What evidence exists to support the efficacy and safety of N-acetylcysteine as an adjunct to corticosteroids in the treatment of severe alcoholic hepatitis?

Comment by InpharmD Researcher

Data on the use of N-acetylcysteine (NAC) in combination with glucocorticoids, specifically for alcoholic hepatitis, are limited with available evidence mostly derived from small studies. In studies combining NAC with prednisolone, dosing typically consisted of an intravenous (IV) loading regimen on day 1 (150 mg/kg over 30 min, 50 mg/kg over 4 h, 100 mg/kg over 16 h in 5% glucose), followed by 100 mg/kg/day on days 2-5 (Tables 1 and 2). NAC was generally well-tolerated and resulted in a significant 1-month survival benefit, but evidence for long-term survival benefit remains inconclusive. Available societal guidelines note IV NAC as an adjunct in severe alcoholic hepatitis, but do not provide specific dosing recommendations.
Background

Both the 2023 American College of Gastroenterology (ACG) Clinical Guideline on Alcohol-Associated Liver Disease and the 2019 American Association for the Study of Liver Diseases (AASLD) Practice Guidance discuss intravenous N-acetylcysteine (NAC) as an adjunct to corticosteroids in severe alcoholic hepatitis, but neither provides a specific dosing recommendation. Both guidelines note that the available evidence is based on studies using a 5-day intravenous NAC infusion administered with prednisolone and suggest that this combination may improve short-term (approximately 28- to 30-day or 1-month) survival; however, a sustained benefit at 3 or 6 months has not been demonstrated. The ACG guideline also notes that earlier studies of NAC monotherapy or NAC combined with other antioxidants did not demonstrate a survival benefit. Although individual randomized trials produced mixed findings, one 2015 network meta-analysis cited by both guidelines supported prednisolone plus 5 days of intravenous NAC as the regimen associated with the greatest short-term survival benefit, with the ACG guideline reporting an 85% reduction in the risk of death at 28 days and the AASLD guideline reporting a relative risk of 0.28 (95% credible interval 0.10 to 0.69). The ACG guideline states that IV NAC may be used as an adjunct to corticosteroids, citing its favorable safety profile, whereas the AASLD guidance considers the combination promising but notes that additional validation is needed. [1], [2], [3]

In a 2017 expert review published by the American Gastroenterological Association (AGA), alcoholic hepatitis is noted as an inflammatory disease, with cytokine production contributing to liver injury; thus, current drug therapies for severe alcoholic hepatitis have anti-inflammatory mechanisms of action. Glucocorticoids (e.g., methylprednisolone 32 mg daily) are considered the standard-of-care, though some patients are unable to tolerate or may have developed resistance to glucocorticoid therapy. In such cases, patients may be treated with pentoxifylline 400 mg three times daily with meals, though data to support this practice is still conflicting. Phosphodiesterase (PDE) inhibitors, particularly PDE-4 inhibitors, have been observed in vivo and in vitro to provide liver protection due to their ability to upregulate anti-inflammatory cytokines, though significant adverse events limit their role in treatment of alcoholic liver disease. Other treatments, such as vitamin E and NAC cocktails, have had limited or mixed success in other published literature; one combination of prednisolone and NAC, given intravenously over the first 5 days of treatment, resulted in significant improvement in 1-month mortality, which found mortality rate to be 16% lower than patients treated with prednisolone alone. Additionally, patients who were treated with prednisolone and NAC presented with fewer infections compared to those on prednisolone monotherapy (19% vs 42%; p= 0.001), as well as a lower incidence of hepatorenal syndrome (HRS; 12% vs 25%; p= 0.02). Though these findings are substantial, more research is required to confirm the benefits and most optimal use case of this regimen. [3], [4]

