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J Cyst Fibros. 2018 Nov;17(6):742-746. doi: 10.1016/j.jcf.2018.08.007. Epub 2018 Sep 7.

Rate and predictors of prescription of lumacaftor - Ivacaftor in the 18 months following approval in the United States.

Author information

1
Division of Respiratory Diseases, Boston Children's Hospital, Boston, MA, United States. Electronic address: Gregory.Sawicki@childrens.harvard.edu.
2
Cystic Fibrosis Foundation, Bethesda, MD, United States.
3
Children's Hospital of Richmond at Virginia Commonwealth U., Richmond, VA, United States.
4
Departments of Biostatistics and Pediatrics, University of Washington, Seattle, WA, United States.

Abstract

RATIONALE:

Lumacaftor-ivacaftor (LUM-IVA) was approved in the US in 2015 for patients with CF aged >12 homozygous for the delF508 mutation, and patients aged 6 to 12 in 2016.

OBJECTIVES:

To examine the rate of initial LUM-IVA prescriptions following approval.

METHODS:

We compared patients eligible for LUM-IVA in the CF Foundation Patient Registry with and without prescriptions in 2015-2016.

RESULTS:

5534 (53%) eligible patients had reported prescriptions. Prescription rate in children ages 6-11 was 19% and 61% among patients ≥12 years old. Individuals ≥12 with prescriptions more likely observed among those with private insurance, clinical trial participation, ages 18-30, FEV1 < 90%, more pulmonary exacerbations, and more use of chronic medications.

CONCLUSIONS:

LUM-IVA uptake was less rapid than what was previously observed for ivacaftor, a CFTR modulator approved for a different population. Age, insurance status, disease severity and use of other therapies differed in those prescribed LUM-IVA in the initial post-approval period.

PMID:
30201329
DOI:
10.1016/j.jcf.2018.08.007

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