Evidence Base

Clinical References

Every clinical trial, guideline, and data source cited in GIST treatment recommendations.

41 references 9 categories
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Clinical Practice Guidelines

4
NCCN Guidelines
Guideline Category 2A

NCCN Clinical Practice Guidelines in Oncology: Gastrointestinal Stromal Tumors (GISTs)

National Comprehensive Cancer Network

Version 1.2026

ESMO/EURACAN/GENTURIS Guidelines
Guideline Category I-A

Gastrointestinal stromal tumours: ESMO-EURACAN-GENTURIS Clinical Practice Guidelines for diagnosis, treatment and follow-up

Casali PG, Blay JY, Abecassis N, et al.

Ann Oncol. 2022;33(1):20-33

AJCC 8th Edition
Guideline

AJCC Cancer Staging Manual, 8th Edition — Gastrointestinal Stromal Tumor

Amin MB, Edge SB, Greene FL, et al.

Springer; 2017

CAP GIST Protocol
Guideline

Protocol for the Examination of Specimens From Patients With Gastrointestinal Stromal Tumor (GIST)

College of American Pathologists

CAP Cancer Protocols, Version 4.3.0.0 (2023)

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Risk Stratification Criteria

3
Miettinen Criteria
Category 2A

Gastrointestinal stromal tumors: pathology and prognosis at different sites

Miettinen M, Lasota J.

Semin Diagn Pathol. 2006;23(2):70-83

Modified NIH Criteria
Category 2A

Risk stratification of patients diagnosed with gastrointestinal stromal tumor

Joensuu H.

Hum Pathol. 2008;39(10):1411-9

NIH Consensus Criteria

Diagnosis of gastrointestinal stromal tumors: A consensus approach

Fletcher CD, Berman JJ, Corless C, et al.

Hum Pathol. 2002;33(5):459-65

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Baseline & Population Data

6
Joensuu Pooled Analysis

Risk of recurrence of gastrointestinal stromal tumour after surgery: an analysis of pooled population-based cohorts

Joensuu H, Vehtari A, Riihimäki J, et al.

Lancet Oncol. 2012;13(3):265-274

Gold Prognostic Nomogram

Development and validation of a prognostic nomogram for recurrence-free survival after complete surgical resection of localised primary gastrointestinal stromal tumour: a retrospective analysis

Gold JS, Gönen M, Gutiérrez A, et al.

Lancet Oncol. 2009;10(11):1045-1052

SEER Cancer Statistics
Registry

Surveillance, Epidemiology, and End Results (SEER) Program: Gastrointestinal Stromal Tumor Survival Statistics

National Cancer Institute, DCCPS, Surveillance Research Program

SEER*Stat Database: Incidence - SEER Research Data, 17 Registries, Nov 2024 Sub (2000-2022), released April 2025

Rossi Natural History Cohort

Natural history of imatinib-naive GISTs: a retrospective analysis of 929 cases with long-term follow-up and development of a survival nomogram based on mitotic index and size as continuous variables

Rossi S, Miceli R, Messerini L, et al.

Am J Surg Pathol. 2011;35(11):1646-1656

DeMatteo 200 GISTs

Two hundred gastrointestinal stromal tumors: recurrence patterns and prognostic factors for survival

DeMatteo RP, Lewis JJ, Leung D, et al.

Ann Surg. 2000;231(1):51-58

DeMatteo Risk Factor Validation

Tumor mitotic rate, size, and location independently predict recurrence after resection of primary gastrointestinal stromal tumor (GIST)

DeMatteo RP, Gold JS, Saran L, et al.

Cancer. 2008;112(3):608-615

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Adjuvant Therapy Trials

15
ACOSOG Z9001
Category 1

Adjuvant imatinib mesylate after resection of localised, primary gastrointestinal stromal tumour: a randomised, double-blind, placebo-controlled trial

DeMatteo RP, Ballman KV, Antonescu CR, et al.

Lancet. 2009;373(9669):1097-104

IMADGIST
Category 2B

A randomized study of 6 versus 3 years of adjuvant imatinib in patients with localized GIST at high risk of relapse

Blay JY, Schiffler C, Bouché O, et al.

Ann Oncol. 2024;35(12):1157-1168

SSGXVIII/AIO (Original)
Category 1

One vs three years of adjuvant imatinib for operable gastrointestinal stromal tumor: a randomized trial

Joensuu H, Eriksson M, Sundby Hall K, et al.

