J Clin Med. 2024 Jun 22;13(13):3652. doi: 10.3390/jcm13133652.

ABSTRACT

Background: Over the past decades, efforts have been made to improve the nutritional well-being of people with cystic fibrosis (pwCF). Due to the correlation observed between nutritional indices and lung function, prevailing recommendations consistently advocate for BMI percentile goals at or above the 50th percentile in pwCF. Recent global trends show a notable increase in overweight and obese statuses among pwCF. This study aims to explore the nutritional status of Italian pwCF. Methods: Data from the Italian CF Patient's Registry were analysed to assess the proportion of individuals categorized as underweight, target weight, overweight, and obese from 2010 to 2021. Patient-level comparison data from 2021 were also examined to identify the potential determinants of overweight and obesity. Results: Analysis spanning 2010 to 2021 reveals a decrease of approximately 40% in underweight status among adults, while the proportion of malnourished patients younger than 18 years remained stable. Conversely, there was a substantial increase of over 70% in overweight status and over 85% in obesity among adults, with minor fluctuations observed among children and adolescents. Patient factors associated with increased obesity incidence included age older than 45 years, male gender, pancreatic sufficiency, possession of at least one CFTR variant conferring residual function, ppFEV1 > 90, and lower prevalence of Pseudomonas aeruginosa colonization. Conclusions: Our study confirms the evolving nutritional status landscape among Italian adult pwCF, with a significant shift towards overweight and obesity over the past decade. These trends highlight the need for proactive measures within CF standards of care to adapt and address the changing needs of patients.

PMID:38999218 | DOI:10.3390/jcm13133652

J Cyst Fibros. 2024 Jul 11:S1569-1993(24)00776-8. doi: 10.1016/j.jcf.2024.06.007. Online ahead of print.

ABSTRACT

INTRODUCTION: Insulin remains the only recommended medical treatment for cystic fibrosis related diabetes (CFRD) Whilst there is an established role for orally bioavailable incretin mimetic agents such as the dipeptidyl peptidase-4 inhibitors (DPP4-I) in Type 2 diabetes mellitus, there exists little data on their utility in CFRD.

AIM: To examine the use of DPP4-I therapy in patients with CFRD at a single large adult cystic fibrosis center.

METHOD: People with CFRD prescribed a DPP4-I were identified from our specialist CFRD clinic and records were retrospectively examined for indication for therapy, tolerability and effectiveness. Analysis of continuous glucose monitoring data Libre 2 was done for these patients (CGM) pre and at least 3 months post therapy was performed.

RESULTS: 23 people with CF (PwCF) with a mean (SD) age of 35.0 ± 2.4 years were included in this analysis . In 21 patients DPP4-I was prescribed as a monotherapy and it was given in combination with insulin in 2 others. Indications for therapy included reactive hypoglycaemia (n = 10) post prandial hyperglycaemia (8), insulin avoidance (3), metformin intolerance (1) and unclear (1). Therapy was well tolerated with no discontinuations due to adverse effects. Significant improvements were noted in Time in Range- Pre vs Post: 78.0 [67.5 - 84.0] vs 89.0 [79.8 - 96.0]%, p = 0.005, Time above Range -Pre vs Post: 19.5 [12.5 - 30.8] vs 6.0 [2.5 - 16.5]%, p = 0.006 and glucose variability Pre versus Post: 28.3 [25.4 - 31.1] vs 26.9 [23.1 - 31.3], p = 0.021, Of the 10 subjects who initiated therapy for hypoglycaemia, 7 reported an improvement in symptoms. No significant difference was found in weight pre and post: 61.5 ± 15.0 kg vs 62.5 ± 15.3 kg, p = 0.326 or Hba1c pre vs post: 41.0 [36.0 - 53.3] mmol/mol versus 40.5 [36.8 - 47.3], p = 0.727.

CONCLUSION: DPP4-I is well tolerated in CFRD and can lead to an improved glycaemic control in these patients with significant improvement in validated CGM metrics.

PMID:38997826 | DOI:10.1016/j.jcf.2024.06.007

J Cyst Fibros. 2024 Jul 11:S1569-1993(24)00787-2. doi: 10.1016/j.jcf.2024.07.003. Online ahead of print.

ABSTRACT

BACKGROUND: Continuing professional development (CPD) is a component of practice that spans all disciplines within cystic fibrosis (CF). E-learning resources theoretically represent flexible, low cost and time efficient methods of CPD. We aimed to explore European Cystic Fibrosis Society (ECFS) members' and CF health professional communities' current views, experiences and perceptions of e-learning and the ECFS education platform (ECFS-EP).

