Clinical Research Studies Research Articles

Retinal Patterns and the Role of Autofluorescence in Choroideremia

Background: Choroideremia is a monogenic inherited retinal dystrophy that manifests in males with night blindness, progressive loss of peripheral vision, and ultimately profound sight loss, commonly by middle age. It is caused by genetic defects of the CHM gene, which result in a deficiency in Rab-escort protein-1, a key element for intracellular trafficking of vesicles, including those carrying melanin. As choroideremia primarily affects the retinal pigment epithelium, fundus autofluorescence, which focuses on the fluorescent properties of pigments within the retina, is an established imaging modality used for the assessment and monitoring of affected patients. Methods and Results: In this manuscript, we demonstrate the use of both short-wavelength blue and near-infrared autofluorescence and how these imaging modalities reveal distinct disease patterns in choroideremia. In addition, we show how these structural measurements relate to retinal functional measures, namely microperimetry, and discuss the potential role of these retinal imaging modalities in clinical practice and research studies. Moreover, we discuss the mechanisms underlying retinal autofluorescence patterns by imaging with a particular focus on melanin pigment. Conclusions: This could be of particular significance given the current progress in therapeutic options, including gene replacement therapy.

From Processed Foods to Ultraprocessed Foods: Evolution of an Industry Model and Impact on Dietary Quality, Health, and Society.

The impact of food technology and product development on the nutritional quality of foods is discussed in the context of food classification schemes, clinical research, and sociocultural studies. Food processing operations are analyzed in terms of their beneficial and detrimental consequences for the nutritional value of foods and ingredients. Several classification schemes are discussed, including dietary guidelines, nutrition information panels, and nutritional scores. The health impact of processed and ultraprocessed foods is discussed in connection with the processing-formulation scheme previously developed by the authors. The importance of product development as a driver for the food industry is highlighted, and formulation-based approaches to improve the healthfulness of industrially produced foods are discussed. Finally, the public perception of processed foods and its impact on the industry are discussed, and the need for a broad engagement among stakeholders to ensure the sustainability of our food system and healthy diets for individuals is emphasized.

Gerontologic Biostatistics and Data Science: Aging Research in the Era of Big Data.

Introduced in 2010, the sub-discipline of gerontologic biostatistics was conceptualized to address the specific challenges of analyzing data from clinical research studies involving older adults. Since then, the evolving technological landscape has led to a proliferation of advancements in biostatistics and other data sciences that have significantly influenced the practice of gerontologic research, including studies beyond the clinic. Data science is the field at the intersection of statistics and computer science, and although the term "data science" was not widely used in 2010, the field has quickly made palpable impacts on gerontologic research. In this Review in Depth, we describe multiple advancements of biostatistics and data science that have been particularly impactful. Moreover, we propose the sub-discipline of "gerontologic biostatistics and data science", or GBDS, which subsumes gerontologic biostatistics into a more encompassing practice. Prominent GBDS advancements that we discuss herein include cutting-edge methods in experimental design and causal inference, adaptations of machine learning, the rigorous quantification of deep phenotypic measurement, and analysis of high-dimensional -omics data. We additionally describe the need for integration of information from multiple studies and propose strategies to foster reproducibility, replicability, and open science. Lastly, we provide information on software resources for GBDS practitioners to apply these approaches to their own work and propose areas where further advancement is needed. The methodological topics reviewed here aim to enhance data-rich research on aging and foster the next generation of gerontologic researchers.

Plain language review: the safety of relugolix combination therapy for advanced prostate cancer.

