Shwachman-Kulczycki Score Research Articles

Adherence to chest physiotherapy, airway clearance techniques and physical exercise by children and adolescents with cystic fibrosis

Introduction: chest physiotherapy, airway clearance techniques, and physical exercise are associated with better outcomes in cystic fibrosis, but adherence to these recommendations remains a challenge. Objective: to assess adherence to chest physiotherapy, airway clearance techniques, and physical exercise by children and adolescents with cystic fibrosis and analyze the factors related to adherence. Methods: retrospective study of children and adolescents with cystic fibrosis from a pediatric referral center in Brazil. Data were collected from electronic records of the multidisciplinary team responsible for caring for these patients at the referral center. Results: the sample included all 83 patients registered at the referral center, with a mean age of 7.88 ± 4.88 years. Among them, 28.9% did not have weekly chest physiotherapy, 66.3% did not practice physical exercise regularly, and only 38.6% practiced airway clearance techniques from 6 to 7 times a week. Higher adherence to airway clearance techniques was associated with higher mean age (p<0.01) and correlated with FEV1 (r=-0.39; p=0.03), FEF25-75% (r =-0.36; p=0.02), FEV1/FVC (r =-0.43; p=0.01) and lower mean Shwachman-Kulczycki Score (r=-0.34; p<0.01). Higher physical exercise adherence was associated with a higher mean age (p<0.01). Individuals using public financing of physiotherapy services had a lower Shwachman-Kulczycki Score (p=0.02) than those using non-public services. The metropolitan region had a higher percentage of individuals using non-public services than other regions (p<0.01). Conclusion: adherence to chest physiotherapy, airway clearance techniques and physical exercise was lower than recommendations. Airway clearance techniques and physical exercise adherence increased with age, and airway clearance techniques adherence was associated with the high severity of disease. These results suggest that patients only increase adherence when they get older and the disease worsens, highlighting the need for strategies to increase adherence early.

Comparing the Efficacy and Safety of Nebulized Gentamicin Plus Amikacin versus Tobramycin in Patients with Cystic Fibrosis.

Cystic fibrosis is an autosomal recessive disease that causes respiratory tract infection. These patients use nebulized antibiotics such as tobramycin and gentamicin plus amikacin. Due to the high price of tobramycin and the inaccessibility of this drug in Iran at different periods, we aimed to compare the efficacy and safety of nebulized plus amikacin and tobramycin in patients with cystic fibrosis. In this analytic cross-sectional study, data were collected from the records of all patients with cystic fibrosis. They were divided into two groups by their type of nebulized antibiotic. Group 1 included 41 patients who received 80 mg gentamicin and 500 mg amikacin as a nebulized antibiotic every other month, whereas, group 2 consisted of 9 patients who received 300 mg nebulized tobramycin. Collected data were pulmonary function parameters, body mass index, the frequency of hospitalization, infection progress, Shwachman-Kulczycki score, and renal complications. The data were compared in terms of efficacy and renal adverse effects by independent t-test and repeated measure ANOVA. A total of 50 cystic fibrosis patients were evaluated and there was no significant difference between group 1 and group 2 in terms of pulmonary function, frequency of hospitalizations, body mass index, Shwachman-Kulczycki score, infection progress, and renal complications. Notably, pulmonary function factors were reduced in both groups over time during their treatment. Nebulized tobramycin and gentamicin plus amikacin had similar efficacy against Pseudomonas aeruginosa in cystic fibrosis and had no serious renal complications.

