Up-to-date and projected estimates of survival for people with cystic fibrosis using baseline characteristics: A longitudinal study using UK patient registry data.
Up-to-date and projected estimates of survival for people with cystic fibrosis using baseline characteristics: A longitudinal study using UK patient registry data.
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DOI：
10.1016/j.jcf.2017.11.019
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发表时间：
2018-03
期刊：
影响因子：
--
通讯作者：
Bilton D
中科院分区：
文献类型：
--
作者：
Keogh RH;Szczesniak R;Taylor-Robinson D;Bilton D
囊性纤维化 (CF) 是白种人中最常见的遗传性疾病，目前影响英国约 10,000 人。近几十年来，随着治疗和护理的不断改进，预后已大大改善。提供最新的生存预测对于患者、临床医生和卫生服务规划非常重要。对 2011 年至 2015 年英国 CF 登记数据进行灵活的参数生存模型，记录了 10,428 人中的 602 例死亡。生存曲线从出生时开始估算；以达到较高年龄为条件；并使用性别基线特征、CFTR 基因型（F508del 零、一、两个拷贝）和诊断时年龄，根据有关未来死亡率趋势的不同假设进行预测。男性与较高的生存率相关，诊断时年龄较大也与此相关，但仅限于 F508del 非纯合子。在出生时诊断的个体中，基因型的生存率没有差异。 F508del 纯合子出生时的中位生存年龄为 46 岁（男性）和 41 岁（女性），出生时诊断的非纯合子的中位生存年龄相似。 5 岁时诊断的 F508del 杂合子的中位生存年龄为 57 岁（男性）和 51 岁（女性）。以存活到 30 岁为条件，纯合子的中位存活年龄升至 52 岁（男性）和 49 岁（女性）。 2006 年至 2015 年间，死亡率每年下降 2%。未来按照这一速度的改善表明，F508del 纯合子婴儿的中位生存年龄为 65 岁（男性）和 56 岁（女性）。如今出生的婴儿以及 30 岁及以上的人中有一半以上预计能活到至少 50 岁。本研究之前的证据我们在 PubMed 中使用术语“（囊性纤维化生存）和（预测或模型或登记或英国或英国）”进行搜索，以确定囊性纤维化 (CF) 个体生存估计的相关研究。我们还考虑了英国囊性纤维化登记处的最新年度报告（囊性纤维化信托基金，2016 年）、Buzzetti 及其同事的综述（2009 年）、MacNeill 的囊性纤维化流行病学章节（2016 年）、MacKenzie 及其同事的研究（2014 年）以及其中的参考文献。对于与 CF 生存相关的因素已有许多研究；大多数人专注于识别风险因素，只有少数提出了估计的生存曲线，这是这项工作的重点。英国最新的生存率研究是 Dodge 及其同事 (2007) 进行的，他们使用了从囊性纤维化诊所和国家死亡登记处获得的数据，并对 2003 年出生的婴儿的生存率进行了估计。我们发现以前的研究没有使用英国囊性纤维化登记数据获得有关生存率的详细信息。 Jackson 及其同事使用登记数据获得了美国和爱尔兰的生存估计（Jackson 等，2011）。 MacKenzie 及其同事使用 2000 年至 2010 年美国囊性纤维化基金会患者登记数据来预测 2010 年出生并诊断为囊性纤维化的儿童的生存率，并考虑了性别、基因型和诊断时的年龄（MacKenzie 等，2014）。之前关于 CF 估计生存率的研究已经过时，或者没有考虑到患者出生时的全部特征。很少有人提出条件生存估计（Dodge et al., 2007）。这项研究的附加价值这是第一项利用英国囊性纤维化登记处产生详细生存统计数据的研究，该登记处是美国以外最大的国家囊性纤维化登记处之一，几乎完全覆盖了英国囊性纤维化人群。主要目标是利用登记处几乎完整的英国 CF 人群的长期随访，以便在现代 CF 护理时代产生准确、精确的预测。估计值是从出生开始的，并以生存到老年为条件。这些是 CF 中的第一个条件估计，还考虑了基因型、性别和诊断时的年龄，这些都使用灵活的方法包含在模型中。还根据死亡率下降趋势提供了不同情景下的预测。我们使用灵活的参数生存模型在该领域是新颖的，我们的方法可用于提供其他慢性疾病和病症的现代生存统计数据。所有现有证据的影响 我们对一系列不同情况下 CF 未来生存率的估计是基于近期英国几乎所有患有该疾病的个体的数据，反映了现代护理时代，并且最适合当今出生的 CF 婴儿的家庭。有条件的估计可以告知已经达到较高年龄的患者及其临床医生。今天出生的婴儿以及当今 30 岁及以上的人中，一半以上预计可以活到 50 岁。基于我们的生存预测的见解可用于了解 CF 医疗保健提供的未来需求。
Cystic fibrosis (CF) is the most common inherited disease in Caucasians, affecting around 10,000 individuals in the UK today. Prognosis has improved considerably over recent decades with ongoing improvements in treatment and care. Providing up-to-date survival predictions is important for patients, clinicians and health services planning. Flexible parametric survival modelling of UK CF Registry data from 2011 to 2015, capturing 602 deaths in 10,428 individuals. Survival curves were estimated from birth; conditional on reaching older ages; and projected under different assumptions concerning future mortality trends, using baseline characteristics of sex, CFTR genotype (zero, one, two copies of F508del) and age at diagnosis. Male sex was associated with better survival, as was older age at diagnosis, but only in F508del non-homozygotes. Survival did not differ by genotype among individuals diagnosed at birth. Median survival ages at birth in F508del homozygotes were 46 years (males) and 41 years (females), and similar in non-homozygotes diagnosed at birth. F508del heterozygotes diagnosed aged 5 had median survival ages of 57 (males) and 51 (females). Conditional on survival to 30, median survival age rises to 52 (males) and 49 (females) in homozygotes. Mortality rates decreased annually by 2% during 2006–2015. Future improvements at this rate suggest median survival ages for F508del homozygous babies of 65 (males) and 56 (females). Over half of babies born today, and of individuals aged 30 and above today, can expect to survive into at least their fifth decade. Evidence before this study We searched