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Efficacy and Safety of G-CSF in Patients With Severe Alcoholic Hepatitis With Null or Partial Response to Steroid

Phase 3
Terminated
Conditions
Alcoholic Hepatitis
Interventions
Drug: steroid
Drug: placebo
Registration Number
NCT02442180
Lead Sponsor
Chuncheon Sacred Heart Hospital
Brief Summary

Steroid is the treatment of choice in patients with severe alcoholic hepatitis. However, null- or partial responder of steroid treatment is recommended to consider liver transplantation. The yearly demand for liver transplants far exceeds the supply of available organs and alcoholic liver disease has been a controversial indication for transplantation. Granulocyte-Colony Stimulating Factor (G-CSF) has been reported to have effect of proliferation of hepatic progenitors in alcoholic steatohepatitis. The aim of this study is to investigate the efficacy of G-CSF in patients with severe alcoholic hepatitis with null or partial response to steroid.

Detailed Description

Severe alcoholic hepatitis is defined as alcoholic hepatitis patients having discriminant function (DF) score over 32 or accompanying hepatic encephalopathy. These patients have shown poor prognosis of 28 day mortality as 30 to 50% without treatment. Steroid (prednisolone 40mg/day for 28 days) is the treatment of choice in patients with severe alcoholic hepatitis. Alcoholic hepatitis with modified DF score greater than or equal to 32 or model for end-stage liver disease (MELD) score over 21 or with hepatic encephalopathy are indications. However, null- or partial responder of steroid treatment is recommended to consider liver transplantation. The yearly demand for liver transplants far exceeds the supply of available organs and alcoholic liver disease has been a controversial indication for transplantation. Even in the responders of steroid treatment, the mortality is still 20% (from 40% without treatment to 20% with steroid treatment). There is a need for development of new treatment for this catastrophic disease. Granulocyte-Colony Stimulating Factor (G-CSF) has been reported to have effect of proliferation of hepatic progenitors in alcoholic steatohepatitis. The aim of this study is to investigate the efficacy of G-CSF in patients with severe alcoholic hepatitis with null or partial response to steroid.

Recruitment & Eligibility

Status
TERMINATED
Sex
All
Target Recruitment
64
Inclusion Criteria

Patients with

  • Clinical significant alcohol intake history (men over 50g within 3 months, women over 40g within 3 months)
  • modified DF score greater than or equal to 32
  • Transjugular liver biopsy shows typical feature of alcoholic hepatitis or meet the clinical diagnosis (total serum bilirubin level over 5 mg/dL, aspartate aminotransferase/alanine aminotransferase ratio >2, aspartate aminotransferase < 300 IU/L)
  • Included patients should meet the all above criteria and Lille score > 0.16 at the day 7 of prednisolone 40mg (or 32 mg of methylprednisolone) daily treatment.
Exclusion Criteria

Patients with

  • hepatitis B surface antigen (HBsAg), anti-hepatitis C virus (anti-HCV), or anti-human immunodeficiency virus (HIV) (+)
  • Malignancy including hepatocellular carcinoma
  • Portal vein thrombosis, hemochromatosis, autoimmune hepatitis, Wilson's disease, alpha-1-antitrypsin deficiency
  • Pregnancy, breast feeding, or who refuses contraception, or who cannot do contraception
  • History of adverse event including allergic response, hypersensitivity to G-CSF
  • Hypovolemic shock due to gastrointestinal hemorrhage or who need packed red blood cell (RBC) transfusion more than 3 units or increased modified discriminant factor (DF) score greater or equal to 32 from below 32 due to gastrointestinal hemorrhage
  • Sepsis or uncontrolled acute infection
  • Hepatic encephalopathy grade 3-4
  • History of steroid or pentoxifylline treatment within 3 months
  • Myeloblast on peripheral blood smear test
  • Critical comorbidities (type I hepatorenal syndrome, serum creatinine >2.5mg/dL, heart failure, pulmonary disease, psychiatric disease, acute pancreatitis etc.)
  • Who refuses to participate in clinical trial

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
G-CSF in null responder to steroidG-CSF (Filgrastim injection)Patients who are randomized to G-CSF treatment group in patients with null responder to prednisolone therapy.
G-CSF + steroid in partial respondersteroidPatients who are randomized to prednisolone plus G-CSF treatment group in patients with partial responder to prednisolone therapy.
Placebo + steroid in partial respondersteroidPatients who are randomized to prednisolone plus placebo treatment group in patients with partial responder to prednisolone therapy.
Placebo + steroid in partial responderplaceboPatients who are randomized to prednisolone plus placebo treatment group in patients with partial responder to prednisolone therapy.
Placebo in null responder to steroidplaceboPatients who are randomized to placebo treatment group in patients with null responder to prednisolone therapy.
G-CSF + steroid in partial responderG-CSF (Filgrastim injection)Patients who are randomized to prednisolone plus G-CSF treatment group in patients with partial responder to prednisolone therapy.
Primary Outcome Measures
NameTimeMethod
2-month survival rate of null responder to steroid treatment and 6-month survival rate of partial responder to steroid treatmentAfter 2 months of G-CSF or placebo treatment in patients with null responder to steroid treatment and after 6 months of G-CSF+steroid or only steroid treatment in patients with partial responder to steroid treatment

Survival status can be determined by the occurrence of mortality regardless of any cause of death.

Secondary Outcome Measures
NameTimeMethod
Hepatic function improvement as assessed by the Child-Pugh scoreday 0,1,3,7,9,11,14,17,20,23,26,29,32,35,60,90,120,150,180

Hepatic function is defined as the Child-Pugh score.

Hepatic function improvement as assessed by the Chronic Liver Failure (CLIF)-Sequential Organ Failure Assessment (SOFA) scoreday 0,1,3,7,9,11,14,17,20,23,26,29,32,35,60,90,120,150,180

Hepatic function is defined as the CLIF-SOFA score.

Hepatic function improvement as assessed by the MELD scoreday 0,1,3,7,9,11,14,17,20,23,26,29,32,35,60,90,120,150,180

Hepatic function is defined as the MELD score.

Hepatic function improvement as assessed by the Fraction of Cluster of differentiation (CD34)+ cell in peripheral bloodday0,7,35

Hepatic function is defined as the CD34+ cell count percentage in circulating blood.

Hepatic function improvement as assessed by the Alcoholic Hepatitis Histology scoreday0,35

Hepatic function is defined as histological scoring system of alcoholic hepatitis (AHHS).

Trial Locations

Locations (1)

Chuncheon Sacred Heart hospital

🇰🇷

Chuncheon, Korea, Republic of

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