Pediatr Pulmonol. 2023 Jul 28. doi: 10.1002/ppul.26602. Online ahead of print.

ABSTRACT

BACKGROUND: Handheld spirometry allows monitoring of lung function at home, of particular importance during the COVID-19 pandemic. Pediatric studies are unclear on whether values are interchangeable with traditional, clinic-based spirometry. We aimed to assess differences between contemporaneous, home (unsupervised) and clinic (supervised) spirometry and the variability of the former. The accuracy of the commercially available spirometer used in the study was also tested.

METHODS: Data from participants in the Clinical Monitoring and Biomarkers to stratify severity and predict outcomes in children with cystic fibrosisc (CLIMB-CF) Study aged ≥ 6 years who had paired (±1 day) clinic and home forced expiratory volume in 1 s (FEV1 ) readings were analyzed. Variability during clinical stability over 6-months was assessed. Four devices from Vitalograph were tested using 1 and 3 L calibration syringes.

RESULTS: Sixty-seven participants (median [interquartile range] age 10.7 [7.6-13.9] years) provided home and clinic FEV1 data pairs. The mean (SD) FEV1 % bias was 6.5% [±8.2%]) with wide limits of agreement (-9.6% to +22.7%); 76.2% of participants recorded lower results at home. Coefficient of variation of home FEV1 % during stable periods was 9.9%. Data from the testing of the handheld device used in CLIMB-CF showed a potential underread.

CONCLUSION: In children and adolescents, home spirometry using hand-held equipment cannot be used interchangeably with clinic spirometry. Home spirometry is moderately variable during clinical stability. New handheld devices underread, particularly at lower volumes of potential clinical significance for smaller patients; this suggests that supervision does not account fully for the discrepancy. Opportunities should be taken to obtain dual device measurements in clinic, so that trend data from home can be utilized more accurately.

PMID:37503909 | DOI:10.1002/ppul.26602

Pediatr Pulmonol. 2023 Jul 28. doi: 10.1002/ppul.26609. Online ahead of print.

NO ABSTRACT

PMID:37503879 | DOI:10.1002/ppul.26609

Pediatr Pulmonol. 2023 Jul 28. doi: 10.1002/ppul.26621. Online ahead of print.

NO ABSTRACT

PMID:37503874 | DOI:10.1002/ppul.26621

Nucleic Acids Res. 2023 Jul 28:gkad622. doi: 10.1093/nar/gkad622. Online ahead of print.

ABSTRACT

Deazaguanine DNA modifications are widespread in phages, particularly in those with pathogenic hosts. Pseudomonas phage iggy substitutes ∼16.5% of its genomic 2'-deoxyguanosine (G) with dPreQ0, and the iggy deazaguanine transglycosylase (DpdA) is unique in having a strict GA target motif, not observed previously. The iggy PreQ0 modification is shown to provide protection against both restriction endonucleases and Cas9 (when present in PAM), thus expanding our understanding of the deazaguanine modification system, its potential, and diversity. Phage iggy represents a new genus of Pseudomonas phages within the Queuovirinae subfamily; which have very little in common with other published phage genomes in terms of nucleotide similarity (<10%) and common proteins (<2%). Interestingly, shared similarity is concentrated in dpdA and preQ0 biosynthesis genes. TEM imaging confirmed a siphovirus morphology with a prolate icosahedral head and a non-contractile flexible tail with one long central tail spike. The observed protective effect of the deazaguanine modification on the iggy DNA may contribute to its broad within-species host range. Phage iggy was isolated on Pseudomonas aeruginosa PAO1, but also infects PDO300, PAK, PA14, as well as 10 of 27 tested environmental isolates and 13 of 20 tested clinical isolates of P. aeruginosa from patients with cystic fibrosis.

PMID:37503841 | DOI:10.1093/nar/gkad622

J Clin Endocrinol Metab. 2023 Jul 28:dgad443. doi: 10.1210/clinem/dgad443. Online ahead of print.

