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Gastrointestinal stromal tumor(GIST)

MedGen UID:
116049
Concept ID:
C0238198
Neoplastic Process
Synonyms: Gastrointestinal stroma tumor; Gastrointestinal Stromal Sarcoma; Gastrointestinal stromal tumor, somatic
SNOMED CT: Gastrointestinal pacemaker cell tumor (128755003); Gastrointestinal stromal tumor (420120006); GIST - Gastrointestinal stromal tumor (420120006); Gastrointestinal stromal tumor (1187383001)
Modes of inheritance:
Autosomal dominant inheritance
MedGen UID:
141047
Concept ID:
C0443147
Intellectual Product
Source: Orphanet
A mode of inheritance that is observed for traits related to a gene encoded on one of the autosomes (i.e., the human chromosomes 1-22) in which a trait manifests in heterozygotes. In the context of medical genetics, an autosomal dominant disorder is caused when a single copy of the mutant allele is present. Males and females are affected equally, and can both transmit the disorder with a risk of 50% for each child of inheriting the mutant allele.
Not genetically inherited
MedGen UID:
988794
Concept ID:
CN307044
Finding
Source: Orphanet
clinical entity without genetic inheritance.
 
Genes (locations): KIT (4q12); SDHB (1p36.13); SDHC (1q23.3)
 
HPO: HP:0100723
Monarch Initiative: MONDO:0011719
OMIM®: 606764
Orphanet: ORPHA44890

Definition

Gastrointestinal stromal tumors are mesenchymal tumors found in the gastrointestinal tract that originate from the interstitial cells of Cajal, the pacemaker cells that regulate peristalsis in the digestive tract. Approximately 70% of GISTs develop in the stomach, 20% in the small intestine, and less than 10% in the esophagus, colon, and rectum. GISTs are typically more cellular than other gastrointestinal sarcomas. They occur predominantly in patients who are 40 to 70 years old but in rare cases may occur in younger persons (Miettinen et al., 1999, 1999). GISTs are also seen as a feature in several syndromes, e.g., neurofibromatosis-1 (NF1; 162200) and GIST-plus syndrome (175510). [from OMIM]

Additional description

From MedlinePlus Genetics
A gastrointestinal stromal tumor (GIST) is a type of tumor that occurs in the gastrointestinal tract, most commonly in the stomach or small intestine. This type of tumor is thought to grow from specialized cells found in the gastrointestinal tract called interstitial cells of Cajal (ICCs) or precursors to these cells. Affected individuals can develop one or more tumors. GISTs are usually found in adults between ages 40 and 70; rarely, children and young adults develop this type of tumor.

Small tumors may cause no signs or symptoms. However, some people with GISTs may experience pain or swelling in the belly area (abdomen), nausea, vomiting, loss of appetite, or weight loss. Sometimes, tumors cause bleeding into the gastrointestinal tract, which may lead to low red blood cell counts (anemia) and, consequently, weakness and tiredness. Bleeding into the intestines may cause black and tarry stools, and bleeding into the throat or stomach may cause vomiting of blood.

Affected individuals with no family history of GIST typically have only one tumor (called a sporadic GIST). People with a family history of GISTs (called familial GISTs) often have multiple tumors and additional signs or symptoms, including noncancerous overgrowth (hyperplasia) of other cells in the gastrointestinal tract and patches of dark skin on various areas of the body. Some affected individuals have a skin condition called urticaria pigmentosa (also known as maculopapular cutaneous mastocytosis), which is characterized by raised patches of brownish skin that sting or itch when touched.

A rare form of GIST, called succinate dehydrogenase (SDH)-deficient GIST, tends to occur in childhood or young adulthood and affects females more commonly than males. In this form, tumors are almost always in the stomach. Individuals with an SDH-deficient GIST have a high risk of developing other types of tumors, particularly noncancerous tumors in the nervous system called paragangliomas and noncancerous lung tumors called pulmonary chondromas. When GISTs occur in combination with paragangliomas, the condition is known as Carney-Stratakis syndrome; the combination of GISTs, paragangliomas, and pulmonary chondromas is known as Carney triad; and the combination of GISTs and pulmonary chondroma is known as incomplete Carney triad.  https://medlineplus.gov/genetics/condition/gastrointestinal-stromal-tumor

