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1.
Wiad Lek ; 73(8): 1790-1795, 2020.
Artigo em Inglês | MEDLINE | ID: mdl-33055353

RESUMO

OBJECTIVE: The aim: The clinical case was studied: comorbidity of mucoviscidosis and congenital dysfunction of adrenal glands cortex. PATIENTS AND METHODS: Materials and methods: The clinical case of combined orphan pathology - cystic fibrosis and congenital dysfunction of adrenal glands cortex (adrenogenital syndrome) has been described. RESULTS: Clinical case: A 2-month child has been diagnosed with mucoviscidosis, of a mixed form, which was genetically confirmed. The proband and the father were found to be heterozygotes for the F508del mutation of the CFTR gene (the father suffers from mucoviscidosis). Congenital dysfunction of the adrenal glands, a viral form, was diagnosed when he was three years old. The child is currently receiving: Creon 100 000 units per day with eating, Colomycin 1 vial per day, Pulmozyme 2.5 mg/2.5 ml daily in the morning for inhalations, Ursofalk 600 mg every day constantly, Hydrocortisone 50 mg/day. CONCLUSION: Conclusions: This clinical case can be attributed to rare, as most such pathological conditions are usually diagnosed in maternity homes along with the prescription of appropriate therapy. This is an example of late diagnosis of the viral form of congenital adrenal dysfunction against the background of cystic fibrosis, indicating the need for earlier detection and timely introduction of substitution therapy to improve favourable prognosis for a disease.


Assuntos
Hiperplasia Suprarrenal Congênita , Fibrose Cística , Glândulas Suprarrenais , Córtex Cerebral , Criança , Pré-Escolar , Comorbidade , Fibrose Cística/complicações , Fibrose Cística/tratamento farmacológico , Regulador de Condutância Transmembrana em Fibrose Cística/genética , Feminino , Humanos , Masculino , Gravidez
2.
Cochrane Database Syst Rev ; 10: CD004730, 2020 10 19.
Artigo em Inglês | MEDLINE | ID: mdl-33075159

RESUMO

BACKGROUND: The Cystic Fibrosis Foundation recommends both short-term and long-acting insulin therapy when cystic fibrosis-related diabetes (CFRD) has been diagnosed. Diagnosis is based on: an elevated fasting blood glucose level greater than 6.94 mmol/L (125 mg/dL); or oral glucose tolerance tests greater than 11.11 mmol/L (200 mg/dL) at two hours; or symptomatic diabetes for random glucose levels greater than 11.11 mmol/L (200 mg/dL); or glycated hemoglobin levels of at least 6.5%. This is an update of a previously published review. OBJECTIVES: To establish the effectiveness of insulin and oral agents for managing diabetes in people with cystic fibrosis in relation to blood sugar levels, lung function and weight management. SEARCH METHODS: We searched the Cochrane Cystic Fibrosis and Genetic Disorders Group's Trials Register comprising references identified from comprehensive electronic database searches and handsearches of relevant journals and abstract books of conference proceedings. We also handsearched abstracts from pulmonary symposia and the North American Cystic Fibrosis Conferences. Date of most recent register search: 10 September 2020. We searched online trials registries; date of most recent searches: 21 March 2020. SELECTION CRITERIA: Randomized controlled trials comparing all methods of pharmacological diabetes therapy in people with diagnosed CFRD. DATA COLLECTION AND ANALYSIS: Two authors independently extracted data and assessed the risk of bias in the included studies. Authors also used GRADE to assess the quality of the evidence. MAIN RESULTS: The searches identified 29 trials (45 references). Four included trials provide results: one short-term single-center cross-over trial (seven adults) comparing insulin with oral repaglinide and no medication in adults with CFRD and normal fasting glucose; one long-term multicenter trial (61 adults with CFRD) comparing insulin with oral repaglinide and placebo; one long-term multicenter trial (67 adults) comparing insulin with oral repaglinide; and one 12-week single-center cross-over trial (20 adults) comparing the long-acting insulin glargine to short-term neutral protamine Hagedorn insulin. Two ongoing trials of newly approved incretin mimics have been noted for possible future inclusion. Downgrading of the quality of the evidence was mainly due to risks of bias across all domains, but particularly due to concerns surrounding allocation concealment and selective reporting. There were also some concerns due to imprecision from small sample sizes and low event rates. Finally, there may be some bias due to the amounts of insulin and repaglinide given not being comparable. Data from one trial comparing insulin to placebo (39 participants) did not show any difference between groups for the primary outcomes of blood glucose levels (very low-quality evidence), lung function (low-quality evidence) or nutritional status (low-quality evidence). Similarly, no differences between groups were seen for the secondary outcomes of number of hypoglycemic episodes (low-quality evidence), secondary infection complications or quality of life (QoL). These results were mirrored in the narrative reports for the second trial in this comparison (seven participants). Data from the one-year trial comparing repaglinide to placebo (38 participants), showed no differences between groups for the primary outcomes of blood glucose levels (very low-quality evidence), lung function (low-quality evidence) and nutritional status (low-quality evidence). Also, no differences were seen between groups for the secondary outcomes of number of hypoglycemic episodes (low-quality evidence), secondary infection complications or QoL. These findings were mirrored in the narrative reports for the second trial (n = 7) in this comparison. Three trials compared insulin to repaglinide (119 participants). Data from one trial (n = 67) showed no difference in blood glucose levels at either 12 months (high-quality evidence) or 24 months; narrative reports from one trial (45 participants) reported no difference between groups, but the second trial (7 participants) reported a beneficial effect of insulin over repaglinide. Two trials (112 participants) found no difference between insulin and repaglinide in lung function or nutritional status (moderate-quality evidence). Two trials (56 participants) reported no difference in the number of hypoglycemic episodes (low-quality evidence). One trial (45 participants) reported no difference between groups in secondary infections and cystic fibrosis QoL. The single trial comparing glargine to neutral protamine Hagedorn insulin did not report directly on the review's primary outcomes, but did report no differences between groups in post-prandial glucose values and weight; neither group reported infectious complications. There was no difference in episodes of hypoglycemia (very low-quality evidence) and while there was no difference reported in QoL, all participants opted to continue treatment with glargine after the trial was completed. Mortality was not reported by any trial in any comparison, but death was not given as a reason for withdrawal in any trial. AUTHORS' CONCLUSIONS: This review has not found any conclusive evidence that any agent has a distinct advantage over another in controlling hyperglycemia or the clinical outcomes associated with CFRD. Given the treatment burden already experienced by people with cystic fibrosis, oral therapy may be a viable treatment option. While some cystic fibrosis centers use oral medications to help control diabetes, the Cystic Fibrosis Foundation (USA) clinical practice guidelines support the use of insulin therapy and this remains the most widely-used treatment method. Randomized controlled trials specifically related to controlling diabetes and its impact on the course of pulmonary disease process in cystic fibrosis continue to be a high priority. Specifically, investigators should evaluate adherence to different therapies and also whether there is benefit in using additional hypoglycemic agents as well as the newly approved incretin mimics. Agents that potentiate insulin action, especially agents with additional anti-inflammatory potential should also be further investigated as adjuvant therapy to insulin.