ABSTRACT

CONTEXT: Elexacaftor/Tezacaftor/Ivacaftor (ETI; Trikafta™) enhances aberrant cystic fibrosis transmembrane conductance regulator (CFTR) function and may improve the insulin secretory defects associated with a deterioration in clinical outcomes in pancreatic insufficient cystic fibrosis (PI-CF).

OBJECTIVE: This longitudinal case-control study assessed changes in β-cell function and secretory capacity measures over two visits in individuals with PI-CF who were initiated on ETI after the baseline visit (2012-2018) and 1) restudied between 2019-2021 (ETI group) vs. 2) those restudied between 2015-2018 and not yet treated with CFTR modulator therapy (controls).

METHODS: Nine ETI participants (mean ± SD age 25 ± 5 years) and eight matched controls were followed up after median(IQR) 5(4-7) and 3(2-3) years, respectively (p < 0.01), with ETI initiation a median of 1 year before follow-up. Clinical outcomes, glucose-potentiated arginine (GPA) and mixed-meal tolerance test (MMTT) measures were assessed with comparisons of within and between group change by non-parametric testing.

RESULTS: Glucose-potentiated insulin and C-peptide responses to GPA deteriorated in controls but not the ETI group, with C-peptide changes different between groups (p < 0.05). Deterioration in basal proinsulin secretory ratio was observed in controls but improved, as did the maximal arginine-induced proinsulin secretory ratio, in the ETI group (p < 0.05 for all comparisons). During MMTT, early insulin secretion improved as evidenced by more rapid insulin secretory rate kinetics.

CONCLUSION: ETI preserves β-cell function in CF through effects on glucose-dependent insulin secretion, proinsulin processing and meal-related insulin secretion. Further work should determine whether early intervention with ETI can prevent deterioration of glucose tolerance in PI-CF.

PMID:37503734 | DOI:10.1210/clinem/dgad443

medRxiv. 2023 Jul 17:2023.07.14.23292662. doi: 10.1101/2023.07.14.23292662. Preprint.

ABSTRACT

Preterm infants (<34 weeks gestation) experience high rates of morbidity and mortality before hospital discharge. Genetic disorders substantially contribute to morbidity and mortality in related populations. The prevalence and clinical impact of genetic disorders is unknown in this population. We sought to determine the prevalence of commonly diagnosed genetic disorders in preterm infants, and to determine the association of disorders with morbidity and mortality. This was a retrospective multicenter cohort study of infants born from 23 to 33 weeks gestation between 2000 and 2020. Genetic disorders were abstracted from diagnoses present in electronic health records. We excluded infants transferred from or to other health care facilities prior to discharge or death when analyzing clinical outcomes. We determined the adjusted odds of pre-discharge morbidity or mortality after adjusting for known risk factors. Of 409,704 infants, 5838 (1.4%) had genetic disorders. Infants with trisomy 13, 18, 21, or cystic fibrosis had greater adjusted odds of severe morbidity or mortality. Of the 17,427 infants who died, 566 (3.2%) had genetic disorders. Of the 65,968 infants with a severe morbidity, 1319 (2.0%) had genetic disorders. Our work demonstrates that genetic disorders are prevalent in preterm infants, especially those with life-threatening morbidities. Clinicians should consider genetic testing for preterm infants with severe morbidity and maintain a higher index of suspicion for life-threatening morbidities in preterm infants with genetic disorders. Prospective genomic research is needed to clarify the prevalence of genetic disorders in this population, and the contribution of genetic disorders to preterm birth and subsequent morbidity and mortality.

PMID:37503109 | PMC:PMC10370234 | DOI:10.1101/2023.07.14.23292662

bioRxiv. 2023 Jul 14:2023.07.14.549068. doi: 10.1101/2023.07.14.549068. Preprint.

