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Original Investigation
May 29, 2020

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

Author Affiliations
  • 1Department of Oncology, Helsinki University Hospital and University of Helsinki, Helsinki, Finland
  • 2Department of Oncology, Skåne University Hospital and Lund University, Lund, Sweden
  • 3Department of Oncology, The Norwegian Radium Hospital, Oslo University Hospital, Oslo, Norway
  • 4Helios Klinikum Berlin-Buch, Sarkomzentrum Berlin-Brandenburg, Berlin, Germany
  • 5Universitätsklinikum Tübingen, Tübingen, Germany
  • 6Schwerpunktpraxis Oncology/Hematology, Düsseldorf, Germany
  • 7Department of Gastroenterology, University of Göttingen, Göttingen, Germany
  • 8Division of Surgical Oncology & Thoracic Surgery, Mannheim University Medical Center, Mannheim, Germany
  • 9Medical Department III, Klinikum rechts der Isar, Technical University of Munich, Munich, Germany
  • 10Klinik für Onkologie und Hämatologie am Krankenhaus Nordwest, Frankfurt, Germany
  • 11Department of Medicine III, University Hospital, LMU Munich, Munich, Germany
  • 12Sarcoma Center, West German Cancer Center, Essen, Germany
  • 13Gerhard-Domagk-Institute of Pathology, University of Münster, Münster, Germany
  • 14Department of Surgery, Sahlgrenska University Hospital, Gothenburg, Sweden
  • 15Department of Oncology and Radiotherapy, Oulu University Hospital, Oulu, Finland
  • 164Pharma Ltd, Turku, Finland
  • 17Department of Cancer Epidemiology, Lund University, Lund, Sweden
JAMA Oncol. 2020;6(8):1241-1246. doi:10.1001/jamaoncol.2020.2091
Visual Abstract. Survival Outcomes Associated With 3 Years vs 1 Year of Adjuvant Imatinib for Patients With High-Risk Gastrointestinal Stromal Tumors
Survival Outcomes Associated With 3 Years vs 1 Year of Adjuvant Imatinib for Patients With High-Risk Gastrointestinal Stromal Tumors
Key Points

Question  Does administration of adjuvant imatinib for 3 years after macroscopically complete surgery prolong overall survival (OS) compared with 1-year administration in patients with localized gastrointestinal stromal tumor (GIST) with high-risk features?

Findings  This secondary analysis of a randomized clinical trial of 397 patients with high-risk GIST found that, after a median follow-up time of 10 years after the study entry, patients randomly allocated to receive 3 years of imatinib had longer recurrence-free survival and OS than patients allocated to 1 year of imatinib: Approximately one-half of deaths were avoided with the longer treatment. No new adverse safety signals were detected.

Meaning  Three years of treatment with adjuvant imatinib was associated with improved OS compared with 1 year of imatinib in patients with high-risk GIST.


Importance  Adjuvant imatinib is associated with improved recurrence-free survival (RFS) when administered after surgery to patients with operable gastrointestinal stromal tumor (GIST), but its influence on overall survival (OS) has remained uncertain.

Objective  To evaluate the effect of adjuvant imatinib on OS of patients who have a high estimated risk for GIST recurrence after macroscopically complete surgery.

Design, Setting, and Participants  In this open-label, randomized (1:1), multicenter phase 3 clinical trial conducted in Finland, Germany, Norway, and Sweden, 400 patients who had undergone macroscopically complete surgery for GIST with a high estimated risk for recurrence according to the modified National Institutes of Health Consensus Criteria were enrolled between February 2004 and September 2008. Data for this follow-up analysis were analyzed from September to November, 2019.

Interventions  Imatinib 400 mg/d administered orally for either 12 months or 36 months after surgery.

Main Outcomes And Measures  The primary end point was RFS; the secondary objectives included OS and treatment safety.

Results  The intention-to-treat cohort consisted of 397 patients (12-month group, 199; 36-month group, 198; 201 men and 196 women; median [IQR] age, 62 (51-69) years and 60 (51-67) years, during a median follow-up time of 119 months after the date of randomization, 194 RFS events and 96 OS events were recorded in the intention-to-treat population. Five-year and 10-year RFS was 71.4% and 52.5%, respectively, in the 36-month group and 53.0% and 41.8% in the 12-month group (hazard ratio [HR], 0.66; 95% CI, 0.49-0.87; P = .003). In the 36-month group, 5-year OS and 10-year OS rates were 92.0% and 79.0%, respectively, and in the 12-month group 85.5% and 65.3% (HR, 0.55; 95% CI, 0.37-0.83; P = .004). The results were similar in the efficacy population, from which 15 patients who did not have GIST in central pathology review and 24 patients who had intra-abdominal metastases removed at surgery were excluded (36-month group, 10-year OS 81.6%; 12-month group, 66.8%; HR, 0.50; 95% CI, 0.32-0.80; P = .003). No new safety signals were detected.

Conclusions and Relevance  Three years of adjuvant imatinib is superior in efficacy compared with 1 year of imatinib. Approximately 50% of deaths may be avoided during the first 10 years of follow-up after surgery with longer adjuvant imatinib treatment.

Trial Registration  ClinicalTrials.gov Identifier: NCT00116935

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