Ramos Kathleen J, Quon Bradley S, Heltshe Sonya L, Mayer-Hamblett Nicole, Lease Erika D, Aitken Moira L, Weiss Noel S, Goss Christopher H
Division of Pulmonary and Critical Care Medicine, Department of Medicine, University of Washington Medical Center, Seattle, WA.
Centre for Heart Lung Innovation, University of British Columbia and St. Paul's Hospital, Vancouver, BC, Canada.
Chest. 2017 Jun;151(6):1320-1328. doi: 10.1016/j.chest.2017.01.019. Epub 2017 Jan 20.
Lung transplantation (LTx) is frequently considered for patients with cystic fibrosis (CF) when the FEV reaches < 30%. This study estimated transplant-free survival for patients with CF and an FEV < 30% and identified predictors of death without LTx.
We conducted a retrospective cohort study using the CF Foundation Patient Registry from January 1, 2003 to December 31, 2013. Adult patients (≥ 18 years) with FEV < 30% prior to LTx were included. We performed Kaplan-Meier survival estimates censored at LTx. Multivariable Cox proportional hazard regression identified predictors of mortality.
There were 3,340 patients with an FEV < 30%. Death without LTx occurred in 1,250 patients (37.4%); 951 patients (28.5%) underwent LTx; 918 patients (27.5%) remained alive without LTx at the end of follow-up; and 221 patients (6.6%) were lost to follow-up. Median transplant-free survival after FEV < 30% was 6.6 years (95% CI, 5.9-7.0). Adjusted predictors of death without LTx included supplemental oxygen use (hazard ratio [HR], 2.1; 95% CI, 1.7-2.6), Burkholderia cepacia infection (HR, 1.8; 95% CI, 1.3-2.6), BMI ≤ 18 (HR, 1.6; 95% CI, 1.3-1.9), female sex (HR, 1.6; 95% CI, 1.2-2.0), CF-related diabetes in patients receiving insulin (HR, 1.4; 95% CI, 1.2-1.8), and ≥ one exacerbation per year (HR, 1.7; 95% CI, 1.3-2.2 vs. 0 exacerbations).
Median survival was > 6.5 years for patients with CF and an FEV < 30%, exceeding prior survival estimates. There was substantial heterogeneity in survival, with some patients with CF dying soon after reaching this lung function threshold and others living for many years. For this reason, we conclude that FEV < 30% remains an important marker of disease severity for patients with CF. Patients with a supplemental oxygen requirement or frequent exacerbations should have prompt referral because of their increased risk of death.
当第一秒用力呼气容积（FEV）降至<30%时，囊性纤维化（CF）患者常被考虑进行肺移植（LTx）。本研究估计了FEV<30%的CF患者的无移植生存期，并确定了未进行LTx死亡的预测因素。
我们进行了一项回顾性队列研究，使用2003年1月1日至2013年12月31日的CF基金会患者登记数据。纳入LTx术前FEV<30%的成年患者（≥18岁）。我们进行了Kaplan-Meier生存估计，并在LTx时进行截尾。多变量Cox比例风险回归确定了死亡的预测因素。
共有3340例FEV<30%的患者。1250例患者（37.4%）未进行LTx死亡；951例患者（28.5%）接受了LTx；918例患者（27.5%）在随访结束时未进行LTx仍存活；221例患者（6.6%）失访。FEV<30%后的中位无移植生存期为6.6年（95%CI，5.9 - 7.0）。未进行LTx死亡的校正预测因素包括使用补充氧气（风险比[HR]，2.1；95%CI，1.7 - 2.6）、洋葱伯克霍尔德菌感染（HR，1.8；95%CI，1.3 - 2.6）、体重指数（BMI）≤18（HR，1.6；95%CI，1.3 - 1.9）、女性（HR，1.6；95%CI，1.2 - 2.0）、接受胰岛素治疗的CF相关糖尿病患者（HR，1.4；95%CI，1.2 - 1.8）以及每年≥1次病情加重（HR，1.7；95%CI，1.3 - 2.2对比0次病情加重）。
FEV<30%的CF患者中位生存期>6.5年，超过了先前的生存估计。生存存在显著异质性，一些CF患者在达到此肺功能阈值后不久死亡，而另一些则存活多年。因此，我们得出结论，FEV<30%仍然是CF患者疾病严重程度的重要标志。有补充氧气需求或频繁病情加重的患者因死亡风险增加应及时转诊。