Assuntos
Fibrose Cística/complicações , Diabetes Mellitus/tratamento farmacológico , Hipoglicemiantes/administração & dosagem , Insulina/administração & dosagem , Administração Oral , Viés , Glicemia/análise , Carbamatos/administração & dosagem , Fibrose Cística/sangue , Diabetes Mellitus/sangue , Diabetes Mellitus/etiologia , Jejum/sangue , Humanos , Hiperglicemia/tratamento farmacológico , Insulina Glargina/administração & dosagem , Insulina Isófana/administração & dosagem , Piperidinas/administração & dosagem , Ensaios Clínicos Controlados Aleatórios como Assunto
3.
PLoS One ; 15(9): e0239658, 2020.
Artigo em Inglês | MEDLINE | ID: mdl-32970760

RESUMO

BACKGROUND: Nebulization of antimicrobial drugs such as tobramycin and colistin is a cornerstone in the treatment of patients with cystic fibrosis (CF) infected with Pseudomonas aeruginosa. However, nebulization has a high treatment burden. The Twincer™ is a dry powder inhaler specifically developed for the inhalation of antibiotics such as colistin. The aim of this study was to compare patient outcomes and experience with colistin dry powder by the Twincer with nebulization of colistin or tobramycin in adult CF patients in a real-life setting. METHODS: This was a retrospective study from 01-01-2015 until 01-07-2018. Effectiveness was evaluated by comparing FEV1 decline and exacerbation rate during a mean of 4.1 years of nebulization therapy prior to the initiation of the Twincer against the same values during a mean of 1.7 years of treatment with the Twincer. RESULTS: Twenty-one patients were evaluated, of whom twelve could be included in the effectiveness analysis, with a total of twenty patient years. Of all patients 71.4% preferred therapy with the Twincer over nebulization. Twincer use resulted in high treatment adherence with an average adherence rate of 92.5%. There was no significant difference in annual decline in FEV1%pred prior to and after start changing from nebulization to the use of the Twincer powder inhaler (median decline -1.56 [-5.57-5.31] and 1.35 [-8.45-6.36]) respectively, p = 0.45 (linear mixed effect model)). No significant difference was found in the number of intravenous or combined total intravenous and oral antibiotic courses during Twincer therapy compared to when using nebulization (1.68 and 2.49 courses during Twincer therapy versus 1.51 and 2.94 courses during nebulization, p = 0.88 and p = 0.63). CONCLUSION: Colistin dry powder inhalation with the Twincer is a more patient friendly alternative to nebulization, and we did not observe significant differences in the clinical outcome, regarding lung function and exacerbation rates.


Assuntos
Antibacterianos/administração & dosagem , Colistina/administração & dosagem , Fibrose Cística/microbiologia , Nebulizadores e Vaporizadores/normas , Infecções por Pseudomonas/tratamento farmacológico , Administração por Inalação , Adolescente , Adulto , Antibacterianos/uso terapêutico , Colistina/uso terapêutico , Fibrose Cística/complicações , Feminino , Humanos , Masculino , Pessoa de Meia-Idade , Infecções por Pseudomonas/complicações
4.
Zhonghua Er Ke Za Zhi ; 58(8): 646-652, 2020 Aug 02.
Artigo em Chinês | MEDLINE | ID: mdl-32842385

