McColley Susanna A, Schechter Michael S, Morgan Wayne J, Pasta David J, Craib Marcia L, Konstan Michael W
Northwestern University Feinberg School of Medicine and Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois.
Children's Hospital of Richmond at Virginia Commonwealth University, Richmond, Virginia.
Pediatr Pulmonol. 2017 Jul;52(7):909-915. doi: 10.1002/ppul.23715. Epub 2017 Apr 24.
Understanding early-life risk factors for childhood death in cystic fibrosis (CF) is important for clinical care, including the identification of effective interventions.
Data from the Epidemiologic Study of Cystic Fibrosis (ESCF) collected 1994-2005 were linked with the Cystic Fibrosis Foundation Patient Registry (CFFPR) demographic and mortality data from 2013. Inclusion criteria were ≥1 visit annually at age 3-5 years and ≥1 FEV measurement at age 6-8 years. Demographic data, nutritional parameters, pulmonary signs and symptoms, microbiology, and FEV were evaluated as risk factors for death before age 18 years. Multivariable Cox proportional hazards regression was used to model the simultaneous effects of risk factors associated with death before age 18 years.
Among 5365 patients enrolled in ESCF who met inclusion criteria, 3880 (72%) were linked to the CFFPR. Among these, 191 (5.7%) died before age 18 years; median age at death was 13.4 ± 3.1 years. Multivariable regression showed clubbing, crackles, female sex, unknown CFTR genotype, minority race or ethnicity, Medicaid insurance (a proxy of low socioeconomic status), Pseudomonas aeruginosa on 2 or more cultures, and weight-for-age <50th percentile were significant risk factors for death regardless of inclusion of FEV at age 6-8 years in the model.
We identified multiple risk factors for childhood death of patients with CF, all of which remained important after incorporating FEV at age 6-8 years. Among the factors identified were the presence of clubbing or crackles at age 3-5 years, signs which are not routinely collected in registries.
了解囊性纤维化（CF）患儿死亡的早期风险因素对临床护理很重要，包括确定有效的干预措施。
1994年至2005年收集的囊性纤维化流行病学研究（ESCF）数据与2013年囊性纤维化基金会患者登记处（CFFPR）的人口统计学和死亡率数据相关联。纳入标准为3至5岁时每年至少就诊1次，6至8岁时至少进行1次FEV测量。人口统计学数据、营养参数、肺部体征和症状、微生物学以及FEV被评估为18岁前死亡的风险因素。多变量Cox比例风险回归用于模拟与18岁前死亡相关的风险因素的同时作用。
在符合纳入标准的5365名ESCF登记患者中，3880名（72%）与CFFPR相关联。其中，191名（5.7%）在18岁前死亡；死亡中位年龄为13.4±3.1岁。多变量回归显示，杵状指、啰音、女性、CFTR基因型未知、少数族裔、医疗补助保险（社会经济地位低的指标）、2次或更多次培养中出现铜绿假单胞菌以及年龄别体重<第50百分位数是死亡的重要风险因素，无论模型中是否纳入6至8岁时的FEV。
我们确定了CF患儿死亡的多个风险因素，在纳入6至8岁时的FEV后，所有这些因素仍然很重要。所确定的因素包括3至5岁时出现杵状指或啰音，这些体征在登记处通常不常规收集。
