The DOI matches its Crossref record; 38 of the 38 references it cites were found in a registry or an index; 19 of the 38 sources its claims cite were checked for support.
Elexacaftor–Tezacaftor–Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele
Open accessAbstract
BACKGROUNDCystic fibrosis is caused by mutations in the gene encoding the cystic fibrosis transmembrane conductance regulator (CFTR) protein, and nearly 90% of patients have at least one copy of the Phe508del CFTR mutation.In a phase 2 trial involving patients who were heterozygous for the Phe508del CFTR mutation and a minimalfunction mutation (Phe508del-minimal function genotype), the next-generation CFTR corrector elexacaftor, in combination with tezacaftor and ivacaftor, improved Phe508del CFTR function and clinical outcomes. METHODSWe conducted a phase 3, randomized, double-blind, placebo-controlled trial to confirm the efficacy and safety of elexacaftor-tezacaftor-ivacaftor in patients 12 years of age or older with cystic fibrosis with Phe508del-minimal function genotypes.Patients were randomly assigned to receive elexacaftor-tezacaftor-ivacaftor or placebo for 24 weeks.The primary end point was absolute change from baseline in percentage of predicted forced expiratory volume in 1 second (FEV 1 ) at week 4. RESULTSA total of 403 patients underwent randomization and received at least one dose of active treatment or placebo.Elexacaftor-tezacaftor-ivacaftor, relative to placebo, resulted in a percentage of predicted FEV 1 that was 13.8 points higher at 4 weeks and 14.3 points higher through 24 weeks, a rate of pulmonary exacerbations that was 63% lower, a respiratory domain score on the Cystic Fibrosis Questionnaire-Revised (range, 0 to 100, with higher scores indicating a higher patient-reported quality of life with regard to respiratory symptoms; minimum clinically important difference, 4 points) that was 20.2 points higher, and a sweat chloride concentration that was 41.8 mmol per liter lower (P<0.001for all comparisons).Elexacaftortezacaftor-ivacaftor was generally safe and had an acceptable side-effect profile.Most patients had adverse events that were mild or moderate.Adverse events leading to discontinuation of the trial regimen occurred in 1% of the patients in the elexacaftor-tezacaftor-ivacaftor group. CONCLUSIONSElexacaftor-tezacaftor-ivacaftor was efficacious in patients with cystic fibrosis with Phe508del-minimal function genotypes, in whom previous CFTR modulator regimens were ineffective.(Funded by Vertex Pharmaceuticals; VX17-445-102 ClinicalTrials.govnumber, NCT03525444.
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Written from the paper’s full text.
TL;DR
In a phase 3, randomized, double-blind, placebo-controlled trial, elexacaftor–tezacaftor–ivacaftor improved lung function, pulmonary exacerbation rate, respiratory symptoms, sweat chloride, and BMI in patients with cystic fibrosis and Phe508del–minimal function genotypes. The regimen was generally safe, with adverse events mostly mild or moderate.
Key findings
- Elexacaftor–tezacaftor–ivacaftor increased percentage of predicted FEV1 by 13.8 points at week 4 and 14.3 points through week 24 relative to placebo (P<0.001 for both).
- The annualized rate of pulmonary exacerbations was 63% lower with active treatment than placebo (rate ratio, 0.37; 95% CI, 0.25 to 0.55; P<0.001).
- CFQ-R respiratory domain score improved by 20.2 points and sweat chloride concentration decreased by 41.8 mmol per liter relative to placebo through week 24 (P<0.001 for both); BMI improved by 1.04 (P<0.001).
- Adverse events occurred in 93.1% of the elexacaftor–tezacaftor–ivacaftor group and 96.0% of the placebo group; 1% of active-treatment patients discontinued because of adverse events, and there were no deaths.
Methods
- Phase 3, multicenter, randomized, double-blind, placebo-controlled trial at 115 sites in 13 countries; 405 patients underwent randomization and 403 received at least one dose (200 active, 203 placebo).
- Eligible patients were 12 years of age or older with cystic fibrosis and Phe508del–minimal function genotypes, screening ppFEV1 40 to 90%, and stable disease during a 28-day screening period; patients were randomly assigned 1:1 to elexacaftor–tezacaftor–ivacaftor or placebo for 24 weeks.