Clinical features

From HPO
Large hands
MedGen UID:
98097
Concept ID:
C0426870
Finding
Constipation
MedGen UID:
1101
Concept ID:
C0009806
Sign or Symptom
Infrequent or difficult evacuation of feces.
Dysphagia
MedGen UID:
41440
Concept ID:
C0011168
Disease or Syndrome
Difficulty in swallowing.
Intestinal obstruction
MedGen UID:
43933
Concept ID:
C0021843
Disease or Syndrome
Blockage or impairment of the normal flow of the contents of the intestine towards the anal canal.
Gastrointestinal stromal tumor
MedGen UID:
116049
Concept ID:
C0238198
Neoplastic Process
Gastrointestinal stromal tumors are mesenchymal tumors found in the gastrointestinal tract that originate from the interstitial cells of Cajal, the pacemaker cells that regulate peristalsis in the digestive tract. Approximately 70% of GISTs develop in the stomach, 20% in the small intestine, and less than 10% in the esophagus, colon, and rectum. GISTs are typically more cellular than other gastrointestinal sarcomas. They occur predominantly in patients who are 40 to 70 years old but in rare cases may occur in younger persons (Miettinen et al., 1999, 1999). GISTs are also seen as a feature in several syndromes, e.g., neurofibromatosis-1 (NF1; 162200) and GIST-plus syndrome (175510).
Neurofibroma
MedGen UID:
45058
Concept ID:
C0027830
Neoplastic Process
A benign peripheral nerve sheath tumor that generally appears as a soft, skin-colored papule or small subcutaneous nodule. Individuals with neurofibromatosis can have numerous neurofibromas.
Urticaria
MedGen UID:
22587
Concept ID:
C0042109
Disease or Syndrome
Raised, well-circumscribed areas of erythema and edema involving the dermis and epidermis. Urticaria is intensely pruritic, and blanches completely with pressure.
Hyperpigmentation of the skin
MedGen UID:
57992
Concept ID:
C0162834
Pathologic Function
A darkening of the skin related to an increase in melanin production and deposition.

Term Hierarchy

CClinical test,  RResearch test,  OOMIM,  GGeneReviews,  VClinVar  
  • CROGVGastrointestinal stromal tumor
Follow this link to review classifications for Gastrointestinal stromal tumor in Orphanet.

Conditions with this feature

Gastrointestinal stromal tumor
MedGen UID:
116049
Concept ID:
C0238198
Neoplastic Process
Gastrointestinal stromal tumors are mesenchymal tumors found in the gastrointestinal tract that originate from the interstitial cells of Cajal, the pacemaker cells that regulate peristalsis in the digestive tract. Approximately 70% of GISTs develop in the stomach, 20% in the small intestine, and less than 10% in the esophagus, colon, and rectum. GISTs are typically more cellular than other gastrointestinal sarcomas. They occur predominantly in patients who are 40 to 70 years old but in rare cases may occur in younger persons (Miettinen et al., 1999, 1999). GISTs are also seen as a feature in several syndromes, e.g., neurofibromatosis-1 (NF1; 162200) and GIST-plus syndrome (175510).
Paragangliomas 4
MedGen UID:
349380
Concept ID:
C1861848
Disease or Syndrome
Hereditary paraganglioma-pheochromocytoma (PGL/PCC) syndromes are characterized by paragangliomas (tumors that arise from neuroendocrine tissues distributed along the paravertebral axis from the base of the skull to the pelvis) and pheochromocytomas (paragangliomas that are confined to the adrenal medulla). Sympathetic paragangliomas cause catecholamine excess; parasympathetic paragangliomas are most often nonsecretory. Extra-adrenal parasympathetic paragangliomas are located predominantly in the skull base and neck (referred to as head and neck PGL [HNPGL]) and sometimes in the upper mediastinum; approximately 95% of such tumors are nonsecretory. In contrast, sympathetic extra-adrenal paragangliomas are generally confined to the lower mediastinum, abdomen, and pelvis, and are typically secretory. Pheochromocytomas, which arise from the adrenal medulla, typically lead to catecholamine excess. Symptoms of PGL/PCC result from either mass effects or catecholamine hypersecretion (e.g., sustained or paroxysmal elevations in blood pressure, headache, episodic profuse sweating, forceful palpitations, pallor, and apprehension or anxiety). The risk for developing metastatic disease is greater for extra-adrenal sympathetic paragangliomas than for pheochromocytomas.
Polyps, multiple and recurrent inflammatory fibroid, gastrointestinal
MedGen UID:
1677803
Concept ID:
C5193005
Disease or Syndrome
GIST-plus syndrome (GISTPS) is an autosomal dominant disorder characterized by incomplete penetrance of multiple mesenchymal tumors of the gastrointestinal tract, including gastrointestinal stromal tumor (GIST), inflammatory fibroid polyps (IFP), and fibroid tumors (FT). Some patients have been reported with coarse facies and skin, broad hands and feet, and premature tooth loss. Isolated GISTs and IFPs are seen in patients with somatic PDGFRA mutations (summary by Manley et al., 2018).

