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ORPHA:90062

Acute liver failure

high confidenceDisorder

Also known as: Acute hepatic failure · Fulminant hepatic failure

Publications

38,143

98.7th percentile

Trials

55

Interventional, condition-specific

Researchers

1,263

Distinct authors in sample

Gene link

—

Readiness

4/6

Stages with a signal

Clinical definition (Orphanet)

A rare hepatic disease characterized by acute onset of severe liver dysfunction without evidence of underlying chronic liver disease. Patients present with nonspecific symptoms like jaundice, upper right abdominal pain, nausea, vomiting, pruritus, fatigue, and fever. The condition may rapidly progress to hepatic , coagulopathy, and life-threatening multiorgan failure. Liver biopsy typically shows massive hepatic necrosis.

How rare: 1-5 / 10 000 — about one to five people per ten thousand (still uncommon, but less ultra-rare).

Orphanet entry

Cross-references

Joined from Mondo / Orphanet. MeSH labels may enter searches; UMLS / OMIM / NCIT are stored for reference.

Additional Mondo synonyms (2)

acute hepatic failure · fulminant hepatic failure

Research stages

Trial readiness signals

Where this condition sits on an open-data research pipeline — not how close a treatment is, and not medical advice. Empty stages often mean “not in these databases under this Mondo ID,” not “impossible.”

4/6 stages with a signal

An interventional trial matched this condition name on ClinicalTrials.gov — see trials below.

  1. Gene identifiedNot found

    No GenCC disease–gene assertion in this build

  2. LiteraturePresent

    38,143 matched papers (22,278 in last 10 years) Source

  3. Phenotype characterisedPresent

    73 HPO annotations (e.g. Jaundice; Hepatocellular necrosis; Elevated circulating hepatic transaminase concentration) Source

  4. Animal modelNot found

    No Alliance genotype “model of” associations via Monarch for these Mondo IDs

  5. Orphan designationPresent

    3 FDA · 11 EMA designations (3 FDA orphan-indication approvals) — e.g. cardiotrophin-1 Source

  6. Interventional trialPresent

    55 matched on ClinicalTrials.gov (8 recruiting in sample)

Biology

Genes and phenotypes

Gene–disease validity from GenCC, plus phenotypes and animal models joined from Monarch Initiative via Mondo ID — not a clinical diagnosis aid.

Do we know what causes it?

Not yet — the cause hasn't been pinned down in GenCC.

No strong gene–disease assertion joined for this Orphanet entity.

Phenotypes (Monarch / HPO)

73

Associated phenotypes · MONDO:0019542

  • Jaundice
  • Hepatocellular necrosis
  • Elevated circulating hepatic transaminase concentration
  • Hepatitis
  • Emotional lability

Showing 5 of 73 — open Monarch for the full list.

Animal models (Monarch / Alliance)

None returned for this Mondo ID. Empty here is not proof that no model organism work exists under another name or gene.

Monarch fetch 2026-07-29

Therapies

Designations, candidates, and chemicals

FDA OOPD and EMA orphan designations, Open Targets clinical candidates, and CTD chemical associations via MyDisease.info. These never change the interventional-trial headline.

Orphan designation (FDA · EMA)

14

Designations · 3 with FDA orphan-indication approval

  • FDA cardiotrophin-1Acute liver failure · 2011-09-13 · Not FDA Approved for Orphan Indication
  • FDA L-ornithine phenylacetateHepatic Encephalopathy Acute liver failure Hyperammonemia · 2010-04-07 · Not FDA Approved for Orphan Indication
  • FDA N-acetylcysteineAcute liver failure · 2002-09-09 · Not FDA Approved for Orphan Indication
  • EMA cardiotrophin-1Treatment of acute liver failure · 30/08/2011 · PositiveEMA designation
  • EMA citric acid monohydrateTreatment of acute liver failure · 27/06/2016 · PositiveEMA designation
  • EMA heterologous human adult liver-derived stem cellsTreatment of acute liver failure · 26/04/2012 · PositiveEMA designation
  • EMA 4-(1H-Pyrrolo[2,3-b]pyridin-2-yl)phenol hydrochloridePrevention of acute liver failure · 21/08/2024 · PositiveEMA designation
  • EMA allogeneic hepatoblastoma cells encapsulated in alginate, ex vivo expandedTreatment of acute liver failure · 21/08/2020 · PositiveEMA designation

Sources: FDA OOPD · EMA orphan designations

Open Targets candidates

9

Drugs / clinical candidates · MONDO_0019542

CTD chemicals (MyDisease.info)

169 associated chemicals · 558 pathways. Therapeutic evidence is listed first when present — not a treatment recommendation.

