Health-related quality of life instruments for clinical trials in AL amyloidosis: report from the Amyloidosis Forum HRQOL Working Group

AA Rizio, MK White, A D'Souza, K Hsu… - Patient Related …, 2023 - Taylor & Francis
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 …

Systematic review of the effect of a one-day versus seven-day recall duration on patient reported outcome measures (PROMs)

T Peasgood, JM Caruana, C Mukuria - The Patient-Patient-Centered …, 2023 - Springer
Background There is ongoing uncertainty around the most suitable recall period for patient-
reported outcome measures (PROMs). Method This systematic review integrates …

Outcomes in facial feminization surgery: a systematic review

K Uhlman, J Gormley, I Churchill, M Huynh… - Facial Plastic Surgery …, 2024 - liebertpub.com
Objective: Review literature on facial feminization surgery (FFS) for the transgender
population and identify whether heterogeneity in reported outcomes and outcome measures …

Psychometric validation of the SF-36 Health Survey in light chain amyloidosis: results from community-based and clinic-based samples

MK White, KL McCausland… - Patient related …, 2017 - Taylor & Francis
Background Light chain (AL) amyloidosis, a rare and life-threatening protein misfolding
disorder, causes organ damage and severely impacts health-related quality of life (HRQoL) …

The use of PROMIS patient-reported outcomes (PROs) to inform light chain (AL) amyloid disease severity at diagnosis

A D'Souza, BE Magnus, J Myers, A Dispenzieri… - Amyloid, 2020 - Taylor & Francis
We sought to evaluate how PROMIS patient-reported outcome (PRO) measures correlated
with disease characteristics in systemic light chain (AL) amyloidosis patients at diagnosis …

Differences in patient‐reported outcomes (PROs) by disease severity in light chain (AL) amyloidosis

A D'Souza, A Szabo, I Akinola… - European Journal of …, 2023 - Wiley Online Library
Objective To assess the impact of organ involvement on patient‐reported outcomes (PROs)
in light chain (AL) amyloidosis. Methods PROs were evaluated using the KCCQ‐12 …

Quality of life and symptoms among patients with relapsed/refractory AL amyloidosis treated with ixazomib‐dexamethasone versus physician's choice

V Sanchorawala, AD Wechalekar, K Kim… - American journal of …, 2023 - Wiley Online Library
Patient‐reported outcomes in AL amyloidosis have not been well‐studied. We analyzed
health‐related quality of life (HRQOL) and AL amyloidosis symptoms data from the phase 3 …

Content validation of the SF-36v2® Health Survey Acute for use in hypoparathyroidism

M Brod, LT Waldman, AD Shu, A Smith - Quality of Life Research, 2023 - Springer
Purpose The purpose of this study was to conduct cognitive debriefing (CD) interviews with
adults diagnosed with chronic hypoparathyroidism (HP) to assess the content validity of the …

Health‐related quality of life in patients with light chain amyloidosis treated with bortezomib, cyclophosphamide, and dexamethasone±daratumumab: Results from the …

V Sanchorawala, G Palladini… - American journal of …, 2022 - Wiley Online Library
In the phase 3 ANDROMEDA trial, patients treated with daratumumab, bortezomib,
cyclophosphamide, and dexamethasone (D‐VCd) had significantly higher rates of organ …

Is there a representative quality‐of‐life questionnaire for patients with AL amyloidosis?—systematic literature review

S Bristogiannis, J Khwaja, Y Lwin… - British Journal of …, 2023 - Wiley Online Library
Systemic AL amyloidosis is an incurable condition with various presentations and may
cause multiple complications related to organ involvement. As survival has improved …