RESUMO

Objective: To analyze the clinical features of cystic fibrosis (CF) associated allergic bronchopulmonary aspergillosis (ABPA) in children. Methods: A retrospective study was performed in 22 children who were diagnosed with CF associated ABPA in Beijing Children's Hospital affiliated to Capital Medical University from March 2010 to March 2020. The clinical features, imaging characteristics, laboratory results and the prognosis were reviewed. Results: A total of 22 cases met the diagnostic criterion, including 12 males and 10 females. The age of diagnosis was (10.4±2.8) years and the age of onset was (5.5±4.4) years. Clinical manifestations included cough and expectoration (22 cases), recurrent wheezing (15 cases), hemoptysis (7 cases), failure to thrive (12 cases), pancreatitis (10 cases), hepatomegaly (7 cases), splenomegaly (4 cases) and steatorrhea (4 cases). CT scans of all the patients showed pulmonary infiltrates and central bronchiectasis, combined with mucoid impaction in 17 cases and high density mucus plug in 12 cases. Eosinophilia was found in 18 patients. Total IgE and serum levels of A. fumigatus-specific IgE were elevated in all 22 patients. Positive culture of sputum or bronchoalvedar lauage fluid for fungus were in 15 cases, with single Aspergillus infection in 8 cases and mixed Aspergillus infection in 3 cases. The predominant bacteria found in the airways were Pseudomonas aeruginosa (17 cases), followed by staphylococcas. aureus (6 cases) and stenotrophomonas. maltophilia (5 cases). Pulmonary function revealed obstructive ventilation dysfunction in 4 cases, mixed dysfunction in 11 cases, and small airway dysfunction in 4 cases. Regarding the treatment, 3 were treated only with systemic corticosteroid, while the remaining 19 cases also received antifungal agents.The follow up continued for 1-7 years, and 6 maintained remission, 10 had recurrent episodes, 1 died, and 5 lost to follow up. Conclusions: CF associated ABPA is extremely rare in China. The overlapping clinical, radiographic, and immunologic features of these two diseases make the diagnosis challenging. Systemic corticosteroids are considered the first-line therapy for these patients, and adjuvant antifungal agents may be helpful. Recurrence rate in our center is high.


Assuntos
Aspergilose Broncopulmonar Alérgica/complicações , Aspergillus fumigatus/isolamento & purificação , Fibrose Cística/complicações , Escarro/microbiologia , Aspergilose Broncopulmonar Alérgica/tratamento farmacológico , Criança , China , Fibrose Cística/diagnóstico , Fibrose Cística/microbiologia , Feminino , Glucocorticoides/uso terapêutico , Humanos , Masculino , Estudos Retrospectivos , Resultado do Tratamento
5.
Radiologe ; 60(9): 774-780, 2020 Sep.
Artigo em Alemão | MEDLINE | ID: mdl-32761355

RESUMO

Although cystic fibrosis (CF) is a multiorgan disease, the extent of CF lung disease is decisive for the course and survival of patients. The optimization of symptomatic therapies has led to a significant improvement in the life expectancy of those affected in recent decades. Regular monitoring of the course of CF lung disease with microbiological, pulmonary function, and imaging examinations is essential for early detection of problems and individualized therapy. With new, causal therapy options in the form of cystic fibrosis transmembrane conductance regulator (CFTR) modulators and early diagnosis through newborn screening, a further normalization of life expectancy and quality of life of CF patients can be expected.


Assuntos
Fibrose Cística , Pneumopatias , Fibrose Cística/complicações , Regulador de Condutância Transmembrana em Fibrose Cística , Humanos , Pulmão , Pneumopatias/diagnóstico , Pneumopatias/etiologia , Qualidade de Vida
6.
Radiologe ; 60(9): 781-790, 2020 Sep.
Artigo em Alemão | MEDLINE | ID: mdl-32776239

RESUMO

Cystic fibrosis (CF) is the most common fatal autosomal recessive disease in the Caucasian population. A mutation in the cystic fibrosis transmembrane regulator protein (CFTR) gene leads to the production of abnormally viscous mucus and secretions in the lungs of these patients. A similar pathology also occurs in other organs. In the abdomen, among others the gastrointestinal tract, the pancreas, and the hepatobiliary system are affected. The involvement of the pancreas leads to its exocrine and endocrine insufficiency. Hepatic manifestations include hepatic steatosis, focal biliary and multilobular cirrhosis, and portal hypertension. Biliary complications include cholelithiasis, microgallbladder, and sclerosing cholangitis. In the gastrointestinal tract, complications such as the distal intestinal obstruction syndrome, invaginations, chronic constipation, wall thickening, and fibrosis in the colon may occur. An important renal manifestation is nephrolithiasis. With currently rapidly increasing life expectancy of patients with cystic fibrosis, complications of extrapulmonary cystic fibrosis manifestations including hepatic and gastrointestinal malignancy could be an increasing cause of morbidity and mortality of these patients. It is therefore important for radiologists to know and recognize these clinical patterns and to monitor these manifestations in follow-up exams. Previous therapy of extrapulmonary manifestations has been largely symptomatic. Fortunately, the new CFTR modulators seem to represent an effective causal therapeutic approach here.


Assuntos
Fibrose Cística , Obstrução Intestinal , Abdome/patologia , Fibrose Cística/complicações , Humanos , Obstrução Intestinal/etiologia , Pulmão , Pâncreas
7.
Radiologe ; 60(9): 823-830, 2020 Sep.
Artigo em Alemão | MEDLINE | ID: mdl-32776240

RESUMO

CLINICAL/METHODOLOGICAL ISSUE: The differentiated assessment of respiratory mechanics, gas exchange and pulmonary circulation, as well as structural impairment of the lung are essential for the treatment of patients with cystic fibrosis (CF). Clinical lung function measurements are often not sufficiently specific and are often difficult to perform. STANDARD RADIOLOGICAL METHODS: The standard procedures for pulmonary imaging are chest X­ray and computed tomography (CT) for assessing lung morphology. In more recent studies, an increasing number of centers are using magnetic resonance imaging (MRI) to assess lung structure and function. However, functional imaging is currently limited to specialized centers. METHODOLOGICAL INNOVATIONS: In patients with CF, studies showed that MRI with hyperpolarized gases and Fourier decomposition/matrix pencil MRI (FD/MP-MRI) are feasible for assessing pulmonary ventilation. For pulmonary perfusion, dynamic contrast-enhanced MRI (DCE-MRI) or contrast-free methods, e.g., FD-MRI, can be used. PERFORMANCE: Functional MRI provides more accurate insight into the pathophysiology of pulmonary function at the regional level. Advantages of MRI over X­ray are its lack of ionizing radiation, the large number of lung function parameters that can be extracted using different contrast mechanisms, and ability to be used repeatedly over time. ACHIEVEMENTS: Early assessment of lung function impairment is needed as the structural changes usually occur later in the course of the disease. However, sufficient experience in clinical application exist only for certain functional lung MRI procedures. PRACTICAL RECOMMENDATIONS: Clinical application of the aforementioned techniques, except for DCE-MRI, should be restricted to scientific studies.


