10 countries. Patients' recruitment started in December 2023. The estimated completion date is December 2033 upon agreement on the achievement of all the registry objectives. The already established collaboration and participation of EHE patient's associations involved in the project will help in promoting the registry and fostering accrual.

Protective Assets Reinforced With Integrated Care and Technology (PARITY): Protocol for a Randomized Controlled Trial.

Black women are significantly more likely to experience severe maternal morbidity and are 3 times as likely to die from pregnancy-related causes compared to White women. Using a strengths-based wellness approach within an integrated supportive care program provided by a community doula could offer pragmatic solutions for Black maternal disparities. The Protective Assets Reinforced with Integrated Care and Technology (PARITY) program consists of a wellness technology platform, including informational links to wellness content and reinforcing motivational SMS text messages, as well as community-based doula support delivered both in person and through the technology platform to improve Black maternal wellness. This pilot randomized controlled trial (RCT) and mixed methods evaluation aims to (1) determine the feasibility and acceptability of the PARITY intervention; (2) investigate the preliminary efficacy of the PARITY intervention on clinical outcomes (maternal blood pressure, gestational weight gain, and cesarean birth); and (3) investigate changes to wellness behavioral outcomes (nutrition, physical activity, sleep, and health care adherence) and empowered strengths (self-efficacy, social support, motivation, resilience, problem-solving, and self-regulation) in the intervention group compared to a control group. A 2-arm RCT and mixed methods evaluation will be conducted. Overall, 60 Black pregnant individuals will be randomized in a ratio of 1:1 to either the intervention or informational control group. Participants in the intervention group will receive access to the technology platform over a 12-week period that ends before birth. Intervention participants will be assigned a doula interventionist, who will meet with them 4 times during the intervention. All participants (intervention and control) will receive a referral for a birth doula at no cost, printed materials about having a healthy pregnancy, and community resources. Feasibility and acceptability will be assessed at the end of the program. Measures will be obtained at baseline (20-28 weeks), the 36th week of pregnancy, birth, and 6-12 weeks post partum. Summary statistics and distribution plots will be used to describe measured variables at each time point. A generalized linear mixed model with a shared random component will be used to analyze the effects of PARITY on clinical, wellness behavioral, and empowered strength outcomes, including baseline nutrition, physical activity, and sleep measures as covariates. For significant effects, post hoc contrasts will be adjusted using the Holm method to maintain comparison-wise error at or <.05. Missing data will be addressed using a pattern-mixture model. The National Institute of Nursing Research funded this pilot RCT. Recruitment, enrollment, and data collection are ongoing, and the estimated study completion date is October 2024. The expected results of this study will provide the feasibility and preliminary efficacy of the PARITY intervention, to be used in a larger trial with a 12-month PARITY program intervention. ClinicalTrials.gov NCT05802615; https://clinicaltrials.gov/study/NCT05802615. DERR1-10.2196/58580.

Acid sphingomyelinase deficiency in France: a retrospective survival study

BackgroundAcid sphingomyelinase deficiency (ASMD) or Niemann–Pick disease types A, A/B, and B is a progressive, life-limiting, autosomal recessive disorder caused by sphingomyelin phosphodiesterase 1 (SMPD1) gene mutations. There is a need to increase the understanding of morbidity and mortality across children to adults diagnosed with ASMD.MethodsThis observational retrospective survey analysed medical records of patients with ASMD with retrievable data from 27 hospitals in France, diagnosed/followed up between 1st January 1990 and 31st December 2020. Eligible records were abstracted to collect demographic, medical/developmental history, and mortality data. Survival outcomes were estimated from birth until death using Kaplan–Meier survival analyses; standardised mortality ratio (SMR) was also explored.ResultsA total of 118 medical records of patients with ASMD (type B [n = 94], type A [n = 15], and type A/B [n = 9]) were assessed. The majority of patients were males (63.6%); the median [range] age at diagnosis was 8.0 [1.0–18.0] months (type A), 1.0 [0–3] year (type A/B), and 5.5 [0–73] years (type B). Overall, 30 patients were deceased at the study completion date; the median [range] age at death for patients with ASMD type A (n = 14) was 1 [0–3.6] year, type A/B (n = 6) was 8.5 [3.0–30.9] years, and type B (n = 10) was 57.6 [3.4–74.1] years. The median [95% confidence interval (CI)] survival age from birth in patients with ASMD type A and type A/B was 2.0 [1.8–2.7] years and 11.4 [5.5–18.5] years, respectively. Survival analysis in ASMD type B was explored using SMR [95% CI] analysis (3.5 [1.6–5.9]), which showed that age-specific deaths in the ASMD type B population were 3.5 times more frequent than those in the general French population. The causes of death were mostly severe progressive neurodegeneration (type A: 16.7%), cancer (type B: 16.7%), or unspecified (across groups: 33.3%).ConclusionsThis study illustrated a substantial burden of illness with high mortality rates in patients with ASMD, including adults with ASMD type B, in France.

Characteristics of effective parent-mediated interventions for parents of children with neurodevelopmental disorders in rural areas: a systematic review protocol

IntroductionParent-mediated interventions are therapeutic approaches that use parent training to enable parents to provide primary support and intervention to their child through the development of necessary skills, knowledge, and resources.Parent-mediated...