RESUMEN
BACKGROUND: Primary ciliary dyskinesia (PCD) is a chronic respiratory illness that places significant strain on the healthcare system due to the complexity and expense of its diagnosis and treatment methods. The diagnostic process typically requires skilled technicians and an assortment of intricate, costly, and time-consuming approaches. Implementing screening tools can enhance efficiency by focusing the diagnostic process on those strongly suspected of having PCD. Tools such as the PCD Rule (PICADAR), North America Criteria Defined Clinical Features (NA-CDCF), the Clinical Index Score (CI), and the newly proposed CInew13 could potentially serve as useful screening tools. This study aims to examine the effectiveness of these tools individually, compare their performance against each other, and assess their results relative to prior research. METHODS: We conducted a diagnostic accuracy test on 83 Egyptian patients referred to Alexandria University Children's Hospital for potential PCD diagnosis between January 2015 and December 2022. The scores obtained from the screening tools were calculated and assessed. RESULTS: Of the initial group, 10 patients were ruled out because they fit other diagnostic parameters. Forty-three cases received a confirmed diagnosis, while 30 did not. Notably, the confirmed cases consistently scored higher on our screening tools than those that remained unconfirmed (p <.001, for all tested scores). We used receiver operating characteristic curves to assess and compare the effectiveness of each tool. The NA-CDCF had the smallest area under curve 0.736 (95% confiedence interval 0.619-0.832); in contrast, the CI score had the largest 0.898 (95% confidence interval 0.808-0.957). CONCLUSION: All the tools tested were effective in identifying suitable patients for PCD testing at statistically significant levels. However, the PICADAR and NA-CDCF scores' performance did not significantly differ in the current study. The CI and CInew13 scores, on the other hand, outperformed both.
RESUMEN
The aim of this study is to study cardio-respiratory effects of nasal high-frequency oscillatory ventilation (NHFOV) vs. NCPAP as an initial mode of ventilation in moderate-late-preterm infants. A randomized controlled trial was conducted in NICU of Alexandria University Maternity Hospital (AUMH). One-hundred late-moderate-preterm infants were randomly assigned to either NHFOV-group (n = 50) or NCPAP-group (n = 50). For both groups, functional echocardiography was performed in the first 24 h to detect hemodynamic changes and respiratory outcome was monitored throughout the hospital stay. The main outcomes were hemodynamic measurements and myocardial function using functional echocardiography of those infants along with the respiratory outcome and complications. Kaplan-Meier survival plot was used representing time course of NCPAP and NHFOV failure. Left ventricular output values were not significantly different in both groups with median 202 ml/kg /min and IQR (176-275) in NCPAP-group and 226 ml/kg/min with IQR (181-286) in NHFOV group. Nevertheless, ejection fraction and fractional shortening were significantly higher in NHFOV-group with P 0.001. The time to weaning, the time to reach 30%-FIO2, the need for invasive ventilation, oxygen support duration, and maximal-FIO2 were significantly more in NCAPAP group. Conclusion: NHFOV is an effective and promising tool of non-invasive-ventilation which can be used as a primary modality of respiratory support in preterm infants with variable forms of respiratory distress syndrome without causing detrimental effect on hemodynamics or significant respiratory complications. Trial registration: NCT05706428 (registered on January 21, 2023). What is Known: ⢠NHFOV might be beneficial as a secondary mode of ventilation and might have an impact on hemodynamics. What is New: ⢠NHFOV can be used as an initial mode of ventilation with CDP beyond the reported pressure limits of CPAP without causing neither CO2 retention nor adverse hemodynamic consequences.
