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加拿大和美国囊性纤维化患者的生存比较:一项基于人群的队列研究。

Survival Comparison of Patients With Cystic Fibrosis in Canada and the United States: A Population-Based Cohort Study.

作者信息

Stephenson Anne L, Sykes Jenna, Stanojevic Sanja, Quon Bradley S, Marshall Bruce C, Petren Kristofer, Ostrenga Josh, Fink Aliza K, Elbert Alexander, Goss Christopher H

机构信息

From St. Michael's Hospital, University of Toronto, and The Hospital for Sick Children, Toronto, Ontario, Canada; University of British Columbia, Vancouver, British Columbia, Canada; Cystic Fibrosis Foundation, Bethesda, Maryland; and University of Washington Medical Center, Seattle, Washington.

出版信息

Ann Intern Med. 2017 Apr 18;166(8):537-546. doi: 10.7326/M16-0858. Epub 2017 Mar 14.

Abstract

BACKGROUND

In 2011, the median age of survival of patients with cystic fibrosis reported in the United States was 36.8 years, compared with 48.5 years in Canada. Direct comparison of survival estimates between national registries is challenging because of inherent differences in methodologies used, data processing techniques, and ascertainment bias.

OBJECTIVE

To use a standardized approach to calculate cystic fibrosis survival estimates and to explore differences between Canada and the United States.

DESIGN

Population-based study.

SETTING

42 Canadian cystic fibrosis clinics and 110 U.S. cystic fibrosis care centers.

PATIENTS

Patients followed in the Canadian Cystic Fibrosis Registry (CCFR) and U.S. Cystic Fibrosis Foundation Patient Registry (CFFPR) between 1990 and 2013.

MEASUREMENTS

Cox proportional hazards models were used to compare survival between patients followed in the CCFR (n = 5941) and those in the CFFPR (n = 45 448). Multivariable models were used to adjust for factors known to be associated with survival.

RESULTS

Median age of survival in patients with cystic fibrosis increased in both countries between 1990 and 2013; however, in 1995 and 2005, survival in Canada increased at a faster rate than in the United States (P < 0.001). On the basis of contemporary data from 2009 to 2013, the median age of survival in Canada was 10 years greater than in the United States (50.9 vs. 40.6 years, respectively). The adjusted risk for death was 34% lower in Canada than the United States (hazard ratio, 0.66 [95% CI, 0.54 to 0.81]). A greater proportion of patients in Canada received transplants (10.3% vs. 6.5%, respectively [standardized difference, 13.7]). Differences in survival between U.S. and Canadian patients varied according to U.S. patients' insurance status.

LIMITATION

Ascertainment bias due to missing data or nonrandom loss to follow-up might affect the results.

CONCLUSION

Differences in cystic fibrosis survival between Canada and the United States persisted after adjustment for risk factors associated with survival, except for private-insurance status among U.S. patients. Differential access to transplantation, increased posttransplant survival, and differences in health care systems may, in part, explain the Canadian survival advantage.

PRIMARY FUNDING SOURCE

U.S. Cystic Fibrosis Foundation.

摘要

背景

2011年,美国报告的囊性纤维化患者的中位生存年龄为36.8岁,而加拿大为48.5岁。由于所使用的方法、数据处理技术和确定偏倚存在固有差异,对各国登记处之间的生存估计进行直接比较具有挑战性。

目的

采用标准化方法计算囊性纤维化的生存估计值,并探讨加拿大和美国之间的差异。

设计

基于人群的研究。

地点

42家加拿大囊性纤维化诊所和110家美国囊性纤维化护理中心。

患者

1990年至2013年期间在加拿大囊性纤维化登记处(CCFR)和美国囊性纤维化基金会患者登记处(CFFPR)随访的患者。

测量

采用Cox比例风险模型比较CCFR(n = 5941)和CFFPR(n = 45448)中患者的生存率。使用多变量模型调整已知与生存相关的因素。

结果

1990年至2013年期间,两国囊性纤维化患者的中位生存年龄均有所增加;然而,在1995年和2005年,加拿大的生存率增长速度快于美国(P < 0.001)。根据2009年至2013年的当代数据,加拿大的中位生存年龄比美国大10岁(分别为50.9岁和40.6岁)。加拿大的调整后死亡风险比美国低34%(风险比,0.66 [95% CI,0.54至0.81])。加拿大接受移植的患者比例更高(分别为10.3%和6.5% [标准化差异,13.7])。美国和加拿大患者的生存差异因美国患者的保险状况而异。

局限性

由于数据缺失或随访的非随机失访导致的确定偏倚可能会影响结果。

结论

在调整了与生存相关的风险因素后,加拿大和美国在囊性纤维化生存方面的差异仍然存在,但美国患者的私人保险状况除外。移植机会的差异、移植后生存率的提高以及医疗保健系统的差异可能部分解释了加拿大的生存优势。

主要资金来源

美国囊性纤维化基金会。

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