The Phe508del Allele and CF Treatment
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Questions and Answers

According to the study results, how much higher was the percentage of predicted FEV1 in patients who received elexacaftor-tezacaftor-ivacaftor compared to placebo at 4 weeks?

  • 8.3 points higher
  • 18.3 points higher
  • 3.8 points higher
  • 13.8 points higher (correct)
  • What was the rate of pulmonary exacerbations in patients who received elexacaftor-tezacaftor-ivacaftor compared to placebo?

  • 43% lower
  • 53% lower
  • 63% lower (correct)
  • 73% lower
  • What was the minimum clinically important difference for the respiratory domain score on the Cystic Fibrosis Questionnaire–Revised?

  • 2 points
  • 6 points
  • 4 points (correct)
  • 8 points
  • How much lower was the sweat chloride concentration in patients who received elexacaftor-tezacaftor-ivacaftor compared to placebo?

    <p>41.8 mmol per liter lower</p> Signup and view all the answers

    What was the publication date of the article?

    <p>October 31, 2019</p> Signup and view all the answers

    Which mutation is responsible for causing cystic fibrosis in nearly 90% of patients?

    <p>Phe508del CFTR mutation</p> Signup and view all the answers

    What is the primary end point of the phase 3 trial conducted to test the efficacy and safety of elexacaftor–tezacaftor–ivacaftor?

    <p>Absolute change from baseline in percentage of predicted FEV1 at week 4</p> Signup and view all the answers

    What are the three drugs used in combination in the phase 2 trial to improve Phe508del CFTR function and clinical outcomes?

    <p>Elexacaftor, tezacaftor, and ivacaftor</p> Signup and view all the answers

    What age group was included in the phase 3 trial to test the efficacy and safety of elexacaftor–tezacaftor–ivacaftor?

    <p>Patients 12 years of age or older</p> Signup and view all the answers

    Where can the full names, academic degrees, and affiliations of the authors be found?

    <p>In the Appendix</p> Signup and view all the answers

    What is the purpose of the phase 3 trial mentioned in the text?

    <p>To confirm the efficacy and safety of elexacaftor-tezacaftor-ivacaftor in patients with cystic fibrosis with Phe508del-minimal function genotypes.</p> Signup and view all the answers

    What were the drugs used in combination in the phase 2 trial to improve Phe508del CFTR function and clinical outcomes?

    <p>Elexacaftor, tezacaftor, and ivacaftor.</p> Signup and view all the answers

    What was the primary end point of the phase 3 trial?

    <p>Absolute change from baseline in percentage of predicted forced expiratory volume in 1 second (FEV1) at week 4.</p> Signup and view all the answers

    How old were the patients included in the phase 3 trial?

    <p>12 years of age or older.</p> Signup and view all the answers

    What is the gene mutation responsible for causing cystic fibrosis in nearly 90% of patients?

    <p>Phe508del CFTR mutation.</p> Signup and view all the answers

    What was the percentage of predicted FEV1 that was higher in patients who received elexacaftor-tezacaftor-ivacaftor compared to placebo at 4 weeks?

    <p>13.8 points higher</p> Signup and view all the answers

    What was the rate of pulmonary exacerbations that was lower in patients who received elexacaftor-tezacaftor-ivacaftor compared to placebo?

    <p>63% lower</p> Signup and view all the answers

    What was the respiratory domain score on the Cystic Fibrosis Questionnaire–Revised that was higher in patients who received elexacaftor-tezacaftor-ivacaftor compared to placebo?

    <p>20.2 points higher</p> Signup and view all the answers

    What was the sweat chloride concentration that was lower in patients who received elexacaftor-tezacaftor-ivacaftor compared to placebo?

    <p>41.8 mmol per liter lower</p> Signup and view all the answers

    How many patients underwent randomization and received at least one dose of active treatment or placebo?

    <p>403 patients</p> Signup and view all the answers

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