METHODS: An online cross-sectional survey was developed by the ECFS Education Committee and circulated via the ECFS conference, emails and within the society subgroups between June and September 2023.

RESULTS: 547 responses were received from 58 countries; 57 % of responders were ECFS members. A wide range of specialities were represented from the multidisciplinary team including 36 % clinicians. The majority of respondents (63 %) spent 6 or more hours a week on their professional education. Online platforms were used either weekly (34 %) or monthly (37 %); 54 % of respondents had used the ECFS-EP and this was rated favourably overall, specifically for content quality. Preferred formats for education were articles and medium length (15-30 min) webinars.

CONCLUSIONS: This multidisciplinary cohort survey illustrates contemporary practice and opinion relating to e-learning and the ECFS-EP. Strengths include the high number of responses and the wide range of countries and specialities represented. Results suggest the ECFS-EP is valued and highlights priority topics, preferred formats and opportunities to optimise awareness. Results support continued provision and oversight of high quality education via an online platform. Continued success will rely on learning from user experience and feedback to inform future practice.

PMID:38997825 | DOI:10.1016/j.jcf.2024.07.003

J Cyst Fibros. 2024 Jul 11:S1569-1993(24)00078-X. doi: 10.1016/j.jcf.2024.05.015. Online ahead of print.

NO ABSTRACT

PMID:38997824 | DOI:10.1016/j.jcf.2024.05.015

J Cyst Fibros. 2024 Jul 11:S1569-1993(24)00786-0. doi: 10.1016/j.jcf.2024.07.002. Online ahead of print.

ABSTRACT

RATIONALE: Cystic Fibrosis (CF) progresses through recurrent infection and inflammation, causing permanent lung function loss and airway remodeling. CT scans reveal abnormally low-density lung parenchyma in CF, but its microstructural nature remains insufficiently explored due to clinical CT limitations. To this end, diffusion-weighted 129Xe MRI is a non-invasive and validated measure of lung microstructure. In this work, we investigate microstructural changes in people with CF (pwCF) relative to age-matched, healthy subjects using comprehensive imaging and analysis involving pulmonary-function tests (PFTs), and 129Xe MRI.

METHODS: 38 healthy subjects (age 6-40; 17.2 ± 9.5 years) and 39 pwCF (age 6-40; 15.6 ± 8.0 years) underwent 129Xe-diffusion MRI and PFTs. The distribution of diffusion measurements (i.e., apparent diffusion coefficients (ADC) and morphometric parameters) was assessed via linear binning (LB). The resulting volume percentages of bins were compared between controls and pwCF. Mean ADC and morphometric parameters were also correlated with PFTs.

RESULTS: Mean whole-lung ADC correlated significantly with age (P < 0.001) for both controls and CF, and with PFTs (P < 0.05) specifically for pwCF. Although there was no significant difference in mean ADC between controls and pwCF (P = 0.334), age-adjusted LB indicated significant voxel-level diffusion (i.e., ADC and morphometric parameters) differences in pwCF compared to controls (P < 0.05).

CONCLUSIONS: 129Xe diffusion MRI revealed microstructural abnormalities in CF lung disease. Smaller microstructural size may reflect compression from overall higher lung density due to interstitial inflammation, fibrosis, or other pathological changes. While elevated microstructural size may indicate emphysema-like remodeling due to chronic inflammation and infection.

PMID:38997823 | DOI:10.1016/j.jcf.2024.07.002

Vitam Horm. 2024;125:311-365. doi: 10.1016/bs.vh.2024.01.003. Epub 2024 Mar 30.

ABSTRACT

Advanced glycation end products (AGEs) are compounds formed via non-enzymatic reactions between reducing sugars and amino acids or proteins. AGEs can accumulate in various tissues and organs and have been implicated in the development and progression of various diseases, including lung diseases. The receptor of advanced glycation end products (RAGE) is a receptor that can bind to advanced AGEs and induce several cellular processes such as inflammation and oxidative stress. Several studies have shown that both AGEs and RAGE play a role in the pathogenesis of lung diseases, such as chronic obstructive pulmonary disease, asthma, idiopathic pulmonary fibrosis, cystic fibrosis, and acute lung injury. Moreover, the soluble form of the receptor for advanced glycation end products (sRAGE) has demonstrated its ability to function as a decoy receptor, possessing beneficial characteristics such as anti-inflammatory, antioxidant, and anti-fibrotic properties. These qualities make it an encouraging focus for therapeutic intervention in managing pulmonary disorders. This review highlights the current understanding of the roles of AGEs and (s)RAGE in pulmonary diseases and their potential as biomarkers and therapeutic targets for preventing and treating these pathologies.

