Ivacaftor in cystic fibrosis adults: Czech experience with six years of follow-up
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...
| Main Authors: | , , , , , , , |
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| Format: | Article |
| Language: | English |
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Palacký University Olomouc, Faculty of Medicine and Dentistry
2016-06-01
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| Series: | Biomedical Papers |
| Subjects: | |
| Online Access: | https://biomed.papers.upol.cz/artkey/bio-201602-0015_Ivacaftor_in_cystic_fibrosis_adults_Czech_experience_with_six_years_of_follow-up.php |
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| author | Libor Fila Lucie Valentova Bartakova Alzbeta Grandcourtova Miloslav Marel Radovan Drnek Alena Bilkova Milan Macek Pavel Drevinek |
| author_facet | Libor Fila Lucie Valentova Bartakova Alzbeta Grandcourtova Miloslav Marel Radovan Drnek Alena Bilkova Milan Macek Pavel Drevinek |
| author_sort | Libor Fila |
| collection | DOAJ |
| description | 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. |
| first_indexed | 2024-12-10T06:10:44Z |
| format | Article |
| id | doaj.art-227fe668bf8c4b7285e574094b698726 |
| institution | Directory Open Access Journal |
| issn | 1213-8118 1804-7521 |
| language | English |
| last_indexed | 2024-12-10T06:10:44Z |
| publishDate | 2016-06-01 |
| publisher | Palacký University Olomouc, Faculty of Medicine and Dentistry |
| record_format | Article |
| series | Biomedical Papers |
| spelling | doaj.art-227fe668bf8c4b7285e574094b6987262022-12-22T01:59:34ZengPalacký University Olomouc, Faculty of Medicine and DentistryBiomedical Papers1213-81181804-75212016-06-01160227627910.5507/bp.2016.029bio-201602-0015Ivacaftor in cystic fibrosis adults: Czech experience with six years of follow-upLibor Fila0Lucie Valentova Bartakova1Alzbeta Grandcourtova2Miloslav Marel3Radovan Drnek4Alena Bilkova5Milan Macek6Pavel Drevinek7Department of Pneumology, 2nd Faculty of Medicine, Charles University in Prague and University Hospital Motol, Prague, Czech RepublicDepartment of Pneumology, 2nd Faculty of Medicine, Charles University in Prague and University Hospital Motol, Prague, Czech RepublicDepartment of Pneumology, 2nd Faculty of Medicine, Charles University in Prague and University Hospital Motol, Prague, Czech RepublicDepartment of Pneumology, 2nd Faculty of Medicine, Charles University in Prague and University Hospital Motol, Prague, Czech RepublicDivision of Health Insurance, University Hospital Motol, Prague, Czech RepublicCystic Fibrosis Centre, University Hospital Motol, Prague, Czech RepublicCystic Fibrosis Centre, University Hospital Motol, Prague, Czech RepublicCystic Fibrosis Centre, University Hospital Motol, Prague, Czech RepublicAims: 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.https://biomed.papers.upol.cz/artkey/bio-201602-0015_Ivacaftor_in_cystic_fibrosis_adults_Czech_experience_with_six_years_of_follow-up.phpcystic fibrosisadultslung diseaseivacaftor |
| spellingShingle | Libor Fila Lucie Valentova Bartakova Alzbeta Grandcourtova Miloslav Marel Radovan Drnek Alena Bilkova Milan Macek Pavel Drevinek Ivacaftor in cystic fibrosis adults: Czech experience with six years of follow-up Biomedical Papers cystic fibrosis adults lung disease ivacaftor |
| title | Ivacaftor in cystic fibrosis adults: Czech experience with six years of follow-up |
| title_full | Ivacaftor in cystic fibrosis adults: Czech experience with six years of follow-up |
| title_fullStr | Ivacaftor in cystic fibrosis adults: Czech experience with six years of follow-up |
| title_full_unstemmed | Ivacaftor in cystic fibrosis adults: Czech experience with six years of follow-up |
| title_short | Ivacaftor in cystic fibrosis adults: Czech experience with six years of follow-up |
| title_sort | ivacaftor in cystic fibrosis adults czech experience with six years of follow up |
| topic | cystic fibrosis adults lung disease ivacaftor |
| url | https://biomed.papers.upol.cz/artkey/bio-201602-0015_Ivacaftor_in_cystic_fibrosis_adults_Czech_experience_with_six_years_of_follow-up.php |
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