  • Acetylcysteine · therapeutic
  • Adrenal Cortex Hormones · therapeutic
  • Caffeine · therapeutic
  • Carnitine · therapeutic
  • Catechin · therapeutic
  • Charcoal · therapeutic
  • Cysteine · therapeutic
  • Diazepam · therapeutic
  • entecavir · therapeutic
  • Epoprostenol · therapeutic
  • gentiopicroside · therapeutic
  • geraniol · therapeutic

Pathways: Glycolysis / Gluconeogenesis; Citrate cycle (TCA cycle); Pentose phosphate pathway; Fructose and mannose metabolism; Fatty acid degradation; Arginine biosynthesis; Alanine, aspartate and glutamate metabolism; Cysteine and methionine metabolism

MyDisease.info · MONDO:0019542

Literature

Is anyone studying this?

38,143

38,143 papers — among the better-studied rare conditions, though still a fraction of common-disease literature (breast cancer: over 700,000). Median papers in the last 10 years for a rare disease in this dataset (publications denominator n=3967) is 59.

38,143 papers since the earliest indexed year in this search — median last-10-year count for a rare disease in this dataset is 59 (publications denominator n=3967).

22,278 in the last 10 years · high confidence · 98.7th percentile (publications denominator)

Phrase hits: 38,128 · MeSH hits: 123

Open Europe PMC search

Who's working on it?

1,263

Distinct author names in 200 sampled papers — named people below.

Who's working on it?

People publishing on this condition (sampled Europe PMC records). Affiliation is the most recent found in that sample.

  1. 01
    Wang Y6 papers · 2026

    School of Food and Biological Engineering, Hefei University of Technology, Hefei 230009, China.

    Papers in Europe PMC
  2. 02
    Li Y5 papers · 2026

    Department of Cardiology, The First Affiliated Hospital of USTC, Division of Life Sciences and Medicine, University of Science and Technology of China (USTC), Hefei, China.

    Papers in Europe PMC
  3. 03
    Zhang Y5 papers · 2026

    People's Hospital of Xinjiang Uygur Autonomous Region, Ürümqi, 830001, China.

    Papers in Europe PMC
  4. 04
    Chen Y4 papers · 2026

    State Key Laboratory of Drug Research, Shanghai Institute of Materia Medica (SIMM), Chinese Academy of Sciences, Shanghai, China; University of Chinese Academy of Sciences, Beijing, China.

    Papers in Europe PMC
  5. 05
    Chen Z4 papers · 2026

    Laboratory of Biomaterials and Translational Medicine, Department of Ultrasound, Center for Nanomedicine, The Third Affiliated Hospital, Sun Yat-sen University, Guangzhou 510630, China.

    Papers in Europe PMC
  6. 06
    He Y4 papers · 2026

    State Key Laboratory of Drug Research, Shanghai Institute of Materia Medica (SIMM), Chinese Academy of Sciences, Shanghai, China; University of Chinese Academy of Sciences, Beijing, China. Electronic address: heyong@simm.ac.cn.

    Papers in Europe PMC
  7. 07
    Li J4 papers · 2026

    Department of Intensive Care Unit, General Hospital of Southern Theatre Command of PLA, Guangzhou, 510010, China.

    Papers in Europe PMC
  8. 08
    Xu Y4 papers · 2026

    Department of Pharmacy, Institute of Clinical Pharmacy, West China Hospital of Sichuan University, Chengdu, Sichuan Province 610041, China.

    Papers in Europe PMC
  9. 09
    Zhang J4 papers · 2026

    Department of Infectious Diseases, Ruijin Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai, China.