Professional guidelines

PubMed

Deprez PH, Moons LMG, OʼToole D, Gincul R, Seicean A, Pimentel-Nunes P, Fernández-Esparrach G, Polkowski M, Vieth M, Borbath I, Moreels TG, Nieveen van Dijkum E, Blay JY, van Hooft JE
Endoscopy 2022 Apr;54(4):412-429. Epub 2022 Feb 18 doi: 10.1055/a-1751-5742. PMID: 35180797
Kelly CM, Gutierrez Sainz L, Chi P
J Hematol Oncol 2021 Jan 5;14(1):2. doi: 10.1186/s13045-020-01026-6. PMID: 33402214Free PMC Article
Akahoshi K, Oya M, Koga T, Shiratsuchi Y
World J Gastroenterol 2018 Jul 14;24(26):2806-2817. doi: 10.3748/wjg.v24.i26.2806. PMID: 30018476Free PMC Article

Curated

NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®), Soft Tissue Sarcoma, 2024

NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®) Gastrointestinal Stromal Tumors, 2023

Recent clinical studies

Etiology

Dashti NK, Shi C
Surg Pathol Clin 2020 Sep;13(3):521-556. Epub 2020 Jul 15 doi: 10.1016/j.path.2020.06.001. PMID: 32773198
Etit D, Kar H, Ekinci N, Yenipazar AE, Çakalağaoğlu F
Balkan Med J 2017 Apr 5;34(2):168-171. doi: 10.4274/balkanmedj.2015.1331. PMID: 28418346Free PMC Article
Keung EZ, Raut CP
Surg Clin North Am 2017 Apr;97(2):437-452. doi: 10.1016/j.suc.2016.12.001. PMID: 28325196
Yamamoto H, Oda Y
Pathol Int 2015 Jan;65(1):9-18. Epub 2014 Nov 20 doi: 10.1111/pin.12230. PMID: 25414046
Beham AW, Schaefer IM, Schüler P, Cameron S, Ghadimi BM
Int J Colorectal Dis 2012 Jun;27(6):689-700. Epub 2011 Nov 29 doi: 10.1007/s00384-011-1353-y. PMID: 22124674Free PMC Article

Diagnosis

Dembiński Ł, Dziekiewicz M, Banasiuk M, Banaszkiewicz A
J Pediatr Gastroenterol Nutr 2023 Aug 1;77(2):e48. Epub 2023 May 17 doi: 10.1097/MPG.0000000000003831. PMID: 37195885
Akahoshi K, Oya M, Koga T, Shiratsuchi Y
World J Gastroenterol 2018 Jul 14;24(26):2806-2817. doi: 10.3748/wjg.v24.i26.2806. PMID: 30018476Free PMC Article
Domansk HA, Walther CS
Monogr Clin Cytol 2017;22:89-91. Epub 2017 Jul 27 doi: 10.1159/000475100. PMID: 28750384
Schaefer IM, Mariño-Enríquez A, Fletcher JA
Adv Anat Pathol 2017 Sep;24(5):259-267. doi: 10.1097/PAP.0000000000000158. PMID: 28632504Free PMC Article
Akçakaya P, Lui WO
Adv Exp Med Biol 2015;889:51-70. doi: 10.1007/978-3-319-23730-5_4. PMID: 26658996

Therapy

Bauer S, Jones RL, Blay JY, Gelderblom H, George S, Schöffski P, von Mehren M, Zalcberg JR, Kang YK, Razak AA, Trent J, Attia S, Le Cesne A, Su Y, Meade J, Wang T, Sherman ML, Ruiz-Soto R, Heinrich MC
J Clin Oncol 2022 Dec 1;40(34):3918-3928. Epub 2022 Aug 10 doi: 10.1200/JCO.22.00294. PMID: 35947817Free PMC Article
Schaefer IM, DeMatteo RP, Serrano C
Am Soc Clin Oncol Educ Book 2022 Apr;42:1-15. doi: 10.1200/EDBK_351231. PMID: 35522913Free PMC Article
Kelly CM, Gutierrez Sainz L, Chi P
J Hematol Oncol 2021 Jan 5;14(1):2. doi: 10.1186/s13045-020-01026-6. PMID: 33402214Free PMC Article
Akahoshi K, Oya M, Koga T, Shiratsuchi Y
World J Gastroenterol 2018 Jul 14;24(26):2806-2817. doi: 10.3748/wjg.v24.i26.2806. PMID: 30018476Free PMC Article
Keung EZ, Raut CP
Surg Clin North Am 2017 Apr;97(2):437-452. doi: 10.1016/j.suc.2016.12.001. PMID: 28325196