Asunto(s)
Hemodinámica , Ventilación de Alta Frecuencia , Recien Nacido Prematuro , Humanos , Recién Nacido , Femenino , Masculino , Hemodinámica/fisiología , Ventilación de Alta Frecuencia/métodos , Ecocardiografía , Síndrome de Dificultad Respiratoria del Recién Nacido/terapia , Síndrome de Dificultad Respiratoria del Recién Nacido/fisiopatología , Resultado del TratamientoRESUMEN
BACKGROUND: Biosimilars constitute a pathway for sustainable financing of healthcare systems in the era of expensive biologics. However, such a pathway is not free of challenges. Since the biosimilars market is expanding in Egypt, there is an urgent need for a policy framework to optimize their use and diffusion in the market. We aim to characterize a national framework based on the experiences of other countries and consultation with local experts. METHODS: A narrative literature review was conducted to identify biosimilars' policy elements worldwide. A workshop was organized with experts to discuss the narrative review findings and create consensus on recommendations. RESULTS: The narrative literature review highlighted the need for biosimilar policy actions in four areas: market authorization, pricing, reimbursement, and uptake. Eighteen experts representing the Egyptian healthcare authorities attended the workshop. The most significant conclusions from the workshop included setting the price of the biosimilar at 30-40% less than its originator's price and establishing financing protocols, in which the more expensive biologics with significant price premiums should be excluded from the formulary. CONCLUSIONS: A summarized national framework policy recommendation for biosimilars was created by local experts from the main public healthcare entities in Egypt. These recommendations coincide with the international policies adopted across different countries that aim to improve patient access while sustaining health expenditure.
RESUMEN
BACKGROUND: Foreign body aspiration (FBA) is a common issue in pediatric emergencies, with regional variations. Various cultures and foods, parents' and physicians' inadequate experience, and lack of bronchoscopy equipment are some attributable factors in the regional variation of FBA. AIM: To more accurately represent the demographic characteristics of aspirated foreign bodies (FBs) across various continents, this review attempted to provide organized information based on the reviewed articles. METHODOLOGY: A search was conducted in PubMed/PubMed Central, EMBASE, and google scholar. From the 36 included articles, information on age, gender, bronchoscopy type, type of FB, location, history of choking, and time elapsed between aspiration and admission were extracted. RESULTS: A total of 14,469 cases were evaluated. According to the findings, children under two accounted for more than 75% of FBAs. Nuts and seeds were the most common FBs in most countries, whereas plastic and metal objects had higher rates in Brazil, and calcified objects were more prevalent in Thailand. The right bronchus was the most frequent location, and rigid bronchoscopy was the most often utilized type of bronchoscope. In addition, a significant percentage of patients were referred to the emergency rooms in the first 12-24 h following the aspiration incident. Moreover, Asian patients reported more choking history, and Europeans had fewer witnesses of FBA. CONCLUSIONS: There is a lack of standardized reporting systems and organized guidelines in pediatric FBA. To select the ideal time for endoscopies and create educational programs, a collaboration between experienced researchers, pediatric pulmonologists, radiologists, and otolaryngologists is required.
Asunto(s)
Obstrucción de las Vías Aéreas , Cuerpos Extraños , Niño , Humanos , Lactante , Estudios Retrospectivos , Broncoscopía , Bronquios , Obstrucción de las Vías Aéreas/epidemiología , Obstrucción de las Vías Aéreas/etiología , Cuerpos Extraños/epidemiologíaRESUMEN
Background: The Egyptian healthcare system is currently in the early phase of health technology assessment (HTA) implementation. The aim of this study is to propose an implementation roadmap based on the national healthcare system status. Methods: A survey was conducted among Egyptian healthcare sector decision-makers to assess the current and future (preferred) HTA implementation status in Egypt based on a widely used international scorecard methodology. Subsequently, interviews were conducted with experts representing middle- and top-tier management in the Egyptian healthcare system to interpret the survey results and recommend specific actions. Results: Experts recommended more capacity-building programs for HTA and health economics. Additionally, they proposed establishing HTA units in separate healthcare authorities and merging them into a single central HTA unit in the long term. Regarding the scope of implementation, experts recommended commencing with the assessment of innovative pharmaceuticals, and thereafter, expanding the scope to cover all health technologies in the long term. Additionally, they recommended using innovative tools such as "multi-criteria decision analysis (MCDA)" for tendering, and "managed entry agreements" for reimbursement decisions. Local burden of diseases and costing studies were also recommended to facilitate the implementation of HTA. Conclusion: Experts agreed that several actions are required for successful HTA implementation in Egypt, including coordination between HTA bodies, application of an explicit MCDA framework, and strengthening of local evidence generation. To implement these actions, investment in technical capacity-building is indispensable. Most experts favored using multiple and soft cost-effectiveness thresholds. Efforts should be made to publish HTA submission guidelines and timelines of the processes.
