UNASSIGNED: Impaired quality of life (QOL) is common in patients with inflammatory bowel disease (IBD). A tool to more quickly identify IBD patients at high risk of impaired QOL improves opportunities for earlier intervention and improves long-term prognosis. The purpose of this study was to use a machine learning (ML) approach to develop risk stratification models for evaluating IBD-related QOL impairments.
UNASSIGNED: An online questionnaire was used to collect clinical data on 2478 IBD patients from 42 hospitals distributed across 22 provinces in China from September 2021 to May 2022. Eight ML models used to predict the risk of IBD-related QOL impairments were developed and validated. Model performance was evaluated using a set of indexes and the best ML model was explained using a Local Interpretable Model-Agnostic Explanations (LIME) algorithm.
UNASSIGNED: The support vector machine (SVM) classifier algorithm-based model outperformed other ML models with an area under the receiver operating characteristic curve (AUC) and an accuracy of 0.80 and 0.71, respectively. The feature importance calculated by the SVM classifier algorithm revealed that glucocorticoid use, anxiety, abdominal pain, sleep disorders, and more severe disease contributed to a higher risk of impaired QOL, while longer disease course and the use of biological agents and immunosuppressants were associated with a lower risk.
UNASSIGNED: An ML approach for assessing IBD-related QOL impairments is feasible and effective. This mechanism is a promising tool for gastroenterologists to identify IBD patients at high risk of impaired QOL.

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OBJECTIVE: Good Clinical Practices (GCP) are essential for patient-centric research. The standard bioethics and GCP training emphasizing a \"one-size-fits-all\" approach may not adequately equip ethics committee members, especially the lay and social scientist members, towards their critical role in reviewing clinical trials and related documentation. This article explores a patient-centered, patient advocates-driven training program focused on raising awareness about research ethics and GCP among patients, advocates and ethics committee members.
METHODS: A patient advocates-driven program called Patient Advocates for Clinical Research (PACER) conducted trainings focused on GCP for patient-centric research for patients, advocates and ethics committee members. Pre- and post-workshop questionnaires were used to assess the participants\' knowledge of GCP.
RESULTS: The workshop was attended by 116 participants. Of these 91 consented to participate in questionnaire evaluation that assessed participants\' knowledge on ethics committee (EC) functionality, research ethics and data confidentiality. Pre-workshop evaluations highlighted knowledge gaps. Only 16.5% were familiar with the primary ethical consideration for vulnerable populations and 69.2% were knowledgeable about data governance. Post-workshop evaluations demonstrated significant overall response improvement of 5.4% (𝜒2=13.890; p<0.001). The understanding of ethical considerations for vulnerable populations rose by 15.4% (p=0.007), and knowledge of data privacy regulations improved by 11.0% (p=0.041).
CONCLUSIONS: The workshop under PACER initiative highlighted the knowledge gaps in understanding the EC functionality, research ethics and data confidentiality. The workshop effectively fostered participants\' understanding of ethical research practices.

Gene editing is a growing gene engineering technique that allows accurate editing of a broad spectrum of gene-regulated diseases to achieve curative treatment and also has the potential to be used as an adjunct to the conventional treatment of diseases. Gene editing technology, mainly based on clustered regularly interspaced palindromic repeats (CRISPR)-CRISPR-associated protein systems, which is capable of generating genetic modifications in somatic cells, provides a promising new strategy for gene therapy for a wide range of human diseases. Currently, gene editing technology shows great application prospects in a variety of human diseases, not only in therapeutic potential but also in the construction of animal models of human diseases. This paper describes the application of gene editing technology in hematological diseases, solid tumors, immune disorders, ophthalmological diseases, and metabolic diseases; focuses on the therapeutic strategies of gene editing technology in sickle cell disease; provides an overview of the role of gene editing technology in the construction of animal models of human diseases; and discusses the limitations of gene editing technology in the treatment of diseases, which is intended to provide an important reference for the applications of gene editing technology in the human disease.

