Ivacaftor Therapy in CF Patients: Single Center Experience

الملخص EN

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.