Project description:Until approximately 15 years ago, sponsors rarely included children in the development of therapeutics. US and European legislation has resulted in an increase in the number of pediatric trials and specific label changes and dosing recommendations, although infants remain an understudied group. The lack of clinical trials in children is partly due to specific challenges in conducting trials in this patient population. Therapeutics in special populations, including premature infants, obese children and children receiving extracorporeal life support, are even less studied. National research networks in Europe and the USA are beginning to address some of the gaps in pediatric therapeutics using novel clinical trial designs. Recent innovations in pediatric clinical trial design, including sparse and scavenged sampling, population pharmacokinetic analyses and 'opportunistic' studies, have addressed some of the historical challenges associated with clinical trials in children.
Project description:BackgroundCost is a barrier to creating educational resources, and new educational initiatives are often limited in distribution. Medical training programs must develop strategies to create and implement cost-effective educational programming.ObjectiveWe developed high-quality medical programming in procedural instruction with efficient economics, reaching the most trainees at the lowest cost.MethodsThe Just-In-Time online procedural program was developed at the University of Toronto in Canada, aiming to teach thoracentesis, paracentesis, and lumbar puncture skills to internal medicine trainees. Commercial vendors quoted between CAD $50,000 and $100,000 to create 3 comprehensive e-learning procedural modules-a cost that was prohibitive. Modules were therefore developed internally, utilizing 4 principles aimed at decreasing costs while creating efficiencies: targeting talent, finding value abroad, open source expansion, and extrapolating efficiency.ResultsProcedural modules for thoracentesis, paracentesis, and lumbar puncture were created for a total cost of CAD $1,200, less than 3% of the anticipated cost in utilizing traditional commercial vendors. From November 2016 until October 2018, 1800 online instructional sessions have occurred, with over 3600 pageviews of content utilized. While half of the instructional sessions occurred within the city of Toronto, utilization was documented in 10 other cities across Canada.ConclusionsThe Just-in-Time online instructional program successfully created 3 procedural modules at a fraction of the anticipated cost and appeared acceptable to residents based on website utilization.
Project description:BackgroundCapacity-building initiatives targeting medical personnel in low- and middle-income countries have focused on individual medical professionals within specific disciplines. However, it has become evident that the enhancement of healthcare quality necessitates more than just individual capacity-building efforts. As a part of The National Radiotherapy and Medical Oncology Center Project in Côte d'Ivoire, the National Cancer Center Korea has initiated a training project for hospital personnel in Côte d'Ivoire.MethodsFrom May to November 2023, 51 hospital personnel from 21 disciplines from Côte d'Ivoire underwent invitation-based training in Korea. A quantitative analysis of the training outcomes for the trainees and a qualitative assessment of the enablers of the training, involving trainees, trainers, and the coordinating team, were conducted.ResultsThe average score at pre- and post-training evaluation of capabilities of the trainees increased from 2.3 to 4.0 (out of 5.0). In addition to medical knowledge and technical skills sharing, trainees collaborated in group activities to develop workflow manuals for disease-, discipline-, and department-specific tasks through a multidisciplinary and cross-functional approach. The qualitative assessment indicated the importance of contextually relevant curriculum development with a special focus on establishing hospital operational plans, maintaining trainee autonomy and motivation, building mutual trust and cooperation between partner countries, engaging stakeholders actively, and providing effective leadership and support structures through a dedicated coordinating team were crucial factors for program success.ConclusionsEffective hospital personnel capacity building programs should not only focus on imparting medical knowledge and technical skills but also actively engage trainees in developing hospital operational plans. Furthermore, ensuring mutual trust and cooperation among various stakeholders, along with the establishment of a dedicated coordinating team, is essential for the success of the program.
Project description:Human inducible pluripotent stem cell (hiPSC)-derived astrocytes (iAs) are critical to study astrocytes in health and disease. They provide several advantages over human fetal astrocytes in research, which include consistency, availability, disease modeling, customization, and ethical considerations. The generation of iAs is hampered by the requirement of Matrigel matrix coating for survival and proliferation. We provide a protocol demonstrating that human iAs cultured in the absence of Matrigel are viable and proliferative. Further, through a side-by-side comparison of cultures with and without Matrigel, we show significant similarities in astrocyte-specific profiling, including morphology (shape and structure), phenotype (cell-specific markers), genotype (transcriptional expression), metabolic (respiration), and functional aspects (glutamate uptake and cytokine response). In addition, we report that, unlike other CNS cell types, such as neuronal progenitor cells and neurons, iAs can withstand the absence of Matrigel coating. Our study demonstrates that Matrigel is dispensable for the culture of human iPSC-derived astrocytes, facilitating an easy, streamlined, and cost-effective method of generating these cells.
