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Efficacy of granulocyte colony stimulating factor in patients with severe alcoholic hepatitis with partial or null
Yuri Cho1, Youn Su Park2, Hwi Young Kim3
1Department of Internal Medicine, CHA Gangnam Medical Center, CHA University School of Medicine, Seoul, Republic of Korea.
Insights
Granulocyte colony stimulating factor (G-CSF) may improve survival in severe alcoholic hepatitis patients unresponsive to steroids. This trial investigates G-CSF as a rescue therapy to enhance liver function and prolong survival in this high-risk group.
Area of Science:
- Hepatology
- Gastroenterology
- Clinical Trials
Background:
- Severe alcoholic hepatitis (AH) has high mortality despite corticosteroid treatment.
- Innovative therapies are needed for AH patients refractory to standard care.
- Granulocyte colony stimulating factor (G-CSF) is explored as a potential novel treatment.
Purpose of the Study:
- To determine if G-CSF treatment increases short-term survival in severe AH patients unresponsive to corticosteroids.
- To assess the efficacy of Lille-score-guided G-CSF treatment.
- To identify patient subgroups who may benefit from G-CSF rescue therapy.
Main Methods:
- Patients with severe AH (Maddrey's discriminant function score ≥ 32) were screened.
- Patients with partial response (Lille score 0.16-0.56) or null response (Lille score ≥ 0.56) to prednisolone were enrolled.
- Randomization to G-CSF or placebo was performed, with survival rates as primary endpoints.
Main Results:
- Preliminary safety data were assessed to ensure study continuation.
- The study aimed to evaluate G-CSF's impact on survival in specific AH patient subgroups.
- Further analysis of risk factors for mortality, including CD34+ cell counts, was planned.
Conclusions:
- G-CSF is hypothesized to prolong short-term survival in severe AH patients refractory to corticosteroids.
- This proof-of-concept trial evaluates G-CSF efficacy in a Lille-score-guided approach.
- The study seeks to identify a specific subgroup of AH patients who would benefit from G-CSF rescue therapy.
Background:
Alcoholic hepatitis (AH) has the most severe presentation among alcohol-related liver diseases. Corticosteroids are the most widely recommended treatment for severe AH. However, more innovative, refined treatment measures are required because of its high mortality despite corticosteroid treatment. This study aims to determine whether granulocyte colony stimulating factor (G-CSF) treatment increases short-term survival in patients with severe AH refractory to corticosteroid treatment.
Methods/Design:
Patients with severe AH whose Maddrey's discriminant function (MDF) score is ≥ 32 and who will be treated with prednisolone (40 mg/day) for 1 week will be screened. Among them, 190 subjects with a partial response (PR) (Lille score 0.16-0.56), and 78 subjects with a null response (NR) (Lille score ≥ 0.56) will be enrolled. Subjects with PR will be randomized to steroid plus placebo or steroid plus 12 G-CSF injections (5 μg/kg/day for 5 days followed by every 3 days) at a ratio of 1:1. Subjects with a NR will be randomized to the placebo or G-CSF group (1:1). Study subjects in the PR group will be treated with prednisolone for 28 days followed by dose tapering for an additional 2 weeks. The primary endpoint is the 2-month survival rate in the NR group and the 6-month survival rate in the PR group. Child-Turcotte-Pugh, model for end-stage liver disease score, and the change in the proportion of peripheral circulating CD34-positive cells will be analyzed as risk factors for mortality. Preliminary safety data for the initial 10 study subjects enrolled in the PR study will be assessed to determine whether the PR study would be continued, according to the G-CSF-mobilized, peripheral-blood stem cell donor assessment protocol of the National Marrow Donor Program.
Discussion:
We hypothesized that G-CSF would prolong short-term survival of patients with severe AH refractory to corticosteroid treatment. This is a proof-of-concept trial designed to assess the efficacy of Lille-score-guided G-CSF treatment. This trial is also designed to identify a special subgroup in whom G-CSF rescue treatment would improve liver function and prolong survival.
Trial Registration:
ClinicalTrials.gov, NCT02442180 . Prospectively registered on 13 May 2015.
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