Barriers to diverse clinical trial participation in Duchenne muscular dystrophy: Engaging Hispanic/Latina caregivers and health professionals.

BACKGROUND: Despite the increasing availability of clinical trials in Duchenne muscular dystrophy, racial/ethnic minorities and other populations facing health disparities remain underrepresented in clinical trials evaluating products for Duchenne. We sought to understand the barriers faced by Hispanic/Latino families specifically and underrepresented groups more generally to clinical trial participation in Duchenne. METHODS: We engaged two participant groups: Hispanic/Latino caregivers of children with Duchenne in the US, including Puerto Rico, and health professionals within the broader US Duchenne community. Caregiver interviews explored attitudes towards and experiences with clinical trials, while professional interviews explored barriers to clinical trial participation among socio-demographically underrepresented families (e.g., low income, rural, racial/ethnic minority, etc.). Interviews were analyzed aggregately and using a thematic analysis approach. An advisory group was engaged throughout the course of the study to inform design, conduct, and interpretation of findings generated from interviews. RESULTS: Thirty interviews were conducted, including with 12 Hispanic/Latina caregivers and 18 professionals. We identified barriers to clinical trial participation at various stages of the enrollment process. In the initial identification of patients, barriers included lack of awareness about trials and clinical trial locations at clinics that were less likely to serve diverse patients. In the prescreening process, barriers included ineligibility, anticipated non-compliance in clinical trial protocols, and language discrimination. In screening, barriers included concerns about characteristics of the trial, as well as mistrust/lack of trust. In consent and recruitment, barriers included lack of timely decision support, logistical factors (distance, time, money), and lack of translated study materials. CONCLUSIONS: Numerous barriers hinder participation in Duchenne clinical trials for Hispanic/Latino families and other populations experiencing health disparities. Addressing these barriers necessitates interventions across multiple stages of the clinical trial enrollment process. Recommendations to enhance participation opportunities include developing clinical trial decision support tools, translating prominent clinical trials educational resources such as ClinicalTrials.gov, fostering trusting family-provider relationships, engaging families in clinical trial design, and establishing ethical guidelines for pre-screening potentially non-compliant patients.

Bowel bladder dysfunction in control children in a pediatric urology office.

INTRODUCTION: Bowel bladder dysfunction (BBD) is common in children. Risk factors for BBD include age, gender, obesity, and behavioral issues such as ADHD. We investigated the modified Swedish Bowel-Bladder questionnaire as a sensitive indicator of BBD in healthy children. OBJECTIVES: We tested the usefulness of the Swedish Bowel-Bladder Questionnaire (BBQ) as an indicator of BBD in children not complaining of bowel/bladder dysfunction at the time of their visit to a pediatric urologist. Our secondary aim was to identify correlations between BBQ scores and risk factors such as gender, BMI, and ADHD. STUDY DESIGN: All families in our Pediatric Urology practice with patients >30 months old who were reportedly toilet trained were provided the Swedish BBQ. Total score as well as sub-scores for storage, emptying, and constipation were prospectively collected. Presenting diagnosis, gender, BMI, and ADHD history were collected. BBQ scores for patients with voiding dysfunction were compared to controls: a) those with genital problems (e.g. hydrocele/undescended testes), b) those with CAKUT (congenital anomalies of the kidneys and urinary tract). BBQ scores were analyzed as a continuous variable vs the potential risk factors (ADHD, obesity, age, and gender) using univariable/multivariable regression analysis. RESULTS: The median BBQ score for the 328 control patients (95 CAKUT and 233 genital) was 2.25 with an IQR: 1 to 6. In contrast, the median BBQ was higher for those with possible voiding dysfunction; n = 282; 9 with an IQR: 5 to 15). Total BBQ score exceeded 6 in 16% (52/328) of control patients. On multivariable analysis, age-adjusted total BBQ scores increased with ADHD in our controls (p = 0.03) but were unaffected by gender or BMI. On multivariable analysis of the voiding dysfunction group, total BBQ scores similarly decreased with age (p < 0.001) and increased with ADHD (p < 0.001) and were affected by gender (p = 0.024). BMI percentile had no significant effect in either cohort. DISCUSSION AND CONCLUSION: The Swedish BBQ was used in a U.S population and demonstrated trends towards increased voiding dysfunction associated with younger age, female gender, and ADHD. We learned that voiding dysfunction in children with ADHD is not associated with constipation and improves over time. Additionally, 16% of children with genital or urinary abnormalities and no voiding complaints have noticeable BBD uncovered by the BBQ. Hence, we conclude that the Swedish BBQ is a sensitive indicator of BBD even in children not complaining of those problems.

