Seminal clinical trials with the triple combination of elexacaftor-tezacaftor-ivacaftor (ETI) demonstrated clinical efficacy in people with cystic fibrosis (pwCF) who carry at least one F508del ...mutation. However, due to exclusion criteria of these clinical trials, the effect of ETI was not studied in a substantial number of pwCF. Thus, we ran a single center trial to evaluate a clinical efficacy of ETI treatment in adult pwCF who were ineligible for enrollment in registration studies.
PwCF on ETI with prior lumacaftor-ivacaftor therapy, severe airway obstruction, well-preserved lung function, or with airway infection with pathogens at risk of more rapid decline in lung function formed the study group, while all the others on ETI formed the control group. Lung function, nutritional status and sweat chloride concentration were assessed before and after initialization of ETI therapy over a 6-month period.
Approximately a half of the ETI-treated pwCF at the adult Prague CF center (49 of 96) were assigned to the study group. Their mean changes in body mass index ( + 1.04 kg/m
) and in sweat chloride concentration (-48.4 mmol/L) were similar to the control group ( + 1.02 kg/m
; -49.7 mmol/L), while the mean change in percent predicted forced expiratory volume in 1 s (ppFEV
; + 10.3 points) was significantly lower than in the control group ( + 15.8 points) (
= 0.0015). In the subgroup analysis, pwCF with severe airway obstruction (ppFEV
<40) and pwCF with well-preserved lung function (ppFEV
>90) showed a less potential for improvement in lung function during the ETI treatment than controls (median change in ppFEV
+ 4.9 points and + 9.5 points, respectively).
PwCF not eligible for inclusion in clinical trials demonstrated improvement in lung function and nutritional status following the initiation of treatment with the ETI combination. Moderate increase in ppFEV
was observed in those with severe airway obstruction or well-preserved lung function.
Ivacaftor is a revolutionary treatment option for cystic fibrosis (CF) patients with G551D and other gating mutations. The aim of this study was to evaluate the clinical status of patients on ...ivacaftor who were followed for up to 6 years together with an evaluation of ivacaftor therapy in one patient with an initial FEV1 less than 40% of predicted value.
Data on development of clinical status and sinopulmonary-related therapies were obtained from patient health records during ivacaftor treatment lasting for up to six years and were compared with an equivalent period before ivacaftor administration.
Five CF adults with a median age 28.6 years (range 21.4-35.6 years) with median FEV1 45% pred. (range 16-85% pred.) were included in the study. Four subjects were also participants in the STRIVE and PERSIST studies. Altogether, twenty-four patient-years of ivacaftor treatment were analyzed. The median FEV1 decline per year decreased from -4.5 to -0.9% pred. (P = 0.043). Reduction in number of days on antibiotic treatment and hospital stays was 21% (P < 0.001) and 75% (P = 0.003), respectively. Improvement and stabilization of lung function was observed for up to six years of treatment. In a patient with severe airway obstruction, an increase in the FEV1 value (30.4% from baseline) was documented during the first twelve months of treatment.
Ivacaftor therapy resulted in improved and stabilized lung function in up to six years of treatment with a reduction in number of days on antibiotic treatment and hospital stays. Its efficiency was also displayed in a patient with severe airway obstruction.
Background Economic data pertaining to cystic fibrosis (CF), is limited in Europe generally, and completely lacking in Central and Eastern Europe. We performed an analysis of all direct costs ...associated with CF relative to key disease features and laboratory examinations. Methods A retrospective prevalence-based cost-of-illness (COI) study was performed in a representative cohort of 242 CF patients in the Czech Republic, which represents about 65 % of all Czech CF patients. Medical records and invoices to health insurance companies for reference year 2010 were analyzed. Results The mean total health care costs were €14,486 per patient, with the majority of the costs going towards medicinal products and devices (€10,321). Medical procedures (€2676) and inpatient care (€1829) represented a much smaller percentage of costs. A generalized linear model showed that the strongest cost drivers, for all cost categories, were associated with patient age and lung disease severity (assessed using the FEV1 spirometric parameter), when compounded by chronic Pseudomonas aeruginosa airway infections. Specifically, maximum total costs are around the age 16 years; a FEV1 increase of 1 % point represented a cost decrease of: 0.9 % (medicinal products), 1.7 % (total costs), 2.8 % (procedures) and 7.0 % (inpatient care). Conclusions COI analysis and regression modeling using the most recent data available can provide a better understanding of the overall economic CF burden. A comparison of our results with other methodologically similar studies demonstrates that although overall costs may differ, FEV1 can nonetheless be utilized as a generally transferable indicator of the relative economic impact of CF.
To assess the biliary manometric perfusion test (BMPT) for evaluating success in treating benign biliary strictures.
During 2003 to 2010, 29 patients were subjected to BMPT after percutaneous balloon ...dilatation treatment. Intrabiliary pressure less than 20cm of water was considered the success threshold. Results of BMPT evaluation were retrospectively compared with a similar group where the standard clinical test was used for evaluating treatment success. The clinical test group included 21 patients treated for biliary strictures from 1994 to 2006.
The two groups were statistically similar by age and gender. The BMPT group was tested without complications and pressure inside the biliary tree was less than 20cm of water in 27 of 29 patients. Subsequently, catheters were removed from all 27. Three patients required re-interventions 13 days, 11 months and 32 months later. Kaplan-Meier survival analysis showed that the probability of biliary patency at 3 year was 82.2%. There was no significant difference between groups by this measure (log rank test, p=0.624).
