Inhaled antibiotics for the treatment of chronic Pseudomonas aeruginosa infection in cystic fibrosis patients: challenges to treatment adherence and strategies to improve outcomes.

BACKGROUND: Inhaled antibiotics (ABs) are recommended for use in the therapy of chronic Pseudomonas aeruginosa infection in patients with cystic fibrosis (CF). The aim of this systematic literature review was to identify level of adherence to inhaled ABs and to determine predictors and consequences of nonadherence in CF. METHODS: A systematic literature search of English-language articles was conducted in April 2015 using Medline and Embase. No publication date limit was applied. The literature screening was conducted by two independent reviewers. All of the included studies were assessed for quality. RESULTS: The search yielded 193 publications, of which ten met the inclusion criteria and underwent data extraction. Seven studies focused on inhaled tobramycin, one on inhaled colistimethate, one on inhaled levofloxacin, and one on inhaled aztreonam lysine. Medication adherence to inhaled ABs was analyzed by pharmacy refill history, daily phone diary, parent and child self-reports, vials counting, or electronic monitoring. In randomized controlled trials (n=3), proportion of adherent patients (>75%-80% of required doses taken) ranged from 86% to 97%; in prospective cohort studies (n=3), adherence rates ranged between 36% and 92%, and in retrospective studies (n=4) it ranged between 60% and 70%. The adherence to inhaled ABs in CF was found to be associated with the complexity of treatment, time of drug administration, age of patients, treatment burden (adverse events, taste), and patient satisfaction. CONCLUSION: The high diversity of adherence data was because of the different study designs (randomized controlled trials vs real-world studies) and the lack of a commonly accepted consensus on the definition of adherence in the reviewed articles. Routine adherence monitoring during CF care, discussing the possible reasons of suboptimal adherence with the patient, and changing treatment regimens on the basis of patient burden can individualize CF therapy for patients and may improve the level of adherence.

Health Related Quality of Life of Children with Chronic Respiratory Conditions.

BACKGROUND: In the management of chronic diseases, treatment approaches have changed in recent decades. Not only are clinical outcomes assessed but also the patients' perception of their quality of life has become an important aspect. OBJECTIVES: The aim of our study was to compare the health-related quality of life (HRQoL) of children with cystic fibrosis (CF) to the HRQoL of asthmatic patients, to assess the level of agreement between parent proxy-report and child self-report and to measure the relationship between spirometry and HRQoL. MATERIAL AND METHODS: 172 children (mean age: 11.61±2.56 years) and their parents completed the questionnaire. The Hungarian version of the Pediatric Quality of Life InventoryTM 4.0 (PedsQLTM 4.0) Generic Core Scale was used to assess HRQoL. Lung function was assessed via spirometry. RESULTS: Significantly lower PedsQLTM scores were measured for CF patients on the psychosocial health (p<0.05), emotional functioning (p<0.005) and school functioning (p<0.01) subscales and the total scale (p<0.05) from the children's self-report. The level of child-parent agreement was fair and moderate in both patient populations [intra-class correlation coefficient range (ICC) asthma=0.29-0.37; ICCCF=0.39-0.59, p<0.001]. The relationship between forced expiratory volume in 1 second (FEV1) and the physical health subscale (r=0.49, p<0.01) was moderate in young (8-12 years) children and also teenagers (13-18 years), with CF (r=0.58, p<0.05) from self-report. We found weak, non-significant correlations between FEV1 and PedsQLTM subscales in children with asthma (8-12 years) (r=-0.01-0.18, ns.). CONCLUSIONS: Children suffering from CF perceive their HRQoL as poorer than children with asthma. In asthmatic patients, it is not sufficient to evaluate clinical outcomes (FEV1); subjective HRQoL should be also estimated in the course of patient care.

Comparison of health-related quality of life among children with cystic fibrosis and their parents in two Eastern European countries.

BACKGROUND: In the optimal care of cystic fibrosis (CF) patients, not only medical parameters are respected but also health-related quality of life (HRQOL). The aim of our study was to compare HRQOL of CF patients from two Eastern European countries. METHODS: 141 patients with CF (6-18 years) and 102 parents completed the Cystic Fibrosis Questionnaire-Revised (CFQ-R). Data about disease severity, type of children's education and questions about parents' employment status were collected. RESULTS: In the patient group, a significant difference was found only in Treatment burden, whereas in the parent group, there were significant differences in Treatment burden, Emotional functioning, Eating and Digestive symptoms between the two countries. School attendance was revealed as an important factor influencing HRQOL. CONCLUSIONS: Observed differences in evaluation of HRQOL may be caused by different therapeutic and diagnostic challenges between countries. To identify possible presence of psychosocial problems, monitoring of HRQOL is recommended.