Advanced prostate cancer is a cancer that began in the prostate (a part of the male body) and has spread to other parts of the body. This is a review of two clinical research studies of patients with advanced prostate cancer who were treated with relugolix combination therapy. Relugolix is a medicine taken by mouth that lowers a male sex hormone, called testosterone. Relugolix is sometimes combined with other medicines such as novel hormonal therapies (NHTs) or chemotherapy to treat advanced prostate cancer. In one study, patients were treated with relugolix combined with an NHT (abiraterone or apalutamide). In a second study, patients were treated with relugolix combined with an NHT (enzalutamide) or chemotherapy (docetaxel). Researchers wanted to understand what possible side effects may happen due to taking these medicines together as prescribed. They also wanted to see if relugolix combination therapy worked to lower testosterone in the same way as relugolix taken alone. Researchers found that most of the side effects of relugolix combined with an NHT or chemotherapy were mild or moderate. Side effects of relugolix combination therapy were similar to the side effects of the medicines when taken alone. However, patients who received relugolix with enzalutamide or docetaxel were more likely to have a serious side effect compared with patients who received relugolix taken alone. Testosterone stayed below 50 nanograms per deciliter (known as castration levels) for patients who received relugolix with NHT or chemotherapy. Patients who receive relugolix combination therapy generally experience mild or moderate side effects, rather than serious side effects. No new safety issues were found during these studies. Patients maintained low testosterone levels. Patients and their doctors should discuss the benefits and possible harms of relugolix combination therapy to treat advanced prostate cancer.

Consideration of the EXiLE test for predicting anaphylaxis after diclofenac etalhyaluronate administration

Diclofenac etalhyaluronate, an active pharmaceutical ingredient in JOYCLU® (JCL), serves as a joint function improvement agent in knee and hip osteoarthritis patients. However, frequent cases of anaphylaxis induced by JCL administration have been reported. Recent clinical research suggests the potential utility of the basophil activation test (BAT) in predicting JCL-induced anaphylaxis. Nonetheless, the BAT is deemed impractical for routine diagnostic testing due to complex procedures involving whole blood stimulation and flow cytometry-based analyses. In the study reported here, an IgE crosslinking-induced luciferase expression (EXiLE) test which uses patient sera without complicated procedures, was performed with patients who had received JCL, with or without subsequent anaphylactic symptoms. The results of this test were then compared with those of the BAT reported in a clinical research study. Of the six BAT-positive JCL-induced anaphylaxis-experienced patients, four were positive in the EXiLE test and all non-experienced patients were negative in both the BAT and EXiLE tests, thus illustrating a high concordance rate of 92.3%. Further validation of testing conditions is expected to improve these rates. Notably, complement inactivation treatment led to a positive EXiLE result in a BAT-negative patient. In conclusion, it appears that the EXiLE test exhibits promise as an alternative to BAT for predicting JCL-induced anaphylaxis, and in so doing offers a simpler diagnostic approach.

Vital pulp therapy: A bibliometric analysis of the 100 most-cited articles from 1966 to 2022

Introduction: Vital pulp therapy (VPT) aims to preserve dental pulp after injury and has gained significant popularity due to advancements in materials and understanding of pulp biology. While bibliometric analyses are common in various fields, none have been conducted specifically for the 100 most-cited articles on VPT. Materials and Methods: This bibliometric study analyzed the 100 most-cited VPT papers using data from Web of Science (WoS-CC), Scopus, and Google Scholar. Articles were manually reviewed to extract key bibliometric data, including titles, citation counts, citation density, authorship, country of origin, publication year, journal, study design, and VPT type. Results: The articles collectively received 19,782 citations, with the oldest from 1966 and the most recent from 2019, having 803 and 104 citations, respectively. Key journals included the Journal of Endodontics, International Endodontic Journal, and Dental Materials. Review articles were the predominant study design, followed by clinical, laboratory, and basic research studies. Eight of the top 20 articles focused on the properties of mineral trioxide aggregate (MTA). Torabinejad and Parirokh were notable contributors before 2010, while Duncan, Bjørndal, and Simon were prominent after 2010. VPT research has increased significantly post-2010, with a notable shift in focus from early studies on calcium hydroxide to more recent studies on MTA’s biocompatibility and its use as a pulp capping agent. Conclusion: This analysis provides a comprehensive list of the top 100 cited VPT articles, offering valuable insights for researchers, students, and clinicians. The study highlights key trends and influential contributions in the field of VPT research.

Analytical measuring interval, linearity, and precision of serology assays for detection of SARS-CoV-2 antibodies according to CLSI guidelines.