Assuntos
Fibrose Cística , Pulmão , Imagem por Ressonância Magnética , Meios de Contraste , Fibrose Cística/complicações , Fibrose Cística/diagnóstico por imagem , Humanos , Pulmão/diagnóstico por imagem , Pulmão/fisiopatologia , Ventilação Pulmonar
8.
Radiologe ; 60(9): 791-801, 2020 Sep.
Artigo em Alemão | MEDLINE | ID: mdl-32621155

RESUMO

With its high detail of morphological changes in lung parenchyma and airways as well as the possibilities for three-dimensional reconstruction, computed tomography (CT) represents a solid tool for the diagnosis and follow-up in patients suffering from cystic fibrosis (CF). Guidelines for standardized CT image acquisition in CF patients are still missing. In the mostly younger CF patients, an important issue is the well-considered use of radiation in CT imaging. The use of intravenous contrast agent is mainly restricted to acute emergency diagnostics. Typical morphological findings in CF lung disease are bronchiectasis, mucus plugging, or signs of decreased ventilation (air trapping) which can be detected with CT even in early stages. Various scoring systems that have become established over time are used to grade disease severity and for structured follow-up, e.g., in clinical research studies. With the technical development of CT, a number of postprocessing software tools were developed to help clinical reporting and overcome interreader differences for a standardized quantification. As an imaging modality free of ionizing radiation, magnetic resonance imaging (MRI) is becoming increasingly important in the diagnosis and follow-up of CF patients and is already frequently a substitute for CT for long-term follow-up at numerous specialized centers.


Assuntos
Fibrose Cística , Pulmão , Tomografia Computadorizada por Raios X , Meios de Contraste , Fibrose Cística/complicações , Fibrose Cística/diagnóstico por imagem , Humanos , Pulmão/diagnóstico por imagem , Imagem por Ressonância Magnética
9.
Radiologe ; 60(9): 813-822, 2020 Sep.
Artigo em Alemão | MEDLINE | ID: mdl-32728856

RESUMO

CLINICAL ISSUE: Disease severity and mortality in patients with cystic fibrosis (CF) is mainly determined by (progressive) pulmonary lung disease. Early diagnosis and therapy are important and of prognostic value to conserve lung function. STANDARD RADIOLOGICAL METHODS: Primary imaging techniques for lung imaging are x­ray and computed tomography (CT) to monitor disease severity and regional distribution. METHODICAL INNOVATIONS: Radiation-free imaging techniques such as magnetic resonance imaging (MRI) have gained interest over the last decade in order to prevent radiation damage. PERFORMANCE: The main findings of CF lung disease are airway wall thickening, bronchiectasis, and mucus plugging, which are found in up to 60% of preschool age children. Pleural abnormalities and consolidations are often associated with pulmonary exacerbation. Young CF patients often show a mosaic pattern as functional changes and also perfusion defects can be seen from birth in 50% of CF patients by contrast-enhanced perfusion imaging, and in up to 90% of adult patients, with varying degrees of severity. Dilated bronchial arteries indicate an increased risk for hemoptysis. ACHIEVEMENTS: Proton MRI is the sole imaging technique that can show structural and functional lung changes in one examination. Structured assessment using a scoring system helps to systematically grade the extent and severity of all CF-associated changes. CONCLUSIONS: Lung MRI for cystic fibrosis has been recently established as a clinical standard examination and is routinely performed at experienced centers. More recently, it has also been used as an endpoint within the framework of clinical studies.


Assuntos
Fibrose Cística , Pulmão , Imagem por Ressonância Magnética , Adulto , Criança , Pré-Escolar , Fibrose Cística/complicações , Fibrose Cística/diagnóstico por imagem , Diagnóstico Precoce , Humanos , Pulmão/diagnóstico por imagem , Tomografia Computadorizada por Raios X
10.
Cochrane Database Syst Rev ; 7: CD008037, 2020 07 16.
Artigo em Inglês | MEDLINE | ID: mdl-32671834

