2000年至2010年及以后囊性纤维化患者的寿命：囊性纤维化基金会患者登记处的生存分析
Longevity of patients with cystic fibrosis in 2000 to 2010 and beyond: survival analysis of the Cystic Fibrosis Foundation patient registry.
作者信息
MacKenzie Todd, Gifford Alex H, Sabadosa Kathryn A, Quinton Hebe B, Knapp Emily A, Goss Christopher H, Marshall Bruce C
出版信息
Ann Intern Med. 2014 Aug 19;161(4):233-41. doi: 10.7326/M13-0636.
BACKGROUND
Advances in treatments for cystic fibrosis (CF) continue to extend survival. An updated estimate of survival is needed for better prognostication and to anticipate evolving adult care needs.
OBJECTIVE
To characterize trends in CF survival between 2000 and 2010 and to project survival for children born and diagnosed with the disease in 2010.
DESIGN
Registry-based study.
SETTING
110 Cystic Fibrosis Foundation-accredited care centers in the United States.
PATIENTS
All patients represented in the Cystic Fibrosis Foundation Patient Registry (CFFPR) between 2000 and 2010.
MEASUREMENTS
Survival was modeled with respect to age, age at diagnosis, gender, race or ethnicity, F508del mutation status, and symptoms at diagnosis.
RESULTS
Between 2000 and 2010, the number of patients in the CFFPR increased from 21,000 to 26,000, median age increased from 14.3 to 16.7 years, and adjusted mortality decreased by 1.8% per year (95% CI, 0.5% to 2.7%). Males had a 19% (CI, 13% to 24%) lower adjusted risk for death than females. Median survival of children born and diagnosed with CF in 2010 is projected to be 37 years (CI, 35 to 39 years) for females and 40 years (CI, 39 to 42 years) for males if mortality remains at 2010 levels and more than 50 years if mortality continues to decrease at the rate observed between 2000 and 2010.
LIMITATIONS
The CFFPR does not include all patients with CF in the United States, and loss to follow-up and missing data were observed. Additional analyses to address these limitations suggest that the survival projections are conservative.
CONCLUSION
Children born and diagnosed with CF in the United States in 2010 are expected to live longer than those born earlier. This has important implications for prognostic discussions and suggests that the health care system should anticipate greater numbers of adults with CF.
PRIMARY FUNDING SOURCE
Cystic Fibrosis Foundation.
背景
囊性纤维化（CF）治疗方法的进步不断延长患者生存期。需要更新生存期估计，以便更好地进行预后评估并预测不断变化的成人护理需求。
目的
描述2000年至2010年期间CF患者的生存趋势，并预测2010年出生并被诊断患有该疾病的儿童的生存期。
设计
基于登记处的研究。
地点
美国110家经囊性纤维化基金会认证的护理中心。
患者
2000年至2010年期间囊性纤维化基金会患者登记处（CFFPR）记录的所有患者。
测量指标
根据年龄、诊断时年龄、性别、种族或族裔、F508del突变状态以及诊断时的症状对生存期进行建模。
结果
2000年至2010年期间，CFFPR中的患者数量从21,000增加到26,000，中位年龄从14.3岁增加到16.7岁，校正死亡率每年下降1.8%（95%CI，0.5%至2.7%）。男性校正死亡风险比女性低19%（CI，13%至24%）。如果死亡率保持在2010年的水平，预计2010年出生并被诊断患有CF的儿童女性中位生存期为37岁（CI，35至39岁），男性为40岁（CI，39至42岁）；如果死亡率继续以2000年至2010年期间观察到的速度下降，则中位生存期超过50岁。
局限性
CFFPR并未涵盖美国所有CF患者，存在失访和数据缺失情况。针对这些局限性的进一步分析表明，生存期预测较为保守。
结论
预计2010年在美国出生并被诊断患有CF的儿童比更早出生的儿童寿命更长。这对预后讨论具有重要意义，并表明医疗保健系统应预见到更多成年CF患者。
主要资金来源
囊性纤维化基金会。
Zotero 插件