Abstract
7050 Background: The potent and highly selective BCR-ABL inhibitor nilotinib has been approved in both the US and Europe for the treatment of patients (pts) with Philadelphia chromosome-positive (Ph+) chronic phase chronic myelogenous leukemia (CML- CP) and CML-AP who are resistant or intolerant to prior therapy, including imatinib. Methods: This phase 2, open-label study evaluated the efficacy and safety of nilotinib in pts with Ph+ CML-AP with imatinib resistance or intolerance. Primary and key secondary endpoints were rate of confirmed hematologic response (HR) and MCyR, respectively. Nilotinib was started at 400 mg twice daily (BID) and escalated to 600 mg BID for inadequate responses. Results: Included in the analysis were 136 pts who received at least 1 dose of nilotinib. Median age was 58 (22–82) yrs; median dose intensity was 781 mg/day; median treatment duration was 210 days. Confirmed HR occurred in 69/129 pts (54%); 34 (26%) had complete HR. Of imatinib-resistant pts, 55/104 pts (53%) had a confirmed HR, and 14/25 imatinib- intolerant pts (56%) had a confirmed HR. A majority of responding pts (82%) maintained HR for at least 12 months. MCyR occurred in 40/129 pts (31%); 24/129 pts (19%) had complete CyR. Of imatinib-resistant pts, 30/104 pts (29%) had a MCyR, and 10/25 imatinib-intolerant pts (40%) had a MCyR. Time to first HR and MCyR was 1 and 2.8 months, respectively. At 12 months, it is estimated that 57% of pts were without progression and the estimated overall survival rate was 81%. At data cutoff, treatment was ongoing for 57/136 pts (42%). The most common grade 3/4 laboratory abnormalities were thrombocytopenia (41%), neutropenia (39%), anemia (25%), and elevated serum lipase (16%). Grade 3/4 non-hematologic AEs were rare and included nausea, fatigue, headache, and diarrhea. Conclusions: The results of this study confirm that nilotinib induces significant and durable responses in CML-AP pts with imatinib resistance or intolerance. There was minimal nilotinib cross- intolerance in patients intolerant to prior imatinib treatment. Nilotinib is well tolerated, with minimal occurrence of grade 3/4 AEs, and is an effective treatment option for this pt population. Author Disclosure Employment or Leadership Consultant or Advisory Role Stock Ownership Honoraria Research Expert Testimony Other Remuneration Novartis Bristol-Myers Squibb, Innovive, Novartis Bristol-Myers Squibb, Novartis Bristol-Myers Squibb, Novartis, Wyeth
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