Reliable and validated serology assays are of increasing importance as the severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) virus continues to evolve and cause outbreaks. Validation of serology assays along with calibration to the International and National Standards (such as anti-SARS-CoV-2 Immunoglobulin WHO International Standard 20/136 or Frederick National Laboratory for Cancer Research's National Serology Standard COVID-NS01097) is critical to ensuring that results from clinical studies are reliable and comparable among various assays and laboratories. We describe the design and execution of a comprehensive study that established the analytical measuring intervals, linearity, precision, and repeatability of four in-house developed serology enzyme-linked immunosorbent assays (SARS-CoV-2 anti-Spike immunoglobin G [IgG] and immunoglobin M [IgM] and anti-Nucleocapsid IgG and IgM) following applicable Clinical and Laboratory Standards Institute (CLSI) guidelines. Overall, this study provides practical guidance on experimental design strategies and data analysis techniques, pertaining to the validation of COVID-19 serology assays according to CLSI guidelines, for use in clinical research studies.

A qualitative study on experiences and perceptions of screening for enrolment in HIV clinical research among volunteers in Kenya

INTRODUCTION Screening volunteers to determine their eligibility to enrol in clinical research is an important phase in clinical research. However, little is known about how volunteers view and experience this phase of research implementation. This study explored volunteers’ perceptions and experiences of screening for enrolment into HIV clinical research studies. MATERIALS AND METHODS A qualitative study was conducted with 44 research participants purposively selected from a sample of 164 participants drawn from six research studies at the Kenya Aids Vaccine Initiative-Institute of Clinical Research (KAVI-ICR) in Nairobi, Kenya. Data was collected between March and June 2014, through in-depth interviews that were audio recorded and transcribed verbatim. Data were managed and thematically analyzed using the Atlas ti software. RESULTS Participants expressed mixed views and experiences about screening. A majority had initial fears about HIV testing and being screened for possible chronic diseases. Discomfort with physical examination, amounts of blood collected and associated pain were reported. On a positive note, participants were appreciative of the free comprehensive screening, and confirmations of being in good health. Those found with minor ailments reported receiving treatment before enrolment. HIV risk reduction behaviours following post-test counselling were also reported by some. CONCLUSIONS Evaluating participants’ experiences of screening for enrolment is important for the design of research that meets ethical requirements and responds to research participants’ fears and concerns for optimal enrolment and retention.

Best Evidence Summary for Management of Older People With Type 2 Diabetes Mellitus Using 'Internet Plus Nursing Services'.

To evaluate and summarise the evidence for the management of older people with Type 2 diabetes mellitus (T2DM) using 'Internet Plus nursing services (IPNS)'. This study was conducted as an evidence summary, adhering strictly to the evidence summary reporting standards established by Fudan University Center for Evidence-based Nursing. We systematically searched for the best available evidence pertaining to the management of older people with T2DM using the IPNS. The literature types encompassed clinical guidelines, expert consensuses, systematic reviews, evidence summaries and original research studies. In order to gather pertinent information, we conducted a comprehensive search across various databases, including UpToDate, BMJ Best Practice, Joanna Briggs Institute, Guidelines International Network, National Institute for Health and Care Excellence, Registered Nurses Association of Ontario, Scottish Intercollegiate Guidelines Network, the Cochrane Library, PubMed, Web of Science, Yi Maitong Guidelines Network, SinoMed, CNKI, WanFang database, Chinese Biomedical Literature Database and China Science and Technology Journal Database. The search spanned from the inception of each database up to July 2023, ensuring an extensive coverage of relevant resources. After rigorous screening and evaluation, our study ultimately identified 19 articles with high-quality research outcomes. These articles consisted of three guidelines, two expert consensus documents, three systematic reviews and eleven original research studies. Through collaborative and in-depth discussions, we extracted and synthesised 27 pieces of evidence related to the application of the IPNS to enhance the T2DM management for older people. We categorised the evidence into five primary themes: mobile terminal design, team building, health education design, interaction and social support, and information feedback. In designing the IPNS for older people with T2DM, utmost attention should be paid to the intricacies of Internet module development preceded by comprehensive guidance. It is imperative to establish multidisciplinary teams to oversee the curation of patient educational content, ensuring its relevance and effectiveness. Leveraging Internet-based information feedback mechanisms is crucial for fostering peer support, assisting in blood glucose control, enhancing self-management capabilities and ultimately improving overall quality of life. Healthcare professionals are supposed to customise the IPNS for this vulnerable population, taking into account institutional resources and local contextual realities. It is highly recommended that clinical medical personnel worldwide adhere to the evidence-based recommendations for better care of older people with T2DM using the IPNS. No patient or public contribution was incorporated in this study. Our study provides structured ideas for the application of IPNS in T2DM management, provides practicable new ways for IPNS to improve self-management of older people with T2DM, and encourages the provision of Internet-based nursing services based on the needs and characteristics of older people, and jointly solves the challenges of T2DM management in order to enhance the effectiveness of nursing services.