Prognosis

Liu X, Chu KM
Clin Transl Oncol 2019 Feb;21(2):145-151. Epub 2018 Jul 12 doi: 10.1007/s12094-018-1914-4. PMID: 30003531
Akahoshi K, Oya M, Koga T, Shiratsuchi Y
World J Gastroenterol 2018 Jul 14;24(26):2806-2817. doi: 10.3748/wjg.v24.i26.2806. PMID: 30018476Free PMC Article
Yamamoto H, Oda Y
Pathol Int 2015 Jan;65(1):9-18. Epub 2014 Nov 20 doi: 10.1111/pin.12230. PMID: 25414046
Joensuu H
Hum Pathol 2008 Oct;39(10):1411-9. doi: 10.1016/j.humpath.2008.06.025. PMID: 18774375
Casper ES
Curr Treat Options Oncol 2000 Aug;1(3):267-73. doi: 10.1007/s11864-000-0039-4. PMID: 12057170

Clinical prediction guides

Gómez-Peregrina D, Cicala CM, Serrano C
Curr Opin Oncol 2024 Jul 1;36(4):282-290. Epub 2024 Apr 19 doi: 10.1097/CCO.0000000000001040. PMID: 38726808
Tariq MU, Din NU, Abdul-Ghafar J, Park YK
Diagn Pathol 2021 Apr 20;16(1):32. doi: 10.1186/s13000-021-01095-2. PMID: 33879215Free PMC Article
Ma C, Tsai H, Su W, Sun L, Shih Y, Wang J
J Postgrad Med 2018 Jul-Sep;64(3):155-163. doi: 10.4103/jpgm.JPGM_693_17. PMID: 29848836Free PMC Article
Etit D, Kar H, Ekinci N, Yenipazar AE, Çakalağaoğlu F
Balkan Med J 2017 Apr 5;34(2):168-171. doi: 10.4274/balkanmedj.2015.1331. PMID: 28418346Free PMC Article
Rini BI
Expert Opin Pharmacother 2007 Oct;8(14):2359-69. doi: 10.1517/14656566.8.14.2359. PMID: 17927489

Recent systematic reviews

Brinch CM, Aggerholm-Pedersen N, Hogdall E, Krarup-Hansen A
Crit Rev Oncol Hematol 2022 Apr;172:103650. Epub 2022 Mar 10 doi: 10.1016/j.critrevonc.2022.103650. PMID: 35283299
Raitio A, Salim A, Mullassery D, Losty PD
Pediatr Surg Int 2021 Sep;37(9):1161-1165. Epub 2021 Jun 3 doi: 10.1007/s00383-021-04931-0. PMID: 34081161Free PMC Article
Zhou Y, Wang X, Si X, Wang S, Cai Z
Asian J Surg 2020 Jan;43(1):1-8. Epub 2019 Mar 8 doi: 10.1016/j.asjsur.2019.02.006. PMID: 30853211
Waidhauser J, Bornemann A, Trepel M, Märkl B
World J Gastroenterol 2019 Aug 14;25(30):4261-4277. doi: 10.3748/wjg.v25.i30.4261. PMID: 31435178Free PMC Article
Herzberg M, Beer M, Anupindi S, Vollert K, Kröncke T
J Pediatr Surg 2018 Sep;53(9):1862-1870. Epub 2018 Mar 29 doi: 10.1016/j.jpedsurg.2018.03.022. PMID: 29685489

Supplemental Content

Table of contents

    Clinical resources

    Practice guidelines

    • PubMed
      See practice and clinical guidelines in PubMed. The search results may include broader topics and may not capture all published guidelines. See the FAQ for details.
    • Bookshelf
      See practice and clinical guidelines in NCBI Bookshelf. The search results may include broader topics and may not capture all published guidelines. See the FAQ for details.

    Curated

    • NCCN, 2024
      NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®), Soft Tissue Sarcoma, 2024
    • NCCN, 2023
      NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®) Gastrointestinal Stromal Tumors, 2023

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