Identifying cystic fibrosis programs in the United States with disproportionately disadvantaged patient populations: Methodology and implications for care.

Introduction: Providing care to under-resourced patients places additional burdens on CF care teams, therefore it is important to identify programs that serve disproportionately disadvantaged populations.

Methods: Using the U.S.

Real-world association between ivacaftor initiation and lung function variability: A registry study.

Background: Increased variability in forced expiratory volume in 1 s of % predicted (FEVpp) has been associated with accelerated lung function decline in individuals with cystic fibrosis (CF). Lung function variability is a leading predictor of decline, but the association between ivacaftor initiation and FEVpp variability has not been characterized.

Methods: We utilized the Cystic Fibrosis Foundation Patient Registry (2008-2020) to quantify this association and identify risk factors of increased variability.

Aging with CF: Characteristics of people with CF aged 40 and older in the United States.

We conducted a descriptive analysis of people with CF 40 years of age and older using CF Foundation Patient Registry data from 2022 to provide a current estimate of the population size and characteristics. We summarized demographic details including biological sex, race, ethnicity, insurance and employment status. Clinical data including body mass index, lung function, respiratory infections, hospitalization rates, prevalence of CF-related complications and CF therapy prescriptions were collated.

Linkage of the CF Foundation Patient Registry with the Scientific Registry of Transplant Recipients database.

Background: The Cystic Fibrosis Foundation Patient Registry (CFFPR) maintains clinical data, including history of solid organ transplant, on people with cystic fibrosis (CF) who obtain care at CF Foundation-accredited care centers. The Scientific Registry of Transplant Recipients (SRTR) database is a collection of national data related to organ transplantation that supports research to evaluate solid organ transplant candidate and recipient outcomes.

Methods: Individuals in the CFFPR were matched to SRTR records using an algorithm that compared names, last four digits of social security numbers, date of birth and date of death.

Impact of the expanded label for elexacaftor/tezacaftor/ivacaftor in people with cystic fibrosis with no F508del variant in the USA.

Background: Elexacaftor/tezacaftor/ivacaftor (ETI), which is approved for people with cystic fibrosis (pwCF) with a F508del variant, was further approved based on data in the USA for those carrying at least one of 177 rare (cystic fibrosis transmembrane conductance regulator) variants.

Methods: PwCF, aged ≥6 years, carrying no F508del variant but with at least one of these 177 rare variants, were identified within the US Cystic Fibrosis Foundation Patient Registry (CFFPR) between 2020 and 2022. The evolution of forced expiratory volume in 1 s (FEV) percentage predicted and rates of pulmonary exacerbations were analysed over the first year following ETI initiation, using a linear regression with generalised estimating equations and a negative binomial model, respectively.

Impact of day-to-day variation in FEV1 on measures of change: A conceptual description.

Clinical trials often demonstrate treatment efficacy through change in forced expiratory volume in one second (FEV), comparing single FEV measurements from post- versus pre-treatment timepoints. Day-to-day variation in measured FEV is common for reasons such as diurnal variation and intermittent health changes, relative to a stable, monthly average. This variation can alter estimation of associations between change in FEV and baseline in predictable ways, through a phenomenon called regression to the mean.

Characteristics of individuals with cystic fibrosis in the United States ineligible for ivacaftor and elexacaftor/tezacaftor/ivacaftor.

Background: We characterized people with cystic fibrosis (CF) ineligible by genotype (not age) for currently approved CFTR modulator therapy using data from the US CF Foundation Patient Registry (CFFPR).

Methods: We summarized clinical characteristics using CFFPR data from 2017 to 2022. Annual rate of change in percent predicted of forced expiratory volume in one second (ppFEV) was estimated using generalized estimating equations.

Treatment of small as well as large declines in lung function enhances recovery to baseline in people with CF.

Background: The benefit of antibiotic treatment of acute drops in FEV percent predicted (FEVpp) has been clearly established, but data from the early 2000s showed inconsistent treatment. Further, there is no empirical evidence for what magnitude of drop is clinically significant.

Methods: We used data from the CF Foundation Patient Registry (CFFPR) from 2016 to 2019 to determine the association between treatment (any IV antibiotics, only oral or newly prescribed inhaled antibiotics, or no antibiotic therapy) following a decline of ≥5% from baseline FEVpp and return to 100% baseline FEVpp days using multivariable logistic regression including an interaction between the magnitude of decline and treatment category.

Forced Expiratory Volume in 1 Second Variability Predicts Lung Transplant or Mortality in People with Cystic Fibrosis in the United States.

Declines in percent predicted forced expiratory volume in 1 second (ppFEV) are an important marker of clinical progression of cystic fibrosis (CF). We examined ppFEV variability in relation to a combined outcome of lung transplant or death. We estimated the association between ppFEV variability and the combined outcome of lung transplant or death.

The impact of switching to race-neutral reference equations on FEV percent predicted among people with cystic fibrosis.

Rationale: The American Thoracic Society recommended switching to race-neutral spirometry reference equations, as race is a social construct and to avoid normalizing disparities in lung function due to structural racism. Understanding the impact of the race-neutral equations on percent predicted forced expiratory volume in one second (ppFEV) in people with cystic fibrosis (PwCF) will help prepare patients and providers to interpret pulmonary function test results.

Objective(s): To quantify the impact of switching from Global Lung Initiative (GLI) 2012 race-specific to GLI 2022 Global race-neutral reference equations on the distribution of ppFEV among PwCF of different races.