ABSTRACT

Stenotrophomonas maltophilia is a Gram-negative emerging opportunistic pathogen often found in respiratory diseases such as cystic fibrosis (CF). Patients with CF experience lifelong polymicrobial infections of the respiratory mucosa. Our prior work showed that P. aeruginosa promotes persistence of S. maltophilia mouse respiratory infections. As is typical for environmental opportunistic pathogens, S. maltophilia has a large genome and a high degree of genetic diversity. In this study, we evaluated the genomic content of S. maltophilia, combining short and long read sequencing to construct complete genomes of 10 clinical isolates which were then compared with the larger phylogeny of S. maltophilia genomic sequence data, and compared colonization/persistence in vivo, alone and in coinfection with P. aeruginosa . We found that while the overall genome size and GC content were fairly consistent, there was considerable variability in arrangement and gene content. Similarly, there was significant variability in S. maltophilia colonization and persistence in vivo in experimental mouse respiratory infection. Ultimately, this study gives us a greater understanding of the genomic diversity of S. maltophilia isolated from patients, and how this genomic diversity relates to interactions with other pulmonary pathogens, and to host disease progression. Identifying the molecular determinants of infection with S. maltophilia can facilitate development of novel antimicrobial strategies for a highly drug-resistant pathogen.

PMID:37503051 | PMC:PMC10369963 | DOI:10.1101/2023.07.14.549068

Res Sq. 2023 Jul 21:rs.3.rs-3112506. doi: 10.21203/rs.3.rs-3112506/v1. Preprint.

ABSTRACT

Background : sodium-dependent glucose cotransporter 1 and 2 (SGLT1/2) belong to the family of glucose transporters, encoded by SLC5A1 and SLC5A2, respectively. SGLT-2 is almost exclusively expressed in the renal proximal convoluted tubule cells. SGLT-1 is expressed in the kidneys but also in other organs throughout the body. Many SGLT inhibitor drugs have been developed based on the mechanism of blocking glucose (re)absorption mediated by SGLT1/2, and several have gained major regulatory agencies' approval for treating diabetes. Intriguingly these drugs are also effective in treating diseases beyond diabetes, for example heart failure and chronic kidney disease. We recently discovered that SGLT-1 is upregulated in the airway epithelial cells derived from patients of cystic fibrosis (CF), a devastating genetic disease affecting greater than 70,000 worldwide. Results : in the present work, we show that the SGLT-1 upregulation is coupled with elevated endoplasmic reticulum (ER) stress response, indicated by activation of the primary ER stress senor inositol-requiring protein 1a(IRE1a) and the ER stress-induced transcription factor X-box binding protein 1 (XBP1), in CF epithelial cells, and in epithelial cells of other stress conditions. Through biochemistry experiments, we demonstrated that XBP1 acts as a transcription factor for SLC5A1 by directly binding to its promoter region. Targeting this ER stress ® SLC5A1 axis by either the ER stress inhibitor Rapamycin or the SGLT-1 inhibitor Sotagliflozin was effective in attenuating the ER stress response and reducing the SGLT-1 levels in these cellular model systems. Conclusions : the present work establishes a causal relationship between ER stress and SGLT-1 upregulation and provides a mechanistic explanation why SGLT inhibitor drugs benefit diseases beyond diabetes.

PMID:37502997 | PMC:PMC10371076 | DOI:10.21203/rs.3.rs-3112506/v1

bioRxiv. 2023 Jul 19:2023.07.17.548927. doi: 10.1101/2023.07.17.548927. Preprint.

ABSTRACT

Excessive alcohol use is thought to increase the risk of respiratory infections by impairing mucociliary clearance (MCC). In this study, we investigate the hypothesis that alcohol reduces the function of CFTR, the protein that is defective in individuals with cystic fibrosis, thus altering mucus properties to impair MCC and the airway's defense against inhaled pathogens.

METHODS: Sprague Dawley rats with wild type CFTR (+/+), matched for age and sex, were administered either a Lieber-DeCarli alcohol diet or a control diet with the same number of calories for eight weeks. CFTR activity was measured using nasal potential difference (NPD) assay and Ussing chamber electrophysiology of tracheal tissue samples. In vivo MCC was determined by measuring the radiographic clearance of inhaled Tc99 particles and the depth of the airway periciliary liquid (PCL) and mucus transport rate in excised trachea using micro-optical coherence tomography (µOCT). The levels of rat lung MUC5b and CFTR were estimated by protein and mRNA analysis.