JAMA. 2012;307(12):1265-1272

SSGXVIII/AIO (10-Year Follow-up)
Category 1

Survival Outcomes Associated With 3 Years vs 1 Year of Adjuvant Imatinib for Patients With High-Risk Gastrointestinal Stromal Tumors: An Analysis of a Randomized Clinical Trial After 10-Year Follow-up

Joensuu H, Eriksson M, Sundby Hall K, Reichardt A, Hermes B, et al.

JAMA Oncol. 2020;6(8):1241-1246

EORTC 62024
Category 1

Time to Definitive Failure to the First Tyrosine Kinase Inhibitor in Localized GI Stromal Tumors Treated With Imatinib As an Adjuvant: A European Organisation for Research and Treatment of Cancer Soft Tissue and Bone Sarcoma Group Intergroup Randomized Trial in Collaboration With the Australasian Gastro-Intestinal Trials Group

Casali PG, Le Cesne A, Poveda Velasco A, et al.

J Clin Oncol. 2015;33(36):4276-4283

EORTC 62024 (Final Analysis)
Category 1

Final analysis of the randomized trial on imatinib as an adjuvant in localized gastrointestinal stromal tumors from the EORTC Soft Tissue and Bone Sarcoma Group, the Australasian Gastro-Intestinal Trials Group, and others

Casali PG, Le Cesne A, Velasco AP, et al.

Ann Oncol. 2021;32(4):533-541

PERSIST-5
Category 2A

Efficacy and Tolerability of 5-Year Adjuvant Imatinib Treatment for Patients With Resected Intermediate- or High-Risk Primary Gastrointestinal Stromal Tumor: The PERSIST-5 Clinical Trial

Raut CP, Espat NJ, Maki RG, et al.

JAMA Oncol. 2018;4(12):e184060

SSGXVIII/AIO (Mutation Subgroup Analysis)
Category 2A

Effect of KIT and PDGFRA Mutations on Survival in Patients With Gastrointestinal Stromal Tumors Treated With Adjuvant Imatinib: An Exploratory Analysis of a Randomized Clinical Trial

Joensuu H, Wardelmann E, Sihto H, et al.

JAMA Oncol. 2017;3(5):602-609

ACOSOG Z9001 (Molecular Correlates)
Category 2A

Pathologic and molecular features correlate with long-term outcome after adjuvant therapy of resected primary GI stromal tumor: the ACOSOG Z9001 trial

Corless CL, Ballman KV, Antonescu CR, et al.

J Clin Oncol. 2014;32(15):1563-1570

SSGXVIII/AIO (Intermediate Analysis)
Category 1

Adjuvant Imatinib for High-Risk GI Stromal Tumor: Analysis of a Randomized Trial

Joensuu H, Eriksson M, Sundby Hall K, et al.

J Clin Oncol. 2016;34(3):244-250

SSGXVIII/AIO (Risk Factor Analysis)
Category 2A

Risk factors for gastrointestinal stromal tumor recurrence in patients treated with adjuvant imatinib

Joensuu H, Eriksson M, Sundby Hall K, et al.

Cancer. 2014;120(15):2325-2333

MetaGIST
Category 1

Comparison of two doses of imatinib for the treatment of unresectable or metastatic gastrointestinal stromal tumors: a meta-analysis of 1,640 patients

Gastrointestinal Stromal Tumor Meta-Analysis Group (MetaGIST)

J Clin Oncol. 2010;28(7):1247-1253

EORTC 62005 (Molecular Correlates)
Category 2A

KIT mutations and dose selection for imatinib in patients with advanced gastrointestinal stromal tumours

Debiec-Rychter M, Sciot R, Le Cesne A, et al.

Eur J Cancer. 2006;42(8):1093-1103

KIT Exon 9 Adjuvant Cohort (Napolitano 2026)
Level IV

Adjuvant Imatinib or Observation in Patients With Gastrointestinal Stromal Tumors With KIT Exon 9 Mutations

Napolitano A, Joensuu H, Rothschild S, Evans D, Heinrich MC, et al.

JAMA Oncol. 2026

Taiwan GIST Registry (High-Risk Adjuvant)
Level IV

A nomogram for predicting recurrence-free survival in patients with high-risk GIST receiving adjuvant imatinib: A nationwide registry study

Yeh CN, Shan YS, Yang CY, Hsiao CF, Tsai CH, et al.