UNASSIGNED: Cardiac arrest (CA) is a common event in the intensive care unit (ICU), which seriously threatens the prognosis of patients. Therefore, it is crucial to determine a simple and effective clinical indicator to judge the prognosis of patients after a CA for later treatments. The purpose of this study was to investigate the relationship between the lactate dehydrogenase to albumin ratio (LAR) and the prognosis of patients after a CA.
UNASSIGNED: The clinical data of participants was obtained from the Medical Information Mart for Intensive Care IV (MIMIC-IV, v2.0; 2008 to 2019). According to the 30-day prognosis, patients were divided into a survivors group (n = 216) and a non-survivors group (n = 304). The optimal LAR threshold was determined using restricted cubic spline (RCS), which divided patients into a high LAR group ( ≥ 15.50, n = 257) and a low LAR group ( < 15.50, n = 263). The ICU hospitalization and 30-day accumulative survival curves of the two groups were plotted following the Kaplan-Meier survival analysis. Multivariate Cox regression was used to analyze the relationship between the LAR and the prognosis of CA patients. Receiver operating characteristic (ROC) curves were drawn to evaluate the predictive efficacy of the LAR on 30-day all-cause mortality, and sensitivity analysis was used to check the reliability of the findings.
UNASSIGNED: A total of 520 patients with CA were enrolled and the 30-day mortality was 58.46%. The LAR in the non-survivors group was higher than in the survivors group. The RCS showed a linear trend relationship between the LAR and the mortality risk in patients during their ICU stay and 30 days; moreover, as the LAR increased, so did the risk of mortality. The Kaplan-Meier survival curve showed that compared with the low LAR group, the cumulative survival rates of ICU hospitalization and 30 days were lower in the high LAR group among CA patients (p < 0.001). Multivariate Cox regression analysis showed that an elevated LAR ( ≥ 15.50) was an independent risk factor for mortality during ICU stay and 30 days (p < 0.005). ROC analysis suggested that the LAR was superior to the sequential organ failure assessment (SOFA) score in predicting the 30-day all-cause mortality in CA patients (area under the curve (AUC) = 0.676, 95% confidence interval [CI]: 0.629-0.723). To verify the reliability of our findings, we performed sensitivity analyses and found that the findings were reliable.
UNASSIGNED: An elevated LAR might be a predictor of mortality in patients following a CA during ICU hospitalization and 30 days, thereby it can be used to provide a reference for the clinical management of these patients.

UNASSIGNED: Hidradenitis supprativa is a chronic inflammatory skin disease that can respond to treatment with laser hair removal.
UNASSIGNED: To assess alexandrite laser hair removal laser as a treatment for hidradenitis suppurativa as measured by the Hidradenitis Supprativa Clinical Response.
UNASSIGNED: We conducted a prospective, randomized, controlled study in adult patients with hidradenitis supprativa. Participants underwent a series of 4 monthly laser treatments to 1 side of the body, with the contralateral side serving as a control. The primary outcome was Hidradenitis Supprativa Clinical Response at week 24, 8 weeks after the final laser treatment.
UNASSIGNED: The percent improvement across treated sites after 4 treatments was 72.73% axillary, 70% inguinal, and 100% inframammary. Across all body regions, Hidradenitis Supprativa Clinical Response was significantly higher for the sites treated with the alexandrite laser compared to the contralateral controls: 75% vs 33.33% (P = .0046, 95% CI: [0.16, 1]).
UNASSIGNED: The limitations of this study include a small sample size from which the data was collected.
UNASSIGNED: The 775-nm alexandrite laser is a safe and effective treatment for hidradenitis supprativa at various anatomic sites in both resolving preexisting lesions and preventing new eruptions.

Imbalance and falls in patients with Parkinson\'s disease (PD) do not only reduce their quality of life but also their life expectancy. Aging-related symptoms as well as disease-specific motor and non-motor symptoms contribute to these conditions and should be treated when appropriate. In addition to an active lifestyle, advanced exercise training is useful and effective, especially for less medically responsive symptoms such as freezing of gait and postural instability at advanced stages. As treadmill training in non-immersive virtual reality, including dual tasks, significantly reduced the number of falls in PD patients, the mechanism(s) explaining this effect should be further investigated. Such research could help to select the most suitable patients and develop the most effective training protocols based on this novel technology. Real-life digital surrogate markers of mobility, such as those describing aspects of endurance, performance, and the complexity of specific movements, can further improve the quality of mobility assessment using wearables.

