Publication: Expanding Nilotinib Access in Clinical Trials (ENACT): An open-label, multicenter study of oral nilotinib in adult patients with imatinib-resistant or imatinib-intolerant Philadelphia chromosome-positive chronic myeloid leukemia in the chronic phase
Issued Date
2012-01-01
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ISSN
10970142
0008543X
0008543X
Other identifier(s)
2-s2.0-83855164150
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Mahidol University
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SCOPUS
Bibliographic Citation
Cancer. Vol.118, No.1 (2012), 118-126
Suggested Citation
Franck E. Nicolini, Anna Turkina, Zhi Xiang Shen, Neil Gallagher, Saengsuree Jootar, Bayard L. Powell, Carmino De Souza, Ming Zheng, Tomasz Szczudlo, Philipp Le Coutre Expanding Nilotinib Access in Clinical Trials (ENACT): An open-label, multicenter study of oral nilotinib in adult patients with imatinib-resistant or imatinib-intolerant Philadelphia chromosome-positive chronic myeloid leukemia in the chronic phase. Cancer. Vol.118, No.1 (2012), 118-126. doi:10.1002/cncr.26249 Retrieved from: https://repository.li.mahidol.ac.th/handle/20.500.14594/13870
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Title
Expanding Nilotinib Access in Clinical Trials (ENACT): An open-label, multicenter study of oral nilotinib in adult patients with imatinib-resistant or imatinib-intolerant Philadelphia chromosome-positive chronic myeloid leukemia in the chronic phase
Abstract
BACKGROUND: Nilotinib is a selective, potent BCR-ABL inhibitor. Previous studies demonstrated the efficacy and safety of nilotinib in Philadelphia chromosome-positive chronic myeloid leukemia patients in chronic phase (CML-CP) or accelerated phase who failed prior imatinib. METHODS: This expanded access trial further characterized the safety of nilotinib 400 mg twice daily in patients with CML-CP (N = 1422). RESULTS: In this large, heavily pretreated population, nilotinib demonstrated significant efficacy, with complete hematologic response and complete cytogenetic response achieved in 43% and 34% of patients, respectively. Responses were rapid, mostly occurring within 6 months, and were higher in patients with suboptimal response to imatinib, with 75% and 50% achieving major cytogenetic response and complete cytogenetic response, respectively. At 18 months, the progression-free survival rate was 80 %. Most patients achieved planned dosing of 400 mg twice daily and maintained the dose > 12 months. Nonhematologic adverse events (AEs) were mostly mild to moderate and included rash (28%), headache (25%), and nausea (17%). Grade 3 or 4 thrombocytopenia (22%), neutropenia (14%), and anemia (3%) were low and managed by dose reduction or brief interruption. Grade 3 or 4 elevations in serum bilirubin and lipase occurred in 4% and 7% of patients, respectively. The incidence of newly occurring AEs decreased over time. Of patients who experienced a dose reduction because of AEs and attempted a re-escalation, 87% successfully achieved re-escalation to the full dose. CONCLUSIONS: This large study confirms that nilotinib was well tolerated and that grade 3 or 4 AEs occurred infrequently and were manageable through transient dose interruptions. Copyright © 2011 American Cancer Society.
