# Changes in Provider Practices for the Management of Cystic Fibrosis (CF) in the Era of Highly Effective Modulator Therapy

> **NIH NIH F32** · JOHNS HOPKINS UNIVERSITY · 2021 · $88,958

## Abstract

Cystic Fibrosis (CF) is caused by mutations in the cystic fibrosis transmembrane conductance regulator
(CFTR) protein, which result in thickened airway mucus, chronic infection, and bronchiectasis. Many daily
therapies are recommended by the CF Foundation (CFF) to treat these complications, including inhaled
hypertonic saline, dornase alfa, and antibiotics, oral azithromycin, and airway clearance therapies. Historically,
adherence to these therapies is quite low, as they are exceedingly time consuming, requiring up to 2-4 hours to
complete daily. Persons with CF also have acute worsening of symptoms, called pulmonary exacerbations
(PEx), that are treated with systemic antibiotics and are associated with progressive lung function decline and
increased mortality. CFTR modulators aim to reverse CFTR dysfunction. With the approval of the CFTR
modulator elexacaftor/tezacaftor/ivacaftor (ETI), 90% of the CF population is eligible for highly effective
modulator therapy (HEMT), defined as CFTR modulator therapy resulting in a substantial increase in percent
predicted FEV1 and improvement in sweat chloride concentrations. Prior to the approval of ETI, only ~5% of
patients were eligible for therapy with HEMT. Our local data show that a majority of patients on ETI report that
they have simplified their chronic therapy regimens following discussion with their CF providers, given the
burden associated with these therapies and improvement in symptoms they experienced after starting ETI.
Likewise, providers at our CF center report prescribing oral antibiotics in the home setting for the treatment of
PEx rather than intravenous (IV) antibiotics in the hospital more frequently for patients on ETI. The details of
how national practice patterns have changed with regards to chronic maintenance therapies and management
of PEx since the approval of ETI remains uncertain. We hypothesize that providers have modified the
management of both chronic and acute therapies for CF such that the approach to CF is less aggressive,
requiring fewer chronic medications and less burdensome treatments for PEx.
 This current study will: (1) utilize a national survey to evaluate CF provider practice patterns, assessing
if providers continue to follow guidelines for chronic and acute therapies in patients on ETI. If modifications
were made, we will assess what chronic therapies were modified and changes in the frequency of oral and IV
antibiotic use and hospitalizations for the management of PEx in patients on ETI; (2) evaluate the management
of PEx prospectively at our center to determine if the proportion of oral vs. IV antibiotics and treatment in the
home vs. hospital setting has changed since the approval of ETI. The results of this proposal address an
important question regarding the evolving management of CF in the era of HEMT and have direct implications
for the clinical care of persons with CF. If our results suggest that provider practices have changed since the
approval of ETI...

## Key facts

- **NIH application ID:** 10312402
- **Project number:** 1F32HL159912-01
- **Recipient organization:** JOHNS HOPKINS UNIVERSITY
- **Principal Investigator:** Alexandra Toporek
- **Activity code:** F32 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2021
- **Award amount:** $88,958
- **Award type:** 1
- **Project period:** 2021-09-23 → 2023-09-22

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10312402

## Citation

> US National Institutes of Health, RePORTER application 10312402, Changes in Provider Practices for the Management of Cystic Fibrosis (CF) in the Era of Highly Effective Modulator Therapy (1F32HL159912-01). Retrieved via AI Analytics 2026-05-21 from https://api.ai-analytics.org/grant/nih/10312402. Licensed CC0.

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