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ORPHA:33402

Pediatric hepatocellular carcinoma

high confidenceDisorder

Also known as: Childhood-onset HCC · Childhood-onset hepatocellular carcinoma · Pediatric HCC

Publications

389

81.4th percentile

Trials

6

Interventional, condition-specific

Researchers

1,222

Distinct authors in sample

Gene link

—

Readiness

2/6

Stages with a signal

Clinical definition (Orphanet)

A rare, aggressive and malignant hepatic tumor arising from the hepatocytes. It develops mainly in children over 10 years of age, either in a cirrhotic background, or more commonly in a non-cirrhotic background (70% of cases).

How rare: How common this is has not been clearly measured.

Orphanet entry

Cross-references

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

Additional Mondo synonyms (8)

Paediatric carcinoma of the liver cell · Pediatric carcinoma of the liver cell · childhood carcinoma of the liver cell · childhood hepatocellular carcinoma · childhood-onset HCC · childhood-onset hepatocellular carcinoma · paediatric HCC · pediatric HCC

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.”

2/6 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

    389 matched papers (241 in last 10 years) Source

  3. Phenotype characterisedNot checked

    Not yet enriched from Monarch / HPO

  4. Animal modelNot checked

    Not yet enriched from Monarch / Alliance

  5. Orphan designationNot checked

    FDA/EMA orphan-drug designation not enriched yet

  6. Interventional trialPresent

    6 matched on ClinicalTrials.gov (1 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)

Not enriched in this build — Monarch phenotype joins were not run for this record.

Animal models (Monarch / Alliance)

Not enriched in this build.

Literature

Is anyone studying this?

389

389 papers have ever been published on this condition (under this name). For scale, breast cancer has over 700,000. Median papers in the last 10 years for a rare disease in this dataset (publications denominator n=5449) is 41.

389 papers since the earliest indexed year in this search — median last-10-year count for a rare disease in this dataset is 41 (publications denominator n=5449).

241 in the last 10 years · high confidence · 81.4th percentile (publications denominator)

Phrase hits: 389 · MeSH hits: 0

Open Europe PMC search

Who's working on it?

1,222

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
    O'Neill AF7 papers · 2026

    Department of Pediatric Oncology, Dana-Farber Cancer Institute/Boston Children's Cancer and Blood Disorders Center and Harvard Medical School, Boston, Massachusetts, USA.

    Papers in Europe PMC
  2. 02
    Towbin AJ7 papers · 2022

    Department of Radiology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.

    Papers in Europe PMC
  3. 03
    Vasudevan SA7 papers · 2026

    Divisions of Pediatric Surgery and Surgical Research, Michael E. DeBakey Department of Surgery, Pediatric Surgical Oncology Laboratory, Texas Children's Surgical Oncology Program, Texas Children's Liver Tumor Program, Dan L. Duncan Cancer Center, Baylor College of Medicine, Houston, TX 77030, USA.

    Papers in Europe PMC
  4. 04
    Bondoc A6 papers · 2025

    Division of Pediatric General and Thoracic Surgery, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio.

    Papers in Europe PMC
  5. 05
    Fuchs J6 papers · 2022

    Department of Pediatric Surgery and Pediatric Urology, University of Tuebingen, Hoppe-Seyler-Str. 3, Tübingen 72076, Germany.

    Papers in Europe PMC
  6. 06
    Geller JI6 papers · 2026

    Division of Oncology, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio.

    Papers in Europe PMC
  7. 07
    Czauderna P5 papers · 2026

    Maciej Murawski and Piotr Czauderna, Medical University of Gdansk, Gdansk, Poland; Viola B. Weeda and Daniel C. Aronson, Emma Children's Hospital Academic Medical Center, Amsterdam, the Netherlands; Rudolf Maibach, International Breast Cancer Study Group Coordinating Center; Arthur Zimmerman, Institute of Pathology, Berne, Switzerland; Bruce Morland, Birmingham Children's Hospital, Birmingham; Derek J. Roebuck, Great Ormond Street Hospital, London; Margaret Childs, University of Nottingham, Nottingham, United Kingdom; Michela Casanova, National Cancer Institute, Milan; Giorgio Perilongo, University of Padova, Padova, Italy; Veronique Laithier, Centre Hospitalier Régional et Universitaire de Besançon, Besançon, France; Rejin Kebudi, İstanbul University, Oncology Institute, İstanbul, Turkey; Marcelo J. Scopinaro, Hospital de Pediatrı´a SAMIC J.P. Garrahan, Buenos Aires, Argentina; Albert Shun, Children's Hospital at Westmead, Sydney, Australia; Benedicte Brichard, Cliniques Universitaires Saint Luc, University of Louvain, Brussels, Belgium; and Beatriz de Camargo, Cancer Hospital A.C. Camargo, São Paulo, Brazil. pczaud@gumed.edu.pl.

    Papers in Europe PMC
  8. 08
    Tiao G5 papers · 2025

    Division of Pediatric General and Thoracic Surgery, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio.

    Papers in Europe PMC
  9. 09
    von Schweinitz D5 papers · 2025

    Department of Pediatric Surgery, Dr. von Hauner Children's Hospital, Ludwig-Maximilians-University Munich, LMU Munich, Lindwurmstr. 2a, 80337, Munich, Germany.

    Papers in Europe PMC
  10. 10
    Aerts I4 papers · 2026

    Oncology Center SIREDO, Institut Curie, PSL Research University, Paris, France.

    Papers in Europe PMC

Clinical research

Is a treatment being tested?

6

interventional trials for this specific condition

6 interventional trials matched this specific condition name; 1 currently recruiting in our sample. 2,356 trials are registered for hepatocellular carcinoma, the broader category — shown separately because they may or may not enrol this specific subtype.

Data as of 27 July 2026

6 interventional trials — more than 73% of diseases in the trials denominator have none at all (5114 of 7003; this disease is at the 89th percentile).

high confidence · 89th percentile (trials denominator)

Recruiting interventional trials

From the matched ClinicalTrials.gov set

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

Broader category: hepatocellular carcinoma

2,356

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

Where to find support

We do not yet link condition-specific patient organisations. These umbrella groups support undiagnosed and ultra-rare families:

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.

"Pediatric hepatocellular carcinoma" OR "Childhood-onset HCC" OR "Childhood-onset hepatocellular carcinoma" OR "Pediatric HCC" OR "Paediatric carcinoma of the liver cell" OR "Paediatric carcinoma of liver cell" OR "Pediatric carcinoma of the liver cell" OR "Pediatric carcinoma of liver cell" OR "childhood carcinoma of the liver cell" OR "childhood carcinoma of liver cell" OR "childhood hepatocellular carcinoma" OR "paediatric HCC"

Run this search on Europe PMC

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

"Pediatric hepatocellular carcinoma" OR "Childhood-onset HCC" OR "Childhood-onset hepatocellular carcinoma" OR "Pediatric HCC" OR "Paediatric carcinoma of the liver cell" OR "Paediatric carcinoma of liver cell" OR "Pediatric carcinoma of the liver cell" OR "Pediatric carcinoma of liver cell" OR "childhood carcinoma of the liver cell" OR "childhood carcinoma of liver cell" OR "childhood hepatocellular carcinoma" OR "paediatric HCC"

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: 6 interventional · 0 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 2026-07-26T23:35:51.852Z