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 transferrable indicator of the relative economic impact of CF.

• MeSH
• cystická fibróza ekonomika   epidemiologie   patofyziologie   MeSH
• dítě MeSH
• dospělí MeSH
• lidé MeSH
• mladiství MeSH
• náklady na zdravotní péči statistika a číselné údaje   MeSH
• osobní újma zaviněná nemocí * MeSH
• předškolní dítě MeSH
• prevalence MeSH
• pseudomonádové infekce ekonomika   epidemiologie   MeSH
• regresní analýza MeSH
• retrospektivní studie MeSH
• spirometrie MeSH
• Check Tag
• dítě MeSH
• dospělí MeSH
• lidé MeSH
• mladiství MeSH
• mužské pohlaví MeSH
• předškolní dítě MeSH
• ženské pohlaví MeSH
• Publikační typ
• časopisecké články MeSH
• Geografické názvy
• Česká republika epidemiologie   MeSH

AIMS: 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. METHODS: 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. RESULTS: 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. CONCLUSION: 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.

• MeSH
• aktivátory chloridových kanálů terapeutické užití   MeSH
• aminofenoly terapeutické užití   MeSH
• chinolony terapeutické užití   MeSH
• cystická fibróza farmakoterapie   patofyziologie   MeSH
• dospělí MeSH
• lidé MeSH
• mladý dospělý MeSH
• následná péče MeSH
• usilovný výdechový objem fyziologie   MeSH
• výsledek terapie MeSH
• Check Tag
• dospělí MeSH
• lidé MeSH
• mladý dospělý MeSH
• mužské pohlaví MeSH
• ženské pohlaví MeSH
• Publikační typ
• časopisecké články MeSH

• Publikační typ
• abstrakt z konference MeSH

Práce na modelu nejčastějšího závažného dědičného vzácného onemocnění u evropských populací, cystické fibrózy, která je považována za model pro ostatní vzácná onemocnění, podává přehled farmakoekonomických studií u tohoto onemocnění.

This study provides a brief overview of pharmacoeconomic studies in cystic fibrosis, the most common genetic disorder in European-derived populations, which serves as a model for other rare diseases.

• MeSH
• analýza nákladů a výnosů MeSH
• cystická fibróza * ekonomika   farmakoterapie   MeSH
• dítě MeSH
• dospělí MeSH
• hospitalizace MeSH
• lidé MeSH
• náklady a analýza nákladů MeSH
• náklady na zdravotní péči * MeSH
• péče o pacienta ekonomika   MeSH
• Pseudomonas aeruginosa MeSH
• sběr dat MeSH
• statistika jako téma MeSH
• Check Tag
• dítě MeSH
• dospělí MeSH
• lidé MeSH
• Publikační typ
• práce podpořená grantem MeSH