Evaluation of the efficiency of nanomicellar formulation of fat-soluble vitamins in patients with cystic fibrosis: the study protocol for a randomized controlled trial

BackgroundCystic fibrosis is an inherited disease, which is caused by the CFTR protein defects due to mutations in the CFTR gene. Along with CFTR dysfunction, exocrine pancreatic insufficiency plays a key role in persistent fat malabsorption in CF patients; therefore, deficiency of fat-soluble vitamins (A, D, E, and K) is still a therapeutic challenge. Even with efficient pancreatic enzyme medication and CF-specific vitamins, many patients with CF have fat-soluble vitamins deficiency. The present study aims to evaluate the efficiency of nanomicelle formulation of fat-soluble vitamins in children with CF in order to achieve the appropriate serum levels of these vitamins.MethodsThis prospective, single-blind control trial will be conducted at the Akbar Children’s Hospital in Mashhad, Iran. Patients with CF will be enrolled based on the eligibility criteria. The control group will receive the standard formulation of fat-soluble vitamins similar to the routine CF treatment, and for the intervention group, the nanomicelle formulation of fat-soluble vitamins will be administered for 3 months. The primary outcome of this study is the measurement of serum levels of fat-soluble vitamins. The secondary outcomes are clinical assessment by the Shwachman-Kulczycki score, anthropometrics, and quality of life. Outcomes will be assessed before and after 3 months.DiscussionDue to persistent fat-soluble vitamin deficiency in CF disease, the nanomicelle formulation could be proposed as a new delivery method of fat-soluble vitamins in the treatment of cystic fibrosis.Trial registrationIranian Registry of Clinical Trials IRCT20220415054541N1. Registered on July 23, 2022.

Neutrophil lymphocyte ratio, mean platelet volume, and immunoreactive trypsinogen as early inflammatory biomarkers for cystic fibrosis in infancy: A retrospective cohort study.

Airway inflammation starts in early life in cystic fibrosis (CF) and limited, objective markers are available to help identify infants with increased inflammation. We aimed to investigate neutrophil, lymphocyte ratio (NLR), mean platelet volume (MPV) and immunoreactive trypsinogen (IRT) to be a possible inflammatory biomarker for CF in infancy. This was a retrospective cohort study in three centers. Between January 2015 and December 2022, children with CF newborn screening (NBS) positivity and diagnosed as CF were included in the study. Correlation analysis were performed with NLR, MPV, IRT and follow-up parameters such as z-scores, modified Shwachman-Kulczycki score (mSKS) at the first, second, third and sixth ages and pulmonary function test (PFT) at the sixth age. A total of 92 children with CF included in the study and 47.8% of them were female. There were no correlations between NLR, MPV and weight and height z-scores for all ages (p > 0.05), a negative correlation was found between MPV and body mass indexes (BMI) z-score at the age of 6 (r = -0.443, p = 0.038). No correlation was found between NLR, MPV and PFT parameters and mSKS at all ages (p > 0.05). There was a negative correlation between first IRT and BMI z-score at 6 years of age (r = -0.381, p = 0.046) and negative correlations between second IRT and weight and BMI z-score at the age of 6 (r = -0.462, p = 0.010; r = -0.437, p = 0.016, respectively). Higher MPV and IRT levels during NBS period are associated with worse nutritional outcome which may reflect chronic inflammation. Children with higher MPV and IRT should be followed up closely in terms of chronic inflammation and nutritional status.

Association between phenotypic and genotypic characteristics and disease severity in individuals with cystic fibrosis.

Objective:To analyze the association between phenotypic and genotypic characteristics and disease severity in individuals with cystic fibrosis treated at a reference center in Minas Gerais, Brazil.Methods:This is a retrospective study that collected clinical and laboratory data, respiratory and gastrointestinal manifestations, type of treatment, Shwachman-Kulczycki score, and mutations from the patients’ medical records.Results:The sample included 50 participants aged one to 33 years, 50% of whom were female. Out of the one hundred alleles of the Cystic Fibrosis Transmembrane Conductance Regulator gene, the most prevalent mutations were DeltaF508 (45%) and S4X (18%). Mutation groups were only associated with pancreatic insufficiency (p=0.013) and not with disease severity (p=0.073). The latter presented an association with colonization by Pseudomonas aeruginosa and Staphylococcus aureus (p=0.007) and with underweight (p=0.036). Death was associated with age at diagnosis (p=0.016), respiratory symptomatology (p=0.013), colonization (p=0.024), underweight (p=0.017), and hospitalization (p=0.003).Conclusions:We could identify the association of mutations with pancreatic insufficiency; the association of Staphylococcus aureus colonization and underweight with disease severity; and the lack of association between mutations and disease severity. Environmental factors should be investigated more thoroughly since they seem to have an important effect on disease severity.

