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2014 ; 2014
(ä): 947923
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Ivacaftor Therapy in CF Patients: Single Center Experience
#MMPMID26556432
Mondal P
; Loyson A
; Lascano J
; Hegde S
Adv Med
2014[]; 2014
(ä): 947923
PMID26556432
show ga
Ivacaftor is the first novel cystic fibrosis pharmaceutical that acts at the
molecular level to potentiate cystic fibrosis transmembrane conductance regulator
(CFTR) function and was first approved for clinical use in 2012. We are sharing
our single center experience of five patients: four from pediatric age group and
one adult patient. All patients had both subjective and objective improvements in
their health. Despite established lung disease, our patients had significant
improvement in both their FEV1 (forced expiratory volume in 1 second) and
FEF25-75 and BMI (body mass index). Larger studies demonstrated only 6.7%
improvement in mean FEV1 after starting Ivacaftor therapy but their patient
population had normal lung function to begin with. In contrast our case series
demonstrates that, in patients with established lung disease and diminished lung
function, Ivacaftor can be expected to result in much higher recovery in lung
function. Mean FEV1 improved by 35% in our case series. Ivacaftor is extremely
expensive, costing $300,000 per patient per year requiring lifelong therapy,
hence requiring prior authorizations from most third-party payers in the USA. The
knowledge shared from our experience will be useful for other clinicians to
petition healthcare policymakers on behalf of their patients.