CFTR调节剂的临床疗效(随访6个月)

Q4 Medicine

Pulmonologiya Pub Date : 2023-04-12 DOI:10.18093/0869-0189-2023-33-2-189-197

E. Kondratyeva, N. Odinaeva, E. Zhekaite, E. Pasnova, I. R. Fatkhullina, T. Maksimycheva, V. Sherman, S. Kutsev

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Respiratory function indicators (FEV1, FVC), sweat test, and anthropometric data were analyzed.Results. In the group of children treated with lumacaftor/ivacaftor, Me (Q1 - Q3) body weight (kg) increased at 6-month follow-up from 40.0 (28.9 - 48.0) to 44.9 (29.3 - 50.8), p < 0.001, and the height (cm) increased from 156.0 (140.0 - 161.0) to 158.0 (143.0 - 162.0),p < 0.001. Me (Q1 - Q3) FEV1 improved from 63.5 (42.3 - 84.8) to 72.0 (56.9 - 82.4) %,p < 0.045. Sweat test (mmol/l) decreased Me (Q1 - Q3) from 115 (101.0 - 123.0) to 86.5 (79.0 - 103.0), p < 0.001. During therapy with elexacaftor/tezacaftor/ ivacaftor, Me (Q1 - Q3) body weight (kg) increased at 6-month follow-up from 44.4 (36.8 - 50.0) to 49.3 (44.1 - 51.9),p < 0.001, and Me (Q1 -Q3) height (cm) increased from 160.5 (152.3 - 165.1) to 163.0 (155.5 - 166.9),p < 0.001. Me (Q1 - Q3) FVC and FEV1 improved: FVC from 78.5 (60.9 - 91.0) to 90.5 (76.8 - 106.8) %,p < 0.001, FEV1 from 73.5 (60.5 - 82.1) to 95.0 (65.3 - 107.0) %,p < 0.001. Sweat test (mmol/l) decreased from 119 (108 - 126) to 75.5 (65.3 - 88);р < 0.001.Conclusion. Health status indicators of of children with CF aged 6 - 18 years were analyzed for 6 months of targeted therapy (lumacaftor/ivacaftor and elexacaftor/tezacaftor/ivacaftor). Positive changes were observed in weight, height, respiratory function, and sweat test.","PeriodicalId":37383,"journal":{"name":"Pulmonologiya","volume":"25 1","pages":""},"PeriodicalIF":0.0000,"publicationDate":"2023-04-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Efficacy of CFTR modulators in clinical practice (6-month follow-up)\",\"authors\":\"E. Kondratyeva, N. Odinaeva, E. Zhekaite, E. Pasnova, I. R. Fatkhullina, T. Maksimycheva, V. Sherman, S. 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引用次数: 0

摘要

囊性纤维化(CF)的致病疗法已经开发出来，可以调节CFTR蛋白并恢复其作为氯离子通道的活性。这种治疗以不同基因型的CFTR调节剂为代表，覆盖85% - 90%的CF患者。目的是通过6个月的随访，分析两种靶向药物在6- 18岁CF患者临床实践中的疗效。该研究是基于对2021 - 2022年“俄罗斯联邦CF患者登记处”数据库的分析进行的。该研究包括178例接受lumacaftor/ivacaftor治疗的患者和158例接受elexaftor /tezacaftor/ivacaftor治疗的患者。分析呼吸功能指标(FEV1、FVC)、汗液试验及人体测量数据。lumacaftor/ivacaftor组6个月随访时Me (Q1 - Q3)体重(kg)从40.0(28.9 ~ 48.0)增加到44.9 (29.3 ~ 50.8)，p < 0.001，身高(cm)从156.0(140.0 ~ 161.0)增加到158.0 (143.0 ~ 162.0)，p < 0.001。Me (Q1 - Q3) FEV1从63.5(42.3 - 84.8)%提高到72.0 (56.9 - 82.4)%，p < 0.045。排汗试验(mmol/l)使Me (Q1 - Q3)从115(101.0 - 123.0)降至86.5 (79.0 - 103.0)，p < 0.001。在elexaftor /tezacaftor/ ivacaftor治疗期间，Me (Q1 -Q3)体重(kg)从44.4(36.8 - 50.0)增加到49.3 (44.1 - 51.9)，p < 0.001, Me (Q1 -Q3)身高(cm)从160.5(152.3 - 165.1)增加到163.0 (155.5 - 166.9)，p < 0.001。Me (Q1 - Q3) FVC和FEV1改善:FVC从78.5(60.9 - 91.0)%提高到90.5 (76.8 - 106.8)%，p < 0.001, FEV1从73.5(60.5 - 82.1)%提高到95.0 (65.3 - 107.0)%，p < 0.001。汗液试验(mmol/l)由119(108 ~ 126)降至75.5 (65.3 ~ 88);对6 ~ 18岁CF患儿进行6个月的靶向治疗(lumacaftor/ivacaftor和eleexaftor /tezacaftor/ivacaftor)后的健康状况指标进行分析。体重、身高、呼吸功能、汗液试验均出现阳性变化。

