Investigational New Drugs

, Volume 30, Issue 6, pp 2377–2383

Sorafenib in patients with metastatic gastrointestinal stromal tumors who failed two or more prior tyrosine kinase inhibitors: a phase II study of Korean gastrointestinal stromal tumors study group


  • S. H. Park
    • Division of Hematology-Oncology, Department of MedicineSungkyunkwan University Samsung Medical Center
  • M. H. Ryu
    • Department of Oncology, Asan Medical CenterUniversity of Ulsan College of Medicine
  • B. Y. Ryoo
    • Department of Oncology, Asan Medical CenterUniversity of Ulsan College of Medicine
  • S. A. Im
    • Department of Internal MedicineSeoul National University Hospital
  • H. C. Kwon
    • Department of Internal Medicine, Dong-A University College of MedicineDong-A University Hospital
  • S. S. Lee
    • Division of Oncology, Department of Internal Medicine, Yonsei University College of MedicineYonsei University Health System
  • S. R. Park
    • Research Institute and HospitalNational Cancer Center
  • B. Y. Kang
    • Department of Oncology/HematologyKyungpook National University Hospital
    • Department of Oncology, Asan Medical CenterUniversity of Ulsan College of Medicine

DOI: 10.1007/s10637-012-9795-9

Cite this article as:
Park, S.H., Ryu, M.H., Ryoo, B.Y. et al. Invest New Drugs (2012) 30: 2377. doi:10.1007/s10637-012-9795-9


Purpose To evaluated the efficacy and safety of sorafenib in patients with advanced gastrointestinal stromal tumors (GIST) who failed to previous standard treatments. Experimental Design Thirty-one patients with measurable metastatic GIST who failed both imatinib and sunitinib were accrued. Sorafenib was administered orally at 400 mg twice daily until disease progression or development of intolerance. The primary endpoint was disease control rate (response + stable disease, DCR) at 24 weeks. Results Sorafenib was well tolerated, with hand-foot skin reaction, fatigue, hypertension, and abdominal pain being the most frequent adverse events. The relative dose intensity of sorafenib during the first 6 months was >80%. Four patients achieved partial response (response rate 13%, 95% CI 1–25%), and 16 (52%) had stable disease. DCR at 24 weeks was measured as 36% (95% CI 19–52%). Median progression-free and overall survivals were 4.9 and 9.7 months, respectively. Progression-free survival of patients with prior use of nilotinib (P = .0085) and with primary genotypes other than KIT exon 11 mutation (P = .0341) was significantly shorter than that of patients without. Conclusions Sorafenib showed antitumor activity in this population of imatinib and sunitinib pretreated GIST. With sorafenib, about one third of patients can maintain disease control for more than 24 weeks.


SorafenibGastrointestinal stromal tumor

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© Springer Science+Business Media, LLC 2012