Childhood Gastrointestinal Stromal Tumors Treatment (PDQ®): Treatment - Health Professional Information [NCI]

Incidence

Gastrointestinal stromal tumors (GIST) are the most common mesenchymal neoplasms of the gastrointestinal tract in adults.[1] These tumors are rare in children.[2] Approximately 2% of all GIST occur in children and young adults.[3,4,5] In one series, pediatric GIST accounted for 2.5% of all pediatric nonrhabdomyosarcomatous soft tissue sarcomas.[6] Previously, these tumors were diagnosed as leiomyomas, leiomyosarcomas, and leiomyoblastomas.

In pediatric patients, GIST are most commonly located in the stomach and almost exclusively affect adolescent females.[5,7,8]

References:

1. Corless CL, Fletcher JA, Heinrich MC: Biology of gastrointestinal stromal tumors. J Clin Oncol 22 (18): 3813-25, 2004.
2. Pappo AS, Janeway K, Laquaglia M, et al.: Special considerations in pediatric gastrointestinal tumors. J Surg Oncol 104 (8): 928-32, 2011.
3. Prakash S, Sarran L, Socci N, et al.: Gastrointestinal stromal tumors in children and young adults: a clinicopathologic, molecular, and genomic study of 15 cases and review of the literature. J Pediatr Hematol Oncol 27 (4): 179-87, 2005.
4. Miettinen M, Lasota J, Sobin LH: Gastrointestinal stromal tumors of the stomach in children and young adults: a clinicopathologic, immunohistochemical, and molecular genetic study of 44 cases with long-term follow-up and review of the literature. Am J Surg Pathol 29 (10): 1373-81, 2005.
5. Benesch M, Wardelmann E, Ferrari A, et al.: Gastrointestinal stromal tumors (GIST) in children and adolescents: A comprehensive review of the current literature. Pediatr Blood Cancer 53 (7): 1171-9, 2009.
6. Cypriano MS, Jenkins JJ, Pappo AS, et al.: Pediatric gastrointestinal stromal tumors and leiomyosarcoma. Cancer 101 (1): 39-50, 2004.
7. Pappo AS, Janeway KA: Pediatric gastrointestinal stromal tumors. Hematol Oncol Clin North Am 23 (1): 15-34, vii, 2009.
8. Benesch M, Leuschner I, Wardelmann E, et al.: Gastrointestinal stromal tumours in children and young adults: a clinicopathologic series with long-term follow-up from the database of the Cooperative Weichteilsarkom Studiengruppe (CWS). Eur J Cancer 47 (11): 1692-8, 2011.

Clinical Features

Most pediatric patients with gastrointestinal stromal tumors (GIST) are diagnosed during the second decade of life with anemia-related gastrointestinal bleeding. In addition, pediatric GIST have a high propensity for multifocality (23%) and nodal metastases.[1,2,3] These features may account for the high incidence of local recurrence seen in this patient population. Despite these features, patients have an indolent course, characterized by multiple tumor recurrences and long survival rates.[2]

References:

1. Pappo AS, Janeway KA: Pediatric gastrointestinal stromal tumors. Hematol Oncol Clin North Am 23 (1): 15-34, vii, 2009.
2. Agaram NP, Laquaglia MP, Ustun B, et al.: Molecular characterization of pediatric gastrointestinal stromal tumors. Clin Cancer Res 14 (10): 3204-15, 2008.
3. Benesch M, Wardelmann E, Ferrari A, et al.: Gastrointestinal stromal tumors (GIST) in children and adolescents: A comprehensive review of the current literature. Pediatr Blood Cancer 53 (7): 1171-9, 2009.