RESUMO

BACKGROUND: Cystic fibrosis is a multi-system disease characterised by the production of thick secretions causing recurrent pulmonary infection, often with unusual bacteria. This leads to lung destruction and eventually death through respiratory failure. There are no antibiotics in development that exert a new mode of action and many of the current antibiotics are ineffective in eradicating the bacteria once chronic infection is established. Antibiotic adjuvants - therapies that act by rendering the organism more susceptible to attack by antibiotics or the host immune system, by rendering it less virulent or killing it by other means, would be a significant therapeutic advance. This is an update of a previously published review. OBJECTIVES: To determine if antibiotic adjuvants improve clinical and microbiological outcome of pulmonary infection in people with cystic fibrosis. SEARCH METHODS: We searched the Cystic Fibrosis Trials Register which is compiled from database searches, hand searches of appropriate journals and conference proceedings. Date of most recent search: 16 January 2020. We also searched MEDLINE (all years) on 14 February 2019 and ongoing trials registers on 06 April 2020. SELECTION CRITERIA: Randomised controlled trials and quasi-randomised controlled trials of a therapy exerting an antibiotic adjuvant mechanism of action compared to placebo or no therapy for people with cystic fibrosis. DATA COLLECTION AND ANALYSIS: Two of the authors independently assessed and extracted data from identified trials. MAIN RESULTS: We identified 42 trials of which eight (350 participants) that examined antibiotic adjuvant therapies are included. Two further trials are ongoing and five are awaiting classification. The included trials assessed ß-carotene (one trial, 24 participants), garlic (one trial, 34 participants), KB001-A (a monoclonal antibody) (two trials, 196 participants), nitric oxide (two trials, 30 participants) and zinc supplementation (two trials, 66 participants). The zinc trials recruited children only, whereas the remaining trials recruited both adults and children. Three trials were located in Europe, one in Asia and four in the USA. Three of the interventions measured our primary outcome of pulmonary exacerbations (ß-carotene, mean difference (MD) -8.00 (95% confidence interval (CI) -18.78 to 2.78); KB001-A, risk ratio (RR) 0.25 (95% CI 0.03 to 2.40); zinc supplementation, RR 1.85 (95% CI 0.65 to 5.26). ß-carotene and KB001-A may make little or no difference to the number of exacerbations experienced (low-quality evidence); whereas, given the moderate-quality evidence we found that zinc probably makes no difference to this outcome. Respiratory function was measured in all of the included trials. ß-carotene and nitric oxide may make little or no difference to forced expiratory volume in one second (FEV1) (low-quality evidence), whilst garlic probably makes little or no difference to FEV1 (moderate-quality evidence). It is uncertain whether zinc or KB001-A improve FEV1 as the certainty of this evidence is very low. Few adverse events were seen across all of the different interventions and the adverse events that were reported were mild or not treatment-related (quality of the evidence ranged from very low to moderate). One of the trials (169 participants) comparing KB001-A and placebo, reported on the time to the next course of antibiotics; results showed there is probably no difference between groups, HR 1.00 (95% CI 0.69 to 1.45) (moderate-quality evidence). Quality of life was only reported in the two KB001-A trials, which demonstrated that there may be little or no difference between KB001-A and placebo (low-quality evidence). Sputum microbiology was measured and reported for the trials of KB001-A and nitric oxide (four trials). There was very low-quality evidence of a numerical reduction in Pseudomonas aeruginosa density with KB001-A, but it was not significant. The two trials looking at the effects of nitric oxide reported significant reductions in Staphylococcus aureus and near-significant reductions in Pseudomonas aeruginosa, but the quality of this evidence is again very low. AUTHORS' CONCLUSIONS: We could not identify an antibiotic adjuvant therapy that we could recommend for treating of lung infection in people with cystic fibrosis. The emergence of increasingly resistant bacteria makes the reliance on antibiotics alone challenging for cystic fibrosis teams. There is a need to explore alternative strategies, such as the use of adjuvant therapies. Further research is required to provide future therapeutic options.


Assuntos
Antibacterianos/administração & dosagem , Infecções Bacterianas/tratamento farmacológico , Fibrose Cística/complicações , Pneumopatias/tratamento farmacológico , Adolescente , Adulto , Anticorpos Monoclonais/uso terapêutico , Infecções Bacterianas/microbiologia , Quimioterapia Adjuvante , Criança , Progressão da Doença , Alho , Humanos , Fragmentos Fab das Imunoglobulinas/uso terapêutico , Pneumopatias/microbiologia , Óxido Nítrico/uso terapêutico , Infecções por Pseudomonas/tratamento farmacológico , Pseudomonas aeruginosa , Ensaios Clínicos Controlados Aleatórios como Assunto , Vitaminas/uso terapêutico , Adulto Jovem , Zinco/administração & dosagem , beta Caroteno/uso terapêutico
11.
Genes Immun ; 21(4): 260-262, 2020 08.
Artigo em Inglês | MEDLINE | ID: mdl-32606316

RESUMO

Cystic fibrosis (CF) is one of the most common autosomal recessive life-limiting conditions affecting Caucasians. The resulting defect in the cystic fibrosis transmembrane conductance regulator protein (CFTR) results in defective chloride and bicarbonate secretion, as well as dysregulation of epithelial sodium channels (ENaC). These changes bring about defective mucociliary clearance, reduced airway surface liquid and an exaggerated proinflammatory response driven, in part, by infection. In this short article we explore the overlap in the pathophysiology of CF and COVID-19 infection and discuss how understanding the interaction between both diseases may shed light on future treatments.


Assuntos
Infecções por Coronavirus/metabolismo , Fibrose Cística/metabolismo , Pneumonia Viral/metabolismo , Animais , Infecções por Coronavirus/complicações , Infecções por Coronavirus/tratamento farmacológico , Infecções por Coronavirus/virologia , Fibrose Cística/complicações , Fibrose Cística/tratamento farmacológico , Regulador de Condutância Transmembrana em Fibrose Cística/metabolismo , Citocinas/metabolismo , Humanos , Pandemias , Peptidil Dipeptidase A/metabolismo , Pneumonia Viral/complicações , Pneumonia Viral/tratamento farmacológico , Pneumonia Viral/virologia
13.
Acta Gastroenterol Belg ; 83(2): 315-318, 2020.
Artigo em Inglês | MEDLINE | ID: mdl-32603052

RESUMO

Most episodes of vomiting, reduced intake and diarrhoea in children can be evaluated and treated without additional tests. However, when the degree of clinical dehydration is not in line with the patient's medical history, other diagnoses should be suspected. In the presence of a hyponatraemic hypochloraemic metabolic alkalosis, cystic fibrosis (CF) should be included in the differential diagnosis, especially if there is failure to thrive even in the absence of respiratory symptoms. Furthermore, young patients diagnosed with CF have a higher risk for an acute electrolyte decompensation caused by increased salt and fluid losses. We present 4 paediatric cases to raise the awareness of electrolyte disturbances in CF patients.


