Nilotinib cannot be recommended for broad use as the first-line treatment of patients with gastrointestinal stromal tumors (GISTs), a recent study published in the journal The Lancet Oncology has concluded.

Because nilotinib has activity against platelet-derived growth factor receptors (PDGFRs), and mutations in PDGFRα are key drivers in most GISTS, researchers sought to compare the efficacy and safety of nilotinib versus imatinib as first-line therapy for patients with advanced GISTs.

For the open-label, multicenter, phase III trial, researchers enrolled 647 patients and randomly assigned them 1:1 to receive oral imatinib 400mg once daily or nilotinib 400mg twice daily.

Continue Reading

Results showed that 2-year progression free survival was 59.2% (95% CI: 50.9-66.5) in the imatinib group compared with 51.6% (95% CI: 43.0-59.5) in the nilotinib group (HR = 1.47; 95% CI: 1.10-1.95).

In regard to safety, the most common grade 3-4 adverse events in the imatinib group were hypophosphatemia, anemia, abdominal pain, and elevated lipase level.

RELATED: Potential Therapy for Rare, Drug-resistant GISTs

In the nilotinib group, the most common grade 3-4 adverse events were anemia, elevated lipase level, increased alanine aminotransferase concentration, and abdominal pain. Serious abdominal pain was experienced by 4% of patients in each group.

Although the findings show that nilotinib is less effective than imatinib for first-line treatment of patients with GIST, the authors conclude that future studies may identify certain patients who would benefit from nilotinib treatment.


  1. Blay J-Y, Shen L, Kang Y-K, et al. Nilotinib versus imatinib as first-line therapy for patients with unresectable or metastatic gastrointestinal stromal tumours (ENESTg1): a randomised phase 3 trial. Lancet Oncol. 2015. [Epub ahead of print]. doi: 10.1016/S1470-2045(15)70105-1.