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Malignant pancreatic tumors are rare in children and adolescents, with an incidence of 0.46 cases per 1 million individuals younger than 30 years.[1,2,3,4] The primary pancreatic tumors of childhood can be classified into the following four categories: References: Cancer in children and adolescents is rare, although the overall incidence has been slowly increasing since 1975.[1] Children and adolescents with cancer should be referred to medical centers that have a multidisciplinary team of cancer specialists with experience treating the cancers that occur during childhood and adolescence. This multidisciplinary team approach incorporates the skills of the following pediatric specialists and others to ensure that children receive treatment, supportive care, and rehabilitation that will achieve optimal survival and quality of life: For specific information about supportive care for children and adolescents with cancer, see the summaries on Supportive and Palliative Care. The American Academy of Pediatrics has outlined guidelines for pediatric cancer centers and their role in the treatment of children and adolescents with cancer.[2] At these centers, clinical trials are available for most types of cancer that occur in children and adolescents, and the opportunity to participate is offered to most patients and their families. Clinical trials for children and adolescents diagnosed with cancer are generally designed to compare potentially better therapy with current standard therapy. Other types of clinical trials test novel therapies when there is no standard therapy for a cancer diagnosis. Most of the progress in identifying curative therapies for childhood cancers has been achieved through clinical trials. Information about ongoing clinical trials is available from the NCI website. Dramatic improvements in survival have been achieved for children and adolescents with cancer. Between 1975 and 2020, childhood cancer mortality decreased by more than 50%.[3,4,5] Childhood and adolescent cancer survivors require close monitoring because side effects of cancer therapy may persist or develop months or years after treatment. For information about the incidence, type, and monitoring of late effects in childhood and adolescent cancer survivors, see Late Effects of Treatment for Childhood Cancer. Childhood cancer is a rare disease, with about 15,000 cases diagnosed annually in the United States in individuals younger than 20 years.[6] The U.S. Rare Diseases Act of 2002 defines a rare disease as one that affects populations smaller than 200,000 people in the United States. Therefore, all pediatric cancers are considered rare. The designation of a rare tumor is not uniform among pediatric and adult groups. In adults, rare cancers are defined as those with an annual incidence of fewer than six cases per 100,000 people. They account for up to 24% of all cancers diagnosed in the European Union and about 20% of all cancers diagnosed in the United States.[7,8] In children and adolescents, the designation of a rare tumor is not uniform among international groups, as follows: Most cancers in subgroup XI are either melanomas or thyroid cancers, with other cancer types accounting for only 2% of the cancers diagnosed in children aged 0 to 14 years and 9.3% of the cancers diagnosed in adolescents aged 15 to 19 years. These rare cancers are extremely challenging to study because of the relatively few patients with any individual diagnosis, the predominance of rare cancers in the adolescent population, and the low number of clinical trials for adolescents with rare cancers. Information about these tumors may also be found in sources relevant to adults with cancer, such as Pancreatic Cancer Treatment and Pancreatic Neuroendocrine Tumors (Islet Cell Tumors) Treatment. References: Incidence Solid pseudopapillary tumor of the pancreas, also known as Frantz tumor, is the most common pediatric pancreatic tumor, accounting for up to 70% of cases in most institutional series.[1,2] This tumor has low malignant potential and most commonly affects females of reproductive age (median age, 21 years), Black people, and East Asian people.[2,3,4] There is no known genetic or hormonal factor to explain the strong female predilection, although it has been noted that all tumors express progesterone receptors.[5] Histology and Genomic Alterations Histologically, solid pseudopapillary tumor of the pancreas is characterized by a combination of solid, pseudopapillary, and cystic changes. The fragility of the vascular supply leads to secondary degenerative changes and cystic areas of hemorrhage and necrosis.[2,3,4] The cells surrounding the hyalinized fibrovascular stalks form the pseudopapillae.[3] A highly specific, paranuclear, dot-like immunoreactivity pattern for CD99 has been described.[6] Variants in the CTNNB1 gene have been identified in more than 90% of these tumors.