    Papers in Europe PMC
  10. 10
    Zhang X4 papers · 2026

    Department of Infectious Diseases, Renmin Hospital of Wuhan University, Wuhan, China.

    Papers in Europe PMC

Clinical research

Is a treatment being tested?

55

interventional trials for this specific condition

55 interventional trials matched this specific condition name; 8 currently recruiting in our sample. 187 trials are registered for liver failure, the broader category — shown separately because they may or may not enrol this specific subtype.

Data as of 11 September 2026

55 interventional trials — more than 77.2% of diseases in the trials denominator have none at all (5501 of 7126; this disease is at the 97.3th percentile).

high confidence · 97.3th percentile (trials denominator)

Recruiting interventional trials

From the matched ClinicalTrials.gov set

55 interventional trials matched after quoted-phrase search and title/condition post-filter.

Broader category: liver failure

187

Interventional trials for the parent category, exclusive of NCT IDs already counted above. Eligibility for this subtype is not guaranteed.

Worth raising with a clinician. How we count trials.

Recruiting under the broader category

Observational and natural-history studies

39 observational studies match this condition. These do not test a treatment and are not counted in the interventional-trial headline, but they are genuine research: natural-history work often defines the endpoints needed for a future rare-disease trial, and families may be able to enroll.

Recruiting or not-yet-recruiting

Open the complete matched search on ClinicalTrials.gov

Other registries (secondary)

Broader net from EU CTIS, ISRCTN, and ICTRP when available — deduped against ClinicalTrials.gov IDs already counted above. Dual-model LLM relevance gates what we keep. These rows are not added to the interventional headline.

raw 78 · after dedupe 78 · already on CT.gov 0 · kept 0 · parent 0 · uncertain 78 · dropped 0 · fetched 2026-07-30

Source notes: ictrp: Error: ICTRP public search unavailable (WHO portal is SPA-only; SOAP needs partnership). Tried: https://apps.who.int/tri

No secondary-registry studies passed dual-model relevance for this condition name (after dedupe).

Uncertain / not reviewed (78)

Where to find support

Condition-specific patient organisations, when Orphanet lists them, are on the disease’s Orphanet page. We also link umbrella groups that support undiagnosed and ultra-rare families.

Orphanet entry for Acute liver failure — check Associations / patient organisations on that page.

India — NPRD

Last verified 2026-07-26

This ORPHAcode is not on our curated NPRD list (direct or Mondo-parent match). That does not decide clinical eligibility; families in India should ask a notified Centre of Excellence about current coverage.

Hand-curated for this project. ORPHAcode mappings are best-effort and may be incomplete or imprecise for umbrella categories. Parent (Mondo) matches mean the policy lists a broader category — confirm eligibility with a Centre of Excellence. Financial entitlements summarised from public policy statements and may change. This is not official government guidance.

How we counted this

Europe PMC query (preferred label + any corrected label + Orphanet and Mondo exact synonyms, stoplisted; unioned with resolved MeSH labels when available). UMLS / OMIM / NCIT cross-references are stored on the overview but are not added to the query string.

"Acute liver failure" OR "Acute hepatic failure" OR "Fulminant hepatic failure"

Run this search on Europe PMC

MeSH descriptor terms unioned into the query: Liver Failure, Acute

ClinicalTrials.gov query (quoted phrases + MeSH via query.cond, plus recall-expansion terms when used):

"Acute liver failure" OR "Acute hepatic failure" OR "Fulminant hepatic failure" OR "Liver Failure, Acute"

Interventional trials matched via: both, phrase, mesh (mesh = registered under a MeSH descriptor no name phrase would catch; recall-expansion = gene / selected parent terms used only for trials).

Study-type breakdown: 55 interventional · 39 observational · 1 expanded access. Only interventional studies enter the trial headline.

Parent-category trials query:

"liver failure"

Query health: ok — strategies attempted: phrase, mesh; with hits: phrase, mesh

Run this search on ClinicalTrials.gov

Confidence reasoning

  • Preferred label is multi-word and distinctive
  • No synonyms dropped by stoplist
  • No label/synonym collisions with other diseases in this corpus

Ingested 2026-07-27T03:36:42.292Z