Biomedical papers, 2016 (vol. 160), issue 2

Biomed Pap Med Fac Univ Palacky Olomouc Czech Repub. 2016, 160(2):276-279 | 10.5507/bp.2016.029

Ivacaftor in cystic fibrosis adults: Czech experience with six years of follow-up

Libor Filaa,b, Lucie Valentova Bartakovaa,b, Alzbeta Grandcourtovaa,b, Miloslav Marela, Radovan Drnekc, Alena Bilkovab,d, Milan Macek Jr.b,e, Pavel Drevinekb,f
a Department of Pneumology, 2nd Faculty of Medicine, Charles University in Prague and University Hospital Motol, Prague, Czech Republic
b Cystic Fibrosis Centre, University Hospital Motol, Prague, Czech Republic
c Division of Health Insurance, University Hospital Motol, Prague, Czech Republic
d Department of Pediatrics, 2nd Faculty of Medicine, Charles University in Prague and University Hospital Motol, Prague, Czech Republic
e Department of Biology and Medical Genetics, 2nd Faculty of Medicine, Charles University in Prague and University Hospital Motol, Prague, Czech Republic
f Department of Medical Microbiology, 2nd Faculty of Medicine, Charles University in Prague and University Hospital Motol, Prague, Czech Republic

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.

Keywords: cystic fibrosis, adults, lung disease, ivacaftor

Received: February 2, 2016; Accepted: May 3, 2016; Prepublished online: May 25, 2016; Published: June 24, 2016


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