Targets and study design for symptom-focused trials aimed at patients with cirrhosis: An expert consensus.



Symptom-focused trials are critically needed for patients with cirrhosis. However, this work would benefit from standard processes and validated measures.


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.


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.


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.





Published Version (Please cite this version)


Publication Info

Patel, Arpan A, Elliot B Tapper, Fasiha Kanwal, Christopher D Woodrell, Lissi Hansen, Jennifer C Lai, Shari Rogal, Cara McDermott, et al. (2023). Targets and study design for symptom-focused trials aimed at patients with cirrhosis: An expert consensus. Hepatology communications, 7(6). p. e0135. 10.1097/hc9.0000000000000135 Retrieved from

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Cara L. McDermott

Assistant Professor in Medicine

Dr. Cara McDermott is an Assistant Professor in Medicine, Division of Geriatrics, at Duke University School of Medicine. She has a K23 award from NIH's National Heart, Lung and Blood Institute on deprescribing fall-risk increasing drugs among multimorbid adults with chronic obstructive pulmonary disease. Her research focuses on identifying gaps in healthcare delivery and using implementation science to adapt and assess evidence-based interventions. Dr. McDermott works to optimize medication use, improve symptom management, and reduce unwanted healthcare use among older adults with multiple chronic conditions, with a particular interest in improving outcomes for patients with cancer, COPD, or dementia and their caregivers.  She is particularly interested in improving shared decision making in this population and medication deprescribing for adults with serious illness. In recent projects, she investigated ways to improve end-of-life care from the perspective of bereaved caregivers and care delivery gaps leading to unwanted healthcare utilization at end of life for patients with multimorbidity. She completed a T32 postdoctoral fellowship with the Cambia Palliative Care Center of Excellence at the University of Washington and a K12 with the University of Washington’s Implementation Sciences Training Program.

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