RESULTS: Alcohol diet was found to decrease CFTR ion transport in the nasal and tracheal epithelium in vivo and ex vivo . This decrease in activity was also reflected in partially reduced full-length CFTR protein levels but not, in mRNA copies, in the lungs of rats. Furthermore, alcohol-fed rats showed a significant decrease in MCC after 8 weeks of alcohol consumption. The trachea from these rats also showed reduced PCL depth, indicating a decrease in mucosal surface hydration that was reflected in delayed mucus transport. Diminished MCC rate was also likely due to the elevated MUC5b expression in alcohol-fed rat lungs.

CONCLUSIONS: Excessive alcohol use can decrease the expression and activity of CFTR channels, leading to reduced airway surface hydration and impaired mucus clearance. This suggests that CFTR dysfunction plays a role in the compromised lung defense against respiratory pathogens in individuals who drink alcohol excessively.

WHAT IS THE CURRENT SCIENTIFIC KNOWLEDGE ON THIS SUBJECT?: Excessive alcohol is associated with delayed mucociliary clearance (MCC) and an increased risk of respiratory infections among heavy drinkers.

WHAT DOES THIS STUDY ADD TO THE FIELD?: Chronic alcohol use reduces CFTR activity and airway surface hydration explaining the mechanisms underlying mucociliary dysfunction. Acquired CFTR dysfunction may be a suitable target to improve host immunity in those affected by prolonged alcohol use.

PMID:37502889 | PMC:PMC10370077 | DOI:10.1101/2023.07.17.548927

J Ovarian Res. 2023 Jul 27;16(1):148. doi: 10.1186/s13048-023-01226-x.

ABSTRACT

BACKGROUND: Over the past two decades, increasing number of people with cystic fibrosis (CF) survive into adulthood. Compared to the general population, sub-fertility is an obstacle for many women with CF (wwCF). Decreased ovarian reserve has been proposed as a possible cause, but limited data is available to support this. The aim of this study was to evaluate the ovarian reserve in wwCF and to correlate this with patients' demographic and clinical data.

METHODS: Reproductive-aged wwCF were enrolled during their routine medical appointments. Assessment included Anti-Mullerian hormone (AMH) levels, routine blood tests and antral follicular count (AFC) evaluation. Additionally, demographic, and clinical information were collected.

RESULTS: A total of wenty-three wwCF were enrolled, with ages ranging from 19 to 40 years (median 27 years). Among the fourteen wwCF who were considering pregnancy, five (35.7%) disclosed undergoing an infertility assessment and receiving fertility treatments. All but one patient had an Anti-Mullerian hormone (AMH) level between the 5th and 95th % for age. Measurement of the antral follicular count (AFC) was possible in 12 of the 23 patients and was ranging 8-40 with a median of 17. The proportion of wwCF presenting below median AMH values was not different in sub-fertile as compared to fertile wwCF (P value 0.54). There were no correlations between AMH levels and disease severity parameters. AMH seemed to be relatively higher in wwCF with mild class mutations, but this was not shown to have statistical significance.

CONCLUSIONS: Our results, in contrast with the limited available published data, do not support the hypothesis that decreased ovarian reserve plays a major role in infertility in wwCF.

PMID:37501150 | DOI:10.1186/s13048-023-01226-x

Pediatr Radiol. 2023 Jul 28. doi: 10.1007/s00247-023-05706-6. Online ahead of print.

ABSTRACT

BACKGROUND: Pediatric radiologists can identify a liver ultrasound (US) pattern predictive of progression to advanced liver disease. However, reliably discriminating these US patterns remains difficult. Quantitative magnetic resonance imaging (MRI) may provide an objective measure of liver disease in cystic fibrosis (CF).

