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

Informations de copyright

© 2023 Rizio et al.

Déclaration de conflit d'intérêts

Avery A. Rizio is an employee of QualityMetric Incorporated, LLC and reports research funding from Amyloidosis Research Consortium. Michelle K. White is an employee of QualityMetric Incorporated, LLC. Anita D’Souza reports research funding from the National Heart, Lung, and Blood Institute (K23 HL141445, R01 HL166339); institutional research funding from AbbVie, Caelum, Celgene, Janssen, Novartis, Takeda, Sanofi, and TeneoBio; Advisory boards of Bristol Myers Squibb, Pfizer, and Janssen; Consultant for BMS/Celgene, Janssen and Prothena. Paula Schmitt reports personal fees from Alnylam Pharmaceuticals, outside the submitted work. Tiffany P. Quock is an employee of Prothena Biosciences Inc. and holds stock in Prothena Corporation plc group. James Signorovitch is an employee of Analysis Group Inc., which has received research funding from ARC. Vaishali Sanchorawala reports research funding from Prothena, Caelum, Millennium-Takeda, Takeda, Celgene, Janssen, Karyopharm, Sorrento, Oncopeptide, Alexion; Advisory boards for AbbVie, Proclara, Prothena, Caelum, Janssen, Regeneron, Protego, Pharmatrace, Telix, Alexion/AstraZeneca; Consultant for Pfizer, Janssen. The authors report no other conflicts of interest in this work.