# Pancreatoblastoma

Source: https://onco.cc/cancers/pancreatoblastoma/  
OnCo record `pancreatoblastoma` (Cancer). Data CC BY-NC 4.0, attribute "Data from OnCo (onco.cc)"; commercial use needs a licence.

## TL;DR

Pancreatoblastoma is the pancreatic cancer of young children, a tumour of immature pancreatic cells that behaves quite unlike adult pancreatic cancer. It grows as a large abdominal mass, often raises the blood marker alpha-fetoprotein, and is treated like the childhood liver cancer hepatoblastoma: chemotherapy to shrink it, then surgery, which cures most children whose tumour has not spread.

## Summary

Pancreatoblastoma is an embryonal tumour that recapitulates fetal pancreatic development, with acinar, ductal and neuroendocrine differentiation and the squamoid nests that define it under the microscope. It presents in preschool children as a large, painless abdominal mass, sometimes with weight loss, vomiting or jaundice, and serum alpha-fetoprotein is raised in most. Alterations in the Wnt pathway (CTNNB1 mutations or APC loss) and loss of heterozygosity at 11p, the Beckwith-Wiedemann locus, are the recurrent genetic findings, and the tumour occurs in children with Beckwith-Wiedemann syndrome and in families with familial adenomatous polyposis. Adult cases occur and behave more aggressively.

Because the disease is so rare, treatment follows consensus from the European Cooperative Study Group for Paediatric Rare Tumours (EXPeRT) and national rare-tumour registries rather than trials. Complete surgical resection is the cornerstone, usually a pancreatoduodenectomy or distal pancreatectomy; for the large tumours that cannot be removed at diagnosis, neoadjuvant chemotherapy with cisplatin and doxorubicin (the PLADO regimen used in hepatoblastoma) shrinks the tumour and makes surgery possible, and alpha-fetoprotein is followed as a response marker. Adjuvant chemotherapy is given after incomplete resection or for metastatic disease, and radiotherapy is reserved for residual disease. Metastases, most often to the liver, occur in a minority at diagnosis and are treated with chemotherapy and resection where possible.

Children with localised, completely resected disease usually do well; outcomes are worse with metastases at diagnosis and in adults. Late effects of platinum and anthracycline chemotherapy and of pancreatic surgery, including diabetes and exocrine insufficiency, need lifelong follow-up. International registries are pooling cases to learn about relapse treatment and the role of Wnt-directed therapy.

## Fields

- Kind: Cancer
- Last checked: 2026-09-18
- Also known as: Infantile pancreatic carcinoma; Childhood pancreatic cancer; Paediatric pancreatoblastoma
- Tags: subtype-page; gastrointestinal
- Group: gastrointestinal
- Burden: The commonest pancreatic cancer of early childhood yet extremely rare, with a few cases a year in most countries, a median age around four years, and occasional cases in adults; it is associated with Beckwith-Wiedemann syndrome and familial adenomatous polyposis.
- Subtypes: Localised pancreatoblastoma, resected at diagnosis; Locally advanced pancreatoblastoma requiring neoadjuvant chemotherapy before resection; Metastatic pancreatoblastoma (liver most often); Syndromic pancreatoblastoma (Beckwith-Wiedemann syndrome, familial adenomatous polyposis); Adult pancreatoblastoma (more aggressive; distinguished from acinar cell carcinoma and ductal PDAC)
- Biomarkers: Serum alpha-fetoprotein (raised in most; response and relapse marker); Squamoid nests and acinar differentiation on histology; trypsin and BCL10 immunohistochemistry; CTNNB1 mutation or APC loss (Wnt pathway); nuclear beta-catenin staining; 11p loss of heterozygosity (Beckwith-Wiedemann locus); Germline APC testing where familial adenomatous polyposis is suspected; CT or MRI of abdomen and chest for staging

## Sections of this record

The page is a hub with ten sections in reading order; large sections have their own page. The same plan as JSON: https://onco.cc/api/v1/cancers/pancreatoblastoma/sections.json