OBJECTIVE: The purpose of this study was to determine if quantitative MRI, including MR elastography, is feasible in children with CF and to determine how quantitative MRI-derived metrics compared to a research US.

MATERIALS AND METHODS: A prospective, multi-institutional trial was performed evaluating CF participants who underwent a standardized MRI. At central review, liver stiffness, fat fraction, liver volume, and spleen volume were obtained. Participants whose MRI was performed within 1 year of US were classified by US pattern as normal, homogeneous hyperechoic, heterogeneous, or nodular. Each MRI measure was compared among US grade groups using the Kruskal-Wallis test.

RESULTS: Ninety-three participants (51 females [54.8%]; mean 15.6 years [range 8.1-21.7 years]) underwent MRI. MR elastography was feasible in 87 participants (93.5%). Fifty-eight participants had an US within 1 year of MRI. In these participants, a nodular liver had significantly higher stiffness (P<0.01) than normal or homogeneous hyperechoic livers. Participants with a homogeneous hyperechoic liver had a higher fat fraction (P<0.005) than others.

CONCLUSION: MR elastography is feasible in children with CF. Participants with a nodular pattern had higher liver stiffness supporting the US determination of advanced liver disease. Participants with a homogeneous hyperechoic pattern had higher fat fractions supporting the diagnosis of steatosis.

PMID:37500799 | DOI:10.1007/s00247-023-05706-6

Anticancer Res. 2023 Aug;43(8):3471-3477. doi: 10.21873/anticanres.16523.

ABSTRACT

BACKGROUND/AIM: GlyH-101 and CaCCinh-A01 are effective blockers of cystic fibrosis transmembrane conductance regulator (CFTR) and Ca2+-activated chloride channels (CaCCs), respectively. Available evidence suggests that GlyH-101 and CaCCinh-A01 can suppress cell proliferation, block invasion and metastasis, and cause several cancer cell types to undergo apoptosis, demonstrating their anti-tumor properties. The aim of this study was to investigate the effect of GlyH-101 and CaCCinh-A01 on HT-29 cell activity and to suggest the possible molecular mechanisms by which inhibitors of CFTR and CaCCs inhibit HT-29 cell activity.

MATERIALS AND METHODS: Human colon HT-29 cancer cells were treated with GlyH-101 or CaCCinh-A01 or GlyH-101 plus CaCCinh-A01 complex. Cell viability was determined by MTT assay, the apoptosis and cell cycle were determined by flow cytometry, and reactive oxygen species (ROS) leves were determined by 2',7'-Dichlorodihydrofluorescein diacetate staining. The expression of proteins related to apoptosis and cell cycle regulation was measured by western blotting.

RESULTS: The proliferative ability of HT-29 cells was dose- and time-dependently reduced by GlyH-101 and CaCCinh-A01. Treatment with GlyH-101 and CaCCinh-A01 resulted in cell necrosis and apoptosis, up-regulated ROS levels, activated the mitochondrial apoptosis pathway, prompted arrest of the cell cycle in S phase, and increased the levels of proteins related to the cell cycle. Additionally, the combination of these two inhibitors had a stronger regulatory effect on HT-29 cell proliferation than either GlyH-101 or CaCCinh-A01 treated alone.

CONCLUSION: GlyH-101 and CaCCinh-A01 inhibited cell proliferation through cell cycle arrest and mitochondrial-related pathways in vitro. The combination of these inhibitors could further enhance their anti-proliferative effects. Our findings propose new lead compounds with anti-colon cancer activity, and also provide new evidence for the effectiveness of chloride channels-targeted therapy in anticancer therapy.

PMID:37500166 | DOI:10.21873/anticanres.16523

Open Respir Arch. 2022 Jan 15;4(2):100155. doi: 10.1016/j.opresp.2022.100155. eCollection 2022 Apr-Jun.

NO ABSTRACT

PMID:37497314 | PMC:PMC10369578 | DOI:10.1016/j.opresp.2022.100155

J Pediatr Gastroenterol Nutr. 2023 Jul 27. doi: 10.1097/MPG.0000000000003898. Online ahead of print.