Assuntos
Alcalose , Fibrose Cística , Desidratação , Hiponatremia , Criança , Fibrose Cística/complicações , Desidratação/complicações , Insuficiência de Crescimento , Humanos , Hiponatremia/etiologia , Vômito
14.
Pediatr Pulmonol ; 55(8): 2128-2134, 2020 08.
Artigo em Inglês | MEDLINE | ID: mdl-32530552

RESUMO

BACKGROUND: We aimed to evaluate anxiety among children with cystic fibrosis (CF) and their mothers related to the COVID-19 pandemic. METHODS: A total of 45 patients with CF and their mothers were enrolled in the study together with 90 age-matched healthy children and their mothers as a control group. The State and Trait Anxiety Inventory (STAI) was administered by teleconference with children aged 13 to 18 years old and their mothers. The STAI for children was administered with children aged 9 to 12 years. Results were compared with age-matched healthy children and their mothers. The relationship between anxiety scores of children with CF and their mothers was evaluated by comparing with clinical data of children with CF. At the conclusion of the teleconference, mothers were asked whether their anxiety had changed as a result of the interview. RESULTS: It was found that healthy children aged 13 to 18 years had higher state anxiety scores than age-matched children with CF. Mothers of children with CF had higher trait anxiety scores, especially those of children aged 0 to 12 years, than mothers of healthy children (P < .05). For mothers of children with CF, state anxiety scores were higher among those whose children had chronic Pseudomonas infection (P < .05). Most mothers of children with CF stated that their anxiety decreased following the interview. CONCLUSION: The COVID-19 pandemic may increase anxiety among mothers of children with CF as well those with healthy children. However, COVID-19 had no effect on the anxiety of children with CF. Informing parents of children with CF about COVID-19 by teleconference may decrease anxiety.


Assuntos
Ansiedade/complicações , Infecções por Coronavirus/complicações , Infecções por Coronavirus/psicologia , Fibrose Cística/complicações , Fibrose Cística/psicologia , Mães , Pneumonia Viral/complicações , Pneumonia Viral/psicologia , Adolescente , Ansiedade/diagnóstico , Ansiedade/terapia , Betacoronavirus , Estudos de Casos e Controles , Criança , Pré-Escolar , Depressão , Feminino , Humanos , Lactente , Recém-Nascido , Masculino , Pandemias , Infecções por Pseudomonas/complicações , Infecções por Pseudomonas/psicologia , Inquéritos e Questionários , Telecomunicações
16.
Cochrane Database Syst Rev ; 6: CD008482, 2020 06 04.
Artigo em Inglês | MEDLINE | ID: mdl-32497260

RESUMO

BACKGROUND: Malabsorption and deficiency of fat-soluble vitamins K may occur in cystic fibrosis, a genetic disorder affecting multiple organs. Vitamin K is known to play an important role in both blood coagulation and bone formation, hence the role of supplementation of vitamin K in this category needs to be reviewed. This is an updated version of the review. OBJECTIVES: To assess the effects of vitamin K supplementation in people with cystic fibrosis and to investigate the hypotheses that vitamin K will decrease deficiency-related coagulopathy, increase bone mineral density, decrease risk of fractures and improve quality of life in people with CF. Also to determine the optimal dose and route of administration of vitamin K for people with CF (for both routine and therapeutic use). SEARCH METHODS: We searched the Cochrane Cystic Fibrosis and Genetic Disorders Group's Trials Register comprising references identified from comprehensive electronic database searches and handsearches of relevant journals and abstract books of conference proceedings. Most recent search: 12 August 2019. SELECTION CRITERIA: Randomised controlled trials of all preparations of vitamin K used as a supplement compared to either no supplementation (or placebo) at any dose or route and for any duration, in patients with cystic fibrosis. DATA COLLECTION AND ANALYSIS: Two authors independently screened papers, extracted trial details and assessed their risk of bias. The quality of the evidence was assessed using the GRADE criteria. MAIN RESULTS: Three trials (total 70 participants, aged 8 to 46 years) assessed as having a moderate risk of bias were included. One trial compared vitamin K to placebo, a second to no supplementation and the third compared two doses of vitamin K. No trial in either comparison reported our primary outcomes of coagulation and quality of life or the secondary outcomes of nutritional parameters and adverse events. Vitamin K versus control Two trials compared vitamin K to control, but data were not available for analysis. One 12-month trial (n = 38) compared 10 mg vitamin K daily or placebo in a parallel design and one trial (n = 18) was of cross-over design with no washout period and compared 5 mg vitamin K/week for four-weeks to no supplementation for four-weeks. Only the 12-month trial reported on the primary outcome of bone formation; we are very uncertain whether vitamin K supplementation has any effect on bone mineral density at the femoral hip or lumbar spine (very low-quality evidence). Both trials reported an increase in serum vitamin K levels and a decrease in undercarboxylated osteocalcin levels. The cross-over trial also reported that levels of proteins induced by vitamin K absence (PIVKA) showed a decrease and a return to normal following supplementation, but due to the very low-quality evidence we are not certain that this is due to the intervention. High-dose versus low-dose vitamin K One parallel trial (n = 14) compared 1 mg vitamin K/day to 5 mg vitamin K/day for four weeks. The trial did report that there did not appear to be any difference in serum undercarboxylated osteocalcin or vitamin K levels (very low-quality evidence). While the trial reported that serum vitamin K levels improved with supplementation, there was no difference between the high-dose and low-dose groups. AUTHORS' CONCLUSIONS: There is very low-quality evidence of any effect of vitamin K in people with cystic fibrosis. While there is no evidence of harm, until better evidence is available the ongoing recommendations by national CF guidelines should be followed.