Asunto(s)
Creación de Capacidad , Evaluación de la Tecnología Biomédica , Evaluación de la Tecnología Biomédica/métodos , Egipto , Encuestas y Cuestionarios , Atención a la SaludRESUMEN
Pediatric eating assessment tool (Pedi-EAT-10Arabic) is a validated and reliable caregiver administered outcome instrument designed for detection of children at high risk of penetration/aspiration. The objective of this study is to translate and validate the Arabic version of Pedi-EAT-10 and to correlate its results with pharyngeal residue and aspiration on fiber optic endoscopic examination of swallowing (FEES). A cross-sectional study including 202 children selected randomly from those attending the swallowing clinic in phoniatrics unit, Otorhinolaryngology department (ORL) at main university hospital between February 2019 and October 2020 complaining of dysphagia. For test-retest reliability, one hundred caregivers refilled the Pedi-EAT-10Arabic after a 2-week period following their first visit. Validity was established by comparing the scores of dysphagia patients to healthy controls. Internal consistency of Pedi-EAT-10Arabic was high (Cronbach's alpha 0.986). Intra class correlation showed excellent test-retest reliability (r = 0.968). The median Pedi-EAT 10Arabic score was significantly higher in dysphagia group compared to healthy controls. (Median 27 IQR 21-34 for cases compared to median zero IQR 0-2 points for healthy controls, P less than 0.001). A strong correlation was found between Pedi-EAT 10Arabic scores and PAS scores with Spearman's correlation coefficient r = 0.803 and P < 0.001. The ROC for evaluating the discriminatory capacity of Pedi-EAT 10 for aspiration showed an AUC of 0.92 (95% CI of 0.89 to 0.96). Conclusion: Pedi-EAT 10Arabic was found to be a valid and reliable screening tool for further instrumental assessment of risk of dysphagia in pediatric population.
Asunto(s)
Trastornos de Deglución , Humanos , Niño , Trastornos de Deglución/diagnóstico , Trastornos de Deglución/etiología , Reproducibilidad de los Resultados , Encuestas y Cuestionarios , Estudios Transversales , DegluciónRESUMEN
BACKGROUND: Cases of foreign body aspiration in children may be encountered in emergency departments. A suggestive history is important in diagnosing aspirated foreign body owing to the difficulty in making a diagnosis on the basis of an abnormal physical examination or chest radiography alone. The aim of this study was to examine the sensitivity and specificity of the presenting symptoms, physical examination, and radiologic findings as predictors of foreign body aspiration in children. In addition, a feasible simple algorithm with a scoring system was generated to indicate bronchoscopic investigation. METHODS: In a retrospective cohort, medical records of patients aged less than 16 years with suspected foreign body aspiration who underwent flexible or rigid bronchoscopy were included. Data including age, sex, symptoms, physical examination findings, radiological features, nature and location of the foreign body, and outcome of the bronchoscopy were collected, and multivariable binary logistic regression analysis was employed for prediction of foreign body aspiration. RESULTS: A total of 203 children were included, and the model showed excellent discrimination power for positive foreign body aspiration (area under the curve = 0.911) with an accuracy, sensitivity, and specificity of 86.2, 90.6, and 76.6%, respectively. The total weighted risk score at a cut-off > 2 showed a significant good power of discrimination (area under the curve = 0.879), with a sensitivity of 79.9% and specificity of 84.4%. Accordingly, a clinical algorithm was recommended. CONCLUSIONS: The proposed scoring system and clinical algorithm might help in decision making with regard to the need and type of bronchoscopy in children presenting with potential foreign body aspiration. However, further prospective multicenter studies should be conducted to validate this scoring system.