Influência de fatores clínicos e socioeconômicos no estado nutricional de pacientes pediátricos com fibrose cística

Objective: To verify the influence of clinical and socioeconomic factors on the nutritional status of pediatric CF patients treated at a referral care facility in the State of Piauí and to describe their general profile. METHODS: This was an observational cross-sectional retrospective descriptive study with secondary data analysis, performed by data from medical records of 23 patients under 16 years of age with confirmed cystic fibrosis, attended at 2018, were collected. RESULTS: There was a slight predominance of male and brown, homozygous (p=0.05), from southeast Piaui, in school age group. Age at diagnosis was 2.0±3.0 years, with 13% asymptomatic. Respiratory symptoms and growth deficits were the main initial manifestations. 47.8% were diagnosed by neonatal screening. Most had good and excellent score for the Shwachman-Kulczycki score, average of 78.5±16.7. Most were eutrophic, with adequate Z-score values. Influence of nutritional status on the Shwachman-Kulczycki score was noted, and the latter was found to be significantly lower (p=0.037) in patients with nutritional deficit. DISCUSSION: There was a delay in the diagnosis of these patients, with low coverage of neonatal screening tests. The socioeconomic and clinical aspects did not influence the nutritional status, except for the severity score that was worse in those with nutritional deficit. CONCLUSION: Nutritional status influenced the severity score, confirming the importance of adequate management and nutritional monitoring for improving the quality of life.

Evaluation of miR-301b and miR-302b Expression in Serum of Cystic Fibrosis Patients and Association with Clinical Scoring System

Background Cystic fibrosis (CF) is an autosomal recessive disorder caused by a mutation in CF transmembrane conductance regulator gene (CFTR). Clinical manifestations of disease and their severity have considerable variations in patients having similar mutation in CFTR gene. This can be due to different polymorphisms, epigenetic changes and microRNAs (miRNAs) as gene modifiers. Considering the proven roles of miR-301b and miR-302b on infection and inflammation, expression of these miRNAs might change in CF patients. Materials and Methods: In this study, 30 CF patients (homozygous for ΔF508 mutation) and 30 healthy individuals were participated and their demographic data were recorded. The whole RNA was extracted from serum samples and cDNA was synthesized. Using Real-Time PCR, expression levels of miR-301b and miR-302b were measured between patient and normal groups. Patient classification was carried out based on Shwachman-Kulczycki score, and expression levels of these miRNAs were determined in these classifications. All statistical analyses were performed using IBM SPSS software V21. Results: Statistical analyses of qRT-PCR results showed a significant increase in serum levels of miR-301b and miR-302b expression (p-Values of 0.02 and 0.03; fold changes of 3.73 and 1.95, respectively) in CF patients compared to healthy controls. A significant increase (p<0.05) in miR-301b expression level was observed in severe, moderate and mild groups, while miR-302b expression level was increased in CF patients of severe and moderate groups according to Shwachman-Kulczycki score. Conclusion Expression levels of miR-301b and miR-302b are different based on clinical scoring system. This data suggests that expressions of these two miRNAs are influenced by infection and inflammation of CF patients. Further studies can lead to development of innovative treatment strategies.

Ultrasound findings of pubertal development in girls with cystic fibrosis and their association with clinical outcomes and Tanner staging.

