Claudio Martin, Gabriela Ileana Malcervelli, Gastón Lucas Martinengo, Patricio Levit, Patricio Servienti, Elisa Malaver, Laura Brion, Vanesa Patronella, Andrea Zumárraga, Jose Zarba
{"title":"布加替尼治疗阿根廷间变性淋巴瘤激酶（ALK）阳性转移性非小细胞肺癌（NSCLC）的安全性和有效性：一项上市后监测研究","authors":"Claudio Martin, Gabriela Ileana Malcervelli, Gastón Lucas Martinengo, Patricio Levit, Patricio Servienti, Elisa Malaver, Laura Brion, Vanesa Patronella, Andrea Zumárraga, Jose Zarba","doi":"10.1007/s40801-025-00484-z","DOIUrl":null,"url":null,"abstract":"<p><strong>Background: </strong>Brigatinib, a next-generation anaplastic lymphoma kinase (ALK) inhibitor was licensed for the treatment of ALK-positive metastatic non-small cell lung cancer in 2016. However, real-world evidence on the safety and effectiveness of brigatinib in Latin America remains limited.</p><p><strong>Objective: </strong>The aim of this study was to assess the safety and effectiveness of brigatinib in the real world in Argentina.</p><p><strong>Methods: </strong>We conducted a non-interventional cohort study of adult patients (aged ≥ 18 years) with a diagnosis of ALK-positive metastatic non-small cell lung cancer not previously treated (first line) or previously treated (second line) with an ALK inhibitor and who received at least one dose of brigatinib between November 2020 and March 2023. The primary outcome was the incidence of treatment-emergent adverse events. Secondary outcomes at the 24-week follow-up were the percentage of patients with an overall best objective response rate of complete or partial response; intracranial objective response rate; progression-free survival; and overall survival.</p><p><strong>Results: </strong>Of the 39 patients included in the study (n = 22 [first line]; n = 17 [second line]), 12 patients (30.7%) experienced treatment-emergent adverse events, with the most frequent being increased levels of transaminases (7.6%), increased level of blood creatine phosphokinase (5%) and hypokalaemia (5%). Most adverse events (85.7%) were mild to moderate. Effectiveness outcomes at 24 weeks in patients treated with brigatinib first line or second line, respectively, were as follows: overall objective response rate: 81.8% and 70.5%; intracranial objective response rate (in patients with brain metastases at baseline): 66.6% and 88.8%; progression-free survival: 93.8% and 82.4%; overall survival: 100% and 87.5%.</p><p><strong>Conclusions: </strong>Brigatinib was demonstrated to be a safe and effective treatment option for ALK-positive metastatic non-small cell lung cancer in routine clinical practice in Argentina.</p><p><strong>Clinical trial registration: </strong>NCT04887519.</p>","PeriodicalId":11282,"journal":{"name":"Drugs - Real World Outcomes","volume":" ","pages":"227-235"},"PeriodicalIF":1.9000,"publicationDate":"2025-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Safety and Effectiveness of Brigatinib in Anaplastic Lymphoma Kinase (ALK) Positive Metastatic Non-Small Cell Lung Cancer (NSCLC) in Argentina: A Post-Marketing Surveillance Study.\",\"authors\":\"Claudio Martin, Gabriela Ileana Malcervelli, Gastón Lucas Martinengo, Patricio Levit, Patricio Servienti, Elisa Malaver, Laura Brion, Vanesa Patronella, Andrea Zumárraga, Jose Zarba\",\"doi\":\"10.1007/s40801-025-00484-z\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><strong>Background: </strong>Brigatinib, a next-generation anaplastic lymphoma kinase (ALK) inhibitor was licensed for the treatment of ALK-positive metastatic non-small cell lung cancer in 2016. However, real-world evidence on the safety and effectiveness of brigatinib in Latin America remains limited.</p><p><strong>Objective: </strong>The aim of this study was to assess the safety and effectiveness of brigatinib in the real world in Argentina.</p><p><strong>Methods: </strong>We conducted a non-interventional cohort study of adult patients (aged ≥ 18 years) with a diagnosis of ALK-positive metastatic non-small cell lung cancer not previously treated (first line) or previously treated (second line) with an ALK inhibitor and who received at least one dose of brigatinib between November 2020 and March 2023. The primary outcome was the incidence of treatment-emergent adverse events. Secondary outcomes at the 24-week follow-up were the percentage of patients with an overall best objective response rate of complete or partial response; intracranial objective response rate; progression-free survival; and overall survival.