Asunto(s)
Algoritmos , Cuerpos Extraños/diagnóstico , Aspiración Respiratoria/diagnóstico , Adolescente , Broncoscopía , Niño , Preescolar , Toma de Decisiones Clínicas , Estudios de Cohortes , Femenino , Humanos , Masculino , Estudios Retrospectivos , Sensibilidad y EspecificidadRESUMEN
Streptococcus pneumoniae remains an important bacterial pathogen, particularly for young children in low- and middle-income countries. A systematic review was conducted of peer-reviewed literature from PubMed published as of May 13, 2020, to identify articles relevant to invasive pneumococcal disease, pneumonia, otitis media (OM), nasopharyngeal carriage (NPC), antimicrobial resistance (AMR), and vaccination coverage in Egypt, with particular focus on children ≤ 18 years of age. A total of 16 relevant articles spanning three decades were included in this review. Among studies reviewed, S. pneumoniae was the causative agent of meningitis in 21-30% of cases among hospitalized children between 1983 and 2003. One study showed that serotypes 6A and 6B predominated among meningitis cases of pediatric patients aged < 5 years. This review also revealed that S. pneumoniae was the most commonly identified bacterial pathogen of acute mastoiditis, a severe complication of acute OM, among children aged 9 months to 11 years. NPC studies showed that approximately 30% of Egyptian children were carriers of S. pneumoniae. AMR, especially to penicillin, continues to be a growing concern in low- and middle-income countries, including among Egyptian children. Several predominant serotypes were identified to be associated with penicillin resistance, such as 6B, 1, 19A, 23F, and 6A. Currently available pneumococcal vaccines (PCVs) such as PCV10 and PCV13 may provide coverage against the most prevalent circulating serotypes among Egyptian children. Comprehensive disease surveillance and immunization programs are needed to ensure that this vulnerable population is sufficiently protected against pneumococcal disease.
RESUMEN
BACKGROUND: Primary ciliary dyskinesia (PCD), a genetically heterogeneous condition enriched in some consanguineous populations, results from recessive mutations affecting cilia biogenesis and motility. Currently, diagnosis requires multiple expert tests. METHODS: The diagnostic utility of multigene panel next-generation sequencing (NGS) was evaluated in 161 unrelated families from multiple population ancestries. RESULTS: Most (82%) families had affected individuals with biallelic or hemizygous (75%) or single (7%) pathogenic causal alleles in known PCD genes. Loss-of-function alleles dominate (73% frameshift, stop-gain, splice site), most (58%) being homozygous, even in non-consanguineous families. Although 57% (88) of the total 155 diagnostic disease variants were novel, recurrent mutations and mutated genes were detected. These differed markedly between white European (52% of families carry DNAH5 or DNAH11 mutations), Arab (42% of families carry CCDC39 or CCDC40 mutations) and South Asian (single LRRC6 or CCDC103 mutations carried in 36% of families) patients, revealing a striking genetic stratification according to population of origin in PCD. Genetics facilitated successful diagnosis of 81% of families with normal or inconclusive ultrastructure and 67% missing prior ultrastructure results. CONCLUSIONS: This study shows the added value of high-throughput targeted NGS in expediting PCD diagnosis. Therefore, there is potential significant patient benefit in wider and/or earlier implementation of genetic screening.
Asunto(s)
Cilios/genética , Trastornos de la Motilidad Ciliar/genética , Pruebas Genéticas , Secuenciación de Nucleótidos de Alto Rendimiento , Alelos , Pueblo Asiatico/genética , Cilios/patología , Trastornos de la Motilidad Ciliar/diagnóstico , Trastornos de la Motilidad Ciliar/patología , Estudios de Cohortes , Etnicidad/genética , Femenino , Homocigoto , Humanos , Masculino , Mutación/genética , FenotipoRESUMEN
Primary ciliary dyskinesia (PCD) is a rare genetic disorder of motile cilia dysfunction generally inherited as an autosomal recessive disease. Genetic testing is increasingly considered an early step in the PCD diagnostic workflow. We used targeted panel next-generation sequencing (NGS) for genetic screening of 33 Egyptian families with clinically highly suspected PCD. All variants prioritized were Sanger confirmed in the affected individuals and correctly segregated within the family. Targeted NGS yielded a high diagnostic output (70%) with biallelic mutations identified in known PCD genes. Mutations were identified in 13 genes overall, with CCDC40 and CCDC39 the most frequently mutated genes among Egyptian patients. Most identified mutations were predicted null effect variants (79%) and not reported before (85%). This study reveals that the genetic landscape of PCD among Egyptians is highly heterogeneous, indicating that a targeted NGS approach covering multiple genes will provide a superior diagnostic yield compared to Sanger sequencing for genetic diagnosis. The high diagnostic output achieved here highlights the potential of placing genetic testing early within the diagnostic workflow for PCD, in particular in developing countries where other diagnostic tests can be less available.