ReviewPatient related outcome measures2023
Health-Related Quality of Life Instruments for Clinical Trials in AL Amyloidosis: Report from the Amyloidosis Forum HRQOL Working Group.
Review in Patient related outcome measures, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 13 papers, 1 of them a synthesis that pooled it.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
13 citing papers in PubMed, 1 synthesis or guideline pooled it, 14 citations in OpenAlex.
- The clinical trials landscape in immunoglobulin light chain amyloidosis: a systematic review.Blood advances · 2024Pooled it
- Disease burden of ATTR amyloidosis based on the SF-36Cardio-oncology (London, England) · 2026Article
- Patient-reported outcomes - the missing link to advancing light chain (AL) amyloidosis clinical research.Blood reviews · 2025Review
- Development of Imaging Endpoints for Clinical Trials in AL and ATTR Amyloidosis: Proceedings of the Amyloidosis Forum.JACC. Cardiovascular imaging · 2025Review
- Functional Status and Quality of Life in Light-Chain Amyloidosis: Advanced Imaging, Longitudinal Changes, and Outcomes.JACC. Heart failure · 2024Article
- Autologous stem cell transplantation in AL amyloidosis: Muddy waters.Blood reviews · 2024Review
- Renal Response Criteria for Clinical Trials in Amyloid Light Chain Amyloidosis.Kidney international reports · 2024Review
- Advancing Transthyretin Amyloidosis Drug Development in an Evolving Treatment Landscape: Amyloidosis Forum Meeting Proceedings.Advances in therapy · 2024Review
- Pathway for Development and Validation of Multi-domain Endpoints for Amyloid Light Chain (AL) Amyloidosis.Therapeutic innovation & regulatory science · 2024Review
- Development and initial validation of the AL-PROfile patient-reported outcome measure in light chain (AL) amyloidosis.European journal of haematology · 2024Article
- Novel and Proven Models of Public, Private, and Public-Private Partnerships in Healthcare: An Update.Handbook of experimental pharmacology · 2024Review
- Peripheral Nervous, Hepatic, and Gastrointestinal Endpoints for AL Amyloidosis Clinical Trials: Report from the Amyloidosis Forum Multi-organ System Working Group.Advances in therapy · 2023Review
- Differences in patient-reported outcomes (PROs) by disease severity in light chain (AL) amyloidosis.European journal of haematology · 2023Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
9 authors at 5 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
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
Indexed as
Identifiers
What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.