Researcher and partner perspectives on the relationship between engagement in research and three uses of patient-centered comparative clinical effectiveness research study findings

BackgroundThe uptake of research findings into clinical practice is critical to providing health care that improves health outcomes for patients. This study explored how Patient-Centered Outcomes Research Institute (PCORI) awardees perceive the relationship between engagement of patients and other partners in research and three uses, or applications, of patient-centered comparative clinical effectiveness research (CER) study findings, which may lead to uptake in clinical practice: (1) Integration into clinical practice guidelines, recognized point-of-care decision tools, or documents that may inform policy; (2) Implementation beyond the study, including at sites outside of the study setting or patient populations; and (3) Active dissemination of findings to specific audiences by parties external to the study team.MethodsThis exploratory qualitative study examined awardee and partner perceptions of what led to each use of study findings and how engaged partners contributed. We purposively selected PCORI-funded research projects with documentation of each use and conducted virtual interviews with 42 individuals (15 PIs or project leads, 2 research team members, and 25 partners) from 17 projects. We conducted thematic analysis of individual projects or project sets, across projects within each use case, and across the three uses.ResultsParticipants described three primary activities in which engaged partners made contributions before, during and after CER studies that facilitated the use of study findings: (1) generating relevant study findings, (2) distributing study findings strategically, and (3) making connections to people or organizations outside the study team. In addition, engagement continued to facilitate the use of study findings during subsequent PCORI-funded implementation and dissemination-specific projects, with partners adapting interventions and creating and tailoring dissemination messages and products. Finally, participants described attributes of teams’ engagement approaches that may have supported partner contributions, including early and ongoing engagement, leveraging partners’ connections and understanding of community needs, and using multiple engagement approaches.ConclusionThis study identified examples of how engagement can help facilitate the use of CER study findings, especially when engagement contributions occur in meaningful ways. Findings from this study suggest a framework for future research on the relationship between engagement in research and uptake of study findings into clinical practice.

Comprehensive analysis of clinical research profiles and study characteristics of oral potentially malignant disorders: an observational study

ObjectiveThe study aims to present an observational study of clinical trials on Oral Potentially Malignant Disorders (OPMDs) and corresponding publications. Study design and settingWe searched the OPMDs-related clinical studies registered in the ClinicalTrials.gov database before March 1, 2024. Subsequently, we investigated the publication status of primary completed studies using PubMed and Google Scholar. ResultsA total of 185 studies were identified for analysis, including 141 interventional studies and 44 observational studies. The most commonly studied disease type was oral lichen planus (OLP), accounting for 113 studies (61.1 %). Interventional studies on OPMDs were predominantly early-phased, blind, randomized, parallel and single-center. The primary purpose of these studies was treatment (106, 75.2 %). Among the treatments, herbal medicine (21, 19.8 %), photodynamic therapy (17, 16.0 %), and glucocorticoids (14, 13.2 %) were of greatest interest. In addition, 58 (63.0 %) of the primarily completed interventional studies were published, with a median time to publication of 33.6 months. Published interventional studies including OLP patients and factorial designs had shorter time to publication. However, these studies were less frequently published in high-impact journals and most primary results were positive. ConclusionsClinical trials on OPMDs predominantly focused on OLP and herbal medicine. The quality of studies is unsatisfactory and publication rate is suboptimal. Improvements are needed in ClinicalTrials.gov registration standards, high-quality study design and more stringent publishing requirements.

Exposure of antral follicles to medroxyprogesterone acetate during stimulation does not cause molecular perturbations in gonadotropin-responsiveness and steroidogenic function of granulosa cells in progestin-primed cycles.