PMID:38997169 | DOI:10.1016/bs.vh.2024.01.003

Am J Med Sci. 2024 Jul 10:S0002-9629(24)01346-6. doi: 10.1016/j.amjms.2024.07.013. Online ahead of print.

ABSTRACT

BACKGROUND: Men with cystic fibrosis (CF) have sexual health concerns such as delayed puberty, infertility, and hypogonadism. The causes and prevalence of hypogonadism have not been well studied. The purpose of this study was to determine the prevalence of a low testosterone concentration in men with CF.

METHODS: This retrospective study was approved by the Emory University Institutional Review Board (IRB). Data were extracted from the electronic medical records of adult men with CF receiving care at the Emory Cystic Fibrosis Center. A total of 129 men with CF were followed at our center from 2016 to 2023. Of these individuals, 76 men with CF (58.9%) had at least one serum total testosterone measurement. Seven individuals were excluded from this study since they were currently receiving testosterone therapy, leaving a final sample size of 69 individuals for the analysis. Demographic data, serum testosterone concentrations, and other factors associated with low testosterone concentrations were collected. Low testosterone was defined as a value below 300 ng/dL. Regression analyses were used to determine factors associated with low testosterone levels.

RESULTS: The mean (± SD) age of the 69 eligible participants was 33.34 ± 10.98 years. The mean testosterone concentration was 421 ± 158.5 ng/dL with 27.54 percent of men with a testosterone value below 300 ng/dL. The mean hemoglobin level was 14.23 ± 2.18 g/dL. Testosterone levels were positively related to hemoglobin levels. Time of day of measurement and age were not associated with testosterone levels.

CONCLUSION: Roughly a quarter of men with CF demonstrated low testosterone in our sample. Low hemoglobin was associated with low testosterone levels in men with CF. Neither time of day nor age influenced testosterone concentrations in this sample.

PMID:38997066 | DOI:10.1016/j.amjms.2024.07.013

Diabetologia. 2024 Jul 12. doi: 10.1007/s00125-024-06223-3. Online ahead of print.

ABSTRACT

People with cystic fibrosis (CF) are at risk for dysglycaemia caused by progressive beta cell dysfunction and destruction due to pancreatic exocrine disease and fibrosis. CF-related diabetes (CFRD) is a unique form of diabetes that has distinctive features from both type 1 and type 2 diabetes. Recent advances in diabetes technology may be of particular benefit in this population given the complex, multi-system organ involvement and challenging health issues that people with CFRD often face. This review summarises how diabetes technologies, such as continuous glucose monitors (CGMs) and insulin delivery devices: (1) have improved our understanding of CFRD, including how hyperglycaemia affects clinical outcomes in people with CF; (2) may be helpful in the screening and diagnosis of CFRD; and (3) offer promise for improving the management of CFRD and easing the burden that this diagnosis can add to an already medically complicated patient population.

PMID:38995399 | DOI:10.1007/s00125-024-06223-3

Pediatr Pulmonol. 2024 Jul 12. doi: 10.1002/ppul.27176. Online ahead of print.

ABSTRACT

BACKGROUND: The benefit of antibiotic treatment of acute drops in FEV1 percent predicted (FEV1pp) 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 FEV1pp and return to 100% baseline FEV1pp days using multivariable logistic regression including an interaction between the magnitude of decline and treatment category.

RESULTS: Overall, 16,495 PWCF had a decline: 16.5% were treated with IV antibiotics, 25.0% non-IV antibiotics, and 58.5% received no antibiotics. Antibiotic treatment was more likely for those with lower lung function, history of a positive PA culture, older age and larger FEV1 decline (p < 0.001). Treatment with IV antibiotics or oral/inhaled antibiotics was associated with a higher odds of recovery to baseline compared to no treatment across all levels of decline, including declines of 5%-10%.

CONCLUSIONS: A large proportion of acute drops in FEV1pp continue to be untreated, especially in younger patients and those with higher baseline lung function. Acute drops as small as 5% predicted are less likely to be recovered if antibiotic treatment is not prescribed. These findings suggest the need for more aggressive antimicrobial treatment of acute drops in FEV1, including those of a magnitude previously believed to be associated with self-recovery.

