A Phase II, Multicenter, Open-Label, Randomized Trial of Pegfilgrastim for Patients With Alcohol-Associated Hepatitis

Authors

John A Tayek
Andrew A Stolz
Danh V Nguyen
M Wayne Fleischman
John A Donovan
Joseph M Alcorn
Daniel C-K Chao
Aliya Asghar
Timothy R Morgan
Southern California Alcoholic Hepatitis (SCAH) Consortium

Publication Date

12-1-2022

Journal

EClinicalMedicine

Abstract

BACKGROUND: In trials conducted in India, recombinant granulocyte colony stimulating factor (GCSF) improved survival in alcohol-associated hepatitis (AH). The aim of this trial was to determine the safety and efficacy of pegfilgrastim, a long-acting recombinant GCSF, in patients with AH in the United States.

METHODS: This prospective, randomized, open label trial conducted between March 2017 and March 2020 randomized patients with a clinical diagnosis of AH and a Maddrey discriminant function score ≥32 to standard of care (SOC) or SOC+pegfilgrastim (0.6 mg subcutaneously) on Day 1 and Day 8 (clinicaltrials.gov NCT02776059). SOC was 28 days of either pentoxifylline or prednisolone, as determined by the patient's primary physician. The second injection of pegfilgrastim was not administered if the white blood cell count exceeded 30,000/mm

FINDINGS: The study was terminated early due to COVID19 pandemic. Eighteen patients were randomized to SOC and 16 to SOC+pegfilgrastim. All patients received prednisolone as SOC. Nine patients failed to receive a second dose of pegfilgrastin due to WBC > 30,000/mm

INTERPRETATION: This phase II trial found no survival benefit at 90 days among subjects with AH who received pegfilgrastim+prednisolone compared with subjects receiving prednisolone alone.

FUNDING: was provided by the United States National Institutes of Health and National Institute on Alcohol Abuse and Alcoholism U01-AA021886 and U01-AA021884.

Keywords

Alcoholic hepatitis, Pegfilgrastim, Phase II, Randomized clinical trial

Comments

Supplementary Materials

PMID: 36267499