Histology and Molecular Features

Histologically, pediatric gastrointestinal stromal tumors (GIST) have a predominance of epithelioid or epithelioid/spindle cell morphology. Unlike adult GIST, the mitotic rate does not appear to accurately predict clinical behavior in pediatric patients.[1,2] Most GIST in the pediatric age range have loss of the succinate dehydrogenase (SDH) complex and consequently, lack SDHB expression by immunohistochemistry.[3,4] In addition, these tumors have minimal large-scale chromosomal changes and overexpress the insulin-like growth factor 1 receptor.[5,6]

Gastrointestinal tumors without a definitive line of differentiation should be evaluated for NTRK alterations.[7] Mesenchymal tumor of the gastrointestinal tract is characterized by the presence of NTRK rearrangements and is a separate entity from GIST. In a report of eight cases of mesenchymal tumors, six occurred in children. Four of these patients had lesions that were enriched for NTRK3 fusions, consistent with the diagnosis of infantile fibrosarcoma of the gastrointestinal tract.[7]

Activating mutations of KIT and PDGFRA, which are seen in 90% of adult GIST, are present in only a small fraction of pediatric GIST.[1,5,8]

The lack of SDHB expression in most pediatric GIST implicates cellular respiration defects in the pathogenesis of this disease and supports the notion that this disease is better categorized as SDH-deficient GIST. Furthermore, about 50% of patients with SDH-deficient GIST have germline mutations of the SDH complex, most commonly involving SDHA.[3] This finding supports the concept that SDH-deficient GIST is a cancer predisposition syndrome, and testing of affected patients for constitutional mutations for the SDH complex should be considered.[9]

SDH-deficient GIST can arise within the context of the following two syndromes:[1,10]

• Carney triad. Carney triad is a syndrome characterized by the occurrence of GIST, lung chondromas, and paragangliomas. In addition, about 20% of patients have adrenal adenomas and 10% have esophageal leiomyomas. GIST are the most common (75%) presenting lesions in these patients. To date, no coding sequence mutations of KIT, PDGFRA, or the SDH genes have been found in these patients.[10,11,12]
• Carney-Stratakis syndrome. Carney-Stratakis syndrome is characterized by paraganglioma and GIST caused by germline mutations of the SDHB, SDHC, and SDHD genes.[4,13]

A small percentage of SDH-deficient GIST lack somatic or germline mutations of the SDH complex. These tumors are characterized by SDHC promoter hypermethylation and gene silencing, and they are categorized as SDH-epimutant GIST.[14]

In an observational study carried out at the National Cancer Institute, 116 patients with presumed wild-type GIST were evaluated, and 95 of these patients had an adequate tumor specimen available for molecular profiling. Among these 95 patients, the investigators identified the following three distinctive subgroups of patients:[15]

• Group 1 (SDH-competent GIST): Group 1 included 11 patients who were designated as SDH competent because of positive staining of SDHB and lack of mutations on sequencing. All of these patients were adults, the median age was 46 years, and 64% were female. The tumors arose primarily in the small bowel (9 of 11). One patient had metastases to the peritoneum, and one patient had multifocal disease. Mutational analysis of these tumors identified mutations in the BRAF, NF1, CBL, KIT, and ARID1A genes. With a median follow-up of 8 years, three of these patients (27%) died of progressive disease.
• Group 2 (SDHX-mutant GIST): Group 2 included 63 patients who were SDH deficient. Mutations were observed in the SDHA (n = 34), SDHB (n = 16), SDHC (n = 12), and SDHD (n = 1) complexes. Of the 38 patients with SDH-mutant GIST who had matching germline and tumor DNA, 31 (82%) had the same mutation detected in the germline and the tumor. This group of patients was younger (median age, 23 years), mostly female (62%), and presented with gastric tumors (100%) and multifocal disease (42%). Metastases at presentation were seen in the lymph nodes (65%), liver (21%), and peritoneum (10%). At a median follow-up from diagnosis of 6 years, only three patients (5%) had died.
• Group 3 (SDHC-epimutant GIST): Group 3 included 21 patients with SDH-deficient tumors, with SDHC promoter methylation and no structural mutations. The median age at diagnosis was younger (age 15 years), and most patients were female (95%). All tumors arose in the stomach; 72% were multifocal. Metastases were present at diagnosis in the liver (37%), peritoneum (5%), and lymph nodes (38%). At a median follow-up of 7 years, only one patient (5%) with an SDH-epimutant tumor died of their disease.