Patients with cystic fibrosis (CF) have a high incidence of pubertal and growth delay. In girls with CF, pubertal delay has an important psychological impact. Still, only a few studies have explored the occurrence of pubertal delay in girls with CF. The aims of this study were to compare the pubertal development of girls with CF compared with healthy controls regarding Tanner staging and pelvic ultrasound and, in girls with CF, correlate the findings with those of spirometry, body mass index, Shwachman-Kulczycki score (SKS), and genotyping. This was a cross-sectional, case-control study including 35 girls with CF aged 6-17 years and following up at the Pediatric Pulmonology Outpatient Clinic of a tertiary hospital. These patients were compared with 59 healthy controls who had undergone pelvic ultrasound as part of another study conducted by the same group. Girls with CF were consecutively enrolled in the study during their annual routine check-up visit. Data collected in the CF group included spirometry and anthropometric results, SKS values, bone age, occurrence of current cystic fibrosis-related diabetes (CFRD) and Pseudomonas aeruginosa colonization, history of meconium ileus, genotype, ultrasound parameters, and Tanner stage. Pelvic ultrasound findings and Tanner stage reflected less pubertal development in girls with CF compared with healthy controls. Pubertal stage in patients with CF who presented CFRD (3.17 ± 1.16), had chronic colonization by Pseudomonas aeruginosa (3.10 ± 1.10), or were homozygous for the F508del mutation (1.91 ± 1.30) was more delayed than in controls (3.41 ± 1.41). Tanner stage correlated with age at menarche, bone age, and anthropometric and ultrasound data. Girls with CF presented a delay in pubertal development evaluated by Tanner stage and ultrasound parameters, which was more evident in the presence of comorbidities.

Evaluation of the health-related quality of life in Turkish cystic fibrosis patients.

Prolongation of life expectancy in cystic fibrosis (CF) to the fourth decade makes health-related quality of life (HRQOL) an important issue in this population. This study aims to evaluate the effects of clinical and socio-demographic characteristics of patients on different aspects of HRQOL in a pediatric CF population. We also intend to assess some measures of reliability and validity of the Turkish CF Questionnaire-Revised (CFQ-R) forms. Fifty-four CF patients and 46 parents / caregivers filled the appropriate CFQ-R forms. Disease severity was evaluated via modified Shwachman-Kulczycki (SK) scores, and additional clinical information was obtained from patient records. Forty-three percent (n=23) of patients were female, the mean age of patients being 13.3±3.96years. Self- and proxy-reported points showed a strong correlation (Spearman's rho>0.6) on more observable domains of HRQOL (e.g. physical functioning). No significant difference was found between sexes on any HRQOL domain. When compared between age groups, treatment burden appeared to worsen with age, while social functioning improved. All three forms of the CFQ-R Turkish version showed acceptable internal consistency reliability and convergence with disease severity measures. The CFQ-R showed a fair correlation with the Shwachman-Kulczycki score and the FEV1% value, which makes its longitudinal use possible in clinical practice. It is important to use self-reported evaluations as well as parent / caregiver-reported HRQOL evaluations, especially in young children. More studies are needed to assess the psychometric properties of Turkish CFQ-R forms.

Evaluation of miR-155 Expression in Cystic Fibrosis Patients

Background: Cystic Fibrosis (CF) is the most fatal genetic disorder among white populations. It is a multi-system disease with various symptoms, which causes many different complications. miRNAs are a class of endogenous small non-coding RNAs of 19-22 nt that regulate mRNAs post-transcriptionally. Different types of miRNAs, including miR-155, are expressed in CF lung epithelial cells. The elevated expression level of miR-155 contributes to the pro-inflammatory expression of IL-8 in CF lung epithelial cells. Therefore, miR-155 may play an important role in the activation of IL-8 in CF. Objectives: The present study aimed to investigate the relationship between miR-155 expression level and the clinical manifestations of CF patients. Methods: The participants of this cross-sectional study included 30 CF patients (according to sweat test and &lt; F508 in the genetic study) with age ranging from 5 to 27 years (M = 17, F = 13) and 30 healthy individuals with age ranging from 8 to 28 (M = 17, F = 13). Using RNA purification Kit, microRNAs, we extracted microRNAs from serum samples, and cDNA was synthesized via the cDNA synthesis kit. Then, the levels of miR-155 were measured by real-time PCR, and the expression levels were compared in different groups of CF patients according to the Shwachman-Kulczycki scoring system. Results: The expression level of miR-155 was elevated in CF patients compared to healthy controls (Fold change: 1.41, P value = 0.056). Interestingly, the elevated level of miR-155 in severe and moderate patients (according to Shwachman-Kulczycki score) showed a significant difference compared to the other patients, where age and sex were not an influential factor. Conclusions: Serum expression levels of miR-155 are different in CF patients, along with the severity of the disease. By suppressing the expression of miR-155, more investigations might lead to the development of new treatment strategies for CF.

