Adaptive multi-interventional trial platform to improve patient care for fibrotic interstitial lung diseases.

BACKGROUND: Fibrotic interstitial lung diseases (fILDs) are a heterogeneous group of lung diseases associated with significant morbidity and mortality. Despite a large increase in the number of clinical trials in the last 10 years, current regulatory-approved management approaches are limited to two therapies that prevent the progression of fibrosis. The drug development pipeline is long and there is an urgent need to accelerate this process. This manuscript introduces the concept and design of an innovative research approach to drug development in fILD: a global Randomised Embedded Multifactorial Adaptive Platform in fILD (REMAP-ILD). METHODS: Description of the REMAP-ILD concept and design: the specific terminology, design characteristics (multifactorial, adaptive features, statistical approach), target population, interventions, outcomes, mission and values, and organisational structure. RESULTS: The target population will be adult patients with fILD, and the primary outcome will be a disease progression model incorporating forced vital capacity and mortality over 12 months. Responsive adaptive randomisation, prespecified thresholds for success and futility will be used to assess the effectiveness and safety of interventions. REMAP-ILD embraces the core values of diversity, equity, and inclusion for patients and researchers, and prioritises an open-science approach to data sharing and dissemination of results. CONCLUSION: By using an innovative and efficient adaptive multi-interventional trial platform design, we aim to accelerate and improve care for patients with fILD. Through worldwide collaboration, novel analytical methodology and pragmatic trial delivery, REMAP-ILD aims to overcome major limitations associated with conventional randomised controlled trial approaches to rapidly improve the care of people living with fILD.

Relación entre Síntomas de Trastornos Témporomandibulares Auto-Reportados y el Movimiento Mandibular de Sujetos Adultos Jóvenes / Relationship Between Symptoms of Self-Reported Temporomandibular Disorders and Mandibular Movement of Young Adult Subjects

RESUMEN: Tanto la limitación del movimiento mandibular como el auto reporte de síntomas de trastornos témporomandibulares (TTM) son utilizados como elementos para el diagnóstico de pacientes en la clínica. Sin embargo, la relación entre la presencia de síntomas y el movimiento mandibular no está del todo clara. Por ello, el objetivo de este estudio fue relacionar la presencia de síntomas asociados a TTM con el movimiento mandibular en una muestra de sujetos adultos jóvenes y sanos. En este estudio exploratorio participaron 40 estudiantes de Odontología, médicamente sanos, con arco dentario maxilar y mandibular continuo y completo (hasta el primer molar). A cada uno se les solicitó responder un cuestionario de auto-reporte de síntomas asociados a TTM recomendado por la American Academy of Orofacial Pain (AAOP). También se realizó un registro y análisis de sus movimientos mandibulares utilizando articulografía electromagnética 3D, con el objetivo de correlacionar la presencia de síntomas con el rango y trayectoria de apertura, y el área comprendida en lo que se describe como polígono de Posselt en el plano frontal y sagital. No se encontraron diferencias significativas entre las respuestas a cada pregunta entre hombres y mujeres. De acuerdo a las respuestas y según Delcanho (1994), 12 participantes resultaron necesitar una evaluación más exhaustiva. En este grupo se encontró que el área del polígono de Posselt en el plano frontal, fue significativamente menor que el de aquellos participantes que no requieren una evaluación más exhaustiva (p=0,003). Los valores de la trayectoria de apertura mandibular varían según de que síntoma se trate. Los principales síntomas que afectarían los valores de los movimientos bordeantes son la "dificultad para abrir la boca" y el "ruido en las articulaciones mandibulares". Con este estudio, hemos puesto de manifiesto que la limitación del movimiento mandibular como signo de TTM debe evaluarse con cautela, debido a que según la presencia de determinados síntomas y otras características de los sujetos (como el sexo), su valor podría variar.

ABSTRACT: Both the limitation of mandibular movement and the self-report of symptoms of temporomandibular disorders (TMD) are used for the diagnosis of patients in clinical diagnosis. However, the relationship between the symptoms and mandibular movement is not entirely clear. Therefore, the objective of this study was to relate the presence of symptoms associated with TMD with mandibular movement in a sample of young and healthy adult subjects. This exploratory study involved 40 medically healthy dentistry students with a continuous and complete maxillary and mandibular arch (up to the first molar). Each subject was asked to answer a self-report questionnaire for symptoms associated with TTM recommended by the American Academy of Orofacial Pain (AAOP). We also recorded and analyzed their mandibular movements using 3D electromagnetic articulography, with the aim of correlating the presence of symptoms with the range and trajectory of opening, and the area included in what is described as a polygon of Posselt in the frontal and sagittal plane. No significant differences were found between the answers to each question between men and women. According to the responses and to Delcanho, 12 participants turned out to need a more exhaustive evaluation. In this group it was found that the area of the Posselt polygon in the frontal plane was significantly smaller than that of those participants who did not require a more extensive evaluation (p = 0.003). The values of the mandibular opening path vary depending on the symptom involved. The main symptoms that would affect the values of the bordering movements are the "difficulty to open the mouth" and "noise in the mandibular joints". With this study, we have shown that the limitation of mandibular movement as a sign of TMD should be assessed with caution, as, according to the presence of certain symptoms and other characteristics of subjects (such as sex), its value may vary.