</p><p><strong>Results: </strong>Of the 39 patients included in the study (n = 22 [first line]; n = 17 [second line]), 12 patients (30.7%) experienced treatment-emergent adverse events, with the most frequent being increased levels of transaminases (7.6%), increased level of blood creatine phosphokinase (5%) and hypokalaemia (5%). Most adverse events (85.7%) were mild to moderate. Effectiveness outcomes at 24 weeks in patients treated with brigatinib first line or second line, respectively, were as follows: overall objective response rate: 81.8% and 70.5%; intracranial objective response rate (in patients with brain metastases at baseline): 66.6% and 88.8%; progression-free survival: 93.8% and 82.4%; overall survival: 100% and 87.5%.</p><p><strong>Conclusions: </strong>Brigatinib was demonstrated to be a safe and effective treatment option for ALK-positive metastatic non-small cell lung cancer in routine clinical practice in Argentina.</p><p><strong>Clinical trial registration: </strong>NCT04887519.</p>\",\"PeriodicalId\":11282,\"journal\":{\"name\":\"Drugs - Real World Outcomes\",\"volume\":\" \",\"pages\":\"227-235\"},\"PeriodicalIF\":1.9000,\"publicationDate\":\"2025-06-01\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Drugs - Real World Outcomes\",\"FirstCategoryId\":\"1085\",\"ListUrlMain\":\"https://doi.org/10.1007/s40801-025-00484-z\",\"RegionNum\":0,\"RegionCategory\":null,\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"2025/2/26 0:00:00\",\"PubModel\":\"Epub\",\"JCR\":\"Q3\",\"JCRName\":\"PHARMACOLOGY & PHARMACY\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Drugs - Real World Outcomes","FirstCategoryId":"1085","ListUrlMain":"https://doi.org/10.1007/s40801-025-00484-z","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"2025/2/26 0:00:00","PubModel":"Epub","JCR":"Q3","JCRName":"PHARMACOLOGY & PHARMACY","Score":null,"Total":0}
Safety and Effectiveness of Brigatinib in Anaplastic Lymphoma Kinase (ALK) Positive Metastatic Non-Small Cell Lung Cancer (NSCLC) in Argentina: A Post-Marketing Surveillance Study.
Background: Brigatinib, a next-generation anaplastic lymphoma kinase (ALK) inhibitor was licensed for the treatment of ALK-positive metastatic non-small cell lung cancer in 2016. However, real-world evidence on the safety and effectiveness of brigatinib in Latin America remains limited.
Objective: The aim of this study was to assess the safety and effectiveness of brigatinib in the real world in Argentina.
Methods: We conducted a non-interventional cohort study of adult patients (aged ≥ 18 years) with a diagnosis of ALK-positive metastatic non-small cell lung cancer not previously treated (first line) or previously treated (second line) with an ALK inhibitor and who received at least one dose of brigatinib between November 2020 and March 2023. The primary outcome was the incidence of treatment-emergent adverse events. Secondary outcomes at the 24-week follow-up were the percentage of patients with an overall best objective response rate of complete or partial response; intracranial objective response rate; progression-free survival; and overall survival.
Results: Of the 39 patients included in the study (n = 22 [first line]; n = 17 [second line]), 12 patients (30.7%) experienced treatment-emergent adverse events, with the most frequent being increased levels of transaminases (7.6%), increased level of blood creatine phosphokinase (5%) and hypokalaemia (5%). Most adverse events (85.7%) were mild to moderate. Effectiveness outcomes at 24 weeks in patients treated with brigatinib first line or second line, respectively, were as follows: overall objective response rate: 81.8% and 70.5%; intracranial objective response rate (in patients with brain metastases at baseline): 66.6% and 88.8%; progression-free survival: 93.8% and 82.4%; overall survival: 100% and 87.5%.
Conclusions: Brigatinib was demonstrated to be a safe and effective treatment option for ALK-positive metastatic non-small cell lung cancer in routine clinical practice in Argentina.
期刊介绍：
Drugs - Real World Outcomes targets original research and definitive reviews regarding the use of real-world data to evaluate health outcomes and inform healthcare decision-making on drugs, devices and other interventions in clinical practice. The journal includes, but is not limited to, the following research areas: Using registries/databases/health records and other non-selected observational datasets to investigate: drug use and treatment outcomes prescription patterns drug safety signals adherence to treatment guidelines benefit : risk profiles comparative effectiveness economic analyses including cost-of-illness Data-driven research methodologies, including the capture, curation, search, sharing, analysis and interpretation of ‘big data’ Techniques and approaches to optimise real-world modelling.
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