Factors influencing quality of life and disease severity in Hungarian children and young adults with cystic fibrosis.

BACKGROUND: The aim of our study was to evaluate factors affecting cystic fibrosis (CF) patients' health-related quality of life (HRQoL) and to assess the level of agreement on HRQol between children and their parents. METHODS: Fifty-nine patients (mean age: 14.03 ± 4.81 years) from 5 Hungarian CF centres completed the survey. HRQoL was measured using The Cystic Fibrosis Questionnaire-Revised (CFQ-R). Parents were asked to fill out a questionnaire about their smoking habits, educational level and history of chronic illness. Disease severity was assessed using the physician-reported Shwachman-Kulczycki (SK) score system. Spirometry, Body Mass Index (BMI) percentile (pc), hospitalisation and Pseudomonas aeruginosa (PA) infection were examined as physiologic parameters of CF, and the impact of these factors on HRQoL was assessed. A multivariate regression analysis was performed to identify the most important factors affecting HRQoL. The level of significance was set to 0.05. RESULTS: Passive smoking and parental educational level and chronic diseases status did not have a significant impact on the patients' HRQoL (p > 0.05). Significantly lower SK scores and spirometry values were found in low BMI pc patients (p < 0.001), in hospitalised (p < 0.01) and in PA-infected patients (p < 0.01), than in the adequate-weight, non-hospitalised and PA culture-negative subgroup. Lower CFQ-R scores were detected in hospitalised patients than in non-hospitalised patients in their Physical functioning domain. PA-infected patients had HRQoL scores that were significantly worse in the Body image (p < 0.01) and Respiratory symptoms (p < 0.05) domains than the PA culture-negative patients. Patients with a low BMI pc (<25th BMI pc) had significantly lower scores in the Eating, Body image and Treatment burden domains, than the adequate-weight patients (>25th BMI pc) (p < 0.01). A strong child-parent agreement was found in the Physical functioning domain (r = 0.77, p < 0.01). CONCLUSIONS: Passive smoking, parental educational level and chronic diseases of parents do not affect the HRQoL of CF patients. In contrast, hospitalisation, PA infection and malnutrition have a significant and negative impact on patients' HRQoL and the clinical severity of the disease. Parents and children were consistent in their scoring of symptoms and behaviours that were observable.

[Quality of life in Hungarian patients with cystic fibrosis]. / Cystás fibrosisban szenvedo betegek életminoségének felmérése Magyarországon.

INTRODUCTION: Cystic fibrosis is a progressive multisystemic disease which affects the quality of life of patients. AIM: The aim of the study was to evaluate quality of life in Hungarian patients with cystic fibrosis. METHODS: Validated Hungarian translation of The Cystic Fibrosis Questionnaire - Revised was used to measure quality of life. Clinical severity was determined on the basis of Shwachman-Kulczycki score. Lung function was measured using spirometry. RESULTS: 59 patients were included from five centres in Hungary. The relationships between 8-13 year-old children self-report and parent proxy report was 0.77 (p<0.001) in physical functioning, 0.07 (p<0.001) in emotional functioning, 0.51 (p<0.001) in eating, 0.21 (p<0.001) in treatment burden, 0.54 (p<0.001) in body image, 0.49 (p<0.001) in respiratory symptoms and 0.40 (p<0.001) in digestive symptoms domains. CONCLUSIONS: In contrast to physical domains weak correlations were observed between answers obtained from children and their parents in psychosocial domains. The perception of both patients and their parents should be assessed when measuring quality of life in paediatric patients with cystic fibrosis.

[Analysis of direct costs during the treatment of tuberculosis in childhood]. / Tuberkulózis kezelése során felmerülo direkt költségek elemzése gyermekkorban.

Incidence and prevalence of tuberculosis (TB) shows a decreasing tendency in Hungary. The medical care of these patients represents a substantial cost to the health services. We assessed the direct costs of paediatric patients with TB in a Hungarian hospital. Patients participating in the study were 9 children, with an average age of 11.17 +/- 6.34 years. In the study group there were two immigrant patients (22, 23%), two (22, 23%) had multidrug-resistant TB (MDR-TB), five patients (62.5%) were contacts, six (66, 67%) had extrapulmonary tuberculosis and three (33, 34% suffered from other co-morbidities. The average direct cost of pulmonary TB was 2920 Euros, direct cost of MDR-TB was between 7300-9120 Euros. MDR-TB increases one and half times the period spent in hospital, two and half times the cost of inpatient care. Extrapulmonary TB with concomitant disease causes 3-7 times higher expenditures to the health service. According to our results we found significant differences between the cost of actual therapy and resources available; thus the compensation needs to be solved.