Evaluation of the psychological status of mothers of children with cystic fibrosis and the relationship between children's clinical status.

In this study, we aimed to determine the psychosocial status of primary care givers of children with cystic fibrosis (CF) and its relationship with the clinical scores of children with CF. Thirty-six patients with CF and their primary care givers were assessed. A personal information form, as well as the Parent Attitude Research Instrument, Maslach Burnout Inventory, Beck Depression Scale and modified Shwachman-Kulczycki Score, were used for data collection. All the CF children's primary care givers were their mothers. The mothers' occupation and educational level affected their child-rearing attitudes (P < 0.05). Furthermore, the frequency of hospital visits and hospitalizations was associated with increased emotional exhaustion, depression and negative attitudes towards child-rearing (P < 0.05). Higher levels of emotional exhaustion in mothers were associated with increased depersonalization and depression, while personal accomplishment was associated with lower levels of depression (P < 0.05). Depression was present in 69.4% of mothers, and its severity was correlated with their children having a poor clinical status. Moreover, increased depression was associated with increased negative attitudes towards child-rearing (P < 0.05). The rate of depression is high in mothers of children with CF. These mothers displayed a negative attitude towards child-rearing, along with the disease, which was more severe in their children. These mothers should undergo routine psychosocial screening, and support should be given to those in need.

Oral prevalence and antifungal susceptibility of Candida species in cystic fibrosis patients

ObjectiveThis study aimed at assessing the oral prevalence ofCandida species in cystic fibrosis patients and the antifungal susceptibility of the isolates. DesignOne 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%). ResultsA 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. ConclusionsPatients with cystic fibrosis were more frequently colonized by Candida species and showed higher oral fungal burden. No antifungal resistant isolates were detected.

Sleep-disordered breathing and markers of morbidity in children and adolescents with cystic fibrosis.

Studies have shown that sleep disorders occur in cystic fibrosis (CF) patients and may be present before daytime clinical manifestations. To evaluate the presence of sleep disorders among children and adolescents with CF, attempting to identify associations with pulmonary function, nutritional status, days in hospital, and days taking antibiotics. Individuals with a diagnosis of CF aged between 6 and 18 years were included. Information on sociodemographic, clinical profile, history of hospitalizations, and use of antibiotics in the last year were collected. Spirometry, bioimpedance, and polysomnography were performed. The presence of nocturnal hypoxemia and obstructive sleep apnea syndrome (OSAS) were evaluated and participants divided according to their presence. Thirty-one patients were included. The prevalence of OSAS was 32.3% and nocturnal hypoxemia was 29.0%. Average nocturnal peripheral oxyhemoglobin saturation (SpO2 ) correlated (P < .001) with forced vital capacity (r = .55) and forced expiratory volume in the first second (r = .62). The higher the percentage of total sleep time (TST) with SpO2 less than 90%, the lower the pulmonary function. Individuals with OSAS and nocturnal hypoxemia had lower spirometric values compared to patients without these disorders, but the nocturnal hypoxemia group also had lower Shwachman-Kulczycki score, longer hospitalization time and antibiotic use. TST with SpO2 less than 90% was associated with length of hospitalization (r2 = .53). Children and adolescents with CF have sleep disorders, including OSAS (32.3%) and nocturnal hypoxemia (29%). Individuals with nocturnal hypoxemia presented lower lung function, worse clinical score, and higher morbidity. TST with SpO2 less than 90% was associated with length of hospitalization.

Upper Airway Findings and Markers of Lung Disease Progression in Patients with Cystic Fibrosis.