Assuntos
Fibrose Cística/sangue , Osteogênese/efeitos dos fármacos , Deficiência de Vitamina K/tratamento farmacológico , Vitamina K/administração & dosagem , Vitaminas/administração & dosagem , Adolescente , Adulto , Biomarcadores/sangue , Coagulação Sanguínea/efeitos dos fármacos , Densidade Óssea , Criança , Fibrose Cística/complicações , Suplementos Nutricionais , Fraturas Ósseas/prevenção & controle , Humanos , Pessoa de Meia-Idade , Osteocalcina/sangue , Precursores de Proteínas/sangue , Protrombina , Qualidade de Vida , Ensaios Clínicos Controlados Aleatórios como Assunto , Vitamina K/sangue , Deficiência de Vitamina K/complicações
17.
PLoS Genet ; 16(6): e1008848, 2020 06.
Artigo em Inglês | MEDLINE | ID: mdl-32530919

RESUMO

Pseudomonas aeruginosa colonizes the airways of cystic fibrosis (CF) patients, causing infections that can last for decades. During the course of these infections, P. aeruginosa undergoes a number of genetic adaptations. One such adaptation is the loss of swimming motility functions. Another involves the formation of the rugose small colony variant (RSCV) phenotype, which is characterized by overproduction of the exopolysaccharides Pel and Psl. Here, we provide evidence that the two adaptations are linked. Using random transposon mutagenesis, we discovered that flagellar mutations are linked to the RSCV phenotype. We found that flagellar mutants overexpressed Pel and Psl in a surface-contact dependent manner. Genetic analyses revealed that flagellar mutants were selected for at high frequencies in biofilms, and that Pel and Psl expression provided the primary fitness benefit in this environment. Suppressor mutagenesis of flagellar RSCVs indicated that Psl overexpression required the mot genes, suggesting that the flagellum stator proteins function in a surface-dependent regulatory pathway for exopolysaccharide biosynthesis. Finally, we identified flagellar mutant RSCVs among CF isolates. The CF environment has long been known to select for flagellar mutants, with the classic interpretation being that the fitness benefit gained relates to an impairment of the host immune system to target a bacterium lacking a flagellum. Our new findings lead us to propose that exopolysaccharide production is a key gain-of-function phenotype that offers a new way to interpret the fitness benefits of these mutations.


Assuntos
Proteínas de Bactérias/genética , Regulação Bacteriana da Expressão Gênica , Interações Hospedeiro-Patógeno/genética , Infecções por Pseudomonas/microbiologia , Pseudomonas aeruginosa/genética , Proteínas de Bactérias/metabolismo , Biofilmes/crescimento & desenvolvimento , Vias Biossintéticas/genética , Fibrose Cística/complicações , Fibrose Cística/microbiologia , Flagelos/metabolismo , Humanos , Mutagênese Sítio-Dirigida , Mutação , Polissacarídeos Bacterianos/biossíntese , Pseudomonas aeruginosa/patogenicidade , Seleção Genética
18.
Radiologe ; 60(9): 831-838, 2020 Sep.
Artigo em Alemão | MEDLINE | ID: mdl-32495009

RESUMO

CLINICAL ISSUE: Abdominal complications are often the first indications for cystic fibrosis (CF), a multiorgan disease. A broad range of abdominal manifestations are associated with the disease, including gastrointestinal abnormalities (such as meconium ileus in newborns and distal intestinal obstruction syndrome in older children) and hepatobiliary alterations (e.g., cholelithiasis, microgallbladder, hepatosteatosis, biliary cirrhosis). A characteristic finding is pancreatic involvement, which leads to exocrine and over the course of time to endocrine insufficiency. STANDARD RADIOLOGICAL METHODS: Ultrasonography is the preferred and often sole modality for a precise diagnosis of abdominal CF manifestations. However, all imaging modalities can be used, depending on the pathology: X­ray, fluoroscopic examinations, computed tomography, magnetic resonance imaging (also with application of magnetic resonance cholangiopancreatography). METHODICAL INNOVATIONS/PERFORMANCE: Scoring systems are useful for standardized diagnostics. Sonographic findings, described using a scoring system, correlate with clinical symptoms, such as pancreatic lipomatosis with abdominal pain (p = 0.018), flatulence (p = 0.006), and gastroesophageal reflux (p = 0.006). EVALUATION/PRACTICAL RECOMMENDATIONS: A standardized approach with structured reporting is important due to the numerous abdominal CF manifestations. To enable precise follow-up analyses, scoring systems based on sonographic findings are excellent.


Assuntos
Abdome , Fibrose Cística , Obstrução Intestinal , Abdome/diagnóstico por imagem , Adolescente , Idoso , Criança , Fibrose Cística/complicações , Fibrose Cística/diagnóstico por imagem , Humanos , Recém-Nascido , Obstrução Intestinal/diagnóstico por imagem , Obstrução Intestinal/etiologia , Pâncreas/diagnóstico por imagem , Tomografia Computadorizada por Raios X
19.
Cochrane Database Syst Rev ; 6: CD009528, 2020 06 10.
Artigo em Inglês | MEDLINE | ID: mdl-32520436