ABSTRACT

OBJECTIVES: Drug-associated acute pancreatitis (DAP) studies typically focus on single acute pancreatitis (AP) cases. We aimed to analyze the (1) characteristics, (2) co-risk factors, and (3) reliability of the Naranjo scoring system for DAP using INSPPIRE-2 (the INternational Study group of Pediatric Pancreatitis: In search for a cuRE-2) cohort study of acute recurrent (ARP) and chronic pancreatitis (CP) in children.

METHODS: Data were obtained from ARP group with ≥1 episode of DAP and CP group with medication exposure +/- DAP. Physicians could report multiple risk factors. Pancreatitis associated with Medication (Med) (ARP+CP) was compared to Non-Medication cases, and ARP-Med vs CP-Med groups. Naranjo score was calculated for each DAP episode.

RESULTS: Of 726 children, 392 had ARP, 334 CP. 51 children (39 ARP and 12 CP) had ≥ 1 AP associated with a medication. 61% had ≥ 1 AP without concurrent medication exposure. The Med group had other risk factors present (where tested): 10/35 (28.6%) genetic, 1/48 (2.1%) autoimmune pancreatitis, 13/51 (25.5%) immune-mediated conditions, 11/50 (22.0%) obstructive/ anatomic, 28/51 (54.9%) systemic risk factors. In Med group, 24/ 51 (47%) had involvement of >1 medication, simultaneously or over different AP episodes. There were 20 ARP and 4 CP cases in "probable" category and 19 ARP and 7 CP in "possible" category by Naranjo scores.

CONCLUSIONS: Medications were involved in 51/ 726 (7%) of ARP or CP patients in INSPPIRE-2 cohort; other pancreatitis risk factors were present in most, suggesting a potential additive role of different risks. The Naranjo scoring system failed to identify any cases as "definitive", raising questions about its reliability for DAP.

PMID:37496124 | DOI:10.1097/MPG.0000000000003898

BMJ Open Respir Res. 2023 Jul;10(1):e001647. doi: 10.1136/bmjresp-2023-001647.

ABSTRACT

OBJECTIVE: Identify prevalence of self-reported swallow, communication, voice and cognitive compromise following hospitalisation for COVID-19.

DESIGN: Multicentre prospective observational cohort study using questionnaire data at visit 1 (2-7 months post discharge) and visit 2 (10-14 months post discharge) from hospitalised patients in the UK. Lasso logistic regression analysis was undertaken to identify associations.

SETTING: 64 UK acute hospital Trusts.

PARTICIPANTS: Adults aged >18 years, discharged from an admissions unit or ward at a UK hospital with COVID-19.

MAIN OUTCOME MEASURES: Self-reported swallow, communication, voice and cognitive compromise.

RESULTS: Compromised swallowing post intensive care unit (post-ICU) admission was reported in 20% (188/955); 60% with swallow problems received invasive mechanical ventilation and were more likely to have undergone proning (p=0.039). Voice problems were reported in 34% (319/946) post-ICU admission who were more likely to have received invasive (p<0.001) or non-invasive ventilation (p=0.001) and to have been proned (p<0.001). Communication compromise was reported in 23% (527/2275) univariable analysis identified associations with younger age (p<0.001), female sex (p<0.001), social deprivation (p<0.001) and being a healthcare worker (p=0.010). Cognitive issues were reported by 70% (1598/2275), consistent at both visits, at visit 1 respondents were more likely to have higher baseline comorbidities and at visit 2 were associated with greater social deprivation (p<0.001).

CONCLUSION: Swallow, communication, voice and cognitive problems were prevalent post hospitalisation for COVID-19, alongside whole system compromise including reduced mobility and overall health scores. Research and testing of rehabilitation interventions are required at pace to explore these issues.

PMID:37495260 | DOI:10.1136/bmjresp-2023-001647

Respirology. 2023 Jul 26. doi: 10.1111/resp.14562. Online ahead of print.

