TY - JOUR
T1 - Targets and study design for symptom-focused trials aimed at patients with cirrhosis
T2 - An expert consensus
AU - Patel, Arpan Arun
AU - Tapper, Elliot B.
AU - Kanwal, Fasiha
AU - Woodrell, Christopher D.
AU - Hansen, Lissi
AU - Lai, Jennifer C.
AU - Rogal, Shari
AU - McDermott, Cara
AU - Rakoski, Mina
AU - Ufere, Nneka N.
N1 - Publisher Copyright:
Copyright © 2023 The Author(s). Published by Wolters Kluwer Health, Inc. on behalf of the American Association for the Study of Liver Diseases.
PY - 2023/6
Y1 - 2023/6
N2 - Background: Symptom-focused trials are critically needed for patients with cirrhosis. However, this work would benefit from standard processes and validated measures. Methods: A writing group was formed among hepatologists, nurses, palliative care providers, pharmacists, and clinical trial experts focused on symptom management in patients with cirrhosis to define the key (1) components of trial design, (2) symptom targets, (3) measurement, and (4) outcomes for each target. From July 2022 to January 2023, panelists participated in an iterative process of developing and arriving at a consensus for each component. The goal was to provide consensus definitions that can be operationalized in future clinical trials, including for patients with cirrhosis. Results: The panel reached a consensus on key reporting features for clinical trials, along with considerations for study design. Nine key symptom targets (muscle cramps, pruritus, pain, fatigue, sexual dysfunction, sleep disorders, depression and anxiety, nausea/vomiting, and dyspnea/breathlessness) were identified. The panel selected instruments that can be considered for clinical trials based on psychometric validation and previous experience. The panel identified ongoing needs, including instrument validation, safety data, evidence about non-pharmacologic interventions, and comparative effectiveness studies. Conclusion: This expert panel identified key design, reporting, and measurement elements to standardize processes and measures in future symptom-focused clinical trials in the context of cirrhosis.
AB - Background: Symptom-focused trials are critically needed for patients with cirrhosis. However, this work would benefit from standard processes and validated measures. Methods: A writing group was formed among hepatologists, nurses, palliative care providers, pharmacists, and clinical trial experts focused on symptom management in patients with cirrhosis to define the key (1) components of trial design, (2) symptom targets, (3) measurement, and (4) outcomes for each target. From July 2022 to January 2023, panelists participated in an iterative process of developing and arriving at a consensus for each component. The goal was to provide consensus definitions that can be operationalized in future clinical trials, including for patients with cirrhosis. Results: The panel reached a consensus on key reporting features for clinical trials, along with considerations for study design. Nine key symptom targets (muscle cramps, pruritus, pain, fatigue, sexual dysfunction, sleep disorders, depression and anxiety, nausea/vomiting, and dyspnea/breathlessness) were identified. The panel selected instruments that can be considered for clinical trials based on psychometric validation and previous experience. The panel identified ongoing needs, including instrument validation, safety data, evidence about non-pharmacologic interventions, and comparative effectiveness studies. Conclusion: This expert panel identified key design, reporting, and measurement elements to standardize processes and measures in future symptom-focused clinical trials in the context of cirrhosis.
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U2 - 10.1097/HC9.0000000000000135
DO - 10.1097/HC9.0000000000000135
M3 - Article
C2 - 37267219
AN - SCOPUS:85185613962
SN - 2471-254X
VL - 7
JO - Hepatology Communications
JF - Hepatology Communications
IS - 6
M1 - e0135
ER -