PMID:38995116 | DOI:10.1002/ppul.27176

J Clin Endocrinol Metab. 2024 Jul 12:dgae474. doi: 10.1210/clinem/dgae474. Online ahead of print.

ABSTRACT

CONTEXT: The pathophysiological mechanisms underlying the natural history of glucose intolerance and its fluctuations in subjects with cystic fibrosis (CF) are still unclear.

OBJECTIVE: To investigate the relationship between longitudinal changes in glucose tolerance and concomitant changes in the main parameters of insulin secretion/metabolism/action determining glucose regulation in CF subjects.

METHODS: Insulin sensitivity and glucose-stimulated insulin secretion (GSIS, a biomarker of beta cell functional mass), as estimated by the Oral Glucose Sensitivity Index (OGIS) and by a sophisticated mathematical model, respectively, and insulin clearance were assessed in 127 CF subjects, aged 10-25 years, who underwent two OGTT tests over at least 1-year follow-up period. Subjects were classified a posteriori as regressors (improved glucose tolerance), stable, or progressors (worsened glucose tolerance). The interplay between beta cell compensatory action and insulin sensitivity over time was analyzed by vector plots of insulin clearance adjusted GSIS (PCadj) versus OGIS.

RESULTS: OGIS decreased in progressors and stable. Insulin clearance decreased in both regressors and progressors. GSIS (beta cell functional mass) improved in regressors and worsened in progressors, whereas it did not change in stable. Vector plot analysis confirmed that glucose regulation changed differently in each group. Multinomial logistic regression analysis showed that baseline glucose tolerance and GSIS changes were the only significant predictors of the changes in glucose tolerance (p<0.02, R2Nagelkerke=0.55), whereas age, gender, z-BMI, CF genotypes, and baseline PCadj were not.

CONCLUSIONS: In CF subjects, changes in beta cell functional mass are associated with favorable or detrimental changes of glucose tolerance over time.

PMID:38994570 | DOI:10.1210/clinem/dgae474

Front Transplant. 2022 Sep 23;1:992985. doi: 10.3389/frtra.2022.992985. eCollection 2022.

ABSTRACT

Cystic fibrosis (CF) is a multisystem disorder and represents the most common inherited condition leading to death in Western countries. Previous reports of chronic kidney disease (CKD) in CF focus on cases post lung, or other solid organ, transplantation but CKD in CF patients pre transplantation is increasingly recognized as a challenging complication of CF. CKD can evolve as a sequel to acute kidney injury for example after prolonged treatment with aminoglycoside antibiotics during episodes of infection. Nephrolithiasis, diabetic nephropathy and a variety of glomerular lesions, such as amyloidosis and Immunoglobulin A nephropathy are also seen. Muscle depletion is common in CF, hence creatinine-based estimates of kidney function may underestimate the degree of renal impairment and lead to delayed diagnosis and management. Improved treatment options for CF patients have resulted in a sustained increase in life expectancy with increasing numbers of CF patients with CKD approaching end-stage renal failure prior to consideration of lung transplantation. We believe that kidney or combined kidney-pancreas transplantation are under-utilized in this population. We provide a brief primer on the landscape of CF and CKD and discuss transplant options. Suitable patients with CF and advanced CKD should be formally assessed for kidney or kidney-pancreas transplantation.

PMID:38994374 | PMC:PMC11235247 | DOI:10.3389/frtra.2022.992985

SSM Qual Res Health. 2024 Jun;5:100412. doi: 10.1016/j.ssmqr.2024.100412. Epub 2024 Feb 24.

