NOVEL-1st: an observational study to assess the safety and efficacy of nilotinib in newly diagnosed patients with Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase in Taiwan
Journal
International journal of hematology
Journal Volume
115
Journal Issue
5
Pages
704
Date Issued
2022-05
Author(s)
Hwang, Wen-Li
Chen, Tsung-Chih
Lin, Hsuan-Yu
Chang, Ming-Chih
Hsiao, Pei-Ching
Bai, Li-Yuan
Kuo, Ching-Yuan
Chen, Yeu-Chin
Liu, Ta-Chih
Gau, Jyh-Pyng
Wang, Po-Nan
Hwang, Wei-Shou
Kuo, Ming-Chung
Liu, Chun-Yu
Liu, Yi-Chang
Ma, Ming-Chun
Su, Nai-Wen
Wang, Chuan-Cheng
Wu, Yi-Ying
Yeh, Su-Peng
Cheng, Hao-Wei
Lee, Yee-Ming
Ku, Fan-Chen
Abstract
Nilotinib has been approved for the treatment of Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase (Ph+ CML-CP). However, the real-world evidence of nilotinib in newly diagnosed untreated Ph+ CML-CP is limited in Taiwan. The NOVEL-1st study was a non-interventional, multi-center study collecting long-term safety and effectiveness data in patients with newly diagnosed and untreated Ph+ CML-CP receiving nilotinib. We enrolled 129 patients from 11 hospitals. Overall, 1,466 adverse events (AEs) were reported; among these, 151 were serious and 524 were nilotinib-related. Common hematological AEs were thrombocytopenia (31.0%), anemia (20.9%), and leukopenia (14.0%); common nilotinib-related AEs were thrombocytopenia (29.5%), anemia (14.7%), and leukopenia (12.4%). Early molecular response, defined as BCR-ABL ≤ 10% at Month 3, was seen in 87.6% of patients. By 36 months, the cumulative rates of complete hematologic response, complete cytogenetic response, major molecular response, molecular response 4.0-log reduction, and molecular response 4.5-log reduction were 98.5, 92.5, 85.8, 65.0, and 45.0%, respectively. Nilotinib is effective and well-tolerated in patients with newly diagnosed Ph+ CML-CP in the real-world setting. Long-term holistic care and a highly tolerable AE profile may contribute to good treatment outcomes in Ph+ CML-CP under first-line treatment with nilotinib.
Subjects
Chronic myeloid leukemia; First-line treatment; Nilotinib; Real-world setting
Type
journal article