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Long-term ventilation (LTV) in children at home, especially invasive ventilation, is not widely available nor practised in low-resource settings (LRS). Barriers to providing LTV include underdeveloped pediatric critical care services, limited expertise in pediatric LTV, limited capacity to screen for sleep-disordered breathing (SDB) and high cost of LTV equipment and consumables. Additional challenges encountered in LRS may be unreliable electricity supply and difficult socioeconomic conditions. Where LTV at home has been successfully implemented, caregivers and families in LRS must often take full responsibility for their child's care as professional home-based nursing care is scarce. Selecting suitable children and families to offer LTV in LRS may therefore face difficult ethical decisions when families are disempowered or incapable of providing 24-h care at home. Early caregiver participation and hands-on training in tracheostomy care and LTV equipment is key to success, irrespective of the caregiver's level of education. The use of overnight oximetry, mobile phone technology, spirometry, and clinical evaluation are simple tools that can aid recognition and monitoring of children needing LTV. As children survive longer supported by LTV, engaging with adult services at an early stage is important to ensure suitable pathways for transition to adult care are in place. Building capacity and expertise in pediatric LTV in LRS requires targeted training of health professionals in related disciplines and advocacy to policymakers and funders that LTV in appropriately selected circumstances is worthwhile, life-changing, and cost-saving., (© 2023 The Authors. Pediatric Pulmonology published by Wiley Periodicals LLC.)

Background: Investigating inconclusive cystic fibrosis (CF) diagnosis in children is difficult without advanced cystic fibrosis transmembrane conductance regulator (CFTR) function tests. This study investigated the utility of beta (β)-adrenergic sweat test to exclude CF in participants with inconclusive diagnosis (CF suspects) in South Africa., Methods: β-adrenergic sweat test and sweat chloride tests (SCT) were performed simultaneously in CF suspects and adult controls (healthy, CFTR heterozygotes and CF). Cholinergic and β-adrenergic induced sweat rate was measured by evaporimetry (transepithelial water loss [TEWL]: g H 2 O/m 2 /h) following intradermal injections. Next-generation sequencing of CFTR was performed in CF suspects. CF diagnosis was defined by genotype., Results: Thirty-seven controls (10 healthy, 14 CF, 13 CFTR heterozygotes) and 32 CF suspects (26 children; 6 adults) were enrolled. Six were excluded from formal analyses due to β-adrenergic sweat test failure. In adults, evaporimetry was superior to SCT for diagnosis of CF with β-adrenergic:cholinergic ratio TEWL ≤ 0.05 achieving 100% sensitivity and specificity. Twenty-two CF suspect children (age range: 3.4-15.6 years) completed β-adrenergic sweat testing of which none had CF confirmed by genotyping: β-adrenergic:cholinergic ratio > 0.05 successfully excluded CF in all but one child who was CFTR heterozygous. Median peak β-adrenergic TEWL and β-adrenergic:cholinergic ratio in CFTR negative and CFTR heterozygous children was significantly lower than adult controls., Conclusion: β-adrenergic sweat test is more accurate than SCT for excluding CF in children with inconclusive diagnosis. Established reference ranges for β-adrenergic sweat test may not be suitable for children due to lower β-adrenergic sweat secretion compared to adults., (© 2022 The Authors. Pediatric Pulmonology published by Wiley Periodicals LLC.)

Introduction: Temporal trends in cystic fibrosis (CF) survival from low-middle-income settings is poorly reported. We describe changes in CF survival after diagnosis over 40 years from a South African (SA) CF center., Methods: An observational cohort study of people diagnosed with CF from 1974 to 2019. Changes in age-specific mortality rates from 2000 (vs. before 2000) were estimated using multivariable Poisson regression. Data were stratified by current age < or ≥10 years and models controlled for diagnosis age, sex, ethnicity, genotype, and Pseudomonas aeruginosa (PA) infection. A second analysis explored the association of mortality with weight and forced expiratory volume in 1 s reported as z-scores (FEV1z-scores) at age 5-8 years., Results: A total of 288 people (52% male; 57% Caucasian; 44% p.Phe508del homozygous) were included (median diagnosis age 0.5 years: Q1, Q3: 0.2, 2.5); 100 (35%) died and 30 (10%) lost to follow-up. Among age >10 years, age-specific mortality from 2000 was significantly lower (adjusted hazard ratio [aHR]: 0.14; 95% confidence interval [CI]: 0.06, 0.29; p < 0.001), but not among age <10 years (aHR: 0.67; 95% CI: 0.28, 1.64; p = 0.383). In children <10 years, Caucasian ethnicity was associated with lower mortality (aHR 0.17; 95% CI: 0.05, 0.63), and longer times since first PA infection with higher mortality (aHR: 1.31; 95% CI: 1.01, 1.68). Mortality was sevenfold higher if FEV1z was <-2.0 at age 5-8 years (aHR: 7.64; 95% CI: 2.58, 22.59)., Conclusion: Overall, CF survival has significantly improved in SA from 2000 in people older than 10 years. However, increased risk of mortality persists in young non-Caucasian children, and with FEV1z <-2.0 at age 5-8 years., (© 2021 Wiley Periodicals LLC.)