RESUMO

BACKGROUND: Clinicians typically select the antibiotics used to treat pulmonary infections in people with cystic fibrosis based on the results of antimicrobial susceptibility testing performed on bacteria traditionally grown in a planktonic mode (grown in a liquid). However, there is considerable evidence to suggest that Pseudomonas aeruginosa actually grows in a biofilm (or slime layer) in the airways of people with cystic fibrosis with chronic pulmonary infections. Therefore, choosing antibiotics based on biofilm rather than conventional antimicrobial susceptibility testing could potentially improve response to treatment of Pseudomonas aeruginosa in people with cystic fibrosis. This is an update of a previously published Cochrane Review. OBJECTIVES: To compare biofilm antimicrobial susceptibility testing-driven therapy to conventional antimicrobial susceptibility testing-driven therapy in the treatment of Pseudomonas aeruginosa infection in people with cystic fibrosis. SEARCH METHODS: We searched the Cochrane Cystic Fibrosis Trials Register, compiled from electronic database searches and handsearching of journals and conference abstract books. Most recent search: 07 April 2020. We also searched two ongoing trials registries and the reference lists of relevant articles and reviews. Most recent searches: 07 April 2020 and 05 September 2017. SELECTION CRITERIA: Randomized controlled trials (RCTs) of antibiotic therapy based on biofilm antimicrobial susceptibility testing compared to antibiotic therapy based on conventional antimicrobial susceptibility testing in the treatment of Pseudomonas aeruginosa pulmonary infection in people with cystic fibrosis. DATA COLLECTION AND ANALYSIS: Two authors independently selected RCTs, assessed their risk of bias and extracted data from eligible trials. Additionally, the review authors contacted the trial investigators to obtain further information. The quality of the evidence was assessed using the GRADE criteria. MAIN RESULTS: The searches identified two multicentre, double-blind RCTs eligible for inclusion in the review with a total of 78 participants (adults and children); one RCT was undertaken in people who were clinically stable, the second was in people experiencing pulmonary exacerbations. Both RCTs prospectively assessed whether the use of biofilm antimicrobial susceptibility testing improved microbiological and clinical outcomes in participants with cystic fibrosis who were infected with Pseudomonas aeruginosa. The primary outcome was the change in sputum Pseudomonas aeruginosa density from the beginning to the end of antibiotic therapy. Although the intervention was shown to be safe, the data from these two RCTs did not provide evidence that biofilm susceptibility testing was superior to conventional susceptibility testing either in terms of microbiological or lung function outcomes. One of the trials also measured risk and time to subsequent exacerbation as well as quality of life measures and did not demonstrate any difference between groups in these outcomes. Both RCTs had an overall low risk of bias and the quality of the evidence using GRADE criteria was deemed to be moderate to high for the outcomes selected. AUTHORS' CONCLUSIONS: The current evidence is insufficient to recommend choosing antibiotics based on biofilm antimicrobial susceptibility testing rather than conventional antimicrobial susceptibility testing in the treatment of Pseudomonas aeruginosa pulmonary infections in people with cystic fibrosis. Biofilm antimicrobial susceptibility testing may be more appropriate in the development of newer, more effective formulations of drugs which can then be tested in clinical trials.


Assuntos
Antibacterianos/uso terapêutico , Biofilmes/efeitos dos fármacos , Fibrose Cística/complicações , Infecções por Pseudomonas/tratamento farmacológico , Pseudomonas aeruginosa/efeitos dos fármacos , Infecções Respiratórias/tratamento farmacológico , Adolescente , Adulto , Biofilmes/crescimento & desenvolvimento , Feminino , Humanos , Masculino , Testes de Sensibilidade Microbiana/métodos , Infecções por Pseudomonas/complicações , Pseudomonas aeruginosa/fisiologia , Ensaios Clínicos Controlados Aleatórios como Assunto , Infecções Respiratórias/microbiologia , Escarro/microbiologia
20.
Arch Oral Biol ; 116: 104772, 2020 Aug.
Artigo em Inglês | MEDLINE | ID: mdl-32474212

RESUMO

OBJECTIVE: This study aimed at assessing the oral prevalence ofCandida species in cystic fibrosis patients and the antifungal susceptibility of the isolates. DESIGN: One hundred patients aged 3-20 years old were included in the study and were divided into three groups: G1 (low severity disease): 25 cystic fibrosis patients with Shwachman-Kulczycki score (SK) between 100 and 71; G2 (high severity disease): 25 cystic fibrosis patients with SK score under 40; and G3 (control): 50 healthy patients age- and gender-matched to cystic fibrosis patients. Stimulated saliva samples were collected and the oral fungal concentrations were assessed. Isolates were identified by phenotypic and genotypic tests. Antifungal susceptibilities to amphotericin B, flucytosine and fluconazole were determined by CLSI methodology. Fungal counts were compared by Kruskal Wallis and Dunn's test (5%). RESULTS: A total of 68 % of Group 1, 80 % of Group 2, and 44 % of controls yielded positive Candida cultures. Oral concentrations of fungi were significantly higher in cystic fibrosis patients in relation to the control group (p < 0.0005). No significant difference was observed between low and high severity cystic fibrosis groups (p > 0.05). C. albicans was most frequently isolated species in all groups. Higher variability of Candida species was observed in the control group. C. dubliniensis and C. tropicalis were only detected among cystic fibrosis groups. All the isolates were susceptible to flucytosine and fluconazole. CONCLUSIONS: Patients with cystic fibrosis were more frequently colonized by Candida species and showed higher oral fungal burden. No antifungal resistant isolates were detected.


Assuntos
Antifúngicos , Fibrose Cística , Adolescente , Antifúngicos/farmacologia , Antifúngicos/uso terapêutico , Candida/efeitos dos fármacos , Criança , Pré-Escolar , Fibrose Cística/complicações , Fibrose Cística/microbiologia , Farmacorresistência Fúngica/efeitos dos fármacos , Fluconazol , Humanos , Testes de Sensibilidade Microbiana , Prevalência , Adulto Jovem
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