Heterogeneity in Survival in Adult Patients With Cystic Fibrosis With FEV1 < 30% of Predicted in the United States
Heterogeneity in Survival in Adult Patients With Cystic Fibrosis With FEV1 < 30% of Predicted in the United States
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DOI：
10.1016/j.chest.2017.01.019
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发表时间：
2017-06-01
期刊：
影响因子：
9.6
通讯作者：
Goss, Christopher H.
中科院分区：
文献类型：
--
作者：
Ramos, Kathleen J.;Quon, Bradley S.;Goss, Christopher H.
背景：当 FEV1 达到 < 30% 时，囊性纤维化 (CF) 患者经常考虑肺移植 (LTx)。本研究估计了 FEV1 < 30% 的 CF 患者的无移植生存率，并确定了不进行 LTx 时死亡的预测因子。 方法：我们使用 CF 基金会患者登记中心从 2003 年 1 月 1 日至 2013 年 12 月 31 日进行了一项回顾性队列研究。纳入了 LTx 前 FEV1 < 30% 的成年患者（>= 18 岁）。我们进行了 LTx 审查的 Kaplan-Meier 生存估计。多变量 Cox 比例风险回归确定了死亡率的预测因子。结果：有 3,340 名患者的 FEV1 < 30%。 1,250 名患者（37.4%）在未接受 LTx 的情况下死亡； 951 名患者（28.5%）接受了 LTx；随访结束时，918 名患者（27.5%）在未进行 LTx 的情况下仍存活； 221 名患者（6.6%）失访。 FEV1 < 30% 后的中位无移植生存期为 6.6 年（95% CI，5.9-7.0）。不使用 LTx 时死亡的调整预测因素包括补充氧气使用（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）、BMI = 每年一次病情加重（HR，1.7；95% CI，1.3-2.2 vs. 0）结论：CF 且 FEV1 < 30% 的患者中位生存期 > 6.5 年，超出了之前的生存估计。生存率存在很大的异质性，一些 CF 患者在达到肺功能阈值后很快死亡，而另一些患者则存活多年。因此，我们得出结论，FEV1 < 30% 仍然是 CF 患者疾病严重程度的重要标志。需要补充氧气或经常病情加重的患者应立即转诊，因为他们的死亡风险增加。
BACKGROUND: Lung transplantation (LTx) is frequently considered for patients with cystic fibrosis (CF) when the FEV1 reaches < 30%. This study estimated transplant-free survival for patients with CF and an FEV1 < 30% and identified predictors of death without LTx.METHODS: 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 FEV1 < 30% prior to LTx were included. We performed Kaplan-Meier survival estimates censored at LTx. Multivariable Cox proportional hazard regression identified predictors of mortality.RESULTS: There were 3,340 patients with an FEV1 < 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 FEV1 < 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 = one exacerbation per year (HR, 1.7; 95% CI, 1.3-2.2 vs. 0 exacerbations).CONCLUSIONS: Median survival was > 6.5 years for patients with CF and an FEV1 < 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 FEV1 < 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.