Cystic fibrosis foundation pulmonary guidelines. Use of cystic fibrosis transmembrane conductance regulator modulator therapy in patients with cystic fibrosis

Ren, Clement L.; Morgan, Rebecca L.; Oermann, Christopher; Resnick, Helaine E.; Brady, Cynthia; Campbell, Annette; DeNagel, Richard; Guill, Margaret; Hoag, Jeffrey; Lipton, Andrew; Newton, Thomas; Peters, Stacy; Willey-Courand, Donna Beth; Naureckas, Edward T.

doi:10.1513/annalsats.201707-539ot

Cited by 75Cystic Fibrosis Research AdvancesInhalation and Respiratory Drug DeliveryClinical practice guidelines implementation

Cystic fibrosis foundation pulmonary guidelines. Use of cystic fibrosis transmembrane conductance regulator modulator therapy in patients with cystic fibrosis

Ren, Clement L., Morgan, Rebecca L., Oermann, Christopher, Resnick, Helaine E., Brady, Cynthia, Campbell, Annette, DeNagel, Richard, Guill, Margaret +6 more·2018·Annals of the American Thoracic Society

DOI 10.1513/annalsats.201707-539ot

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Abstract

Abstract Rationale Cystic fibrosis (CF) transmembrane conductance regulator (CFTR) modulators are a new class of medications targeting the underlying defect in CF. Ivacaftor (IVA) and IVA combined with lumacaftor (LUM; IVA/LUM) have been approved by the U.S. Food and Drug Administration (FDA) for use in patients with CF. However, the FDA label for these medications encompasses patient groups that were not studied as part of the drug approval process. CF clinicians, patients, and their families have recognized a need for recommendations to guide the use of these medications. Objective Develop evidence-based guidelines for CFTR modulator therapy in patients with CF. Methods A multidisciplinary committee of CF caregivers and patient representatives was assembled. A methodologist, an epidemiologist, a medical librarian, and a biostatistician were recruited to assist with the literature search, evidence grading, and generation of recommendations. The committee developed clinical questions using the Patient-Intervention-Comparison-Outcome format. A systematic review was conducted to find relevant publications. The evidence was then evaluated using the GRADE (Grading of Recommendations, Assessment, Development, and Evaluation) approach, and recommendations were made based on this analysis. Results For adults and children aged 6 years and older with CF due to gating mutations other than G551D or R117H, the guideline panel made a conditional recommendation for treatment with IVA. For those with the R117H mutation, the guideline panel made a conditional recommendation for treatment with IVA for 1) adults aged 18 years or older, and 2) children aged 6–17 years with a forced expiratory volume in 1 second (FEV1) less than 90% predicted. For those with the R117H mutation, the guideline panel made a conditional recommendation against treatment with IVA for 1) children aged 12–17 years with an FEV1 greater than 90% predicted, and 2) children less than 6 years of age. Among those with two copies of F508del, the guideline panel made a strong recommendation for treatment with IVA/LUM for adults and children aged 12 years and older with an FEV1 less than 90% predicted; and made a conditional recommendation for treatment with IVA/LUM for 1) adults and children aged 12 years or older with an FEV1 greater than 90% predicted, and 2) children aged 6–11 years. Conclusions Using the GRADE approach, we have made recommendations for the use of CFTR modulators in patients with CF. These recommendations will be of help to CF clinicians, patients, and their families in guiding decisions regarding use of these medications.

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Small molecules that restore chloride transport function of mutant CFTR have shown promising clinical results, but their narrow application range, poor improvements in lung function, and side effects limit their utilization.
“Some small molecules that aim to restore the chloride transport function of the mutant CFTR have shown promising clinical results. However, their narrow application range, poor improvements in lung function, and side effects limit their utilization.[188–190]”
From RNA delivery biomaterials for the treatment of genetic and rare diseases (2019) · cites this paper as [188]

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Authors: Ren, C. L., Morgan, R. L., Oermann, C., Resnick, H. E., Brady, C., Campbell, A., DeNagel, R., Guill, M., Hoag, J., Lipton, A., Newton, T., Peters, S., Willey-Courand, D. B., & Naureckas, E. T.
Affiliations: Indiana University School of Medicine (US)
McMaster University (CA)
Children’s Mercy–Kansas City, Kansas City, Missouri
Resnick, Chodorow, and Associates, Silver Spring, Maryland
Children’s Minnesota - St. Paul Hospital (US)
Kansas City University (US)
Patient Community Advisor, New York, New York
Dartmouth College (US)
Drexel University (US)
Walter Reed National Military Medical Center (US)
Miller Children's & Women's Hospital (US)
South Dakota State University (US)
University of Texas Health Science Center at San Antonio, San Antonio, Texas; and
University of Chicago (US)
Corresponding author: Ren, Clement L.
ORCID: Ren, Clement L.
Morgan, Rebecca L.
Oermann, Christopher
Resnick, Helaine E.
Naureckas, Edward T.
Venue: Annals of the American Thoracic Society
Publisher: Oxford University Press (OUP)
Published: 2018-03-01
Volume: 15
Issue: 3
Pages: 271-280
ISSN: 2325-6621 (ISSN-L)
2329-6933
DOI: 10.1513/annalsats.201707-539ot
OpenAlex: W2784491391
Cited by: 74
References: 38
Citations by year: 2025: 7 · 2024: 12 · 2023: 8 · 2022: 6 · 2021: 9 · 2020: 7 · 2019: 17 · 2018: 8
FWCI: 7.72
Type: journal article
Language: en
Open access: closed
Licence: academic.oup.com/pages/standard-publication-reuse-rights (vor)
Locations: Annals of the American Thoracic Society (publishedVersion)
PubMed (publishedVersion)
Topics: Health Sciences › Medicine › Pulmonary and Respiratory Medicine › Cystic Fibrosis Research Advances
Health Sciences › Medicine › Pulmonary and Respiratory Medicine › Inhalation and Respiratory Drug Delivery
Health Sciences › Medicine › Public Health, Environmental and Occupational Health › Clinical practice guidelines implementation
Keywords: cystic fibrosis, CFTR modulator therapy, ivacaftor, lumacaftor/ivacaftor, F508del mutation, R117H mutation, G551D mutation, FEV1, treatment eligibility, children and adolescents, adults, evidence-based recommendations, GRADE, patient and family involvement, patient-reported outcomes, lung function
MeSH terms: Aminophenols, Aminopyridines, Cystic Fibrosis, Forced Expiratory Volume, Humans, Mutation, Quinolones, Cystic Fibrosis Transmembrane Conductance Regulator, Benzodioxoles

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Cystic fibrosis foundation pulmonary guidelines. Use of cystic fibrosis transmembrane conductance regulator modulator therapy in patients with cystic fibrosis

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