Dolor al administrar medicamento por inyección intramuscular: ventroglútea vs dorsoglútea / Pain in the administration of drugs by intramuscular injection: ventrogluteal vs dorsogluteal / Dor na administração intramuscular de drogas: ventroglúteo vs dorsogluteal

ResumenIntroducción. La inyección intramuscular es una técnica que causa dolor e incomodidad a las personas. Con el objetivo de determinar la zona de punción menos dolorosa para la administración de medicamentos intramusculares en glúteo, se realizó una revisión de literatura en busca de la evidencia de mayor calidad. Método. Se empleó el método propuesto por el enfoque de la Práctica Clínica de Enfermería Basada en la Evidencia. Se recopiló estudios de EBSCOhost, Google, Google Académico, Cochrane Library y PubMed. Se aplicó los filtros propuestos para la selección: se sometió los dos artículos seleccionados a lectura crítica mediante la plataforma FLC 2.0 con la cual se valoró la calidad de los estudios. El riesgo de sesgo se valoró con The Cochrane Collaboration's tool for assessing risk of bias. A través del sistema de clasificación GRADE se determinó la calidad de la evidencia y la fuerza de la recomendación de la intervención.Resultado. Los artículos incluidos en el estudio respondieron parcialmente a la pregunta clínica planteada. La percepción de dolor durante la administración de inyectables en el sitio ventroglúteo es menor que en el dorsoglúteo, los hallazgos tuvieron un nivel de evidencia moderado y la fuerza de recomendación fue 1B.Conclusión. No hay evidencia contundente para preferir la elección del sitio ventroglúteo sobre el dorsoglúteo para disminuir el dolor asociado a la administración intramuscular de analgésicos antiinflamatorios, aunque el primer sitio posee mayores ventajas en comparación con el segundo.

AbstractIntroduction. Intramuscular injection is a technique that causes pain and discomfort to people. In order to determine the least painful puncture site for the administration of intramuscular gluteal medications, a literature review was conducted in search of the highest quality evidence.Method.The method used in this research was the Evidence-Based Nursing approach. The studies were collected from EBSCOhost, Google, Google Scholar, Cochrane Library and PubMed. Filters of the selection criteria were applied to the papers and then were subjected to critical appraisal using the web platform FLC 2.0 leaving 2 articles remaining. The Cochrane Collaboration's tool for assessing risk of bias was applied and afterwards the GRADE approach was used to determine the quality of the evidence and strength of recommendation.Result. Papers included in the study responded partially the clinical question posed. The ventrogluteal IM injection site seems to be less painful than in the dorsogluteal site during the administration of analgesic antiinflammatory drugs, the findings had a moderate level of evidence and strength of recommendation was 1B. Conclusion. No strong evidence was found to prefer the ventrogluteal site rather than the dorsogluteal site in order to reduce pain associated with intramuscular administration of anti-inflammatory analgesics drugs. Although the first injection site has major advantages compared to the second

ResumoIntrodução: A injeção intramuscular é uma técnica que causa dor e desconforto para as pessoas. Com o objetivo de determinar o local da punção menos doloroso para a administração de medicamentos intramusculares no glúteo, realizou-se uma revisão de literatura em busca de evidência de maior qualidade.Materiais e método: Empregou-se o método proposto pelo enfoque de Prática Clínica de Enfermagem Baseada em Evidência. Realizaram-se estudos de EBSCOhost, Google, Google Acadêmico, Cochrane Library e PubMed. Aplicaram-se os filtros propostos para a seleção: foram submetidos dois artigos selecionados para a leitura crítica mediante a plataforma FLC 2.0 com a qual se estabeleceu um valor para a qualidade dos estudos. O risco de inclinação se determina com The Cochrane Collaboration's tool for assessing risk of bias. Através do sistema de classificação GRADE se determinou a qualidade de evidência e a necessidade da recomendação da intervenção. Resultados: Os artigos incluídos no estudo responderam parcialmente a pergunta clínica planteada. A percepção da dor durante a administração de injetáveis no local ventroglúteo é menor que no dorsoglúteo, as descobertas tiveram um nível de evidência moderado e a necessidade de recomendação foi 1B.Conclusões:Não há evidência contundente para preferir a escolha do local ventroglúteo sobre o dorsoglúteo para diminuir a dor associada a administração intramuscular de analgésicos antiinflamatórios, embora o primeiro local possua maiores vantagens em comparação com o segundo.

Site-Specific Fluorescence Polarization for Studying the Disaggregation of α-Synuclein Fibrils by Small Molecules.

Fibrillar aggregates of the protein α-synuclein (αS) are one of the hallmarks of Parkinson's disease. Here, we show that measuring the fluorescence polarization (FP) of labels at several sites on αS allows one to monitor changes in the local dynamics of the protein after binding to micelles or vesicles, and during fibril formation. Most significantly, these site-specific FP measurements provide insight into structural remodeling of αS fibrils by small molecules and have the potential for use in moderate-throughput screens to identify small molecules that could be used to treat Parkinson's disease.

La anestesia epidural: sus principales complicaciones (revisión bibliográfica) / Epidural anesthesia: its main complications (bibliographical revision)

La anestesia epidural es una técnica que ha adquirido mucho auge recientemente para diferentes tipos de cirugía; por su gran versatilidad y por presentar menos riesgos para el paciente. Sin embargo, existen gran cantidad de complicaciones tanto técnicas como clínicas que deben valorarse e informarse al paciente antes de seleccionar este método. Se debe siempre poner en una balanza el riesgo-beneficio que conlleva esta técnia para cada paciente. De esta forma se puede evitar que sufran complicaciones las cuales aunque no son muy frecuentes si pueden llegar a ser devastadoras; como por ejemplo: cuando se produce un bloqueo completo con pérdida de fuerza muscular que ocasionaría un paro respiratorio, un hematoma o absceso epidural, complicaciones en la labor de parto, alteraciones neurológicas entre otras.