Of the 95 patients that were evaluated at this clinic, 18 patients had syndromic GIST (i.e., Carney triad or Carney-Stratakis syndrome). Among the Carney triad patients, two patients had the complete triad, five patients had SDH mutations, and six patients had epimutant tumors. Seven patients with Carney-Stratakis syndrome had SDH-mutant GIST (n = 6) or SDH-epimutant GIST (n = 1).[15]

References:

1. Pappo AS, Janeway KA: Pediatric gastrointestinal stromal tumors. Hematol Oncol Clin North Am 23 (1): 15-34, vii, 2009.
2. Miettinen M, Lasota J: Gastrointestinal stromal tumors: review on morphology, molecular pathology, prognosis, and differential diagnosis. Arch Pathol Lab Med 130 (10): 1466-78, 2006.
3. Miettinen M, Lasota J: Succinate dehydrogenase deficient gastrointestinal stromal tumors (GISTs) - a review. Int J Biochem Cell Biol 53: 514-9, 2014.
4. Miettinen M, Wang ZF, Sarlomo-Rikala M, et al.: Succinate dehydrogenase-deficient GISTs: a clinicopathologic, immunohistochemical, and molecular genetic study of 66 gastric GISTs with predilection to young age. Am J Surg Pathol 35 (11): 1712-21, 2011.
5. Janeway KA, Liegl B, Harlow A, et al.: Pediatric KIT wild-type and platelet-derived growth factor receptor alpha-wild-type gastrointestinal stromal tumors share KIT activation but not mechanisms of genetic progression with adult gastrointestinal stromal tumors. Cancer Res 67 (19): 9084-8, 2007.
6. Tarn C, Rink L, Merkel E, et al.: Insulin-like growth factor 1 receptor is a potential therapeutic target for gastrointestinal stromal tumors. Proceedings of the National Academy of Sciences 105 (24): 8387-92, 2008. Also available online. Last accessed June 04, 2019.
7. Atiq MA, Davis JL, Hornick JL, et al.: Mesenchymal tumors of the gastrointestinal tract with NTRK rearrangements: a clinicopathological, immunophenotypic, and molecular study of eight cases, emphasizing their distinction from gastrointestinal stromal tumor (GIST). Mod Pathol 34 (1): 95-103, 2021.
8. Agaram NP, Laquaglia MP, Ustun B, et al.: Molecular characterization of pediatric gastrointestinal stromal tumors. Clin Cancer Res 14 (10): 3204-15, 2008.
9. Janeway KA, Kim SY, Lodish M, et al.: Defects in succinate dehydrogenase in gastrointestinal stromal tumors lacking KIT and PDGFRA mutations. Proc Natl Acad Sci U S A 108 (1): 314-8, 2011.
10. Otto C, Agaimy A, Braun A, et al.: Multifocal gastric gastrointestinal stromal tumors (GISTs) with lymph node metastases in children and young adults: a comparative clinical and histomorphological study of three cases including a new case of Carney triad. Diagn Pathol 6: 52, 2011.
11. Carney JA: Carney triad: a syndrome featuring paraganglionic, adrenocortical, and possibly other endocrine tumors. J Clin Endocrinol Metab 94 (10): 3656-62, 2009.
12. Benesch M, Wardelmann E, Ferrari A, et al.: Gastrointestinal stromal tumors (GIST) in children and adolescents: A comprehensive review of the current literature. Pediatr Blood Cancer 53 (7): 1171-9, 2009.
13. Pasini B, McWhinney SR, Bei T, et al.: Clinical and molecular genetics of patients with the Carney-Stratakis syndrome and germline mutations of the genes coding for the succinate dehydrogenase subunits SDHB, SDHC, and SDHD. Eur J Hum Genet 16 (1): 79-88, 2008.
14. Killian JK, Miettinen M, Walker RL, et al.: Recurrent epimutation of SDHC in gastrointestinal stromal tumors. Sci Transl Med 6 (268): 268ra177, 2014.
15. Boikos SA, Pappo AS, Killian JK, et al.: Molecular Subtypes of KIT/PDGFRA Wild-Type Gastrointestinal Stromal Tumors: A Report From the National Institutes of Health Gastrointestinal Stromal Tumor Clinic. JAMA Oncol 2 (7): 922-8, 2016.