A 2026 systematic review and meta-analysis evaluated 52 randomized controlled trials involving 5,121 participants with severe alcohol-associated hepatitis to assess the efficacy and safety of pharmacologic therapies. Among the included studies, the addition of NAC to corticosteroids was associated with a significant reduction in 28-day mortality compared with corticosteroids alone (RR, 0.35; 95% CI, 0.16–0.78; 174 participants; 1 study), although no statistically significant benefit was observed for 90-day mortality (RR, 0.66; 95% CI, 0.41–1.08). Combination therapy was also associated with lower rates of infection (RR, 0.45; 95% CI, 0.27–0.75; moderate-certainty evidence) and progression to hepatorenal syndrome (RR, 0.48; 95% CI, 0.24–0.94; low-certainty evidence), with no significant difference in liver transplantation rates. The authors suggested that low-certainty evidence supports corticosteroids as first-line therapy for eligible patients with severe alcohol-associated hepatitis, while the evidence supporting adjunctive therapies, including NAC, ranges from low to moderate certainty and requires further confirmation. [5]

Background References: [1] Jophlin LL, Singal AK, Bataller R, et al. ACG Clinical Guideline: Alcohol-Associated Liver Disease. Am J Gastroenterol. 2024;119(1):30-54. doi:10.14309/ajg.0000000000002572
[2] Crabb DW, Im GY, Szabo G, Mellinger JL, Lucey MR. Diagnosis and Treatment of Alcohol-Associated Liver Diseases: 2019 Practice Guidance From the American Association for the Study of Liver Diseases. Hepatology. 2020;71(1):306-3​​33. doi:10.1002/hep.30866
[3] Singh S, Murad MH, Chandar AK, et al. Comparative Effectiveness of Pharmacological Interventions for Severe Alcoholic Hepatitis: A Systematic Review and Network Meta-analysis. Gastroenterology. 2015;149(4):958-70.e12. doi:10.1053/j.gastro.2015.06.006
[4] Mitchell MC, Friedman LS, McClain CJ. Medical Management of Severe Alcoholic Hepatitis: Expert Review from the Clinical Practice Updates Committee of the AGA Institute. Clin Gastroenterol Hepatol. 2017;15(1):5-12. doi:10.1016/j.cgh.2016.08.047
[5] Islam AH, Alvizuri C, Desalegn H, et al. Pharmacological Strategies for the Management of Severe Alcohol-associated Hepatitis: A Systematic Review and Meta-Analysis. Clin Gastroenterol Hepatol. 2026;24(3):606-620. doi:10.1016/j.cgh.2025.05.016
Literature Review

A search of the published medical literature revealed 2 studies investigating the researchable question:

What evidence exists to support the efficacy and safety of N-acetylcysteine as an adjunct to corticosteroids in the treatment of severe alcoholic hepatitis?

Level of evidence

C - Multiple studies with limitations or conflicting results  Read more→



Please see Tables 1-2 for your response.


Glucocorticoids plus N-Acetylcysteine in Severe Alcoholic Hepatitis
Design

Multicenter, randomized, controlled trial

N= 174

Objective To investigate whether combination therapy with glucocorticoids plus N-acetylcysteine improves survival in patients with severe acute alcoholic hepatitis
Study Groups

Prednisolone plus N-acetylcysteine (n= 85)

Prednisolone only (n= 89)

