Longevity of patients with cystic fibrosis in 2000 to 2010 and beyond: survival analysis of the Cystic Fibrosis Foundation patient registry.
Longevity of patients with cystic fibrosis in 2000 to 2010 and beyond: survival analysis of the Cystic Fibrosis Foundation patient registry.
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DOI：
10.7326/m13-0636
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发表时间：
2014-08-19
影响因子：
39.2
通讯作者：
Marshall BC
中科院分区：
文献类型：
--
作者：
MacKenzie T;Gifford AH;Sabadosa KA;Quinton HB;Knapp EA;Goss CH;Marshall BC
囊性纤维化（CF）治疗的进展继续延长生存期。为了更好地预测和预测不断变化的成人护理需求，需要对生存率进行更新估计。描述2000年至2010年CF存活率的趋势，并预测2010年出生和诊断患有该病的儿童的存活率。基于注册的研究。在美国有110个囊性纤维化基金会认可的护理中心。2000年至2010年期间囊性纤维化基金会患者登记处（CFFPR）中的所有患者。根据年龄、诊断时的年龄、性别、人种或种族、F508del突变状态和诊断时的症状对生存率进行建模。在2000年至2010年期间，CFFPR中的患者数量从21000例增加到26000例，中位年龄从14.3岁增加到16.7岁，调整后的死亡率每年下降1.8%（95%CI，0.5%至2.7%）。男性的调整后死亡风险比女性低19%（CI，13%至24%）。如果死亡率保持在2010年的水平，2010年出生并诊断为CF的儿童的中位生存期预计为女性37年（CI，35至39岁），男性40年（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，则超过50年。CFFPR未纳入美国的所有CF患者，并观察到失访和数据缺失。针对这些局限性的其他分析表明，生存预测是保守的。2010年在美国出生并被诊断为CF的儿童预计比更早出生的儿童寿命更长。这对预后讨论有重要意义，并表明卫生保健系统应预期更多的成年CF患者。囊性纤维化基金会。
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. To characterize trends in CF survival between 2000 and 2010 and to project survival for children born and diagnosed with the disease in 2010. Registry-based study. 110 Cystic Fibrosis Foundation–accredited care centers in the United States. All patients represented in the Cystic Fibrosis Foundation Patient Registry (CFFPR) between 2000 and 2010. Survival was modeled with respect to age, age at diagnosis, gender, race or ethnicity, F508del mutation status, and symptoms at diagnosis. 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. 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. 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. Cystic Fibrosis Foundation.