Special Considerations for the Treatment of Children With Cancer

Cancer in children and adolescents is rare, although the overall incidence has been slowly increasing since 1975.[1] Referral to medical centers with multidisciplinary teams of cancer specialists experienced in treating cancers that occur in childhood and adolescence should be considered. This multidisciplinary team approach incorporates the skills of the following health care professionals and others to ensure that children receive treatment, supportive care, and rehabilitation that will achieve optimal survival and quality of life:

• Primary care physicians.
• Pediatric surgeons.
• Radiation oncologists.
• Pediatric medical oncologists/hematologists.
• Rehabilitation specialists.
• Pediatric nurse specialists.
• Social workers.
• Child-life professionals.
• Psychologists.

For 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 pediatric patients with cancer.[2] At these pediatric cancer 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. Most of the progress made in identifying curative therapy 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. 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] Also, the designation of a pediatric rare tumor is not uniform among international groups, as follows:

• A consensus effort between the European Union Joint Action on Rare Cancers and the European Cooperative Study Group for Rare Pediatric Cancers estimated that 11% of all cancers in patients younger than 20 years could be categorized as very rare. This consensus group defined very rare cancers as those with annual incidences of fewer than 2 cases per 1 million people. However, three additional histologies (thyroid carcinoma, melanoma, and testicular cancer) with incidences of more than 2 cases per 1 million people were also included in the very rare group because there is a lack of knowledge and expertise in the management of these tumors.[9]
• The Children's Oncology Group (COG) defines rare pediatric cancers as those listed in the International Classification of Childhood Cancer subgroup XI, which includes thyroid cancers, melanomas and nonmelanoma skin cancers, and multiple types of carcinomas (e.g., adrenocortical carcinomas, nasopharyngeal carcinomas, and most adult-type carcinomas such as breast cancers, colorectal cancers, etc.).[10] These diagnoses account for about 5% of the cancers diagnosed in children aged 0 to 14 years and about 27% of the cancers diagnosed in adolescents aged 15 to 19 years.[4]

  Most cancers in subgroup XI are either melanomas or thyroid cancers, with other cancer types accounting for only 2% of the cancers in children aged 0 to 14 years and 9.3% of the cancers in adolescents aged 15 to 19 years.

These rare cancers are extremely challenging to study because of the low number of patients with any individual diagnosis, the predominance of rare cancers in the adolescent population, and the lack 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 Gastrointestinal Stromal Tumors Treatment.

References:

1. Smith MA, Seibel NL, Altekruse SF, et al.: Outcomes for children and adolescents with cancer: challenges for the twenty-first century. J Clin Oncol 28 (15): 2625-34, 2010.
2. American Academy of Pediatrics: Standards for pediatric cancer centers. Pediatrics 134 (2): 410-4, 2014. Also available online. Last accessed December 15, 2023.
3. Smith MA, Altekruse SF, Adamson PC, et al.: Declining childhood and adolescent cancer mortality. Cancer 120 (16): 2497-506, 2014.
4. National Cancer Institute: NCCR*Explorer: An interactive website for NCCR cancer statistics. Bethesda, MD: National Cancer Institute. Available online. Last accessed December 15, 2023.
5. Surveillance Research Program, National Cancer Institute: SEER*Explorer: An interactive website for SEER cancer statistics. Bethesda, MD: National Cancer Institute. Available online. Last accessed August 18, 2023.
6. Ward E, DeSantis C, Robbins A, et al.: Childhood and adolescent cancer statistics, 2014. CA Cancer J Clin 64 (2): 83-103, 2014 Mar-Apr.
7. Gatta G, Capocaccia R, Botta L, et al.: Burden and centralised treatment in Europe of rare tumours: results of RARECAREnet-a population-based study. Lancet Oncol 18 (8): 1022-1039, 2017.
8. DeSantis CE, Kramer JL, Jemal A: The burden of rare cancers in the United States. CA Cancer J Clin 67 (4): 261-272, 2017.
9. Ferrari A, Brecht IB, Gatta G, et al.: Defining and listing very rare cancers of paediatric age: consensus of the Joint Action on Rare Cancers in cooperation with the European Cooperative Study Group for Pediatric Rare Tumors. Eur J Cancer 110: 120-126, 2019.
10. Pappo AS, Krailo M, Chen Z, et al.: Infrequent tumor initiative of the Children's Oncology Group: initial lessons learned and their impact on future plans. J Clin Oncol 28 (33): 5011-6, 2010.