- Overview (on the hub): The TL;DR, the family this cancer belongs to, the organ, who gets it and what the state of the art is. https://onco.cc/cancers/pancreatoblastoma/#overview [4 state-of-the-art points]
- Types and stages (on the hub): Anatomy, the subtypes and how they differ, how it is staged, and where advanced disease spreads. https://onco.cc/cancers/pancreatoblastoma/#what-it-is [5 subtypes]
- Symptoms and diagnosis (on the hub): How this cancer shows itself, how the diagnosis is confirmed, and the biomarkers clinicians test for. https://onco.cc/cancers/pancreatoblastoma/#finding-it [6 biomarkers]
- Treatment (on the hub): The standard of care by setting, the medicines, surgery and radiotherapy named in it, and the regimens behind them. https://onco.cc/cancers/pancreatoblastoma/#treating-it [5 settings, 3 decisions with options]
- Trials and papers (on the hub): Trials recruiting now, the landmark trials, the trials held by this cancer's subtypes, the key papers and what they mean, the latest literature, and the milestones year by year. https://onco.cc/cancers/pancreatoblastoma/#evidence [4 key papers, 5 milestones]
- Biology and targets (on the hub): The molecular landscape: the targets and how often each appears, the pathways, the mechanics stages and the preclinical models. https://onco.cc/cancers/pancreatoblastoma/#science [1 target, 2 pathways]
- Countries and centres (own page): Cases by country, the UK and NHS pathway and other country lenses, the expert centres with trials on record, and the centres named on this cancer's subtypes. https://onco.cc/cancers/pancreatoblastoma/where-you-are/
- Decisions and support (on the hub): The decisions you may face, the aids that walk through them, the warnings on record, the first sixty days and the questions to ask. https://onco.cc/cancers/pancreatoblastoma/#living-with-it [15 questions, 6 red cards]
- Pipeline and open problems (own page): Everything in development, the medicines held by this cancer's subtypes, the open problems and what is being done about them, the roadmaps, and what changed on this record. https://onco.cc/cancers/pancreatoblastoma/coming/ [3 medicines, 4 open problems]
- Data (own page): Every connected record, the notes, the JSON, Markdown and RDF twins, and where the record came from and when it was checked. https://onco.cc/cancers/pancreatoblastoma/data/ [26 connected records]

## Standard of care

- Diagnosis and staging: Imaging of abdomen and chest, serum alpha-fetoprotein and biopsy; genetic review for Beckwith-Wiedemann syndrome and familial adenomatous polyposis. ([CT (computed tomography)](https://onco.cc/technologies/ct/), [MRI](https://onco.cc/technologies/mri/), [Germline (hereditary) testing](https://onco.cc/technologies/germline-testing/))
- Resectable at diagnosis: Complete resection (pancreatoduodenectomy or distal pancreatectomy); adjuvant chemotherapy after incomplete resection. ([Whipple procedure (pancreaticoduodenectomy)](https://onco.cc/terms/whipple/), [Resection margins (R0 / R1 / R2)](https://onco.cc/terms/resection-margins/))
- Unresectable at diagnosis: Neoadjuvant cisplatin and doxorubicin (PLADO, as in hepatoblastoma) followed by delayed resection when the tumour shrinks. ([Cisplatin](https://onco.cc/drugs/cisplatin/), [Doxorubicin](https://onco.cc/drugs/doxorubicin/), [Neoadjuvant / adjuvant / perioperative](https://onco.cc/terms/neoadjuvant-adjuvant/))
- Metastatic or relapsed: Cisplatin and doxorubicin-based chemotherapy with resection of residual disease where possible; radiotherapy for unresectable residual tumour; international registry enrolment. ([Cisplatin](https://onco.cc/drugs/cisplatin/), [Doxorubicin](https://onco.cc/drugs/doxorubicin/), [Metastasis](https://onco.cc/terms/metastasis/))
- Survivorship: Lifelong follow-up for pancreatic insufficiency, diabetes and the late effects of platinum and anthracycline chemotherapy. ([Oncofertility and fertility preservation](https://onco.cc/technologies/fertility-preservation/), [Cancer cachexia](https://onco.cc/terms/cachexia/))

## State of the art

- EXPeRT consensus recommendations (2021) give a shared treatment pathway across Europe for a tumour no single country sees often.
- Hepatoblastoma-style neoadjuvant chemotherapy makes most large tumours resectable.
- Recognition of the Wnt pathway and Beckwith-Wiedemann link explains the biology and guides genetic counselling.
- International rare-tumour registries are pooling cases to study relapse and late effects.