[7] Diagnosis Solid pseudopapillary tumor of the pancreas is a very friable tumor, and tumor rupture and hemoperitoneum have been reported.[2,3,4] Tumors can occur throughout the pancreas and are often exophytic. On imaging, the mass shows typical cystic and solid components, with intratumoral hemorrhage and a fibrous capsule.[3] A retrospective review of the National Cancer Database identified 21 pediatric patients (younger than 18 years) and 348 adult patients with solid pseudopapillary neoplasm of the pancreas.[8] When compared with their adult counterparts, the children had similar disease severity at presentation, received similar treatments, and experienced equivalent postoperative outcomes. Treatment of Solid Pseudopapillary Tumor of the Pancreas The outcomes of patients with solid pseudopapillary tumors of the pancreas are excellent, with 10-year survival rates exceeding 95%.[5] Treatment options for solid pseudopapillary tumor of the pancreas include the following: Surgery Treatment of solid pseudopapillary tumor of the pancreas is primarily surgical. However, preoperative and operative spillage is not unusual.[9] Whipple procedures (pancreaticoduodenectomy) are often necessary, but non-Whipple, pancreatic-sparing resections may be possible with a pancreatico-jejunostomy procedure. Surgery is usually curative, although local recurrences occur in 5% to 15% of cases.[4] A retrospective review of the Italian Pediatric Rare Tumor Registry identified 43 pediatric patients diagnosed with solid pseudopapillary tumor of the pancreas between 2000 and 2018.[10][Level of evidence C1] The median age at diagnosis was 13.2 years (range, 7–18 years). Only one patient presented with metastatic disease. A study identified 38 children aged 0 to 18 years who were diagnosed with pseudopapillary solid tumor of the pancreas between 2008 and 2022 in a German registry.[11] Patients were a median age of 14.5 years at diagnosis (range, 8–18 years), with a female preponderance (81.6%). The pancreatic tail was the most frequent location of the tumor. All patients underwent surgical resection. Chemotherapy Single-agent gemcitabine is reportedly effective in cases of unresectable or metastatic disease.[12] Metastatic disease, usually in the liver, may occur in up to 15% of cases.[2,3,4,5,6] References: Incidence and Risk Factors Pancreatoblastoma accounts for 10% to 20% of all pancreatic tumors during childhood. It is the most common pancreatic tumor of young children and typically presents in the first decade of life, with a median age at diagnosis of 5 years.[1,2] Patients with Beckwith-Wiedemann syndrome have an increased risk of developing pancreatoblastoma. This syndrome is identified in up to 60% of children who develop pancreatoblastoma during early infancy and in 5% of children who develop pancreatoblastoma later in life.[3] Pancreatoblastoma has also been associated with familial adenomatous polyposis syndromes.[4] Clinical Presentation Although approximately one-half of pancreatoblastoma cases originate in the head of the pancreas, jaundice is uncommon. Close to 80% of the tumors secrete alpha-fetoprotein, which can be used to measure response to therapy and monitor for recurrence.[2] In some cases, the tumor may secrete adrenocorticotropic hormone or antidiuretic hormone, and patients may present with Cushing syndrome and the syndrome of inappropriate antidiuretic hormone secretion.[3] Metastases are present in 30% to 40% of patients, usually involving liver, lungs, and lymph nodes.[2] Histology and Molecular Features Pancreatoblastoma is thought to arise from the persistence of the fetal analogue of pancreatic acinar cells. Pathological examination shows an epithelial neoplasm with an arrangement of acinar, trabecular, or solid formations separated by dense stromal bands.[1] These tumors will often have activation of WNT signaling (most commonly caused by somatic variants in CTNNB1). IGF2 gene alterations have also been frequently observed in individuals with pancreatoblastoma. These findings suggest that pancreatoblastoma might result from the disruption of normal pancreas differentiation.[5,6] Treatment of Pancreatoblastoma The European Cooperative Study Group for Pediatric Rare Tumors within the PARTNER project (Paediatric Rare Tumours Network - European Registry) has published consensus guidelines for the diagnosis and treatment of childhood pancreatoblastoma.[7] Using a multimodality approach, close to 80% of patients can be cured.[2] Treatment options for pancreatoblastoma include the following: Surgery Surgery is the main treatment for pancreatoblastoma, and a complete surgical resection is required for cure. Because of the common origin of the tumor in the head of the pancreas, a Whipple procedure is usually required.