{"doi":"10.1002/cncr.32623","title":"Long‐term results of a phase 2 trial of nilotinib 400 mg twice daily in newly diagnosed patients with chronic‐phase chronic myeloid leukemia","abstract":"BACKGROUND: Nilotinib is a potent, second-generation inhibitor of BCR-ABL1 tyrosine kinase and has been approved as frontline and salvage therapy for patients with chronic-phase chronic myeloid leukemia (CP-CML). METHODS: In this single-institution, phase 2 study, 122 patients with newly diagnosed CP-CML received nilotinib 400 mg twice daily. The median follow-up on study was 78.3 months (interquartile range, 58.4-96.5 months). RESULTS: Fifty-six percent of patients remained on therapy at the last follow-up. Both the complete cytogenetic response rate and the major molecular response (MR) rate were 91%. Seventy-five percent and 59% of patients achieved a ≥4.5-log reduction in BCR-ABL1 transcripts (MR4.5) and a sustained MR4.5 beyond 2 years, respectively. The estimated event-free survival and overall survival rates at 5 years were 89% and 93%, respectively, and the corresponding rates at 10 years were 85% and 88%, respectively. Treatment discontinuation due to toxicity occurred in 19% of patients, mostly because of cardiovascular events (10%) and biochemical abnormalities (6%). The top 3 nonhematologic toxicities were rash (55%), elevated bilirubin (57%), and elevated aminotransferases (48%). Hematologic toxicity was transient and mild. Ischemic cardiovascular adverse events occurred in 8% of patients. Four patients (3%) progressed to accelerated or blast phase while on therapy, and 7 patients (6%) died on study. CONCLUSIONS: The current data confirm the long-term efficacy of nilotinib 400 mg twice daily in patients with CP-CML. A majority of patients can achieve sustained MR4.5.","journal":"Cancer","year":2020,"id":68905,"datarank":0.4636563680037475,"base_score":3.091042453358316,"endowment":3.091042453358316,"self_citation_contribution":0.4636563680037475,"citation_network_contribution":0.0,"self_endowment_contribution":0.4636563680037475,"citer_contribution":0.0,"corpus_percentile":null,"corpus_rank":null,"citation_count":21,"citer_count":0,"citers_with_citation_signal":0,"citers_with_endowment":0,"datacite_reuse_total":0,"is_dataset":false,"is_dataset_confidence":0.8693,"is_data_producer":false,"deposit_databanks":null,"is_oa":true,"file_count":0,"downloads":0,"has_version_chain":false,"published_date":"2020-01-01","fair_score":null,"fair_percentile":null,"algorithm_id":"datarank_citation_only_1hop_v6","ranking_scope":"data_only","authors":[{"id":230406,"name":"Jörge E. Cortes","orcid":"0000-0002-8636-1071","position":1,"is_corresponding":false},{"id":33069,"name":"Keyur P. Patel","orcid":"0000-0001-5081-2427","position":2,"is_corresponding":false},{"id":364890,"name":"Susan O’Brien","orcid":"0000-0003-3386-6540","position":3,"is_corresponding":false},{"id":350728,"name":"Graciela M. Nogueras‐González","orcid":"0000-0002-6350-2206","position":4,"is_corresponding":false},{"id":18373,"name":"Marina Konopleva","orcid":"0000-0002-9347-2212","position":5,"is_corresponding":false},{"id":232854,"name":"Srđan Verstovšek","orcid":"0000-0002-6912-8569","position":6,"is_corresponding":false},{"id":230408,"name":"Guillermo Garcia‐Manero","orcid":"0000-0002-3631-2482","position":7,"is_corresponding":false},{"id":232855,"name":"Alessandra Ferrajoli","orcid":"0000-0001-9547-4193","position":8,"is_corresponding":false},{"id":232841,"name":"Tapan M. Kadia","orcid":"0000-0002-9892-9832","position":9,"is_corresponding":false},{"id":365823,"name":"Farhad Ravandi‐Kashani","orcid":null,"position":10,"is_corresponding":false},{"id":232842,"name":"Gautam Borthakur","orcid":"0000-0001-7679-6453","position":11,"is_corresponding":false},{"id":365824,"name":"Sara Dellasala","orcid":null,"position":12,"is_corresponding":false},{"id":232851,"name":"Zeev Estrov","orcid":"0000-0002-1623-3613","position":13,"is_corresponding":false},{"id":230404,"name":"Elias Jabbour","orcid":"0000-0003-4465-6119","position":14,"is_corresponding":false},{"id":109598,"name":"Hagop M. Kantarjian","orcid":"0000-0002-1908-3307","position":15,"is_corresponding":false},{"id":232852,"name":"Lucia Masárová","orcid":"0000-0001-6624-4196","position":0,"is_corresponding":true}],"reference_count":18,"raw_metadata":{"citation_network_status":"fetched"},"created_at":"2026-07-18T21:41:52.279368Z","pmid":"31999850","pmcid":null,"fwci":null,"citation_percentile":null,"influential_citations":0,"oa_status":null,"license":null,"views":0,"total_file_size_bytes":0,"version_count":0,"fair_f":null,"fair_a":null,"fair_i":null,"fair_r":null,"fair_zscore":null,"fair_rationale":null,"fair_model":null,"fair_agent_version":null,"fair_fulltext_source":null,"fair_has_llm":null,"fair_computed_at":null,"clinical_trials":[],"software_tools":[],"db_accessions":[],"linked_datasets":[],"topics":[]}