PDF(Pubmed)
Abstract：
Background/Objectives: Cystic fibrosis is a genetically determined disease that significantly influences and shortens life. Treatment with CFTR modulators (CFTR-T) is a new hope for patients. It can change the predictive values of a poor prognosis (e.g., exacerbation rate and FEV1 value). The aim of the study was to analyse exacerbation incidence and spirometry data before and after one year (+/- 2 weeks) of CFTR-T in 85 CF patients at the CF Centre in Poznań. To our knowledge, this is the first analysis of CFTR-T efficiency in the Central-Eastern Europe population. Methods: We retrospectively analysed the spirometry and exacerbation data of 85 CF adult patients (both men and women), who in the middle of 2022 began treatment with CFTR modulators. Results: The one-year ratio of hospitalisation caused by severe exacerbations lowered from 1.25 to 0.21 per patient per year. We also saw a 66% decline in ambulatory exacerbations. The median FEV1% increased by 9.60% in absolute values and by 460 mL. Even in the group with very severe obstruction (FEV1 < 35%), there was an increase in median FEV1% of 5.9 in absolute values. We also proved the increase in FVC% (median 17.10% in absolute value and 600 mL) in the study group. Conclusions: After one year of treatment, an impressive improvement was observed in two important predictive values of poor prognosis: exacerbation rate and FEV1 values. Further observation is needed to determine how long the improvement will be present and its influence on quality of life and life expectancy.
摘要：
背景/目的：囊性纤维化是一种遗传决定的疾病，显著影响和缩短寿命。用CFTR调节剂(CFTR-T)治疗是患者的新希望。它可以改变不良预后的预测值(例如，恶化率和FEV1值)。该研究的目的是分析波兹南CF中心85名CF患者在CFTR-T治疗一年（+/-2周）前后的恶化发生率和肺活量测定数据。据我们所知,这是对中东欧人口CFTR-T效率的首次分析。方法：我们回顾性分析85例CF成年患者（男性和女性）的肺活量测定和恶化数据，2022年中期开始使用CFTR调节剂进行治疗。结果：严重加重导致的一年住院率从每年1.25降至0.21。我们还看到非卧床恶化率下降了66%。中位数FEV1%的绝对值增加了9.60%，增加了460mL。即使在严重梗阻组（FEV1<35%），中位FEV1%的绝对值增加5.9%.我们还证明了研究组中FVC％的增加（绝对值和600mL的中位数为17.10％）。结论:经过一年的治疗,在不良预后的两个重要预测值中观察到了令人印象深刻的改善:加重率和FEV1值.需要进一步观察以确定改善的时间及其对生活质量和预期寿命的影响。