## Open problems

- No prospective trial exists or is likely; treatment is extrapolated from hepatoblastoma and pooled registries.
- Relapsed and metastatic disease has no established second-line therapy.
- Adult pancreatoblastoma is often misdiagnosed and behaves worse than the childhood form.
- Survivors face pancreatic insufficiency and diabetes after major pancreatic surgery in early life.

## Sources

- Wikipedia: https://en.wikipedia.org/wiki/Pancreatoblastoma
- Wikipedia: https://en.wikipedia.org/wiki/Pancreatoblastoma
- EXPeRT consensus on pancreatoblastoma (2021): https://doi.org/10.1002/pbc.29112

## Connected records

- cancers: [Childhood cancers (all types)](https://onco.cc/cancers/childhood-cancers/), [Hepatoblastoma](https://onco.cc/cancers/hepatoblastoma/), [Pancreatic acinar cell carcinoma](https://onco.cc/cancers/pancreatic-acinar-cell-carcinoma/), [Pancreatic ductal adenocarcinoma](https://onco.cc/cancers/pancreatic/), [Pancreatic neuroendocrine tumours](https://onco.cc/cancers/pancreatic-net/), [Rare cancers of childhood (NCI PDQ umbrella)](https://onco.cc/cancers/rare-childhood-cancers/), [Resectable pancreatic ductal adenocarcinoma](https://onco.cc/cancers/resectable-pdac/), [Solid pseudopapillary neoplasm of the pancreas](https://onco.cc/cancers/solid-pseudopapillary-neoplasm/)
- technologies: [CT (computed tomography)](https://onco.cc/technologies/ct/), [Cytotoxic chemotherapy](https://onco.cc/technologies/cytotoxic-chemotherapy/), [Germline (hereditary) testing](https://onco.cc/technologies/germline-testing/), [MRI](https://onco.cc/technologies/mri/), [Oncofertility and fertility preservation](https://onco.cc/technologies/fertility-preservation/)
- targets: [KRAS](https://onco.cc/targets/kras/)
- drugs: [Cisplatin](https://onco.cc/drugs/cisplatin/), [Doxorubicin](https://onco.cc/drugs/doxorubicin/), [Vincristine](https://onco.cc/drugs/vincristine/)
- pathways: [Pancreatic cancer (KEGG map)](https://onco.cc/pathways/pancreatic-cancer-signalling/), [Wnt / β-catenin](https://onco.cc/pathways/wnt/)
- terms: [Cancer cachexia](https://onco.cc/terms/cachexia/), [Grade](https://onco.cc/terms/tumour-grade/), [Metastasis](https://onco.cc/terms/metastasis/), [Neoadjuvant / adjuvant / perioperative](https://onco.cc/terms/neoadjuvant-adjuvant/), [Pancreatic enzyme replacement therapy (PERT, pancreatin, Creon) for pancreatic exocrine insufficiency: why and how to take it](https://onco.cc/terms/pancreatic-enzyme-replacement/), [Resection margins (R0 / R1 / R2)](https://onco.cc/terms/resection-margins/), [Whipple procedure (pancreaticoduodenectomy)](https://onco.cc/terms/whipple/)
- key papers: [Bien 2011: pancreatoblastoma, a report from the European cooperative study group for paediatric rare tumours (EXPeRT)](https://onco.cc/key-papers/paper-bien-pancreatoblastoma-expert-european-cooperative-ejc-2011/), [Dhebri 2004: diagnosis, treatment and outcome of pancreatoblastoma](https://onco.cc/key-papers/paper-dhebri-pancreatoblastoma-diagnosis-treatment-outcome-pancreatology-2004/), [Klimstra 1995: pancreatoblastoma, a clinicopathologic study and review of the literature](https://onco.cc/key-papers/paper-klimstra-pancreatoblastoma-clinicopathologic-study-ajsp-1995/), [Pancreatoblastoma in children: EXPeRT/PARTNER diagnostic and therapeutic recommendations](https://onco.cc/key-papers/paper-bien-pediatr-blood-cancer/)

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JSON: https://onco.cc/api/v1/entities/pancreatoblastoma.json