Does medroxyprogesterone acetate (MPA) exposure in progestin-primed ovarian stimulation (PPOS) cycles cause molecular perturbations in the steroidogenic function and gonadotropin responsiveness of the granulosa cells? PPOS cycles are identical to traditional GnRH antagonist cycles not only for clinical IVF characteristics but also for gonadotropin receptor expression, response to gonadotropins, and steroidogenic function at the molecular level. PPOS is increasingly used as an alternative to GnRH antagonists due to the inhibitory effect of progesterone on LH release by reducing GnRH pulsatility at the hypothalamic level. Although a growing body of evidence from clinical studies did not indicate significant differences between PPOS and antagonist protocols for IVF cycle characteristics and obstetrical outcomes, it is still unknown whether exposure of the antral follicle cohort to progesterone or its synthetic derivatives during ovarian stimulation causes any subtle molecular aberrations in terms of steroidogenesis and gonadotropin responsiveness. To address this issue, detailed comparative molecular analyses were conducted in the luteinized mural granulosa cells (GCs) obtained from normal responding IVF patients undergoing PPOS and antagonist cycles. A clinical translational research study was conducted with IVF patients. This study included 55 normal responding IVF patients who underwent ovarian stimulation with either PPOS using MPA (5 mg twice daily) or GnRH antagonist cetrorelix acetate. Recombinant forms of FSH and hCG were used for ovarian stimulation and ovulation triggering, respectively. Luteinized mural GCs obtained during the oocyte retrieval procedure were used for the experiments. Cell culture, quantitative real-time PCR, immunoblotting, confocal time-lapse live cell imaging, and hormone assays were used. Demographic and IVF cycle characteristics of the patients undergoing ovarian stimulation with PPOS and GnRH antagonist were similar, including ovarian response, mature oocyte yield, and fertilization rates. Molecular analyses revealed that the expression of the enzymes involved in sex-steroid synthesis (StAR, SCC, 3β-HSD, 17β-HSD, aromatase) and the uptake/storage/utilization of cholesterol (LDL receptor, Hormone-sensitive lipase, hydroxy-methyl glutaryl Co-enzyme-A reductase, and Sterol O-acyltransferase1) in the GCs of the PPOS cycles were comparable to those of the antagonist cycles. The expression of the receptors for gonadotropins, estrogen, and progesterone hormones was also similar. Basal and hCG-induced increases in 3β-HSD expression and progesterone production and basal and FSH-induced increases in aromatase expression and E2 output of the GCs from PPOS patients did not exhibit any meaningful differences when compared with GCs from antagonist cycles. Furthermore, basal and hCG-induced up-regulation in the LDL receptor expression and cholesterol uptake did not differ between the groups. Confocal imaging also revealed similar patterns of expression for the steroidogenic enzymes and their co-localization with mitochondria. Lastly, the expression of the other important genes regulating cumulus expansion, ovulation, and luteal function [Relaxin, ADAMTS-1, and epidermal growth factor (EGF)-like growth factor amphiregulin] in the GCs of the PPOS and antagonist cycles were similar. N/A. Caution should be exercised when interpreting our data which was derived from normally responding patients whose ovulation was triggered with hCG. It is unclear whether the molecular parameters assessed vary according to infertility etiologies, magnitude of ovarian response, mode of trigger, and any other underlying ovarian pathologies or systemic diseases. MPA was the progestin used for PPOS and whether these findings can be generalized to other progestins is unknown. This study provides reassuring molecular evidence that exposure of antral follicle cohorts to MPA during the follicular growth phase does not have any detrimental effects on steroidogenic, ovulatory, and luteal functions when compared with GnRH antagonist cycles. This study was funded by the School of Medicine, the Graduate School of Health Sciences of Koc University and Koç University Research Center for Translational Medicine (KUTTAM), and equally funded by the Republic of Turkey Ministry of Development Research Infrastructure Support Program. All authors declare no conflict of interest. N/A.