PubMed with terms “(cystic fibrosis survival) and (projection OR model OR registry OR United Kingdom OR UK)” to identify relevant studies on survival estimates for individuals with cystic fibrosis (CF). We also considered the most recent annual report from the UK Cystic Fibrosis Registry (Cystic Fibrosis Trust, 2016), a review by Buzzetti and colleagues (2009), the chapter on Epidemiology of Cystic Fibrosis by MacNeill (2016), the study of MacKenzie and colleagues (2014), and references therein. There have been many studies of factors associated with survival in CF; most have focused on identifying risk factors, and only a few have presented estimated survival curves, which are the focus of this work. The most recent study of survival in the UK is by Dodge and colleagues (2007), who used data obtained from CF clinics and the national death register, and gave an estimate of survival for babies born in 2003. We found no previous studies that have obtained detailed information on survival using UK Cystic Fibrosis Registry data. Jackson and colleagues obtained survival estimates for the US and Ireland using registry data (Jackson et al., 2011). MacKenzie and colleagues used US Cystic Fibrosis Foundation Patient Registry data from 2000 to 2010 to project survival for children born and diagnosed with CF in 2010, accounting for sex, genotype and age at diagnosis (MacKenzie et al., 2014). Previous studies on estimated survival in CF have become out of date or have not accounted for the full range of patient characteristics available at birth. Few have presented conditional survival estimates (Dodge et al., 2007). Added value of this study This is the first study to yield detailed survival statistics using the UK Cystic Fibrosis Registry, which is one of the largest national CF registries outside of the US and has almost complete coverage of the UK CF population. The primary goal was to leverage the long-term follow-up of the nearly complete UK CF population available in the Registry for the purposes of producing accurate, precise predictions in the modern era of CF care. Estimates are presented from birth and conditional on survival to older ages. These are the first conditional estimates in CF to also account for genotype, sex and age at diagnosis, which were each included in the modelling using a flexible approach. Projections are also provided under different scenarios based on downward trends in mortality rates. Our use of flexible parametric survival models is novel in this field, and our approach could be used to provide modern survival statistics for other chronic diseases and disorders. Implications of all the available evidence Our estimates of future survival in CF under a range of different scenarios are based on data on nearly all individuals living with the disease in the UK in recent times, reflective of a modern era of care, and are most appropriate for the families of babies being born in the present day with CF. Conditional estimates inform patients who have already reached an older age, and their clinicians. Over half of babies born today, and of individuals aged 30 years and above alive today, can expect to survive into their fifth decade. Insights based on our survival projections can be used to inform future needs in CF health care provision.
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影响因子：
76.2
作者：
Taylor-Robinson, David C.;Smyth, Rosalind L.;Diggle, Peter J.;Whitehead, Margaret
通讯作者：
Whitehead, Margaret
影响因子：
2
作者：
Thiébaut, ACM;Bénichou, J
通讯作者：
Bénichou, J
影响因子：
2.2
作者：
Downing, Melanie;Pollitt, Rodney
通讯作者：
Pollitt, Rodney
影响因子：
7.7
作者：
Taylor-Robinson D;Archangelidi O;Carr SB;Cosgriff R;Gunn E;Keogh RH;MacDougall A;Newsome S;Schlüter DK;Stanojevic S;Bilton D;CF-EpinNet collaboration
通讯作者：
CF-EpinNet collaboration
影响因子：
5.8
作者：
Dijk, F. Nicole;Fitzgerald, Dominic A.
通讯作者：
Fitzgerald, Dominic A.