The manometric test is an alternative for evaluating success in treating benign biliary strictures. It is simple, less time-consuming, economical, safe, effective and more comfortable for patients than the clinical test.
We introduce a simple method for estimating the changes of nitrate concentrations in surface waters regarding the land use modification. Stream and drainage water nitrate concentrations in ...prevailingly agricultural catchments of three different scales located in the Crystalline complex of the Czech massif, Czech Republic, were included in this study. Water quality samples were collected through the years 1992-2006 at monthly and bi-monthly intervals. For the catchment land use analysis, the satellite images LANDSAT 7 (CORINE Land Cover) and digitised cadastre maps of the land register were processed using ESRI ArcMap GIS; both sources were corrected by field survey. We demonstrate on three different basin - scale studies a strong relation between the arable land ratio within a catchment and the coherent stream water nitrate concentration. The results acquired from all the evaluated catchments showed that every 10% decrease of ploughed land proportion in a catchment lowers the nitrate concentration C90 value (90% probability of non-exceedance) in average by 6.38 mg/L.
Úvod. Karcinom prsu je nejčastěji diagnostikovaný zhoubný nádor u žen. V České republice se vyskytuje u 20% žen, které onemocní zhoubným nádorem. Ročně onemocní na karcinom prsu přes 5000 žen a kolem ...1900 žen na něj zemře. Od zavedení celoplošného screeningu nádorů prsu v České republice v roce 2002 se očekávalo zvýšení podílu nádorů nižších stadií a snížení úmrtnosti žen na karcinom prsu. Po zavedení screeningu se i přes stoupající incidenci karcinomu prsu úmrtnost nezvyšovala, spíše stagnovala nebo se mírně snížila. Cíl. Zhodnotit na souboru pacientek s karcinomem prsu zjištěným v rámci screeningového a diagnostického vyšetření, zda screening snižuje úmrtnost žen na karcinom prsu. Metoda. V období od ledna 2003 do prosince 2009 byl ve Fakultní nemocni Královské Vinohrady v Praze diagnostikován karcinom prsu u 466 žen. Celkem bylo u těchto žen zjištěno 476 karcinomů prsu. Do screeningové skupiny pacientek s diagnostikovaným zhoubným nádorem prsu bylo zařazeno 160 žen ve věkovém rozmezí 45-74 let se 161 zhoubným nádorem. Jedna pacientka měla v době stanovení diagnózy oboustranný nádor. Do diagnostické skupiny pacientek se zjištěným zhoubným nádorem prsu byla zařazena 181 pacientka. U této skupiny se prokázalo 186 zhoubných nádorů, neboť u pěti žen byl ve sledovaném období diagnostikovaný bilaterální karcinom prsu. Všechny pacientky absolvovaly mamografii, ultrazvuk a core-cut biopsii z prsu. U obou skupin pacientek byla hodnocena průměrná velikost zjištěných nádorů a statisticky byla zhodnocena úmrtnost žen na karcinom prsu. ýsledky. Průměrná velikost zhoubného nádoru prsu ve screeningové skupině byla 15,3 mm a v diagnostické skupině 25,4 mm. Během více jak 7letého sledování zemřely na karcinom prsu tři ženy ze screeningové skupiny (160 žen) a 13 žen z diagnostické skupiny (181 žena). Při porovnání screeningové a diagnostické skupiny byla prokázána statisticky významně nižší úmrtnost ve screeningové skupině než v diagnostické skupině. Závěr. V průběhu více jak 7letého sledování skupiny pacientek s karcinomem prsu bylo prokázáno, že zavedený screening nádorů prsu snižuje úmrtnost na karcinom prsu. Dosažené výsledky podporují pokračování screeningového programu nádoru prsu v České republice.
Introduction. Breast cancer is the most frequently diagnosed major cancer among women. In the Czech Republic it is found in 20% of women suffering with cancer. Annually, about 5.000 women ill with breast cancer and around 1900 women die. Nationwide breast cancer screening has been introduced in 2002 in Czech Republic and there was expected that it should increase the proportion of lower stage cancers and reduce the mortality of women with breast cancer. The introduction of screening has shown that despite the rising incidence of breast cancer, mortality did not increase, rather stagnated or slightly decreased. Aim. To evaluate the group of patients with breast cancer identified in the screening and diagnostic examinations, whether screening can reduce the mortality of women of breast cancer. Method. During the period from January 2003 to December 2009 in the Faculty Hospital Královské Vinohrady in Prague there were total found 476 breast cancers in 466 women. In the screening group of patients diagnosed with breast cancer were enrolled 160 women with 161 breast cancers. One patient had at the time of diagnosis bilateral carcinoma. To the diagnostic group were included 181 women with breast cancer, with a total of 186 tumours. Bilateral breast cancer was diagnosed in 5 women in the period. Mammography, ultrasound and core-cut biopsy from breast tissue were used in all patients with the breast cancer. In both groups of patients was also evaluated the average size of diagnosed tumors and statisticly was evalueted the mortality of breast cancer. Results. The average size of a malignant tumor in the screening group was 15.3mms and 25.4 mm in the diagnostic group. During more than 7 years of monitoring, 3 women from the screening group (160 women) died of breast cancer. In the diagnostic group (181 women) 13 women died of breast cancer. A comparison of screening and diagnostic group was confirmed statistically significantly lower mortality rate in the screening group than in the diagnostic group. Conclusion. During more than 7 years old follow up of patients with breast cancer has been shown that breast cancer screening can reduce mortality of breast cancer. Achieved results support the continuation of breast cancer screening program in the Czech Republic.
Bílková A, Zemanová M, Janík V, Vránová J.
Literatura 19
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