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ORPHA:210159

Adult hepatocellular carcinoma

high confidenceDisorder

Also known as: Adult HCC

Publications

608

81.7th percentile

Trials

62

Interventional, condition-specific

Researchers

1,409

Distinct authors in sample

Gene link

—

Readiness

3/6

Stages with a signal

Clinical definition (Orphanet)

A rare carcinoma of the liver characterized by one to several or many nodules occurring anywhere within the liver, composed of neoplastic epithelial cells with hepatocellular differentiation. The vast majority of tumors are associated with chronic liver disease (such as hepatitis B or C, or steatohepatitis) or exposure to a variety of exogenous agents. Patients may present with signs and symptoms related to the tumor, as well as to the underlying condition. Common manifestations include right upper quadrant abdominal pain, weight loss, , jaundice, and ascites. Symptomatic tumors generally have poor prognosis.

How rare: 1-9 / 100 000 — about one to nine people per hundred thousand.

Orphanet entry

Cross-references

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

Additional Mondo synonyms (9)

adult HCC · adult hepatocellular carcinoma · adult hepatoma · adult primary carcinoma of liver cell · adult primary carcinoma of the liver cell · adult primary hepatocellular carcinoma · adult primary hepatoma · adult primary liver cell carcinoma · hepatocellular carcinoma of adults

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.”

3/6 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

    608 matched papers (432 in last 10 years) Source

  3. Phenotype characterisedPresent

    1 HPO annotations (e.g. Adult onset) Source

  4. Animal modelNot found

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

  5. Orphan designationNot found

    No FDA or EMA orphan-drug designation matched this disease via UMLS or preferred name Source

  6. Interventional trialPresent

    62 matched on ClinicalTrials.gov

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)

1

Associated phenotypes · MONDO:0016216

  • Adult onset

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)

No designation matched this disease via UMLS or preferred name on the FDA OOPD mirror or EMA orphan register. Absence here is not proof that none exists under another wording.

Open Targets candidates

No drugs or clinical candidates returned for this Mondo ID on Open Targets.

CTD chemicals (MyDisease.info)

No CTD chemical associations returned for this Mondo ID.

Literature

Is anyone studying this?

608

608 papers have been published on this condition. That is a real research literature — still far smaller than common diseases (breast cancer: over 700,000 papers). Median papers in the last 10 years for a rare disease in this dataset (publications denominator n=3967) is 59.

608 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).

432 in the last 10 years · high confidence · 81.7th percentile (publications denominator)

Phrase hits: 608 · MeSH hits: 0

Open Europe PMC search

Who's working on it?

1,409

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 Z6 papers · 2026

    Department of Immunotherapy, Affiliated Cancer Hospital of Zhengzhou University & Henan Cancer Hospital, Zhengzhou, China.

    Papers in Europe PMC
  2. 02
    Zhang J6 papers · 2026

    Department of Pediatric Surgery, The First Affiliated Hospital of Zhengzhou University, Zhengzhou, Henan 450052, China.

    Papers in Europe PMC
  3. 03
    Chen Z5 papers · 2025

    Department of Pediatric Surgery, Capital Institute of Pediatrics, Beijing 100020, China.

    Papers in Europe PMC
  4. 04
    Li L5 papers · 2025

    Children's Research Institute, Departments of Pediatrics and Internal Medicine, Center for Regenerative Science and Medicine, Simmons Comprehensive Cancer Center, University of Texas Southwestern Medical Center, Dallas, Texas, USA.

    Papers in Europe PMC
  5. 05
    Zhou J5 papers · 2025

    Medical Oncology Department of Gastrointestinal Cancer, Liaoning Cancer Hospital & Institute, Cancer Hospital of Dalian University of Technology, Liaoning, Shenyang, China.

    Papers in Europe PMC
  6. 06
    Wang T4 papers · 2026

    Department of Pharmacy, Second Affiliated Hospital of Zhejiang University School of Medicine, Hangzhou, China.

    Papers in Europe PMC
  7. 07
    Yang J4 papers · 2025

    Department of Liver Surgery, Liver Transplantation Center, West China Hospital of Sichuan University, Chengdu, Sichuan Province, China.

    Papers in Europe PMC
  8. 08
    Zhang W4 papers · 2026

    Department of Colorectal Surgery, Shanghai Changhai Hospital, Naval Medical University, Shanghai, China.

    Papers in Europe PMC
  9. 09
    Zhang X4 papers · 2026

    Department of Immunotherapy, Affiliated Cancer Hospital of Zhengzhou University & Henan Cancer Hospital, Zhengzhou, China.

    Papers in Europe PMC
  10. 10
    Casazza G3 papers · 2022

    Department of Clinical Sciences and Community Health - Laboratory of Medical Statistics, Biometry and Epidemiology "G.A. Maccacaro", Università degli Studi di Milano, Milan, Italy.

    Papers in Europe PMC

Clinical research

Is a treatment being tested?

62

interventional trials for this specific condition

62 interventional trials matched this specific condition name; none in our sample are currently recruiting. 2,300 trials are registered for hepatocellular carcinoma, the broader category — shown separately because they may or may not enrol this specific subtype.

Data as of 11 September 2026

62 interventional trials — more than 77.2% of diseases in the trials denominator have none at all (5501 of 7126; this disease is at the 97.6th percentile).

high confidence · 97.6th percentile (trials denominator)

Recruiting interventional trials

From the matched ClinicalTrials.gov set

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

No currently recruiting studies in the matched set. Open the same search on ClinicalTrials.gov.

Broader category: hepatocellular carcinoma

2,300

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

1 observational study matches 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.

None of the matched observational studies is currently listed as 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 65 · after dedupe 65 · already on CT.gov 0 · kept 0 · parent 0 · uncertain 65 · 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 (65)

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 Adult hepatocellular carcinoma — 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.

"Adult hepatocellular carcinoma" OR "Adult HCC" OR "adult hepatoma" OR "adult primary carcinoma of liver cell" OR "adult primary carcinoma of the liver cell" OR "adult primary hepatocellular carcinoma" OR "adult primary hepatoma" OR "adult primary liver cell carcinoma" OR "hepatocellular carcinoma of adults" OR "hepatocellular carcinoma of the adults"

Run this search on Europe PMC

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

"Adult hepatocellular carcinoma" OR "Adult HCC" OR "adult hepatoma" OR "adult primary carcinoma of liver cell" OR "adult primary carcinoma of the liver cell" OR "adult primary hepatocellular carcinoma" OR "adult primary hepatoma" OR "adult primary liver cell carcinoma" OR "hepatocellular carcinoma of adults" OR "hepatocellular carcinoma of the adults"

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

Study-type breakdown: 62 interventional · 1 observational · 0 expanded access. Only interventional studies enter the trial headline.

Parent-category trials query:

"hepatocellular carcinoma"

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

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 2026-07-27T09:30:59.071Z