Background/aims: This study evaluated the outcomes of the patients with metastatic gastrointestinal stromal tumor who failed imatinib and sunitinib treatment, and were treated with nilotinib within a compassionate use program.
Methodology: Patients who had previously received imatinib and sunitinib and had experienced disease progression or drug intolerance with this therapy were included. Demographic and clinical characteristics of the patients, response to nilotinib and duration of response were evaluated along with adverse events.
Results: Data of 7 patients (6 males and 1 female) who received nilotinib for at least 30 days were analyzed. Median age of the patients was 68 years (range, 47-70). Complete response was achieved in one patient. One patient experienced partial response, 2 patients stable disease and 3 patients had progressive disease with nilotinib treatment. No life threatening adverse events were observed. Three patients experienced grade 2-3 asthenia. No electrocardiographic changes were observed throughout the treatment period.
Conclusions: Nilotinib is active and may represent an alternative treatment choice in patients with gastrointestinal stromal tumor resistant to imatinib and sunitinib.