[8,9] Chemotherapy For large, unresectable, or metastatic tumors, preoperative chemotherapy is indicated. Pancreatoblastoma often responds to chemotherapy. A cisplatin-based regimen is usually recommended. The PLADO regimen, which includes cisplatin and doxorubicin, is most commonly used. Treatment is modeled after the management of hepatoblastoma, with two to three cycles of preoperative therapy, followed by resection and adjuvant chemotherapy.[2,4,10,11] Patients with relapsed or persistent pancreatoblastoma have responded to treatment with gemcitabine (one case) [12] and vinorelbine and oral cyclophosphamide (two cases).[13] Other treatments Although radiation therapy has been used for unresectable tumors and relapsed cases, its role in the treatment of microscopic disease after surgery has not been defined.[4] High-dose chemotherapy with autologous hematopoietic stem cell rescue has been reported to be effective in selected cases.[10,14] References: Incidence Islet cell tumors represent approximately 15% of pediatric pancreatic tumors in most series.[1,2,3] These tumors usually present in middle age and may be associated with multiple endocrine neoplasia type 1 (MEN1) syndrome. Less than 5% of islet cell tumors occur in children.[4] For more information, see Childhood Multiple Endocrine Neoplasia (MEN) Syndromes Treatment. A single referral center in Russia retrospectively identified 22 children (aged 5–16 years) who were diagnosed with insulinomas.[5] Five patients (23%) had multiple pancreatic lesions. All children underwent surgical treatment. Two patients were diagnosed with metastatic insulinomas. One of them had metastases at the time of insulinoma diagnosis, while the other was diagnosed with liver metastases 8 years after surgery. Eight children (36%) were found to carry MEN1 variants (inherited, n = 5; de novo, n = 1; no data, n = 2). Children with MEN1 syndrome had a significantly higher number of pancreatic tumors than children without MEN1 syndrome. All of the MEN1 carriers developed additional MEN1 symptoms during the following 2 to 13 years. After five patients were diagnosed with inherited MEN1 syndrome, previously undiscovered MEN1 manifestations were found in seven family members. Clinical Presentation, Risk Factors, and Diagnosis The most common type of functioning islet cell tumor is insulinoma, followed by gastrinoma. Nonfunctioning tumors are extremely rare in pediatrics, except when associated with MEN1 syndrome. Islet cell tumors are typically solitary. When multiple tumors are present, a diagnosis of MEN1 syndrome should be considered. On imaging, these tumors are usually small and well defined. Somatostatin receptor scintigraphy is useful for detecting the location of islet cell tumors. However, only 60% to 70% of tumors express somatostatin receptors.[4] Treatment of Islet Cell Tumors Treatment options for islet cell tumors include the following: Treatment of islet cell tumors includes medical therapy for control of the syndrome and complete surgical resection.[6] For patients with malignant tumors and unresectable or metastatic disease, chemotherapy and mTOR inhibitors are recommended. The management of these tumors in children follows the consensus guidelines established for adult patients.[3,7] For more information, see Pancreatic Neuroendocrine Tumors (Islet Cell Tumors) Treatment. References: Incidence and Risk Factors Pancreatic carcinomas are extremely rare in children. These malignancies represent less than 5% of pediatric pancreatic tumors and include the following:[1,2] For more information, see the following summaries: Clinical Presentation Presenting symptoms are nonspecific and are related to local tumor growth. However, 4% to 15% of adult patients with acinar cell carcinoma may present with a lipase hypersecretion syndrome, manifesting as peripheral polyarthropathy and painful subcutaneous nodules. Treatment of Pancreatic Carcinoma For information about the treatment of pancreatic carcinoma, see Pancreatic Cancer Treatment. References: Information about National Cancer Institute (NCI)–supported clinical trials can be found on the NCI website. For information about clinical trials sponsored by other organizations, see the ClinicalTrials.gov website. The PDQ cancer information summaries are reviewed regularly and updated as new information becomes available. This section describes the latest changes made to this summary as of the date above. This summary was comprehensively reviewed. This summary is written and maintained by the PDQ Pediatric Treatment Editorial Board, which is editorially independent of NCI. The summary reflects an independent review of the literature and does not represent a policy statement of NCI or NIH. More information about summary policies and the role of the PDQ Editorial Boards in maintaining the PDQ summaries can be found on the About This PDQ Summary and PDQ® Cancer Information for Health Professionals pages. Purpose of This Summary This PDQ cancer information summary for