NO ABSTRACT

PMID:37495234 | DOI:10.1111/resp.14562

J Cyst Fibros. 2023 Jul 24:S1569-1993(23)00832-9. doi: 10.1016/j.jcf.2023.07.003. Online ahead of print.

NO ABSTRACT

PMID:37495469 | DOI:10.1016/j.jcf.2023.07.003

J Cyst Fibros. 2023 Jul 24:S1569-1993(23)00830-5. doi: 10.1016/j.jcf.2023.07.002. Online ahead of print.

ABSTRACT

BACKGROUND: Our understanding of the natural history of cystic fibrosis liver disease (CFLD) is limited, leading to uncertainty for patients their families and clinicians when liver abnormalities are identified.

AIM: to determine the incidence of CFLD, identify risk factors and document the natural history of liver abnormalities in cystic fibrosis (CF).

METHODS: The Irish longitudinal study of CFLD (ILSCFLD) prospectively enrolled 95% of children with CF in 2007. Their liver disease status was classified as (i) advanced liver disease with portal hypertension (CFLD). (ii) nonspecific cystic fibrosis liver disease (NSCFLD) (iii) no liver disease (NoLD) RESULTS: 480/522 (91.9%) children were followed for a median 8.53 years IQR 1.28, of whom 35 (7.29%) had CFLD, 110 (22.9%) NSCFLD and 335 (69.79%) had NoLD. At follow-up 28/445 (6.29%) participants without CFLD at baseline, progressed to CFLD (Incidence 7.51/1000 person years (Pyrs) (95%CI 4.99-10.86). Of these 25/28(89.28%) were <10 years. No participant >10 years of age without clinical or radiological evidence of liver disease at baseline progressed to CFLD. During follow-up 18/35(51.43%) participants with CFLD died or received a transplant, MTx rate 7.75/100 Pyrs (95%CI 4.59-12.25) compared to NSCFLD 2.33/100 Pyrs (95%CI 1.44-3.56) and NoLD 1.13/100 Pyrs (95%CI 0.77-1.59). CFLD was an independent risk factor for mortality in CF. Children with CFLD also had a shorter life expectancy.

CONCLUSION: The incidence of CFLD was highest in children under10 years. Children over10 years, with normal hepatic function did not develop CFLD. Research to identify the cause and improve outcome should focus on young children.

PMID:37495468 | DOI:10.1016/j.jcf.2023.07.002

R I Med J (2013). 2023 Aug 3;106(7):58-63.

ABSTRACT

While smoking prevalence has decreased among the general population, the use of electronic cigarettes (E- cigarettes) has risen significantly and can cause significant lung injury. We sought to determine if persons with cystic fibrosis (PwCF) have similar rates of E-cigarette use as compared with age-matched peers, and to understand perceptions of E-cigarette safety through a survey-based study. A total of 29 PwCF and 26 age-matched control patients participated in this study. There was no significant difference between PwCF and control patients regarding perceptions of the negative impact of E-cigarette use on one's health. Overall, both PwCF and control patients reported a good quality of life. PwCF were equally likely to identify E-cigarettes as harmful to one's lung health as healthy controls but were significantly more likely to have heard of EVALI. While small, our study has demonstrated the need for further education of both PwCF and healthy young adults.

PMID:37494629

Pediatr Pulmonol. 2023 Jul 26. doi: 10.1002/ppul.26603. Online ahead of print.

ABSTRACT

The field of rare and diffuse pediatric lung disease continues to evolve and expand rapidly as clinicians and researchers make advancements in the diagnosis and treatment of children's interstitial and diffuse lung disease, non-cystic fibrosis bronchiectasis, and primary ciliary dyskinesia. Papers published on these topics in Pediatric Pulmonology and other journals in 2022 describe newly recognized disorders, elucidate disease mechanisms and courses, explore potential biomarkers, and assess novel treatments. In this review, we will discuss these important advancements and place them in the context of existing literature.

PMID:37493100 | DOI:10.1002/ppul.26603