Exp Ther Med. 2026

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Neoadjuvant Therapy

3
RTOG 0132/ACRIN 6665
Category 2B

Phase II trial of neoadjuvant/adjuvant imatinib mesylate (IM) for advanced primary and metastatic/recurrent operable gastrointestinal stromal tumor (GIST): early results of RTOG 0132/ACRIN 6665

Eisenberg BL, Harris J, Blanke CD, et al.

J Surg Oncol. 2009;99(1):42-47

MDACC Neoadjuvant Phase II
Category 2B

A randomized, phase II study of preoperative plus postoperative imatinib in GIST: evidence of rapid radiographic response and temporal induction of tumor cell apoptosis

McAuliffe JC, Hunt KK, Lazar AJ, et al.

Ann Surg Oncol. 2009;16(4):910-919

RTOG 0132 (Long-term Update)
Category 2B

Clinical outcomes of Phase II trial of neoadjuvant/adjuvant imatinib mesylate for advanced primary and recurrent/metastatic gastrointestinal stromal tumor: long-term follow-up results of RTOG 0132

Wang D, Zhang Q, Blanke CD, et al.

Ann Surg Oncol. 2012;19(4):1074-1080

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Targeted Therapy

3
NAVIGATOR
Category 2A

Avapritinib in advanced PDGFRA D842V-mutant gastrointestinal stromal tumour (NAVIGATOR): a multicentre, open-label, phase 1 trial

Heinrich MC, Jones RL, von Mehren M, et al.

Lancet Oncol. 2020;21(7):935-946

Cassier PDGFRA Outcomes
Category 2A

Outcome of patients with platelet-derived growth factor receptor alpha-mutated gastrointestinal stromal tumors in the tyrosine kinase inhibitor era

Cassier PA, Fumagalli E, Rutkowski P, et al.

Clin Cancer Res. 2012;18(16):4458-4464

NAVIGATOR (Long-term Update)
Category 2A

Avapritinib in unresectable or metastatic PDGFRA D842V-mutant gastrointestinal stromal tumours: Long-term efficacy and safety data from the NAVIGATOR phase I trial

Jones RL, Serrano C, von Mehren M, et al.

Eur J Cancer. 2021;145:132-142

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Surgical Assessment

5
EORTC 62024 (Surgical Quality)
Category 2A

Quality of Surgery and Outcome in Localized Gastrointestinal Stromal Tumors Treated Within an International Intergroup Randomized Clinical Trial of Adjuvant Imatinib

Gronchi A, Bonvalot S, Poveda Velasco A, et al.

JAMA Surg. 2020;155(6):e200397

Hohenberger Rupture Series
Category 2A

Pattern of recurrence in patients with ruptured primary gastrointestinal stromal tumour

Hohenberger P, Ronellenfitsch U, Oladeji O, et al.

Br J Surg. 2010;97(12):1854-1859

Hølmebakk Rupture Definition
Category 2A

Definition and clinical significance of tumour rupture in gastrointestinal stromal tumours of the small intestine

Hølmebakk T, Bjerkehagen B, Boye K, et al.

Br J Surg. 2016;103(6):684-691

Hølmebakk Gastric Rupture Outcomes
Category 2A

Recurrence-free survival after resection of gastric gastrointestinal stromal tumours classified according to a strict definition of tumour rupture: a population-based study

Hølmebakk T, Bjerkehagen B, Boye K, et al.

Ann Surg Oncol. 2018;25(5):1133-1139

Boye Genotype-Rupture Association
Category 2A

Genotype and risk of tumour rupture in gastrointestinal stromal tumour

Boye K, Berner JM, Hølmebakk T, et al.

Br J Surg. 2018;105(2):e169-e175

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Molecular Pathology

1
ConticaGIST
Category 2A

Tumor genotype is an independent prognostic factor in primary gastrointestinal stromal tumors of gastric origin: a multi-institutional analysis based on ConticaGIST

Wozniak A, Rutkowski P, Schöffski P, et al.

Clin Cancer Res. 2014;20(23):6105-6116

Clinical Utility Evidence

1
Guerin Physician Underestimation Study

Physician Underestimation of the Risk of Gastrointestinal Stromal Tumor Recurrence After Resection

Guerin A, Sasane M, Gauthier G, et al.

JAMA Oncol. 2015;1(6):797-805

Disclaimer: This page lists data sources for clinical decision support purposes. All treatment recommendations should be discussed with the treating oncology team. References are sourced from PubMed, NCCN, ESMO, and other peer-reviewed publications.