Introduction Cystic fibrosis (CF) is a genetic disease that limits the quality of life mainly due to respiratory symptoms. The relationship between findings of the upper airways and CF lung disease is not yet completely understood. Objective The aim of the present study is to describe the most frequent nasal findings and pathogens in patients with CF and investigate the association between the findings of the upper respiratory tract and markers of lung disease progression. Methods Retrospective study in patients with CF from the Pediatric Pulmonology Department who underwent otorhinolaryngological evaluation between 2015 and 2017. Nasal endoscopy and nasal swab collection were part of the evaluation. The severity markers used were: percentage of predicted forced expiratory volume in the first second (FEV1%), body mass index (BMI) and the Shwachman-Kulczycki (SK) clinical score. Results A total of 48 patients with CF were included. The mean of the predicted percentage of FEV1% was 83.36 ± 30.04. The average 14 and SK score 89.11 ± 10.50. The bacteriology of the nasal swab was positive in 27 (54.1%) patients. Staphylococcus aureus was positive in 18 patients, Pseudomonas aeruginosa in 5, Pseudomonas cepacea in 3 and Stenotrophomonas maltophila in 1 patient. Nasal polyps were found in nine participants. Nasal polyps were found in nine participants and were associated with lower SK score. Conclusion The pathogens found in the upper airway were, in order: S. aureus , P. aeruginosa , P. cepacea e S. maltophila . The presence of polyps in the nasal cavity showed statistical significance and appears to have association with the prognostic factor measured by the SK score.

Evaluation of Copeptin during Pulmonary Exacerbation in Cystic Fibrosis.

Copeptin was found to be a stable biomarker of inflammation and stress response in cardiac, renal, metabolic, and respiratory conditions such as pneumonia. The aim of this study was to investigate the copeptin levels in biological fluids (serum and sputum supernatant) of cystic fibrosis pediatric patients during pulmonary exacerbation and remission and to investigate the possible influence of copeptin levels on disease severity and quality of life. Copeptin serum concentrations were measured in 28 pediatric cystic fibrosis (CF) patients: 13 in stable condition and 15 during pulmonary exacerbation. In 10 CF patients, copeptin was also measured in the sputum. In all the patients, we assessed complete blood count, BMI, sputum culture, lung function, and chest imaging (with Brasfield score). The severity of symptoms was assessed using the Shwachman-Kulczycki (SK) score, and the quality of life was assessed with the Cystic Fibrosis Quality of Life Questionnaire-Revised (CFQ-R). Copeptin concentrations in serum and sputum supernatant was measured using an ELISA kit. Statistical analysis was done in Statistica v.12. Serum and sputum copeptin levels were higher in CF patients during pulmonary exacerbation than in a stable period, but the differences were not significant (p = 0.58 and p = 0.13, respectively). Copeptin did not correlate significantly with any clinical, laboratory, or spirometry markers of exacerbation. There was, however, a significant inverse correlation between the serum copeptin level and symptoms severity (r = ‐0.77, p = 0.008) and radiological changes (r = ‐0.5626, p = 0.036) during pulmonary exacerbation in pediatric CF patients. Copeptin also inversely correlated with the quality of life domains in CF patients: vitality and eating habits, mostly loss of appetite (p = 0.031 and p = 0.016, respectively). Copeptin may be useful to identify patients with a higher risk of deterioration to improve their outcomes.

Avaliação da qualidade de vida dos pacientes com fibrose cística atendidos em um hospital de referência de Minas Gerais