A feasibility trial of olanzapine for young people with Anorexia Nervosa (OPEN): clinicians’ perspectives

BackgroundThe OPEN feasibility trial testing olanzapine in anorexia nervosa (AN) in young people (YP) was not successful due to poor recruitment. This study aims to understand clinicians’ views and experiences of using olanzapine in AN and the challenges in implementing the trial in National Health Service (NHS) clinical settings.MethodsWe conducted qualitative interviews with eating disorders (ED) clinicians involved with the study (n = 11). Framework analysis was applied to qualitative data to identify barriers and facilitators to recruitment and study implementation. A web-based semi-structured Qualtrics survey was administered to ED clinicians (n = 24). Findings from the survey were used to corroborate and expand on the information derived from qualitative interviews.ResultsQualitative analysis identified four main themes: (1) Acknowledging Service User (SU) / Family Concerns, (2) Prioritising person-centred care, (3) Limited Service Capacity and (4) Study eligibility criteria. Subthemes are outlined accordingly. Clinicians appeared confident addressing SU concerns around olanzapine in clinical discussions, but timing was critical, and olanzapine was considered one aspect of treatment that needed to align with their holistic approach. Service pressures restricted opportunities for recruitment and the ability to offer regular review. At the same time, some YP were ineligible for the trial, as they were already taking olanzapine, or needed to be prescribed it more promptly than the study procedures allowed. Survey findings underlined confidence in prescribing and informing on olanzapine, the various possible benefits of olanzapine besides weight gain, and the importance of therapeutic alliances and informed consent. Both data sets highlight the need for further evidence on long-term safety, side effects and efficacy of olanzapine use for AN. Where clinical service capacity is at a premium, research implementation is not prioritised, particularly in intensive clinical settings.ConclusionsFindings provide first-hand insight into individual and systemic challenges with research implementation in the NHS, which need to be considered when designing future clinical research studies. We emphasise a person-centred approach when discussing olanzapine to consider a holistic recovery from AN beyond weight-gain as an isolated outcome for improvement.

Boosting efficiency in a clinical literature surveillance system with LightGBM.

Given the suboptimal performance of Boolean searching to identify methodologically sound and clinically relevant studies in large bibliographic databases, exploring machine learning (ML) to efficiently classify studies is warranted. To boost the efficiency of a literature surveillance program, we used a large internationally recognized dataset of articles tagged for methodological rigor and applied an automated ML approach to train and test binary classification models to predict the probability of clinical research articles being of high methodologic quality. We trained over 12,000 models on a dataset of titles and abstracts of 97,805 articles indexed in PubMed from 2012-2018 which were manually appraised for rigor by highly trained research associates and rated for clinical relevancy by practicing clinicians. As the dataset is unbalanced, with more articles that do not meet the criteria for rigor, we used the unbalanced dataset and over- and under-sampled datasets. Models that maintained sensitivity for high rigor at 99% and maximized specificity were selected and tested in a retrospective set of 30,424 articles from 2020 and validated prospectively in a blinded study of 5253 articles. The final selected algorithm, combining a LightGBM (gradient boosting machine) model trained in each dataset, maintained high sensitivity and achieved 57% specificity in the retrospective validation test and 53% in the prospective study. The number of articles needed to read to find one that met appraisal criteria was 3.68 (95% CI 3.52 to 3.85) in the prospective study, compared with 4.63 (95% CI 4.50 to 4.77) when relying only on Boolean searching. Gradient-boosting ML models reduced the work required to classify high quality clinical research studies by 45%, improving the efficiency of literature surveillance and subsequent dissemination to clinicians and other evidence users.

Abstract B120: Understanding Barriers and Facilitators Affecting Participation in Cancer Clinical Research Studies for Racially and Ethnically Diverse Populations in Rural North Carolina

