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The primary endpoint was overall survival (OS) from the start of reference treatment (avapritinib for NAVIGATOR patients or first-line TKI for treatment of unresectable\/metastatic GIST for Study 1002 patients); the secondary endpoint was progression-free survival (PFS). Adjusted Kaplan\u2013Meier survival curves were compared by Cox regression.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>Fifty-six (NAVIGATOR) and 19 (Study 1002) patients with PDGFRA D842V-mutant GIST were evaluated; of the 56 patients from NAVIGATOR, a subgroup of patients treated with either 300\u2009mg (recommended phase 2 dose) or 400\u2009mg (maximum tolerated dose) avapritinib starting dose (<jats:italic>n<\/jats:italic>\u2009=\u200938) were analyzed separately. Patient characteristics were adjusted for imbalances by propensity score between the study groups. Inverse probability of treatment weighting-adjusted Kaplan\u2013Meier analysis of OS showed median OS was not reached for NAVIGATOR patients treated with any of the avapritinib doses tested and was 12.6\u2009months for Study 1002 patients; OS rate at 6\/48\u2009months was 100%\/63% in NAVIGATOR and 56%\/17% in Study 1002 (<jats:italic>P\u2009=<\/jats:italic>\u00a00.0001). In the 300\/400\u2009mg subgroup, adjusted OS rates at 6\/36\u2009months were 100%\/73 and 68%\/20% in Study 1002 (<jats:italic>P\u2009=<\/jats:italic>\u00a00.0016). Adjusted median PFS was 29.5\u2009months in NAVIGATOR and 3.4\u2009months in Study 1002.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusions<\/jats:title><jats:p>In this indirect, retrospective analysis, avapritinib demonstrated more durable survival outcomes compared with other TKIs in patients with unresectable\/metastatic PDGFRA D842V-mutant GIST.<\/jats:p><\/jats:sec><jats:sec><jats:title>Trial registration<\/jats:title><jats:p>The NAVIGATOR trial was registered at ClinicalTrials.gov as per July 2015, Identifier:<jats:ext-link xmlns:xlink=\"http:\/\/www.w3.org\/1999\/xlink\" ext-link-type=\"uri\" xlink:href=\"https:\/\/clinicaltrials.gov\/ct2\/show\/NCT02508532\">NCT02508532<\/jats:ext-link>.<\/jats:p><\/jats:sec>","DOI":"10.1186\/s12885-021-08013-1","type":"journal-article","created":{"date-parts":[[2021,3,19]],"date-time":"2021-03-19T10:18:12Z","timestamp":1616149092000},"update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":26,"title":["Clinical efficacy comparison of avapritinib with other tyrosine kinase inhibitors in gastrointestinal stromal tumors with PDGFRA D842V mutation: a retrospective analysis of clinical trial and real-world data"],"prefix":"10.1186","volume":"21","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-6158-890X","authenticated-orcid":false,"given":"Margaret","family":"von Mehren","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michael C.","family":"Heinrich","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hongliang","family":"Shi","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sergio","family":"Iannazzo","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Raymond","family":"Mankoski","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sa\u0161a","family":"Dimitrijevi\u0107","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Gerard","family":"Hoehn","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Silvia","family":"Chiroli","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Suzanne","family":"George","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"297","published-online":{"date-parts":[[2021,3,19]]},"reference":[{"issue":"33","key":"8013_CR1","doi-asserted-by":"publisher","first-page":"5352","DOI":"10.1200\/JCO.2007.15.7461","volume":"26","author":"MC Heinrich","year":"2008","unstructured":"Heinrich MC, Maki RG, Corless CL, Antonescu CR, Harlow A, Griffith D, Town A, McKinley A, Ou WB, Fletcher JA, et al. 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The study was conducted in accordance with the International Conference on Harmonisation\/Good Clinical Practice Guidelines, the ethical principles of the Declaration of Helsinki, and applicable national and local regulatory requirements. All patients provided written informed consent.Study 1002 was conducted at sites that granted institutional review board approval and in full compliance with the principles of the Declaration of Helsinki. Due to the retrospective nature of the study, ethical considerations were limited to patient confidentiality and privacy. A list of NAVIGATOR and Study 1002 institutional review boards or independent ethics committees of each study site is provided in supplementary Tables\u00a0and, respectively. Since <i>PDGFRA<\/i>-driven GIST is rare, de-identification of data may not have provided full protection of patient confidentiality. Therefore, a limited data set approach was used to allow disclosure of protected health information as defined by the Health Insurance Portability and Accountability Act for research purposes. Individual patient consent was not obtained due to the non-interventional and retrospective nature of the study.","order":2,"name":"Ethics","group":{"name":"EthicsHeading","label":"Ethics approval and consent to participate"}},{"value":"The manuscript does not contain data from any individual person, and so consent for publication is not applicable for inclusion in this report.","order":3,"name":"Ethics","group":{"name":"EthicsHeading","label":"Consent for publication"}},{"value":"Dr. von Mehren reported receiving other support from Arog Pharmaceuticals and Deciphera Pharmaceuticals outside the submitted work and other support from Blueprint Medicines Corporation during the conduct of the study. Dr. von Mehren and Dr. Heinrich both report funding from GIST Cancer Research Fund and the Jonathan David Foundation. Dr. Heinrich reports grants and personal fees from Blueprint Medicines Corporation, and a US Veterans Administration Merit Review Grant (I01 BX000338, MCH) during the conduct of the study, grants and personal fees from Deciphera Pharmaceuticals, outside the submitted work, other from MolecularMD, during the conduct of the study and personal fees and other from Novartis; in addition, Dr. Heinrich has a patent \u201cTreatment of Gastrointestinal Stromal Tumors\u201d licensed to Novartis, and having a patent \u201cActivating Mutations of PDGFRA\u201d issued. At the time of development of this study, Sergio Iannazzo was employed by Hetegy, a health economics consulting company which received consulting fees from Blueprint Medicines Corporation. Sergio Iannazzo is currently an employee and shareholder of Blueprint Medicines Corporation. Dr. George reports personal fees from Bayer, Blueprint Medicines Corporation, Daiichi Sankyo, Deciphera Pharmaceuticals, Eli Lilly, Exelixis, MORE Health, Research to Practice and UpToDate, and grants from ARIAD, Bayer, Blueprint Medicines Corporation, Deciphera Pharmaceuticals, Novartis, Pfizer, outside the submitted work; stock in Abbott Laboratories and Allergan; expert testimony for Bayer; relationship with Research to Practice; and royalties from UpToDate. Hongliang Shi, Raymond Mankoski, Sa\u0161a Dimitrijevi\u0107, Gerard Hoehn and Silvia Chiroli are employees and shareholders of Blueprint Medicines Corporation.","order":4,"name":"Ethics","group":{"name":"EthicsHeading","label":"Competing interests"}}],"article-number":"291"}}