Treatment of Childhood Gastrointestinal Stromal Tumors

Treatment options for pediatric gastrointestinal stromal tumors (GIST) include the following:

1. Observation.
2. Surgery.
3. Targeted therapy.

Once the diagnosis of pediatric GIST is established, patients should be referred to medical centers with expertise in the treatment of GIST.[1,2]

Given the indolent course of the disease in pediatric patients, it is reasonable to avoid extensive initial surgeries and to withhold subsequent resections unless needed to address symptoms such as obstruction or bleeding.[3,4]

Tumor samples are evaluated for mutations in KIT (exons 9, 11, 13, 17), PDGFRA (exons 12, 14, 18), and BRAF (V600E).[1,2] Treatment options for GIST depend on whether a mutation is detected.

GIST with a KIT or PDGFRA mutation: Pediatric patients who harbor KIT or PDGFRA mutations are managed like adults. For more information, see Gastrointestinal Stromal Tumors Treatment.

Succinate dehydrogenase (SDH)-deficient GIST: Approximately one-half of all patients with wild-type GIST are SDH deficient.[5] For most pediatric patients with SDH-deficient GIST, surgical resection of localized disease is recommended because of its indolent course. Extensive surgery and repeated surgical resections should be avoided.

This approach is supported by a study of 76 patients with wild-type GIST who underwent surgery for newly diagnosed and recurrent disease.[5]

• Only 9% of patients experienced a fatal event, whereas 71% (54 patients) developed tumor recurrence or progression at a median of 2.5 years.
• For this population, the 1-year event-free survival (EFS) rate was 73%, the 5-year EFS rate was 24%, and the 10-year EFS rate was 16%.
• Factors associated with an increased risk of recurrence included metastatic disease and elevated mitotic rate. SDH status and extent of surgical resection did not influence the risk of recurrence.
• Among 33 patients who underwent reoperation for recurrent disease, each subsequent resection was associated with a lower EFS rate.

In patients with SDH-deficient GIST, responses to imatinib, regorafenib, vandetanib, and sunitinib are uncommon.[3,6,7,8]

1. In a review of ten patients who were treated with imatinib mesylate, one patient experienced a partial response and three patients had stable disease.[3]
2. In the phase III SWOG Cancer Research Network intergroup S0033 (NCT00009906) trial, 20 tumors from patients presumed to have wild-type disease were resequenced.[8]
   • Twelve of these tumors were identified as being SDH mutant, and only one patient (8.3%) experienced a partial response to imatinib.[9]
3. In another study, sunitinib appeared to show more activity.[10]
   • In six children with imatinib-resistant GIST, one patient had a partial response, and five patients had stable disease.