OBJETIVO: Avaliar a qualidade de vida (QV) dos pacientes com fibrose cística (FC) acompanhados por um centro de referência de Minas Gerais mediante a aplicação de questionário específico e validado.MÉTODOS: Estudo transversal, de análise quantitativa, com aplicação do questionário de QV em FC, escore de Shwachman-Kulczycki e valores espirométricos. A amostra do estudo foi composta por 26 pacientes com idade de 6 a 29 anos e 13 pais de crianças com idade de 6 a 13 anos. Os participantes foram divididos em três grupos: grupo I (6 a 13 anos), grupo II (pais ou cuidadores de crianças de 6 a 13 anos), grupo III (≥14 anos). Os escores foram apresentados por médias e medianas e analisados por meio do teste de correlação linear de Pearson. Foi adotado o nível de significância de 5%.RESULTADOS: Os pacientes com FC acompanhados neste hospital de referência de Minas Gerais apresentavam boa percepção de QV e escore de Shwachman-Kulczycki excelente. No grupo I, foram encontrados valores menores para o domínio digestório (67,49%) e maiores para o domínio execução de tratamento (83,84%). No grupo II observou-se menores pontuações no domínio peso (65,70%) e maiores na imagem corporal (84,61%). No grupo III, valores menores para o domínio social (71,12%) e maiores para alimentação (92,47%). Com relação aos valores espirométricos, encontrou-se valores significativos correlacionando a relação entre o volume expiratório forçado no primeiro segundo e a capacidade vital forçada (VEF1/CVF) aos domínios físico/percepção da saúde e funcionalidade no grupo III.CONCLUSÕES: Os pacientes com FC acompanhados no centro de referência deste estudo apresentaram boa percepção de QV, o que pode ser verificado, na maioria dos domínios analisados, por escores superiores a 50. O uso de questionários específicos para avaliação de QV é um método de baixo custo e de fácil aplicação, devendo ser verdadeiramente incluído à rotina dos serviços de saúde que atendem a população diagnosticada com FC.

Sinonasal computed tomography in pediatric cystic fibrosis: do we know the indications?

ObjectivesTo investigated the correlation between sinonasal computed tomography (SNCT) findings and sinonasal symptoms, genotype, chronic colonization by Pseudomonas aeruginosa, forced expiratory volume in the first second (FEV1), body mass index (BMI), Shwachman-Kulczycki (SK) score, and Bhalla score in patients with cystic fibrosis. MethodsWe retrospectively reviewed the medical records of 61 patients aged 2–16 years who received care at the Pediatric Cystic Fibrosis Center of our institution over a 10-year period. SNCT findings were graded using the Lund-Mackay scoring system. For patients who had SNCT scans performed at two different time points, the findings from both examinations were compared. ResultsPatients with chronic P. aeruginosa colonization and patients with atelectasis had higher Lund-Mackay scores (p = 0.04 and p = 0.01, respectively). There was no difference in Lund-Mackay scores between sinonasal symptomatic and asymptomatic patients (p = 0.45). Among patients who had two SNCT scans available (n = 11), those with no evidence of bronchiectasis on chest CT had decreased Lund-Mackay scores compared to those with evidence of bronchiectasis, who had an increase in their scores (p = 0.03). Variations in the Bhalla score were positively and variations in the SK score were negatively correlated with variations in the Lund-Mackay score (r = 0.74, p = 0.01; and r = −0.85, p < 0.01). ConclusionsAssociations between SNCT findings, chronic P. aeruginosa colonization, SK score, and chest CT findings were demonstrated. Further studies with larger sample sizes are needed to evaluate patient follow-up and assess the benefits of the sinonasal treatment strategy adopted for patients who exhibit pulmonary deterioration despite controlling for other factors associated with exacerbation.

Perfil dos pacientes com fibrose cística atendidos em um hospital universitário de referência de Minas Gerais/Brasil

Introduo: Os avanos no manejo da fibrose cstica (FC) permitiram um aumento significativo da expectativa de vida dos indivduos fibrocsticos Objetivo: Caracterizar o perfil epidemiolgico e clnico dos pacientes com FC assistidos no ambulatrio de um hospital universitrio de referncia no tratamento da doena. Mtodos: Por meio da consulta aos pronturios dos pacientes em acompanhamento, foi realizada uma pesquisa descritiva, retrospectiva, entre janeiro a agosto de 2015. Resultados: A mdia de idade dos pacientes no perodo da avaliao e a mdia de idade ao diagnstico foram de 10,066,93 e 1,713,28 anos, respectivamente. A maior parte da populao era do sexo feminino, caucasiana, eutrficos, residentes fora do municpio de referncia, possua insuficincia pancretica, apresentava colonizao bacteriana e realizava exerccios respiratrios, conforme orientados no ambulatrio. A mdia do escore de Shwachman-Kulczycki foi de 88,6311,09. Concluso: Este perfil favorvel evidencia que uma boa adeso ao tratamento e a compreenso acerca das orientaes oferecidas pela equipe multiprofissional contriburam para a manuteno do bom estado geral e estabilizao dos sintomas.

