20 Questions
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?
13.8 points higher
What was the rate of pulmonary exacerbations in patients who received elexacaftor-tezacaftor-ivacaftor compared to placebo?
63% lower
What was the minimum clinically important difference for the respiratory domain score on the Cystic Fibrosis Questionnaire–Revised?
4 points
How much lower was the sweat chloride concentration in patients who received elexacaftor-tezacaftor-ivacaftor compared to placebo?
41.8 mmol per liter lower
What was the publication date of the article?
October 31, 2019
Which mutation is responsible for causing cystic fibrosis in nearly 90% of patients?
Phe508del CFTR mutation
What is the primary end point of the phase 3 trial conducted to test the efficacy and safety of elexacaftor–tezacaftor–ivacaftor?
Absolute change from baseline in percentage of predicted FEV1 at week 4
What are the three drugs used in combination in the phase 2 trial to improve Phe508del CFTR function and clinical outcomes?
Elexacaftor, tezacaftor, and ivacaftor
What age group was included in the phase 3 trial to test the efficacy and safety of elexacaftor–tezacaftor–ivacaftor?
Patients 12 years of age or older
Where can the full names, academic degrees, and affiliations of the authors be found?
In the Appendix
What is the purpose of the phase 3 trial mentioned in the text?
To confirm the efficacy and safety of elexacaftor-tezacaftor-ivacaftor in patients with cystic fibrosis with Phe508del-minimal function genotypes.
What were the drugs used in combination in the phase 2 trial to improve Phe508del CFTR function and clinical outcomes?
Elexacaftor, tezacaftor, and ivacaftor.
What was the primary end point of the phase 3 trial?
Absolute change from baseline in percentage of predicted forced expiratory volume in 1 second (FEV1) at week 4.
How old were the patients included in the phase 3 trial?
12 years of age or older.
What is the gene mutation responsible for causing cystic fibrosis in nearly 90% of patients?
Phe508del CFTR mutation.
What was the percentage of predicted FEV1 that was higher in patients who received elexacaftor-tezacaftor-ivacaftor compared to placebo at 4 weeks?
13.8 points higher
What was the rate of pulmonary exacerbations that was lower in patients who received elexacaftor-tezacaftor-ivacaftor compared to placebo?
63% lower
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?
20.2 points higher
What was the sweat chloride concentration that was lower in patients who received elexacaftor-tezacaftor-ivacaftor compared to placebo?
41.8 mmol per liter lower
How many patients underwent randomization and received at least one dose of active treatment or placebo?
403 patients
Test your knowledge on the groundbreaking study published in the New England Journal of Medicine on Elexacaftor-Tezacaftor-Ivacaftor treatment for Cystic Fibrosis with a single Phe508del allele. Explore the findings and implications of this research.
Make Your Own Quizzes and Flashcards
Convert your notes into interactive study material.
Get started for free