ABSTRACT

This article offers the case of cystic fibrosis (CF), a multi-system disease, to illustrate how individuals with chronic illness cultivate and apply embodied knowledge to optimize their well-being. We identified three interrelated processes that occur when disease chronicity and menstrual cyclicity meet: 1) knowledge production with a period-tracking app; 2) application of embodied knowledge to manage life with menstrual-related CF symptoms; 3) cultivation of the body-self as a menstruating woman with CF. These dynamic processes capture how cis-gender women with CF attune to their bodies, navigate their illness, and situate themselves within their lifeworlds. Genetic conditions like CF are apt for studying these processes because adults have managed their disease for decades, with longitudinal experience that often exceeds that of their clinicians. Our evidence elucidates the co-constitutive nature of chronic disease, gendered subjectivity, and biological processes in flux. We explored the menstrual cyclicity of chronic disease symptoms by having 72 participants track their CF symptoms across 4 menstrual cycles on a customized period-tracking app. We performed semi-structured interviews with 20 participants to understand how they interpreted these cyclical CF symptoms. We learned that digital tracking attuned participants to monthly fluctuations in CF symptoms. They applied this knowledge to manage their lives and shape their sense of self. We argue that women with CF produce distinct embodied knowledge during their reproductive years, shaping their illness experience, disease management, overall health, quality of life, and selfhood. The dynamics we describe may reflect broader patterns by which women with other chronic illnesses experience their bodies and understand themselves in the world.

PMID:38993933 | PMC:PMC11238905 | DOI:10.1016/j.ssmqr.2024.100412

J Am Acad Dermatol. 2024 Jul 9:S0190-9622(24)01038-7. doi: 10.1016/j.jaad.2024.06.075. Online ahead of print.

NO ABSTRACT

PMID:38992504 | DOI:10.1016/j.jaad.2024.06.075

J Am Acad Dermatol. 2024 Jul 9:S0190-9622(24)01039-9. doi: 10.1016/j.jaad.2024.07.008. Online ahead of print.

NO ABSTRACT

PMID:38992503 | DOI:10.1016/j.jaad.2024.07.008

Lancet. 2024 Jul 8:S0140-6736(24)00909-7. doi: 10.1016/S0140-6736(24)00909-7. Online ahead of print.

ABSTRACT

BACKGROUND: International guidelines have recommended cognitive behavioural therapy, including acceptance and commitment therapy (ACT), as it offers validated benefits for managing fibromyalgia; however, it is inaccessible to most patients. We aimed to evaluate the effect of a 12-week, self-guided, smartphone-delivered digital ACT programme on fibromyalgia management.

METHODS: In the PROSPER-FM randomised clinical trial conducted at 25 US community sites, adult participants aged 22-75 years with fibromyalgia were recruited and randomly assigned (1:1) to the digital ACT group or an active control group that offered daily symptom tracking and monitoring and access to health-related and fibromyalgia-related educational materials. Randomisation was done with a web-based system in permuted blocks of four at the site level. We used a blind-to-hypothesis approach in which participants were informed they would be randomly assigned to one of two potentially effective therapies under evaluation. Research staff were not masked to group allocation, with the exception of a masked statistics group while preparing statistical programming for the interim analysis. The primary endpoint was patient global impression of change (PGIC) response rate at week 12. Analyses were by intention to treat. The trial was registered with ClinicalTrials.gov, NCT05243511 (now fully closed).

FINDINGS: Between Feb 8, 2022, and Feb 2, 2023, 590 individuals were screened, of whom 275 (257 women and 18 men) were randomly assigned to the digital ACT group (n=140) and the active control group (n=135). At 12 weeks, 99 (71%) of 140 ACT participants reported improvement on PGIC versus 30 (22%) of 135 active control participants, corresponding to a difference in proportions of 48·4% (95% CI 37·9-58·9; p<0·0001). No device-related safety events were reported.

INTERPRETATION: Digital ACT was safe and efficacious compared with digital symptom tracking in managing fibromyalgia in adult patients.

FUNDING: Swing Therapeutics.

PMID:38991582 | DOI:10.1016/S0140-6736(24)00909-7

PLoS Pathog. 2024 Jul 11;20(7):e1012394. doi: 10.1371/journal.ppat.1012394. Online ahead of print.