Objectives: Forced expiratory volume in 1 second (FEV1) is an important predictor of outcome in cystic fibrosis (CF). This study aimed to describe the change in lung function, nutritional status, and mortality of children with CF at a single center in Cape Town, South Africa, and identify factors associated with poor lung function and mortality., Methods: A retrospective study was conducted of children aged between 5 and 18 years between January 2007 and December 2016. At least two separate best annual FEV1 measurements were required for inclusion in the study., Results: A total of 143 children were followed up from which 107 study participants (median diagnosis age 5.5 months) were included. There was no statistically significant improvement from 2007 to 2016 in population mean FEV1 (2.5 ± 1.70 to -1.9 ± 1.70 [P = .1]) and body mass index (-0.7 ± 1.2 to -0.4 ± 1.2 [P = .3]) Z scores. FEV1 Z score declined by 0.17 per year. No significant correlation between FEV1 and age of diagnosis, sex, ethnicity, genotype, geographical location, pancreatic status, or infections was identified. On multiple stepwise regression analysis, FEV1 at age 6 was found to be the only independent predictor of mortality (adjusted odds ratio [95% CI] 0.5 [0.3-0.8]; P = .005)., Conclusion: FEV1 at age 6 was an independent predictor for CF-related mortality. Measurement of lung function in preschool children in SA with CF using more sensitive methods than spirometry is important to identify children at risk of poor outcomes., (© 2020 Wiley Periodicals, Inc.)

Objectives: Chronic Pseudomonas aeruginosa (Pa) infection in cystic fibrosis (CF) can be prevented with early eradication treatment. In resource-constrained environments, low-cost, off-label nebulized antibiotics, including intravenous gentamicin solution, are often used for eradication therapy. This study aimed to describe the characteristics and clinical course of children with CF and early Pa infection, treated with a Pa eradication protocol combining inhaled gentamicin and systemic antibiotics., Study Design: Retrospective descriptive study., Patient-Subject Selection: All children (0-18 years) attending a CF clinic in South Africa, with early Pa infections between January 2005 and March 2015, who received nebulized gentamicin-based Pa eradication treatment., Methodology: Data were described and compared between those with successful versus unsuccessful eradication, using descriptive and inferential statistics appropriate to normality of distribution., Results: One hundred and forty-nine children were managed in the CF Clinic over the study period, of whom 44 (29.5%; 28 [63.6%] male) had early Pa infections treated with a gentamicin-based eradication regimen. Thirty-nine (88.6%) patients had successful Pa eradication at 12 months follow-up; of which 28 (71.8%) had Pa reinfection at a median of 37.0 (21.0-101.0) months after initial treatment. Six patients (13%) acquired chronic Pa infection during the median follow-up period of 77 months. Older age was associated with Pa eradication failure and chronic Pa infection. There were no clinically significant adverse events associated with gentamicin inhalational therapy., Conclusions: Nebulized gentamicin solution combined with systemic antibiotics appears to be safe and has comparable efficacy to other strategies in eradicating early Pa infections in children with CF., (© 2019 Wiley Periodicals, Inc.)

Pulmonary hydatid cysts in children frequently develop complications such as infection and air leaks. Surgery combined with medical therapy remains the standard form of treatment. We report a 7-year-old child who developed rupture of a large pulmonary hydatid cyst following chest trauma; conservative management resulted in successful expulsion of the cyst.