Abstract Increasing representation of minoritized racial and ethnic populations in cancer clinical research studies has been declared a priority in the US for many years. Additionally, it is widely recognized that people living in rural areas of the US have limited access to cancer clinical research studies, and solutions are needed to extend participation opportunities to rural cancer patients. Underrepresentation of minoritized racial and ethnic populations and rural communities perpetuates health inequities, and solutions are needed to remove barriers and implement facilitators for participation. However, a review of existing literature found a lack of attention on the intersection of race and rurality and the needs and values of these populations as it pertains to cancer research. To address this concern, we used a sequential mixed method, community-engaged approach to explore the barriers and facilitators affecting participation in cancer clinical research from the perspectives of cancer survivors and their caregivers, as well as cancer healthcare professionals in rural North Carolina (NC). We used existing publicly available data to identify and characterize four of the most rural, racially and ethnically diverse, and medically underserved counties in NC. The principal investigator collaborated with cancer centers, churches, and American Indian tribal leaders in NC to administer surveys to cancer survivors and caregivers to assess their beliefs and experiences regarding cancer clinical research. Eligible participants included persons who had been diagnosed with cancer or a caregiver of someone diagnosed with cancer. In addition, we conducted key informant interviews with cancer health care professionals providing cancer care in the selected rural NC counties. We analyzed surveys and interview responses with descriptive statistics. Forty-three cancer survivors/caregivers completed surveys and twenty-two cancer healthcare professionals completed semi-structured interviews. Cancer survivors and caregivers indicated that the lack of invitation to participate in cancer clinical research was their greatest barrier to participation, whereas cancer healthcare professionals cited the lack of transportation as the perceived greatest barrier for patients. Cancer survivors, caregivers and healthcare professionals agreed that having support from clinical research personnel would be amongst the most helpful facilitators, along with opportunities to participate in clinical research studies close to home. Cancer healthcare professionals suggested the lack of trust in clinical research was a barrier for their patients; however, cancer survivors and caregivers in this study indicated lack of trust was not a significant barrier to their participation. Recommendations to address barriers and promote facilitators to participation in cancer clinical research include the importance of engaging the community, addressing unconscious bias, decentralizing clinical trials, and providing recruitment materials in culturally responsive formats. Citation Format: Amy Garrett, Karine Dubé, Lisa Spees, Ronny Bell, Marjory Charlot, Sandra Greene. Understanding Barriers and Facilitators Affecting Participation in Cancer Clinical Research Studies for Racially and Ethnically Diverse Populations in Rural North Carolina [abstract]. In: Proceedings of the 17th AACR Conference on the Science of Cancer Health Disparities in Racial/Ethnic Minorities and the Medically Underserved; 2024 Sep 21-24; Los Angeles, CA. Philadelphia (PA): AACR; Cancer Epidemiol Biomarkers Prev 2024;33(9 Suppl):Abstract nr B120.

Abstract B119: Evaluation of a navigator training program to increase clinical trials and genomic research participation among adult African Americans and Hispanics residing in the Southern United States

Abstract Introduction: Patient Navigators (PNs) have have proven to be effective along the cancer care continuum. However, emerging data suggest that the scope of patient navigation should be expanded to include other contexts such as the recruitment of racial/ethnic minorities for cancer clinical trials. Because the processes of recruitment and retention are often linked, the same barriers hindering minority recruitment often hinder minority retention. The potential benefits of studying generalizability and statistical power for subgroup analyses may be lost with disproportionately high attrition among the enrolled minorities. The dual focus of the training curriculum on both recruitment and retention distinguishes it from the few previous efforts focused on the traditional PN model for minority recruitment in clinical trials and genomic research studies. Methods: A two-phased navigator training program was implemented. Components of the training program was adapted in collaboration, with the Joint Cancer Advisory Council and Bioethics Co-Leaders, from the existing UAB IMPACT Patient Navigation Training Curriculum. A systematic literature review will be conducted to identify the most salient genomic educational and resource needs and concerns of AAs and Hispanic populations regarding participation in genomic-focused research and clinical trials. These findings informed the adaptation of the training curriculum based on principles of adult education. Results: We conducted a joint training for the Clinical Trials and Genomic Navigators at each Partnership institution (MSM, TU, UAB) for the proposed project. The Co-Leaders from the Partnership Outreach Cores, the Bioethics Shared Resource, and the Joint Cancer Advisory Council planned and assigned responsibilities and dates for completion of the training activities. The initial training was the GW Navigator On-line Training Program. A supplemental training was held at one of the partnering sites, a safety-net hospital (Grady Cancer Center for Excellence). The in-person training featured an overview presentation on Genetic Risk Assessment in Oncology by a licensed genetic counselor, followed by a case study approach to enrollment and the implementation of a research study with training materials such as tablets provided by the training conductors. An evaluation was completed by the team for training conductors. The third training was held at Tuskegee University and ttopics included the importance of consent training. For this presentation, we will present the qualitative and quantitative findings. Conclusions: The training outcomes were standardized and included input from the Bioethics Co-Leaders and JCAR members. We measured changes over time (pre vs. posttest) in: 1) knowledge and skills of clinical trials and genomic research; 2) ability to identify barriers and solutions to participation in research, 3) ability to implement the two toolkits, and 4) level of ease in identifying open protocol research studies and clinical trials. Citation Format: Osato Eke, Makeeta Rayton, Teresa Hall, Lleana Barrios-Zermeno, Jennier Culbertson, Angela Morris, Vivian Carter, Yu-Mei Schoenberger, Victoria Churchill, Desiree Rivers, Stephen Sodeke, Mohamed Mubasher, Roland Matthews, Brian Rivers. Evaluation of a navigator training program to increase clinical trials and genomic research participation among adult African Americans and Hispanics residing in the Southern United States [abstract]. In: Proceedings of the 17th AACR Conference on the Science of Cancer Health Disparities in Racial/Ethnic Minorities and the Medically Underserved; 2024 Sep 21-24; Los Angeles, CA. Philadelphia (PA): AACR; Cancer Epidemiol Biomarkers Prev 2024;33(9 Suppl):Abstract nr B119.