Unlike recommendations for adults, the use of adjuvant imatinib cannot be recommended in children with SDH-deficient GIST.[11]

References:

1. Demetri GD, Benjamin RS, Blanke CD, et al.: NCCN Task Force report: management of patients with gastrointestinal stromal tumor (GIST)--update of the NCCN clinical practice guidelines. J Natl Compr Canc Netw 5 (Suppl 2): S1-29; quiz S30, 2007.
2. Janeway KA, Weldon CB: Pediatric gastrointestinal stromal tumor. Semin Pediatr Surg 21 (1): 31-43, 2012.
3. Pappo AS, Janeway KA: Pediatric gastrointestinal stromal tumors. Hematol Oncol Clin North Am 23 (1): 15-34, vii, 2009.
4. Pappo AS, Janeway K, Laquaglia M, et al.: Special considerations in pediatric gastrointestinal tumors. J Surg Oncol 104 (8): 928-32, 2011.
5. Weldon CB, Madenci AL, Boikos SA, et al.: Surgical Management of Wild-Type Gastrointestinal Stromal Tumors: A Report From the National Institutes of Health Pediatric and Wildtype GIST Clinic. J Clin Oncol 35 (5): 523-528, 2017.
6. Neppala P, Banerjee S, Fanta PT, et al.: Current management of succinate dehydrogenase-deficient gastrointestinal stromal tumors. Cancer Metastasis Rev 38 (3): 525-535, 2019.
7. Demetri GD, van Oosterom AT, Garrett CR, et al.: Efficacy and safety of sunitinib in patients with advanced gastrointestinal stromal tumour after failure of imatinib: a randomised controlled trial. Lancet 368 (9544): 1329-38, 2006.
8. Demetri GD, von Mehren M, Blanke CD, et al.: Efficacy and safety of imatinib mesylate in advanced gastrointestinal stromal tumors. N Engl J Med 347 (7): 472-80, 2002.
9. Heinrich MC, Rankin C, Blanke CD, et al.: Correlation of Long-term Results of Imatinib in Advanced Gastrointestinal Stromal Tumors With Next-Generation Sequencing Results: Analysis of Phase 3 SWOG Intergroup Trial S0033. JAMA Oncol 3 (7): 944-952, 2017.
10. Janeway KA, Albritton KH, Van Den Abbeele AD, et al.: Sunitinib treatment in pediatric patients with advanced GIST following failure of imatinib. Pediatr Blood Cancer 52 (7): 767-71, 2009.
11. Dematteo RP, Ballman KV, Antonescu CR, et al.: Adjuvant imatinib mesylate after resection of localised, primary gastrointestinal stromal tumour: a randomised, double-blind, placebo-controlled trial. Lancet 373 (9669): 1097-104, 2009.

Treatment Options Under Clinical Evaluation for Childhood Gastrointestinal Stromal Tumors

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.

Latest Updates to This Summary (01 / 03 / 2023)

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.

Editorial changes were made to this summary.

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.

About This PDQ Summary

Purpose of This Summary

This PDQ cancer information summary for health professionals provides comprehensive, peer-reviewed, evidence-based information about the treatment of pediatric gastrointestinal stromal tumors. 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:

• be discussed at a meeting,
• be cited with text, or
• replace or update an existing article that is already cited.

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 Gastrointestinal Stromal Tumors Treatment are:

• Denise Adams, MD (Children's Hospital Boston)
• Karen J. Marcus, MD, FACR (Dana-Farber Cancer Institute/Boston Children's Hospital)
• William H. Meyer, MD
• Paul A. Meyers, MD (Memorial Sloan-Kettering Cancer Center)
• Thomas A. Olson, MD (Aflac Cancer and Blood Disorders Center of Children's Healthcare of Atlanta - Egleston Campus)
• Alberto S. Pappo, MD (St. Jude Children's Research Hospital)
• D. Williams Parsons, MD, PhD (Texas Children's Hospital)
• Arthur Kim Ritchey, MD (Children's Hospital of Pittsburgh of UPMC)
• Carlos Rodriguez-Galindo, MD (St. Jude Children's Research Hospital)
• Stephen J. Shochat, MD (St. Jude Children's Research Hospital)

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.

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The preferred citation for this PDQ summary is:

PDQ® Pediatric Treatment Editorial Board. PDQ Childhood Gastrointestinal Stromal Tumors Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/soft-tissue-sarcoma/hp/child-gist-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 31661204]

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Last Revised: 2024-01-03