CFTR modulator monotherapy for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del)

Matthew Heneghan; Sherie Smith; Nikki Jahnke; Dr Sarah Nevitt; Kevin W Southern; supported by the Cochrane Cystic Fibrosis Review Group

doi:10.1002/14651858.cd016290

May 7, 2026Cochrane library

CFTR modulator monotherapy for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del)

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Key Points

To evaluate the benefits and harms of CFTR modulator monotherapy in individuals with cystic fibrosis and class II gene variants, primarily F508del.
Included 10 randomized controlled trials (N=424) assessing eight CFTR modulators.
Participants were homozygous for F508del, with the longest study lasting 29 days.
Outcomes assessed included adverse effects and time to pulmonary exacerbation.
No deaths reported, but no clinically significant quality of life improvements observed.
Insufficient evidence for lung function effects and no differences in adverse effect risks compared to placebo.
Certainty of evidence was rated very low for comparisons between lumacaftor and placebo, and cavosonstat and placebo.

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Implication

Randomized trial evaluates CFTR modulator monotherapy effects on lung function and exacerbation rates in cystic fibrosis patients, indicating a lack of clinical benefit.

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Cite This Study

Heneghan et al. (2026) studied this question.

synapsesocial.com/papers/69fecfcdb9154b0b82876cc6 https://doi.org/10.1002/14651858.cd016290

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CFTR modulator monotherapy for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del)

Key Points

No abstract yet

Implication

Discussion

Cite This Study

CFTR modulator monotherapy for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del)

Key Points

No abstract yet

Implication

Discussion

Cite This Study

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