Inclusion Criteria Age ≥18 years; average alcohol intake >50 g per day during the 3 months before enrollment; Maddrey’s discriminant function of 32 or more; liver histologic findings consistent with alcoholic hepatitis
Exclusion Criteria Hepatorenal syndrome, hepatocellular carcinoma, uncontrolled bacterial infection or gastrointestinal hemorrhage in the previous 4 days, infection with HCV, HBV, HIV, autoimmune hepatitis, hemochromatosis, Wilson’s disease, alpha1-antitrypsin deficiency, acetaminophen-induced hepatitis, cancer, N-acetylcysteine allergy, serious cardiac, respiratory, or neurologic disease
Methods Patients received 40 mg of oral prednisolone per day for 28 days. Prednisolone–N-acetylcysteine group received intravenous N-acetylcysteine on day 1 (150, 50, and 100 mg/kg in 250, 500, and 1000 ml of 5% glucose solution over 30 minutes, 4 hours, and 16 hours, respectively) and on days 2 through 5 (100 mg/kg/day in 1000 ml of 5% glucose solution). Prednisolone-only group received an infusion in 1000 ml of 5% glucose solution per day on days 1 through 5. 
Duration 2004 through 2009
Outcome Measures 6-month survival, 1 and 3-month survival, hepatitis complications, adverse events related to N-acetylcysteine use
Baseline Characteristics   Prednisolone Only (n= 89) Prednisolone–N-Acetylcysteine (n= 85)
Age, years 52.2±8.5 52.8±8.7
Male sex 50 (56%) 55 (65%)
Alcohol intake, g/day 108.8±55.3 107.2±58.0
AUDIT score 22.6±7.4 22.5±7.4
CAGE score 3.2±0.9 3.3±0.9
Child–Pugh score 11.3±1.3 11.0±1.5
Maddrey’s discriminant function 58.2±20.0 54.0±18.2
Hepatic encephalopathy 39 (44%) 38 (45%)
Ascites 62 (70%) 59 (69%)

Abbreviations: Alcohol Use Disorders Identification Test (AUDIT)

The CAGE questionnaire includes 4 questions on use of alcoholic beverages. A score of 2 or more indicates alcohol abuse (score range, 0 to 4)

Results   Prednisolone Only (n= 89) Prednisolone–N-Acetylcysteine (n= 85) p-value
6-month mortality 38% 27% 0.07
1-month mortality 24% 8% 0.006
3-month mortality 34% 22% 0.06
Death due to hepatorenal syndrome 22% 9% 0.02
Infections 42% 19% 0.001
Adverse Events Infections were less frequent in the prednisolone–N-acetylcysteine group (19%) compared to the prednisolone-only group (42%); rash after first injection of N-acetylcysteine occurred in 4% of patients in the combination group. 
Study Author Conclusions Although combination therapy with prednisolone plus N-acetylcysteine increased 1-month survival among patients with severe acute alcoholic hepatitis, 6-month survival, the primary outcome, was not improved.
Critique The study was well-designed as a multicenter, randomized, controlled trial, providing robust data on the short-term benefits of combination therapy. However, the lack of significant improvement in 6-month survival and the potential for a lack of power to detect differences in longer-term outcomes are limitations. Additionally, the open-label design may introduce bias, and the study did not explore the potential benefits of extending N-acetylcysteine treatment beyond 5 days. 
Table 1 References:
[6] Nguyen-Khac E, Thevenot T, Piquet MA, et al. Glucocorticoids plus N-acetylcysteine in severe alcoholic hepatitis. N Engl J Med. 2011;365(19):1781-1789. doi:10.1056/NEJMoa1101214
A Combination of N‑Acetylcysteine and Prednisone Has No Benefit Over Prednisone Alone in Severe Alcoholic Hepatitis: A Retrospective Analysis
Design

Retrospective cohort analysis

N= 68

Objective To assess whether there were any survival advantages with a combination treatment of intravenous N-acetylcysteine (NAC) and prednisone over prednisone alone in those with severe alcoholic hepatitis
Study Groups

Prednisone (n= 21)

Prednisone + NAC (n= 47)

Inclusion Criteria Patients admitted with acute alcoholic hepatitis (AH) with a discriminant function (DF) score ≥32 who received treatment with prednisone or prednisone and NAC
Exclusion Criteria Patients with major gastrointestinal bleeding or septic shock were not studied
Methods

Study investigators reviewed the electronic records of patients admitted with AH between January 1, 2013, and February 28, 2019.