Quality of life amongst adolescents and young adults with cystic fibrosis: correlations with clinical outcomes

OBJECTIVES:The current study sought to evaluate the quality of life of young patients with cystic fibrosis and correlate these results with the clinical parameters indicative of disease severity.METHODS:This cross-sectional study applied the validated Portuguese version of a cystic fibrosis specific quality of life questionnaire to clinically stable patients aged 14 to 21 years old. The correlations between the questionnaire domain scores and forced expiratory volume in one second (FEV1) values, the Shwachman-Kulczycki score, and body mass index were assessed, and correlations were considered as significant when p<0.05.RESULTS:A total of 31 patients (11 females; 16.4±2.3 years old) were evaluated, and the median scores on the questionnaire domains ranged from 66.7 to 100. A significant correlation was found between body mass index and the weight (r=0.43, p=0.016) and the eating questionnaire domains (r=0.44, p=0.013); between FEV1 and the physical (r=0.53, p=0.002) and treatment burden (r=0.41, p=0.023) domains; and between the Shwachman-Kulczycki score and the physical (r=0.39, p=0.03), health (r=0.41, p=0.023), and role (r=0.37, p=0.041) domains. A significant difference was found amongst patients with FEV1 values above or below 60% of the predicted value with regard to the role and health domains. No differences in the scores were found according to gender.CONCLUSIONS:The current cystic fibrosis specific quality of life questionnaire scores exhibited wide variability across all domains; however, they indicated a relatively satisfactory quality of life amongst the patients studied. Certain domains exhibited significant correlations with clinical parameters; thus, this instrument has consistent associations with clinical outcomes.

Burkholderia cepacia complex in cystic fibrosis in a Brazilian reference center.

The Burkholderia cepacia complex (BCC) can cause a severe decline in lung function in cystic fibrosis (CF). Our objective was to determine the BCC prevalence and to evaluate its clinical impact on CF. Clinical and laboratory variables were determined for CF patients with BCC (Group-A=50 patients) and without BCC (Group-B=134 patients). The microorganisms were identified by biochemical tests, the Vitek2®Compact test, recA-PCR and recA-nested-PCR with species-specific primers and DNA sequencing. The patients were evaluated by the Shwachman-Kulczycki score (SKCS), Bhalla score (BS), spirometry and body mass index (BMI). The BCC prevalence was 22.5%. The most common species were Burkholderia multivorans (30%), Burkholderia cepacia (24%), Burkholderia cenocepacia IIIA (10%), B. cenocepacia IIIB (2%) and Burkholderia vietnamiensis (2%). There was difference between the groups in nutritional status (p=0.02) and general activity (p=0.026). There was difference in total BS points (p=0.04) and the following parameters: bronchiectasis severity (p=0.007), peribronchial thickening (p=0.013), bronchiectasis extent (p=0.01) and general aspects of the affected bronchial zone (p=0.02). The respiratory disorder classifications were as follows: obstructive-4.8% (Group-A) and 23.8% (Group-B); restrictive-9.5% (Group-A and Group-B); obstructive+restrictive-19% (Group-A) and 1.6% (Group-B); and obstructive+restrictive with a decreased forced expiratory flow-47.6% (Group-A) and 30.2% (Group-B) (p=0.02). Nutritional status was a minor contributing factor to weight, height and BMI in the Group-A (p=0.02). The BCC prevalence, particularly the prevalence of B. multivorans, was higher in this study. The SKCS, BS, spirometry and nutritional status results showed that BCC has a negative impact on clinical status. Phenotypic methods are useful for the identification of presumptive BCC. The Vitek2®Compact test showed accuracy in BCC identification. PCR, nested-PCR, and recA sequencing showed specificity in BCC species identification.