Patient and family views on research priorities and design of clinical trials and research studies in pediatric multiple sclerosis.

This survey study aimed to (1) identify patient/family research priorities in pediatric-onset multiple sclerosis (POMS), and (2) delineate optimized methods for research study/clinical trials design, engagement, and implementation. Participants were as follows: (1) parents of a child (<18 years) with POMS enrolled in a national registry, (2) adolescents (13-17 years) with POMS in the registry, and (3) adults (18-40 years) with POMS receiving care at a registry affiliated clinic. Of 293 eligible participants, 192 completed surveys. Experiences with health care and medications were generally positive but there remain areas of priority improvement. Incentives to participate in clinical trials included medications previously tested and in pill form, bloodwork/study visits required ⩾ every 3 months, cognitive testing ⩽1 hour, compensation for travel and time, ability to continue current multiple sclerosis (MS) medication, option to take study medication if on placebo, and individualized study feedback. Priorities for clinical research were (1) psychosocial impact, (2) cognitive/academic impact, (3) environmental risk, and (4) nutrition. Results highlighted the importance of a holistic approach to study design and a focus on the impact of disease on daily life to best engage patients and families in POMS clinical trials and research.

The application of cell products for the treatment of critical limb ischemia in patients with diabetes mellitus: a review of the literature

The number of patients with diabetes mellitus (DM) has been progressively increasing worldwide over the past decades, and many international organizations consider DM as a public health emergency of the 21st century.Critical limb ischemia (CLI) is the most severe stage of peripheral arterial disease (PAD) in DM and is characterized by a high risk of limb loss without revascularization. Traditional treatment tactics include open and endovascular revascularization surgical techniques. However, in patients not eligible for revascularization and in cases where performed surgical treatment performed has been ineffective, there are almost no therapeutic alternatives, often leading to amputations and death. As of today, one of the newest non-surgical treatment options is cell therapy. Among different cells, mesenchymal stromal cells (MSCs) are potentially one of the most prospective for use in this patient population.This article provides an overview of clinical trials using cell therapy in patients with CLI.To analyze publications, electronic databases PubMed, SCOPUS, ClinicalTrials, and ScienceDirect were searched to identify published data from clinical trials, research studies, and review articles on cell therapy for critical lower extremity ischemia. After the search, 489 results were received.As a result of systematic selection, 22 clinical trials were analyzed.According to the analyzed literature data, the use of cell products in this category of patients is effective and safe. Cell therapy can stimulate the formation of new vessels and enhances collateral circulation; it is also reported improved distal perfusion, increased pain-free walking distance, decreased amputation rates, and increased survival rates.Nevertheless, further study of the potential use of this category of drugs is needed.

Development of a core outcome set of clinical research on the integration of traditional Chinese and Western medicine for spinal metastases: a study protocol

BackgroundIn recent years, the incidence of spinal metastasis (SM) has been increasing steadily. In response to this serious public health problem, researchers have made progress by using the integration of...

Conduct of Large, Multisite, Comparative Clinical Effectiveness Research Studies: Learnings From the Patient-Centered Outcomes Research Institute's Palliative Care Learning Network.

Conduct of Large, Multisite, Comparative Clinical Effectiveness Research Studies: Learnings From the Patient-Centered Outcomes Research Institute's Palliative Care Learning Network.