Patients were categorized into those who received oral prednisone (40 mg/day) or a combination of prednisone and intravenous (IV) NAC. The NAC dosage was as follows:

Day 1: 150 mg/kg in 5% glucose over 30 minutes, followed by 50 mg/kg over 240 minutes, and 10 mg/kg over the next 16 hours.

Days 2–5: 10 mg/kg per 24 hours in 1,000 mL of 5% glucose solution.

Treatment choice was at the discretion of the on-call hepatologist, and all patients stayed in the hospital for 5–7 days. If patients had a Lille score <0.45, prednisone was continued for 28 days, followed by a rapid taper.

Duration January 1, 2013, to February 28, 2019
Outcome Measures

Primary: 30-day and 90-day transplant-free all-cause mortality

Secondary: Treatment effect on Lille score (with poor treatment response defined as a Lille score ≥0.45)

Baseline Characteristics   Prednisone (n= 21) Prednisone + NAC (n= 47) p-value
Age, years 44.4 ± 8.7 48.5 ± 10.5 0.126
Female 13 (62%) 26 (55%) 0.612

Race

White

Balck

 

10 (48%)

10 (48%)

 

36 (77%)

9 (19%)

0.049

 

 

Encephalopathy

11 (52%)

23 (49%)

0.793

Cirrhosis 12 (57%) 32 (68%) 0.383
Ascites 18 (86%) 35 (74%) 0.301
Sepsis 6 (29%) 11 (23%) 0.649
GI bleeding 3 (14%) 3 (6%) 0.288
Renal failure 6 (29%) 18 (38%) 0.438
T. bili 14.3 ± 10.8 17.8 ± 9.1 0.075
Albumin 2.84 ± 0.45 2.66 ± 0.56 0.195
AST 222 ± 406 343 ± 1105 0.189
ALT 106 ± 280 135 ± 552 0.502
ALP 203 ± 112 176 ± 75 0.686
PT 21.3 ± 3.0 24.1 ± 6.7 0.224
Cr 1.16 ± 0.83 1.59 ± 1.65 0.468
MELD score 25.5 ± 6.4 29.2 ± 6.4 0.029
Biopsy 7 (33%) 17 (36%) 0.821

Lille score

Mean score

≥0.45

<0.45

 

0.30 ± 0.32

6 (29%)

15 (71%)

 

0.40 ± 0.34

16 (34%)

31 (66%)

<0.001

0.004

Abbreviations: ALP= alkaline phosphatase. ALT= alanine transaminase. AST= aspartate aminotransferase. Cr= creatinine. GI= gastrointestinal. MELD= Model for end-stage liver diseases. PT= prothrombin time. T bili= total bilirubin.
Results   Prednisone (n= 21) Prednisone + NAC (n= 47) p-Value
30-day mortality 1 (5%) 8 (16%) -*
90-day mortality 2 (9.5%) 12 (25.5%) 0.14*

*Not statistically significant.

The overall 30-day and 90-day mortality in both groups was 13.2% (9/68) and 20.6% (14/68), respectively.

The type of treatment regimen had no effect on Lille score (OR 0.84, 95% CI 0.25–2.78).

Adverse Events There were no serious adverse events with either NAC or prednisone treatment, and no patients discontinued treatment because of adverse events.
Study Author Conclusions The combination treatment of NAC + prednisone is not better than prednisone alone in patients with severe alcoholic hepatitis.
Critique The study's retrospective design limits the ability to establish causality. The small sample size and potential selection bias, as treatment choice was at the discretion of the treating hepatologist, may affect the generalizability of the findings. However, the study provides valuable insights into the lack of benefit of adding NAC to prednisone in severe alcoholic hepatitis.
Table 2 References:
[7] Amjad W, Alukal J, Doycheva I, et al. A Combination of N-Acetylcysteine and Prednisone Has No Benefit Over Prednisone Alone in Severe Alcoholic Hepatitis: A Retrospective Analysis. Dig Dis Sci. 2020;65(12):3726-3733. doi:10.1007/s10620-020-06142-4