Clinical outcomes at 9-10 years of age in children born with cystic fibrosis transmembrane conductance regulator related metabolic syndrome.

Background And Objectives: There are limited data on cystic fibrosis (CF) transmembrane conductance regulator-related metabolic syndrome (CRMS) outcomes beyond infancy. The goal of this study was to analyze outcomes of infants with CRMS up to the age of 9-10 years using the CF Foundation Patient Registry (CFFPR).

Methods: We analyzed data from the CFFPR for individuals with CF and CRMS born between 2010 and 2020.

Enhancing immunogenic responses through CDK4/6 and HIF2α inhibition in Merkel cell carcinoma.

Approximately 50% of Merkel cell carcinoma (MCC) patients facing this highly aggressive skin cancer initially respond positively to PD-1-based immunotherapy. Nevertheless, the recurrence of MCC post-immunotherapy emphasizes the pressing need for more effective treatments. Recent research has highlighted Cyclin-dependent kinases 4 and 6 (CDK4/6) as pivotal cell cycle regulators gaining prominence in cancer studies.

Impact of loss to follow-up on survival estimation for cystic fibrosis.

Purpose: Deaths among those lost to follow-up (LTFU) in the Cystic Fibrosis Foundation Patient Registry (CFFPR) are critically important to the epidemiology of cystic fibrosis (CF). Unreported deaths could impact estimates of survival if LTFU is associated with disease trajectory.

Methods: We characterized the LTFU population (1986-2017) from the CFFPR and identified deaths via linkage with the National Death Index (NDI).

Cystic fibrosis prevalence in the United States and participation in the Cystic Fibrosis Foundation Patient Registry in 2020.

Background: The Cystic Fibrosis Foundation Patient Registry (CFFPR) collects data on individuals with cystic fibrosis (CF) in the United States (US). In 2012, the US CF population was estimated at 33,292 to 34,327 individuals, with 81-84% CFFPR participation.

Methods: In this study, we update these estimates via simulation to account for uncertainty in CF incidence by race or Hispanic ethnicity, initiation of CF newborn screening (NBS) programs by state, and updated cumulative survival for CF births 1968-2020.

The prevalence of onchocerciasis in Africa and Yemen, 2000-2018: a geospatial analysis.

Background: Onchocerciasis is a disease caused by infection with Onchocerca volvulus, which is transmitted to humans via the bite of several species of black fly, and is responsible for permanent blindness or vision loss, as well as severe skin disease. Predominantly endemic in parts of Africa and Yemen, preventive chemotherapy with mass drug administration of ivermectin is the primary intervention recommended for the elimination of its transmission.

Methods: A dataset of 18,116 geo-referenced prevalence survey datapoints was used to model annual 2000-2018 infection prevalence in Africa and Yemen.

Real-world Associations of US Cystic Fibrosis Newborn Screening Programs With Nutritional and Pulmonary Outcomes.

Importance: Newborn screening (NBS) for cystic fibrosis (CF) has been universal in the US since 2010, but its association with clinical outcomes is unclear.

Objective: To describe the real-world effectiveness of NBS programs for CF in the US on outcomes up to age 10 years.

Design, Setting, And Participants: This was a retrospective cohort study using CF Foundation Patient Registry data from January 1, 2000, to December 31, 2018.

Predicting the environmental suitability for onchocerciasis in Africa as an aid to elimination planning.

Recent evidence suggests that, in some foci, elimination of onchocerciasis from Africa may be feasible with mass drug administration (MDA) of ivermectin. To achieve continental elimination of transmission, mapping surveys will need to be conducted across all implementation units (IUs) for which endemicity status is currently unknown. Using boosted regression tree models with optimised hyperparameter selection, we estimated environmental suitability for onchocerciasis at the 5 × 5-km resolution across Africa.

The effect of HIV infection and exposure on cognitive development in the first two years of life in Malawi.

Objectives: To assess longitudinal patterns and determinants of cognitive development in infants living with HIV, infants exposed to maternal HIV infection, and HIV-unexposed infants.

Methods: Prospective, community-based cohort study of 555 Malawian infants aged 8 weeks to 24 months, using multivariable linear mixed-effects regression models with random intercepts to analyze repeated measures of cognitive function.

Results: At 3 months of age, cognitive scores on the Bayley Scales of Infant Development (BSID 3rd edition) were lower in the 96 HIV-infected infants (mean = 14.

Mapping the global distribution of podoconiosis: Applying an evidence consensus approach.

Background: Podoconiosis is a type of elephantiasis characterised by swelling of the lower legs. It is often confused with other causes of tropical lymphedema and its global distribution is uncertain. Here we synthesise the available information on the presence of podoconiosis to produce evidence consensus maps of its global geographical distribution.

A database of geopositioned onchocerciasis prevalence data.

Onchocerciasis is a neglected tropical disease with numerous symptoms and side effects, and when left untreated can lead to permanent blindness or skin disease. This database is an attempt to combine onchocerciasis prevalence data from peer-reviewed publications into a single open-source dataset. The process followed to extract and format the information has been detailed in this paper.

Ecological and Socioeconomic Predictors of Transmission Assessment Survey Failure for Lymphatic Filariasis.

The transmission assessment survey (TAS) is recommended to determine whether cessation of mass drug administration (MDA) for lymphatic filariasis (LF) is warranted. Ministries of health typically implement TASs in evaluation units (EUs) that have had more than five rounds of annual MDA. Under TAS guidelines, sample size calculations determine a decision value: if the number of individuals testing positive exceeds this threshold, then MDA continues in the EU.