Up-to-date and projected estimates of survival for people with cystic fibrosis using baseline characteristics: A longitudinal study using UK patient registry data

Abstract

BackgroundCystic 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.MethodsFlexible 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.FindingsMale 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).InterpretationOver half of babies born today, and of individuals aged 30 and above today, can expect to survive into at least their fifth decade.Research in contextEvidence before this studyWe 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 e...

Evaluation of a five-year predicted survival model for cystic fibrosis in later time periods

“…Survival of patients with cystic fibrosis (CF) has improved worldwide [1][2][3][4][5][6] . Improved care over the last 80 years increased the expectation of life at birth from a median of six months to approaching 50 years 2,7,8 .…”

“…However, this comparison is overly stringent and cannot account for individual circumstances that markedly change outcomes. If truly desired, one might look instead to the aggregated median age at death which reflects the age-distribution of the contemporary CF population, current mortality rates and the improving conditional survival from all ages as a result of rapidly improving therapies 3,9 . Recently, this measurement rose to nearly 30 years 2 .…”

Cystic fibrosis year in review 2018, part 1

“…In addition, geographic variation was seen, with the worst survival for Hispanic patients in the Midwest . While these survival estimates included patients in specific subgroups, a study from the UK CF Registry showed a more accurate reflection of survival, based on age at diagnosis, current age, sex, or mutation class, were developed …”

“…69 While these survival estimates included patients in specific subgroups, a study from the UK CF Registry showed a more accurate reflection of survival, based on age at diagnosis, current age, sex, or mutation class, were developed. 70 Although understanding survival statistics can inform patients and families, evaluation of predictors of survival can lead to more concrete changes in care that may alter survival outcomes. In prior years, the French have published predictors of mortality over the next 3 years, 71 and this model was validated in the Canadian CF Registry, with the prognostic score accurately predicting death or lung transplantation in the Canadian population.…”

Cytological appearance of pancreatic cystosis on fine‐needle aspiration

“…Although the pulmonary changes are the most serious complications of this disease, the wide distribution of the CFTR accounts for the involvement of pancreas, hepatobiliary system, kidneys, and gastrointestinal tracts. Median survival among patients with CF is around 40 years and, with continued improvements in therapy, these patients will be presenting to other medical specialties with extrapulmonary manifestations of the disease, which will need to be recognized and treated appropriately.…”