A quality management system for clinical and public health research operations is indispensable because it ensures the integrity and reliability of research outcomes. By implementing a robust quality management practice in research implementation and operation, research teams can uphold the highest standard of research conduct, thereby enhancing the credibility and trustworthiness of research findings. This paper elucidates the significance and role of a quality management system in clinical and public health research operations and its efficacy in minimising and eliminating protocol deviations and highlights the key steps in setting up a quality management system for research operations.

BACKGROUND: The integrity and reliability of clinical research outcomes rely heavily on access to vast amounts of data. However, the fragmented distribution of these data across multiple institutions, along with ethical and regulatory barriers, presents significant challenges to accessing relevant data. While federated learning offers a promising solution to leverage insights from fragmented data sets, its adoption faces hurdles due to implementation complexities, scalability issues, and inclusivity challenges.
OBJECTIVE: This paper introduces Federated Learning for Everyone (FL4E), an accessible framework facilitating multistakeholder collaboration in clinical research. It focuses on simplifying federated learning through an innovative ecosystem-based approach.
METHODS: The \"degree of federation\" is a fundamental concept of FL4E, allowing for flexible integration of federated and centralized learning models. This feature provides a customizable solution by enabling users to choose the level of data decentralization based on specific health care settings or project needs, making federated learning more adaptable and efficient. By using an ecosystem-based collaborative learning strategy, FL4E encourages a comprehensive platform for managing real-world data, enhancing collaboration and knowledge sharing among its stakeholders.
RESULTS: Evaluating FL4E\'s effectiveness using real-world health care data sets has highlighted its ecosystem-oriented and inclusive design. By applying hybrid models to 2 distinct analytical tasks-classification and survival analysis-within real-world settings, we have effectively measured the \"degree of federation\" across various contexts. These evaluations show that FL4E\'s hybrid models not only match the performance of fully federated models but also avoid the substantial overhead usually linked with these models. Achieving this balance greatly enhances collaborative initiatives and broadens the scope of analytical possibilities within the ecosystem.
CONCLUSIONS: FL4E represents a significant step forward in collaborative clinical research by merging the benefits of centralized and federated learning. Its modular ecosystem-based design and the \"degree of federation\" feature make it an inclusive, customizable framework suitable for a wide array of clinical research scenarios, promising to revolutionize the field through improved collaboration and data use. Detailed implementation and analyses are available on the associated GitHub repository.

UNASSIGNED: This study aimed to gain insights into pediatric clinical trials conducted in children\'s hospitals in China and provide valuable references for the development of children\'s hospitals and the research and development of pediatric drugs.
UNASSIGNED: A comprehensive search was performed on the Chinese Clinical Trial Registry (Chi CTR) and ChinaDrugTrials.org.cn to collect information on all clinical trials involving subjects under 18 years, including those conducted in children\'s hospitals. The retrieval period was extended until 31 December 2022.
UNASSIGNED: A total of 459 pediatric clinical trials were collected, comprising 299 from Chi CTR and 160 from the Drug Clinical Trial Registration and Information Publicity Platform (Information Platform). Post-marketing drug studies and phase III clinical trials accounted for the majority of research stages. These trials covered a wide range of diseases/systems, with a particular focus on respiratory system disorders, tumors, endocrine disorders, and nutritional or metabolic diseases. Chemical drugs constituted the most extensively studied category, while traditional Chinese medicine/natural drugs received comparatively less attention. Clinical trial activities were primarily geographically focused on the eastern coastal regions of China, with multicenter trials being the most predominant. Ethics committee approval was obtained for 427 studies.
UNASSIGNED: The pediatric clinical trials conducted by children\'s hospitals in China have shown an overall upward trend; however, there is limited research focusing on traditional Chinese medicine, along with significant regional and institutional imbalances. Furthermore, there is still room for improvement regarding ethical review processes. It is recommended that children\'s hospitals enhance their scientific research capabilities while optimizing resource allocation to meet medical service demands effectively. Additionally, fostering more research-focused children\'s hospitals will contribute to the high-quality development of children\'s health in China.