health professionals provides comprehensive, peer-reviewed, evidence-based information about the treatment of pediatric pancreatic cancer. It is intended as a resource to inform and assist clinicians in the care of their patients. It does not provide formal guidelines or recommendations for making health care decisions. Reviewers and Updates This summary is reviewed regularly and updated as necessary by the PDQ Pediatric Treatment Editorial Board, which is editorially independent of the National Cancer Institute (NCI). The summary reflects an independent review of the literature and does not represent a policy statement of NCI or the National Institutes of Health (NIH). Board members review recently published articles each month to determine whether an article should: Changes to the summaries are made through a consensus process in which Board members evaluate the strength of the evidence in the published articles and determine how the article should be included in the summary. The lead reviewers for Childhood Pancreatic Cancer Treatment are: Any comments or questions about the summary content should be submitted to Cancer.gov through the NCI website's Email Us. Do not contact the individual Board Members with questions or comments about the summaries. Board members will not respond to individual inquiries. Levels of Evidence Some of the reference citations in this summary are accompanied by a level-of-evidence designation. These designations are intended to help readers assess the strength of the evidence supporting the use of specific interventions or approaches. The PDQ Pediatric Treatment Editorial Board uses a formal evidence ranking system in developing its level-of-evidence designations. Permission to Use This Summary PDQ is a registered trademark. Although the content of PDQ documents can be used freely as text, it cannot be identified as an NCI PDQ cancer information summary unless it is presented in its entirety and is regularly updated. However, an author would be permitted to write a sentence such as "NCI's PDQ cancer information summary about breast cancer prevention states the risks succinctly: [include excerpt from the summary]." The preferred citation for this PDQ summary is: PDQ® Pediatric Treatment Editorial Board. PDQ Childhood Pancreatic Cancer Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/pancreatic/hp/child-pancreatic-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 31661209] Images in this summary are used with permission of the author(s), artist, and/or publisher for use within the PDQ summaries only. Permission to use images outside the context of PDQ information must be obtained from the owner(s) and cannot be granted by the National Cancer Institute. Information about using the illustrations in this summary, along with many other cancer-related images, is available in Visuals Online, a collection of over 2,000 scientific images. Disclaimer Based on the strength of the available evidence, treatment options may be described as either "standard" or "under clinical evaluation." These classifications should not be used as a basis for insurance reimbursement determinations. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. Contact Us More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website's Email Us. Last Revised: 2024-08-13 This information does not replace the advice of a doctor. Ignite Healthwise, LLC disclaims any warranty or liability for your use of this information. Your use of this information means that you agree to the Terms of Use and Privacy Policy. Learn how we develop our content. Healthwise, Healthwise for every health decision, and the Healthwise logo are trademarks of Ignite Healthwise, LLC.Topic Contents
Childhood Pancreatic Cancer Treatment (PDQ®): Treatment - Health Professional Information [NCI]
General Information About Childhood Pancreatic Cancer
Special Considerations for the Treatment of Children With Cancer
Solid Pseudopapillary Tumor of the Pancreas
Pancreatoblastoma
Islet Cell Tumors
Pancreatic Carcinoma
Treatment Options Under Clinical Evaluation for Childhood Pancreatic Cancer
Latest Updates to This Summary (08 / 13 / 2024)
About This PDQ Summary
Our Health Library information does not replace the advice of a doctor. Please be advised that this information is made available to assist our patients to learn more about their health. Our providers may not see and/or treat all topics found herein. This information does not replace the advice of a doctor. Ignite Healthwise, LLC disclaims any warranty or liability for your use of this information. Your use of this information means that you agree to the Terms of Use and Privacy Policy. Learn how we develop our content. Healthwise, Healthwise for every health decision, and the Healthwise logo are trademarks of Ignite Healthwise, LLC.Childhood Pancreatic Cancer Treatment (PDQ®): Treatment - Health Professional Information [NCI]