Limits...
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

FEF25–75 trend. Similar to trend in FEV1 (see Figure 2), there was significant improvement in FEF 25–75% (P < 0.01). The adult patient (number 5) had only modest improvement suggesting that the patient already had irreversible lung damage.
© Copyright Policy
Related In: Results  -  Collection

License
getmorefigures.php?uid=PMC4590953&req=5

fig3: FEF25–75 trend. Similar to trend in FEV1 (see Figure 2), there was significant improvement in FEF 25–75% (P < 0.01). The adult patient (number 5) had only modest improvement suggesting that the patient already had irreversible lung damage.

Mentions: The mean BMI among pediatric patients was 16.2 kg/m2 at baseline which improved to 20.9 kg/m2 at 15 months (Figure 1). The mean FEV1 changed from 56.2 to 99.7 percent predicted (Figure 2) and the mean FEF25–75 from 40.2 to 90 percent predicted (Figure 3). The adult patient was a 45-year-old female who had been previously diagnosed with advanced lung disease when Ivacaftor was started. Her FEV1 was 23% at 3 months after beginning of the therapy, which changed to 42% at 15 months after Ivacaftor. Her BMI was 29.6 kg/m2 at baseline which did not change significantly with time. Our results yield a P value of less than 0.01 for all the three data sets analyzed separately for FEV1, FEF25–75, and BMI. The adult patient was known to be pancreatic sufficient and her obesity was a contributing factor to her diabetes mellitus. Her HbA1c improved from 7.4% to 6.6% without changing any insulin regimen after Ivacaftor was started. She had one hospitalization for CF exacerbation in the year before starting Ivacaftor though she did not require any hospitalization after Ivacaftor therapy.


Ivacaftor Therapy in CF Patients: Single Center Experience.

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

FEF25–75 trend. Similar to trend in FEV1 (see Figure 2), there was significant improvement in FEF 25–75% (P < 0.01). The adult patient (number 5) had only modest improvement suggesting that the patient already had irreversible lung damage.
© Copyright Policy
Related In: Results  -  Collection

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

fig3: FEF25–75 trend. Similar to trend in FEV1 (see Figure 2), there was significant improvement in FEF 25–75% (P < 0.01). The adult patient (number 5) had only modest improvement suggesting that the patient already had irreversible lung damage.
Mentions: The mean BMI among pediatric patients was 16.2 kg/m2 at baseline which improved to 20.9 kg/m2 at 15 months (Figure 1). The mean FEV1 changed from 56.2 to 99.7 percent predicted (Figure 2) and the mean FEF25–75 from 40.2 to 90 percent predicted (Figure 3). The adult patient was a 45-year-old female who had been previously diagnosed with advanced lung disease when Ivacaftor was started. Her FEV1 was 23% at 3 months after beginning of the therapy, which changed to 42% at 15 months after Ivacaftor. Her BMI was 29.6 kg/m2 at baseline which did not change significantly with time. Our results yield a P value of less than 0.01 for all the three data sets analyzed separately for FEV1, FEF25–75, and BMI. The adult patient was known to be pancreatic sufficient and her obesity was a contributing factor to her diabetes mellitus. Her HbA1c improved from 7.4% to 6.6% without changing any insulin regimen after Ivacaftor was started. She had one hospitalization for CF exacerbation in the year before starting Ivacaftor though she did not require any hospitalization after Ivacaftor therapy.

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