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Ivacaftor Therapy in CF Patients: Single Center Experience.

Mondal P, Loyson A, Lascano J, Hegde S - Adv Med (2014)

Bottom Line: All patients had both subjective and objective improvements in their health.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.

View Article: PubMed Central - PubMed

Affiliation: Department of Pediatrics, University of Florida, 1600 SW Archer Road, Suite HD 604, P.O. Box 100296, Gainesville, FL 32610, USA.

ABSTRACT
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.

No MeSH data available.


Related in: MedlinePlus

CT of the chest before and after Ivacaftor. Two representative chest CT slices of patient number 4 ((a) and (b), 16-year-old male) at the same anatomic region before and after Ivacaftor therapy. They demonstrate decreased mucus impaction in the latter scan despite the continued presence of bronchiectasis. Decreased mucus impaction probably explains improved lung function (Figures 2 and 3) despite irreversible nature of bronchiectasis. In the bottom panels ((c) and (d)) are the chest CT of another patient (14-year-old female) who already had established bronchiectasis in the peripheral airways before Ivacaftor (c) which has been resolved 18 months after Ivacaftor therapy (d).
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fig4: CT of the chest before and after Ivacaftor. Two representative chest CT slices of patient number 4 ((a) and (b), 16-year-old male) at the same anatomic region before and after Ivacaftor therapy. They demonstrate decreased mucus impaction in the latter scan despite the continued presence of bronchiectasis. Decreased mucus impaction probably explains improved lung function (Figures 2 and 3) despite irreversible nature of bronchiectasis. In the bottom panels ((c) and (d)) are the chest CT of another patient (14-year-old female) who already had established bronchiectasis in the peripheral airways before Ivacaftor (c) which has been resolved 18 months after Ivacaftor therapy (d).

Mentions: Ivacaftor also had an impact on the sputum microbiology (Table 1). Two of our patients had methicillin resistant Staphylococcus aureus (MRSA) before, which could not be isolated after Ivacaftor therapy. One of them, who used to grow nonmucoid pseudomonas before in bronchoalveolar lavage (BAL), is now growing only methicillin-sensitive Staphylococcus aureus (MSSA), since we have started Ivacaftor. In two patients, where pre- and post-chest CT scans were available, there was significant improvement in the radiology findings, which showed decreased mucus impaction, improved bronchial wall thickening, and partial reversal of bronchiectasis despite established lung disease (Figure 4). During the same period, his FEV1 has improved from 26% predicted to 50% after Ivacaftor therapy. The vitamin D level did not change significantly.


Ivacaftor Therapy in CF Patients: Single Center Experience.

Mondal P, Loyson A, Lascano J, Hegde S - Adv Med (2014)

CT of the chest before and after Ivacaftor. Two representative chest CT slices of patient number 4 ((a) and (b), 16-year-old male) at the same anatomic region before and after Ivacaftor therapy. They demonstrate decreased mucus impaction in the latter scan despite the continued presence of bronchiectasis. Decreased mucus impaction probably explains improved lung function (Figures 2 and 3) despite irreversible nature of bronchiectasis. In the bottom panels ((c) and (d)) are the chest CT of another patient (14-year-old female) who already had established bronchiectasis in the peripheral airways before Ivacaftor (c) which has been resolved 18 months after Ivacaftor therapy (d).
© Copyright Policy
Related In: Results  -  Collection

License
Show All Figures
getmorefigures.php?uid=PMC4590953&req=5

fig4: CT of the chest before and after Ivacaftor. Two representative chest CT slices of patient number 4 ((a) and (b), 16-year-old male) at the same anatomic region before and after Ivacaftor therapy. They demonstrate decreased mucus impaction in the latter scan despite the continued presence of bronchiectasis. Decreased mucus impaction probably explains improved lung function (Figures 2 and 3) despite irreversible nature of bronchiectasis. In the bottom panels ((c) and (d)) are the chest CT of another patient (14-year-old female) who already had established bronchiectasis in the peripheral airways before Ivacaftor (c) which has been resolved 18 months after Ivacaftor therapy (d).
Mentions: Ivacaftor also had an impact on the sputum microbiology (Table 1). Two of our patients had methicillin resistant Staphylococcus aureus (MRSA) before, which could not be isolated after Ivacaftor therapy. One of them, who used to grow nonmucoid pseudomonas before in bronchoalveolar lavage (BAL), is now growing only methicillin-sensitive Staphylococcus aureus (MSSA), since we have started Ivacaftor. In two patients, where pre- and post-chest CT scans were available, there was significant improvement in the radiology findings, which showed decreased mucus impaction, improved bronchial wall thickening, and partial reversal of bronchiectasis despite established lung disease (Figure 4). During the same period, his FEV1 has improved from 26% predicted to 50% after Ivacaftor therapy. The vitamin D level did not change significantly.

Bottom Line: All patients had both subjective and objective improvements in their health.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.

View Article: PubMed Central - PubMed

Affiliation: Department of Pediatrics, University of Florida, 1600 SW Archer Road, Suite HD 604, P.O. Box 100296, Gainesville, FL 32610, USA.

ABSTRACT
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.

No MeSH data available.


Related in: MedlinePlus