Project description:BackgroundIn some current healthcare settings, there is a noticeable absence of national institutions committed to the synthesis and use of evidence in healthcare decision- and policy-making. This absence creates a need to broaden the responsibilities of healthcare providers to include knowledge brokering and advocacy in order to optimize knowledge translation to other stakeholders, especially policy-makers. However, this process requires practitioners and researchers to acquire certain types of knowledge and skills. This article introduces two innovative methods for capacity building in knowledge translation (KT).MethodsDuring a workshop aimed at preparing 21 trainers in evidence-based medicine, two innovative methods were used: (1) debate and (2) a knowledge translation project (KTP). The main objective of the debates approach was to strengthen participants' critical thinking abilities by requiring them to search for and appraise evidence and defend their arguments. The KTP was used to introduce participants to the essential steps of knowledge translation and to suggest an extended role for healthcare practitioners, i.e., using evidence to manage not only individual patients but also to a community of patients. Participants' performances were assessed according to a pre-designed scheme. At the end of the workshop, participants' opinions and experiences with the innovative teaching methods were evaluated based on their answers to a questionnaire and the results of small-group discussions.ResultsThe participants performed well in both the debate and KTP methods. During post-workshop group discussions, they indicated that the debate approach had added a new dimension to their evidence-based medicine skills by adding purpose and motivation. However, they felt that their performances would have been better if they had been offered practical demonstrations of how to conduct the debate. The participants indicated that the KTP enhanced their understanding of the relationships between evidence and implementation, and motivated them to investigate public health problems in addition to individual patient problems. However, some participants maintained that these issues fell outside the scope of their role as doctors.ConclusionDebates and evidence implementation through KTP are generally well accepted by healthcare practitioners as methods by which they can improve their skills in KT.
Project description:Small cell lung cancer (SCLC) is an aggressive, complex disease with a distinct biology that contributes to its poor prognosis. Management of SCLC is still widely limited to chemotherapy and radiation therapy, and research recruitment still poses a considerable challenge. Here, we review the current standard of care for SCLC and advances made in utilizing immunotherapy. We also highlight research in the development of targeted therapies and emphasize the importance of a team-based approach to make clinical advances. Building an integrative network between an academic site and community practice sites optimizes biomarker and drug target discovery for managing and treating a difficult disease like SCLC.
Project description:Accurate and efficient molecular recognition plays a crucial role in the fields of molecular detection and diagnostics. Conventional trial-and-error-based molecular recognition approaches have always been challenged in distinguishing minimal differences between targets and non-targets, such as single nucleotide polymorphisms (SNPs) of oligonucleotides. To address these challenges, here, a novel concept of dynamic addressing analysis is proposed. In this concept, by dissecting the regions of the target and creating a corresponding recognizer, it is possible to eliminate the inaccuracy and inefficiency of recognition. To achieve this concept, a Dynamic Addressing Molecular Robot (DAMR), a DNA-based dynamic addressing device is developed which is capable of dynamically locating targets. DAMR is designed to first bind to the conserved region of the target while addressing the specific region dynamically until accurate recognition is achieved. DAMR has provided an approach for analyzing low-resolution targets and has been used for analyzing SNP of miR-196a2 in both cell and serum samples, which has opened new avenues for effective and efficient molecular recognition.
Project description:In China, the focus of drug research and development has gradually shifted from generic to innovative drugs. Using the Chinese Clinical Trials Registry and Information Transparency Platform, we retrospectively analyzed clinical trials of innovative pediatric drugs conducted in mainland China over the last decade. The goal of this work was to better understand the characteristics of and historical changes in innovative pediatric drug research and development (R&D) in China and to provide effective data support for policy makers and other stakeholders. This study included 198 innovative pediatric drug clinical trials. The data showed that, although some progress has been made in the R&D of innovative pediatric drugs in China, many factors limiting this progress still exist, such as concentrated R&D areas, inadequate pediatric participants, and unbalanced source distributions. The level of innovative pediatric drug R&D in China currently lags behind the global level and has not kept pace with anti-neoplastic drug R&D in China. To promote the innovative development of pediatric drugs in China, the Chinese government must develop an R&D supervision framework, improve the motivation and innovation capabilities of pharmaceutical companies, and optimize the source distribution between regions.
Project description:BackgroundHome parenteral nutrition (HPN) is a life-sustaining therapy for short bowel syndrome (SBS) and other severe digestive diseases, but complications are common. We evaluated a predischarge HPN hands-on training course to reduce complications in children with SBS, including hospital readmissions.MethodsWe conducted a prospective, nonrandomized controlled research study between April 1, 2014, and April 30, 2017. Eligible participants were children aged <18 years old with SBS and anticipated HPN dependence duration ≥6 months. Excluded participants had a previous history of discharge with a central venous catheter (CVC), HPN, or intravenous fluids or strictly palliative goals of care. An intervention group practiced hands-on HPN within the hospital room for 24 hours using infusion equipment. The groups received standard teaching (CVC care, home infusion pump operation, HPN preparation and administration).ResultsNine children were assigned to the intervention group and 12 served as controls. The median age was 8.4 months, and length of stay (LOS) was 82 days. All participants experienced ≥1 event, with a total of 47 issues related to HPN. There were no significant associations between group assignment and 30-day postdischarge events. Each additional week of LOS was associated with 11% increase in the odds of an emergency department visit (OR 1.11; 95% CI, 1.01-1.26) and 16% increase in the odds of readmission (OR 1.16; 95% CI, 1.04-1.37).ConclusionsPostdischarge events remained widespread despite HPN bedside interventions offered by this pilot intervention. With refinement of HPN discharge processes, quality benchmarks are needed.