Health-Related Quality of Life Instruments for Clinical Trials in AL Amyloidosis: Report from the Amyloidosis Forum HRQOL Working Group.

Systemic AL (light chain) amyloidosis is a rare protein misfolding disorder associated with plasma cell dyscrasia affecting various organs leading to organ dysfunction and failure. The Amyloidosis Forum is a public-private partnership between the Amyloidosis Research Consortium and the US Food and Drug Administration Center for Drug Evaluation and Research with the goal of accelerating the development of effective treatments for AL amyloidosis. In recognition of this goal, 6 individual working groups were formed to identify and/or provide recommendations related to various aspects of patient-relevant clinical trial endpoints. This review summarizes the methods, findings, and recommendations of the Health-Related Quality of Life (HRQOL) Working Group. The HRQOL Working Group sought to identify existing patient-reported outcome (PRO) assessments of HRQOL for use in clinical trials and practice deemed relevant across a broad spectrum of patients with AL amyloidosis. A systematic review of the AL amyloidosis literature identified 1) additional signs/symptoms not currently part of an existing conceptual model, and 2) relevant PRO instruments used to measure HRQOL. The Working Group mapped content from each identified instrument to areas of impact in the conceptual model to determine which instrument(s) provide coverage of relevant concepts. The SF-36v2® Health Survey (SF-36v2; QualityMetric Incorporated, LLC) and Patient-Reported Outcomes Measurement Information System-29 Profile (PROMIS-29; HealthMeasures) were identified as instruments relevant to patients with AL amyloidosis. Existing evidence of reliability and validity was evaluated with a recommendation for future work focused on estimating clinically meaningful within-patient change thresholds for these instruments. For sponsors, the context of use-including specific research objectives, trial population, and investigational product under study-should inherently drive selection of the appropriate PRO instrument and endpoint definitions to detect meaningful change and enable patient-focused drug development.

A criança e a dança: observação clínica em grupo sobre o processo de individuação / Children and dance: a clinical observation in group about the process of individuation / El niño y la danza: observación clínica grupal sobre el proceso de individuación

O artigo analisa um conjunto de observação clínica de grupo de crianças,fundamentada na abordagem da Psicologia Analítica. Promove uma reflexão sobre adança como manifestação expressiva e criativa que propicia experiências significativasde aprendizado. Pressupõe um modelo educacional que visa despertar a potencialidadeda criança, desenvolver trabalhos corporais e inserir na educação aprendizados pormeio da vivência aliada à expressão livre. Investiga a percepção da criança a respeitodo ato de dançar. É desenvolvido um plano de trabalho denominado “Atividade de Dançae Expressão Livre” realizado em quatro encontros com oito crianças do ensinoFundamental. Os procedimentos constituem-se na aplicação de desenho com o tema “Oque é dançar para você” feito antes e depois das atividades de dança e uma entrevistacom cada criança, a fim de compreender a sua percepção sobre à dança. Os resultadosestimulam futuras pesquisas, posto que é observado que as crianças, além do prazer departicipar das atividades e dançar, expressam sentimentos de vergonha e de liberdade,de interagir com colegas e de ampliar a consciência de seu corpo. Há indícios deampliação de autopercepção corporal e de autoimagem, assim como maior percepçãode si e do outro. Conclui-se ser necessário efetivar pesquisa com maior número departicipantes, em grupo experimental e controle, para que o trabalho proposto possaconstituir um modelo de projeto a ser aplicado na educação formal(AU)

The article develops a clinical observation group based on an AnalyticalPsychological approach. The article promotes a reflection about dance as an expressiveand creative manifestation that allows meaningful experiences of learning. It refersto an educational model with the purpose of: awakening the potential of a child, developingbody work, and inserting in the child’s education the learning through body experienceconnected to the intellect. The objective is to investigate in the group the children’sperception about the act of dancing. A work plan called “Dance Activity and FreeExpression” is developed in four meetings with eight children from “Fundamental OneEducation”. The procedures involve the application of drawings before and after the dance activities with the theme “What is dancing for you”, and interviews with each oneof the children, in order to understand their perception related to dance. The resultsstimulate future researches since it was observed that the children, besides the pleasureof attending the activities and dance, exposed some feelings such as shame and freedom,they interacted with their classmates and broadened the awareness of the body. Therewas an indication of enlargement in body self perception and self image, and strongerperception of self and other. The conclusion shows the need of researches with abigger number of participants, experimental group and control group, in order to constructa model of project to be applied in formal education(AU)

Este artículo analiza un conjunto de observación clínica a un grupo deniños, basado en el enfoque de la Psicología Analítica. Promueve una reflexión sobrela danza como manifestación expresiva y creativa que ofrece experiencias significativasde aprendizaje. Presupone un modelo educativo que tiene como objetivo despertar elpotencial del niño, el desarrollar trabajos corporales e incluir en la educación,aprendizajes a través de la vivencia de la libre expresión. Se investiga la percepcióndel niño sobre el acto de bailar. Se desarrolla un plan de trabajo llamado “ Actividad dela Danza y la Libre Expresión”, realizado en cuatro reuniones con ocho niños delenseñanza primaria. Los procesos son constituidos por la aplicación del diseño con eltema “ ¿Qué es la danza para ti “ realizado antes y después de las actividades dedanza, así como una entrevista con cada niño participante, para poder entender supercepción acerca de la danza. Los resultados estimulan investigaciones futuras, yaque se observa que los niños, además del placer de participar en las actividades dedanza, expresan sentimientos de vergüenza y de libertad, de interactuar con suscompañeros y aumentar la conciencia de su cuerpo. Existe evidencia de la expansiónde la autopercepción corporal y de la autoimagen, así como una mayor conciencia desí mismo y del otro. Se concluye que es necesario realizar investigación con un númeromayor de participantes en grupos experimental y de control, para que el trabajopropuesto pueda se constituir un modelo a ser aplicado en la educación formal(AU)