ABSTRACT

Staphylococcus aureus is a facultative intracellular pathogen of human macrophages, which facilitates chronic infection. The genotypes, pathways, and mutations influencing that phenotype remain incompletely explored. Here, we used two distinct strategies to ascertain S. aureus gene mutations affecting pathogenesis in macrophages. First, we analyzed isolates collected serially from chronic cystic fibrosis (CF) respiratory infections. We found that S. aureus strains evolved greater macrophage invasion capacity during chronic human infection. Bacterial genome-wide association studies (GWAS) identified 127 candidate genes for which mutation was significantly associated with macrophage pathogenesis in vivo. In parallel, we passaged laboratory S. aureus strains in vitro to select for increased infection of human THP-1 derived macrophages, which identified 15 candidate genes by whole-genome sequencing. Functional validation of candidate genes using isogenic transposon mutant knockouts and CRISPR interference (CRISPRi) knockdowns confirmed virulence contributions from 37 of 39 tested genes (95%) implicated by in vivo studies and 7 of 10 genes (70%) ascertained from in vitro selection, with one gene in common to the two strategies. Validated genes included 17 known virulence factors (39%) and 27 newly identified by our study (61%), some encoding functions not previously associated with macrophage pathogenesis. Most genes (80%) positively impacted macrophage invasion when disrupted, consistent with the phenotype readily arising from loss-of-function mutations in vivo. This work reveals genes and mechanisms that contribute to S. aureus infection of macrophages, highlights differences in mutations underlying convergent phenotypes arising from in vivo and in vitro systems, and supports the relevance of S. aureus macrophage pathogenesis during chronic respiratory infection in CF. Additional studies will be needed to illuminate the exact mechanisms by which implicated mutations affect their phenotypes.

PMID:38991026 | DOI:10.1371/journal.ppat.1012394

Eur Arch Paediatr Dent. 2024 Jul 11. doi: 10.1007/s40368-024-00885-8. Online ahead of print.

ABSTRACT

PURPOSE: Good oral health is important for children and adolescents with cystic fibrosis (CF). The purpose of this scoping review is to describe the existing evidence base regarding oral health in children and adolescents with CF and provide recommendations for future research.

METHODS: Using a scoping review framework, a comprehensive search was undertaken using medline, embase, and PubMed. The search strategy included broad terms relating to CF, oral health, and children and adolescents and included only papers written in English.

RESULTS: 61 articles were included. Topics investigated included dental caries, enamel defects, periodontal health, dental staining, oral health related quality of life, dental management, and dental development of children and adolescents with CF.

CONCLUSION: Dental outcomes of children and adolescents with CF differ from the healthy population. The current literature describing dental health in children and adolescents with CF includes predominately descriptive analyses. A shift to hypothesis-based studies to explore causal relationships that explain the differences in dental outcomes seen in the CF population offers an opportunity to better understand the problems faced by children and adolescents with CF. Research that actively engages stakeholders, including children and adolescents with CF and their families will enable evidence-based recommendations to improve their oral health.

PMID:38990412 | DOI:10.1007/s40368-024-00885-8

Pediatr Pulmonol. 2024 Jul 11. doi: 10.1002/ppul.27173. Online ahead of print.

NO ABSTRACT

PMID:38990108 | DOI:10.1002/ppul.27173

Int Forum Allergy Rhinol. 2024 Jul 11. doi: 10.1002/alr.23413. Online ahead of print.

ABSTRACT

Chitosan is a promising drug delivery vector for therapeutics owing to its biocompatibility. Once crosslinked with chitosan, prolonged drug release was noted regardless of hydrophilicity. Hydrophilic drugs may require different strategies to obtain a sustained release profile.

PMID:38990106 | DOI:10.1002/alr.23413

Pediatr Pulmonol. 2024 Jul 11. doi: 10.1002/ppul.27175. Online ahead of print.

ABSTRACT

BACKGROUND: Racial and ethnic disparities in pediatric lung transplantation (LTx) related to the shifting cystic fibrosis (CF) population receiving highly effective modulator therapy (HEMT) has not been well investigated.

METHODS: The UNOS Registry was queried for patients age 1-25 years undergoing bilateral LTx between 1 January 2012 and 31 December 2021. Race and ethnicity were classified as non-Hispanic White, non-Hispanic Black, Hispanic, or none of the above. The primary outcome was posttransplant mortality. Trends in the association between race/ethnicity and mortality were examined using transplant year as a continuous variable and stratifying year based on introduction of HEMT (triple combination therapy) in November 2019.

RESULTS: In the study sample (N = 941), 7% of patients were non-Hispanic Black, 15% were Hispanic, and 2% were some other racial or ethnic group. One hundred (11%) received LTx after approval of triple combination therapy, and 407 (43%) died during follow-up. We identified a statistically significant disparity in mortality hazard (hazard ratio: 1.91; 95% confidence interval: 1.31, 2.80) in non-Hispanic Black compared to non-Hispanic White patients in the pre-triple combination therapy era.

CONCLUSIONS: We found higher mortality hazard among non-Hispanic Black compared to non-Hispanic White children undergoing LTx in the United States. Further monitoring of LTx outcomes to identify and address disparities is needed in the current era of triple combination therapy for CF.

PMID:38990104 | DOI:10.1002/ppul.27175