AI Article Synopsis
- Systemic AL amyloidosis is a rare disorder caused by misfolded proteins that affects multiple organs, leading to serious health issues.
- The Amyloidosis Forum, a collaboration between researchers and the FDA, aims to speed up the development of treatments for this condition through focused working groups addressing clinical trial outcomes.
- The HRQOL Working Group has reviewed existing patient-reported outcome tools and recommends using the SF-36v2 and PROMIS-29 to assess health-related quality of life in clinical trials, while emphasizing the need for future research on their effectiveness.
Article Abstract
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.
Download full-text PDF |
Source |
|---|---|
| http://www.ncbi.nlm.nih.gov/pmc/articles/PMC10202704 | PMC |
| http://dx.doi.org/10.2147/PROM.S399658 | DOI Listing |
Publication Analysis
Top Keywords
Similar Publications
Post COVID-19 infection and quality of life of healthcare workers at Sahloul University Hospital of Sousse in Tunisia.
Qual Life Res
January 2025
Occupational Medicine Department, University Hospital Sahloul, Sousse, Tunisia.
Background: Since the COVID-19 pandemic, health care workers (HCWs) faced an enormous physical and mental burden, sometimes altering their quality of life due mainly to persistent challenges stemming from their frontline position.
Aims: Todetermine the prevalence of post-COVID-19 syndrome, and its impact on the Health-Related Quality of Life (HRQoL) among HCWs.
Methods: This is an exhaustive cross-sectional study with analytical scope, conducted among all HCWs of the University Hospital Sahloul of Sousse, Tunisia, who have contracted COVID-19 between September 2020 and 30 March 2021 (N=529 cases).
Global prevalence and moderating factors of malnutrition in colorectal cancer survivors: A meta-analysis.
J Cancer Surviv
January 2025
School of Nursing, College of Nursing, Taipei Medical University, Taipei, Taiwan.
Purpose: This meta-analysis aims to estimate the global prevalence of severe, moderate, overall malnutrition and moderating factors of malnutrition in colorectal cancer (CRC) survivors.
Methods: A comprehensive search was conducted in Embase, CINAHL, Medline-OVID, PubMed, Scopus, and Web of Science from inception to February 8, 2024, without language, region, or publication date restrictions. A generalized linear mixed model and random-effects model were used to examine the pooled prevalence, and moderator analyses were implemented to investigate variations in the pooled prevalence.
Baricitinib Provides Significant Improvements in Quality of Life and Functioning in Adults with Moderate-to-Severe Atopic Dermatitis with Baseline Body Surface Area ≤ 40% and Severe Itch.
Dermatol Ther (Heidelb)
January 2025
Department of Dermatology, University of Tsukuba, Tsukuba, Japan.
Introduction: Patients with moderate-to-severe atopic dermatitis (AD), a body surface area (BSA) of ≤ 40%, and an itch numerical rating scale (NRS) score of ≥ 7 ("BARI itch dominant") have been characterized as an important group to consider for the oral janus kinase (JAK) 1/2 inhibitor baricitinib (BARI). Herein we aim to evaluate quality of life (QoL) and functioning outcomes in adult patients with BSA ≤ 40% and itch NRS ≥ 7 at baseline (BL) who received BARI 4 mg in the topical corticosteroid (TCS) combination trial BREEZE-AD7.
Materials: BREEZE-AD7 was a randomized, double-blind, placebo-controlled, parallel-group outpatient study involving adult patients with moderate-to-severe AD who received once-daily placebo or 2-mg or 4-mg BARI in combination with TCS for 16 weeks.
Executive function profiles in survivors of neonatal critical illness - a latent profile analysis in school-aged children born very preterm or with complex congenital heart disease and in typically developing peers.
Child Neuropsychol
January 2025
Child Development Center, University Children's Hospital Zurich, Zurich, Switzerland.
Executive function (EF) impairments are prevalent in survivors of neonatal critical illness such as children born very preterm (VPT) or with complex congenital heart disease (cCHD). This paper aimed to describe EF profiles in school-aged children born VPT or with cCHD and in typically developing peers, to identify child-specific and family-environmental factors associated with these profiles and to explore links to everyday-life outcomes. Data from eight EF tests assessing working memory, inhibition, cognitive flexibility, switching, and planning in = 529 children aged between 7 and 16 years was subjected into a latent profile analysis.
View Article and Find Full Text PDFSelf-Management in Children and Adolescents With Chronic Illness: An Evolutionary Analysis of the Concept.
J Adv Nurs
January 2025
Faculty of Health Sciences and Nursing, Center for Interdisciplinary Research in Health (CIIS), Universidade Católica Portuguesa, Porto, Portugal.
Aim: To increase conceptual clarity regarding the self-management of school-age children and adolescents with chronic illnesses in a community context.
Design: Concept Analysis: Rodgers' evolutionary approach.
Data Sources: Search conducted in the Cumulative Index to Nursing and Allied Health Literature, Psychology and Behavioural Sciences Collection, Nursing and Allied Health Collection, Academic Search Complete, Cochrane, Web of Science, Medical Literature Analysis and Retrieval System Online, Scopus, Repositório Científico de Acesso Aberto de Portugal, ProQuest Dissertations and Theses, Joanna Briggs Institute Evidence Synthesis.
Want AI Summaries of new PubMed Abstracts delivered to your In-box?
Enter search terms and have AI summaries delivered each week - change queries or unsubscribe any time!