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Efficacy of CFTR modulators in clinical practice (6-month follow-up)

Pathogenetic therapy for the treatment of cystic fibrosis (CF) has been developed that modulates the CFTR protein and restores its activity as a chloride channel. This treatment is represented by CFTR modulators for various genotypes covering 85 - 90% of patients with CF.The aim was to analyze the efficacy of two targeted drugs in patients aged 6 - 18 years with CF in clinical practice during a 6-month follow-up.Methods. The study was conducted based on the analysis of the database “CF Patient Registry of the Russian Federation” for 2021 - 2022. The study included 178 patients receiving lumacaftor/ivacaftor and 158 patients receiving elexacaftor/tezacaftor/ivacaftor. Respiratory function indicators (FEV1, FVC), sweat test, and anthropometric data were analyzed.Results. In the group of children treated with lumacaftor/ivacaftor, Me (Q1 - Q3) body weight (kg) increased at 6-month follow-up from 40.0 (28.9 - 48.0) to 44.9 (29.3 - 50.8), p < 0.001, and the height (cm) increased from 156.0 (140.0 - 161.0) to 158.0 (143.0 - 162.0),p < 0.001. Me (Q1 - Q3) FEV1 improved from 63.5 (42.3 - 84.8) to 72.0 (56.9 - 82.4) %,p < 0.045. Sweat test (mmol/l) decreased Me (Q1 - Q3) from 115 (101.0 - 123.0) to 86.5 (79.0 - 103.0), p < 0.001. During therapy with elexacaftor/tezacaftor/ ivacaftor, Me (Q1 - Q3) body weight (kg) increased at 6-month follow-up from 44.4 (36.8 - 50.0) to 49.3 (44.1 - 51.9),p < 0.001, and Me (Q1 -Q3) height (cm) increased from 160.5 (152.3 - 165.1) to 163.0 (155.5 - 166.9),p < 0.001. Me (Q1 - Q3) FVC and FEV1 improved: FVC from 78.5 (60.9 - 91.0) to 90.5 (76.8 - 106.8) %,p < 0.001, FEV1 from 73.5 (60.5 - 82.1) to 95.0 (65.3 - 107.0) %,p < 0.001. Sweat test (mmol/l) decreased from 119 (108 - 126) to 75.5 (65.3 - 88);р < 0.001.Conclusion. Health status indicators of of children with CF aged 6 - 18 years were analyzed for 6 months of targeted therapy (lumacaftor/ivacaftor and elexacaftor/tezacaftor/ivacaftor). Positive changes were observed in weight, height, respiratory function, and sweat test.

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来源期刊

Pulmonologiya Medicine-Pulmonary and Respiratory Medicine

CiteScore

1.40

自引率

0.00%

发文量

期刊介绍： The aim of this journal is to state a scientific position of the Russian Respiratory Society (RRS) on diagnosis and treatment of respiratory diseases based on recent evidence-based clinical trial publications and international consensuses. The most important tasks of the journal are: -improvement proficiency qualifications of respiratory specialists; -education in pulmonology; -prompt publication of original studies on diagnosis and treatment of respiratory diseases; -sharing clinical experience and information about pulmonology service organization in different regions of Russia; -information on current protocols, standards and recommendations of international respiratory societies; -discussion and consequent publication Russian consensus documents and announcement of RRS activities; -publication and comments of regulatory documents of Russian Ministry of Health; -historical review of Russian pulmonology development. The scientific concept of the journal includes publication of current evidence-based studies on respiratory medicine and their discussion with the participation of Russian and foreign experts and development of national consensus documents on respiratory medicine. Russian and foreign respiratory specialists including pneumologists, TB specialists, thoracic surgeons, allergists, clinical immunologists, pediatricians, oncologists, physiologists, and therapeutists are invited to publish article in the journal.