{"entity":{"id":"pancreatoblastoma","kind":"cancer","name":"Pancreatoblastoma","aka":["Infantile pancreatic carcinoma","Childhood pancreatic cancer","Paediatric pancreatoblastoma"],"tldr":"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.\n\nBecause 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.\n\nChildren 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.","asOf":"2026-09-18","wikipedia":"https://en.wikipedia.org/wiki/Pancreatoblastoma","links":[{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Pancreatoblastoma"},{"label":"EXPeRT consensus on pancreatoblastoma (2021)","url":"https://doi.org/10.1002/pbc.29112"}],"tags":["subtype-page","gastrointestinal"],"related":["pancreatic-acinar-cell-carcinoma","resectable-pdac","pancreatic-net","hepatoblastoma","rare-childhood-cancers","childhood-cancers"],"cancers":[],"sections":[],"technologies":["cytotoxic-chemotherapy","ct","mri","germline-testing","fertility-preservation"],"targets":["kras"],"drugs":["cisplatin","doxorubicin","vincristine"],"companies":[],"institutions":[],"pathways":["wnt","pancreatic-cancer-signalling"],"terms":["whipple","resection-margins","neoadjuvant-adjuvant","metastasis","tumour-grade"],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"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"],"standardOfCare":[{"setting":"Diagnosis and staging","approach":"Imaging of abdomen and chest, serum alpha-fetoprotein and biopsy; genetic review for Beckwith-Wiedemann syndrome and familial adenomatous polyposis.","refs":["ct","mri","germline-testing"],"guideline":{"version":"EXPeRT consensus on pancreatoblastoma (Pediatric Blood and Cancer 2021)","url":"https://doi.org/10.1002/pbc.29112"}},{"setting":"Resectable at diagnosis","approach":"Complete resection (pancreatoduodenectomy or distal pancreatectomy); adjuvant chemotherapy after incomplete resection.","refs":["whipple","resection-margins"],"guideline":{"version":"EXPeRT consensus on pancreatoblastoma (Pediatric Blood and Cancer 2021)","url":"https://doi.org/10.1002/pbc.29112"}},{"setting":"Unresectable at diagnosis","approach":"Neoadjuvant cisplatin and doxorubicin (PLADO, as in hepatoblastoma) followed by delayed resection when the tumour shrinks.","refs":["cisplatin","doxorubicin","neoadjuvant-adjuvant"],"guideline":{"version":"EXPeRT consensus on pancreatoblastoma (Pediatric Blood and Cancer 2021)","url":"https://doi.org/10.1002/pbc.29112"}},{"setting":"Metastatic or relapsed","approach":"Cisplatin and doxorubicin-based chemotherapy with resection of residual disease where possible; radiotherapy for unresectable residual tumour; international registry enrolment.","refs":["cisplatin","doxorubicin","metastasis"],"guideline":{"version":"EXPeRT consensus on pancreatoblastoma (Pediatric Blood and Cancer 2021)","url":"https://doi.org/10.1002/pbc.29112"}},{"setting":"Survivorship","approach":"Lifelong follow-up for pancreatic insufficiency, diabetes and the late effects of platinum and anthracycline chemotherapy.","refs":["fertility-preservation","cachexia"],"guideline":{"version":"EXPeRT consensus on pancreatoblastoma (Pediatric Blood and Cancer 2021)","url":"https://doi.org/10.1002/pbc.29112"}}],"stateOfArt":["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."],"history":[{"year":1957,"title":"Becker describes an infantile pancreatic carcinoma later named pancreatoblastoma","refs":["whipple"]},{"year":1977,"title":"Horie coins the term pancreatoblastoma for the childhood tumour with squamoid nests","refs":["tumour-grade"]},{"year":2001,"title":"Beta-catenin and APC alterations link pancreatoblastoma to the Wnt pathway and to familial adenomatous polyposis","refs":["wnt"]},{"year":2011,"title":"EXPeRT pooled analysis defines outcomes and the role of neoadjuvant chemotherapy and complete resection","refs":["cisplatin","doxorubicin"]},{"year":2021,"title":"EXPeRT consensus recommendations for diagnosis and treatment","refs":["whipple","cisplatin"]}],"pipeline":[],"openProblems":["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."],"parent":"pancreatic"},"route":"/cancers/pancreatoblastoma/","neighbours":{"cancer":[{"id":"childhood-cancers","kind":"cancer","name":"Childhood cancers (all types)","route":"/cancers/childhood-cancers/"},{"id":"hepatoblastoma","kind":"cancer","name":"Hepatoblastoma","route":"/cancers/hepatoblastoma/"},{"id":"pancreatic-acinar-cell-carcinoma","kind":"cancer","name":"Pancreatic acinar cell carcinoma","route":"/cancers/pancreatic-acinar-cell-carcinoma/"},{"id":"pancreatic","kind":"cancer","name":"Pancreatic ductal adenocarcinoma","route":"/cancers/pancreatic/"},{"id":"pancreatic-net","kind":"cancer","name":"Pancreatic neuroendocrine tumours","route":"/cancers/pancreatic-net/"},{"id":"rare-childhood-cancers","kind":"cancer","name":"Rare cancers of childhood (NCI PDQ umbrella)","route":"/cancers/rare-childhood-cancers/"},{"id":"resectable-pdac","kind":"cancer","name":"Resectable pancreatic ductal adenocarcinoma","route":"/cancers/resectable-pdac/"}],"technology":[{"id":"ct","kind":"technology","name":"CT (computed tomography)","route":"/technologies/ct/"},{"id":"cytotoxic-chemotherapy","kind":"technology","name":"Cytotoxic chemotherapy","route":"/technologies/cytotoxic-chemotherapy/"},{"id":"germline-testing","kind":"technology","name":"Germline (hereditary) testing","route":"/technologies/germline-testing/"},{"id":"mri","kind":"technology","name":"MRI","route":"/technologies/mri/"},{"id":"fertility-preservation","kind":"technology","name":"Oncofertility and fertility preservation","route":"/technologies/fertility-preservation/"}],"target":[{"id":"kras","kind":"target","name":"KRAS","route":"/targets/kras/"}],"drug":[{"id":"cisplatin","kind":"drug","name":"Cisplatin","route":"/drugs/cisplatin/"},{"id":"doxorubicin","kind":"drug","name":"Doxorubicin","route":"/drugs/doxorubicin/"},{"id":"vincristine","kind":"drug","name":"Vincristine","route":"/drugs/vincristine/"}],"pathway":[{"id":"pancreatic-cancer-signalling","kind":"pathway","name":"Pancreatic cancer (KEGG map)","route":"/pathways/pancreatic-cancer-signalling/"},{"id":"wnt","kind":"pathway","name":"Wnt / β-catenin","route":"/pathways/wnt/"}],"term":[{"id":"cachexia","kind":"term","name":"Cancer cachexia","route":"/terms/cachexia/"},{"id":"tumour-grade","kind":"term","name":"Grade","route":"/terms/tumour-grade/"},{"id":"metastasis","kind":"term","name":"Metastasis","route":"/terms/metastasis/"},{"id":"neoadjuvant-adjuvant","kind":"term","name":"Neoadjuvant / adjuvant / perioperative","route":"/terms/neoadjuvant-adjuvant/"},{"id":"resection-margins","kind":"term","name":"Resection margins (R0 / R1 / R2)","route":"/terms/resection-margins/"},{"id":"whipple","kind":"term","name":"Whipple procedure (pancreaticoduodenectomy)","route":"/terms/whipple/"}]}}