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

Publication ,  Journal Article
Patel, AA; Tapper, EB; Kanwal, F; Woodrell, CD; Hansen, L; Lai, JC; Rogal, S; McDermott, C; Rakoski, M; Ufere, NN
Published in: Hepatol Commun
June 1, 2023

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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Published In

Hepatol Commun

DOI

EISSN

2471-254X

Publication Date

June 1, 2023

Volume

7

Issue

6

Location

United States

Related Subject Headings

  • Liver Cirrhosis
  • Humans
  • Consensus
  • 3202 Clinical sciences
 

Citation

APA
Chicago
ICMJE
MLA
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Patel, A. A., Tapper, E. B., Kanwal, F., Woodrell, C. D., Hansen, L., Lai, J. C., … Ufere, N. N. (2023). Targets and study design for symptom-focused trials aimed at patients with cirrhosis: An expert consensus. Hepatol Commun, 7(6). https://doi.org/10.1097/HC9.0000000000000135
Patel, Arpan A., Elliot B. Tapper, Fasiha Kanwal, Christopher D. Woodrell, Lissi Hansen, Jennifer C. Lai, Shari Rogal, Cara McDermott, Mina Rakoski, and Nneka N. Ufere. “Targets and study design for symptom-focused trials aimed at patients with cirrhosis: An expert consensus.Hepatol Commun 7, no. 6 (June 1, 2023). https://doi.org/10.1097/HC9.0000000000000135.
Patel AA, Tapper EB, Kanwal F, Woodrell CD, Hansen L, Lai JC, et al. Targets and study design for symptom-focused trials aimed at patients with cirrhosis: An expert consensus. Hepatol Commun. 2023 Jun 1;7(6).
Patel, Arpan A., et al. “Targets and study design for symptom-focused trials aimed at patients with cirrhosis: An expert consensus.Hepatol Commun, vol. 7, no. 6, June 2023. Pubmed, doi:10.1097/HC9.0000000000000135.
Patel AA, Tapper EB, Kanwal F, Woodrell CD, Hansen L, Lai JC, Rogal S, McDermott C, Rakoski M, Ufere NN. Targets and study design for symptom-focused trials aimed at patients with cirrhosis: An expert consensus. Hepatol Commun. 2023 Jun 1;7(6).

Published In

Hepatol Commun

DOI

EISSN

2471-254X

Publication Date

June 1, 2023

Volume

7

Issue

6

Location

United States

Related Subject Headings

  • Liver Cirrhosis
  • Humans
  • Consensus
  • 3202 Clinical sciences