- The primary end point was absolute change from baseline in ppFEV1 at week 4, analyzed with a mixed-effects model for repeated measures; pulmonary exacerbations were analyzed with negative binomial regression.
Limitations
- The paper does not include a formal limitations section.
- Evident from the text: the intervention period was 24 weeks, so long-term durability and safety beyond 24 weeks are not established; patients could enroll in an ongoing 96-week open-label extension.
- Evident from the text: eligibility was restricted to patients 12 years or older with Phe508del–minimal function genotypes and screening ppFEV1 40 to 90%, limiting generalizability to younger children, other CFTR genotypes, or patients outside that lung-function range.
Model-written summary. Check it against the paper before relying on it.
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Claims the paper makes
19 of 38 cited sources checked · 12 supported · 0 flagged · accuracy not yet measured
The sources with a verdict support this claim; the rest weren’t checked yet, have no full text we could read, or their verdicts aren’t rated yet.
In the paper
“For persons with cystic fibrosis who are homozygous for the Phe508del CFTR mutation, the combination of a single corrector, either lumacaftor or tezacaftor, with the potentiator ivacaftor improves clinical outcomes, including lung function and the rate of pulmonary exacerbations.9,11”
§ Elexacaftor–Tezacaftor–Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele > Abstract > CONCLUSIONS
What the sources say
- Supportedcites [9] Wainwright CE, Elborn JS, Ramsey BW, Lumacaftor–ivacaftor in patients with cystic fibrosis homozygous for Phe508del CFTR. N Engl J Med 2015; 373: 220– 31. 25981758 PMC4764353 source
Split into 2 atoms — 2 confirmed, 0 partially supported, 0 disputed, 0 misattributed, 0 unverifiable. Expand the claim to see per-atom verdicts.
- Not checked — no full text of the sourcecites [11] Taylor-Cousar JL, Munck A, McKone EF, Tezacaftor–ivacaftor in patients with cystic fibrosis homozygous for Phe508del. N Engl J Med 2017; 377: 2013– 23. 29099344 source
Strength:plain· not yet calibratedCausal:direct· not yet calibratedTests✓ Asserted: passes✓ Checkable: passes? Load-bearing: unclear✓ Based: passes- Supported
The cited sources support only part of this claim — read each source’s verdict below.
In the paper
“The combination includes the next-generation corrector elexacaftor plus the corrector tezacaftor and the potentiator ivacaftor to more fully restore the function of Phe508del CFTR.18”
§ Elexacaftor–Tezacaftor–Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele > Abstract > CONCLUSIONS
What the sources say
- Partly supportedcites [18] Keating D, Marigowda G, Burr L, VX-445-tezacaftor–ivacaftor in patients with cystic fibrosis and one or two Phe508del alleles. N Engl J Med 2018; 379: 1612– 20. 30334692 PMC6289290 sourceExperimental — accuracy not yet measured
The Background identifies VX-445 as a next-generation corrector used with tezacaftor and ivacaftor to restore Phe508del CFTR function. The in vitro Results report greater chloride transport with the triple combination than with one- or two-drug treatments. Our copy does not identify VX-445 by the name elexacaftor, so that specific identification cannot be verified from this source alone. (LLM, conf=0.78)
Tests✓ Asserted: passes✓ Checkable: passes✓ Load-bearing: passes✓ Based: passes- Partly supported
Every source the paper cites for this claim was checked and supports it.
In the paper
“In this 24-week trial of triple-combination CFTR modulator therapy in patients with cystic fibrosis who have a single Phe508del allele, elexacaftor–tezacaftor–ivacaftor treatment resulted in improvements in lung function, the rate of pulmonary exacerbations, sweat chloride concentration, CFQ-R respiratory domain scores, and BMI and was generally safe with an acceptable side-effect profile,”
§ Elexacaftor–Tezacaftor–Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele > DISCUSSION
What the sources say
- Supportedcites [18] Keating D, Marigowda G, Burr L, VX-445-tezacaftor–ivacaftor in patients with cystic fibrosis and one or two Phe508del alleles. N Engl J Med 2018; 379: 1612– 20. 30334692 PMC6289290 source
The Methods describe this source as a phase 2 trial of VX-445 (elexacaftor)–tezacaftor–ivacaftor. Its Results report improvements in lung function, sweat chloride concentration, and CFQ-R respiratory scores; the Discussion reports an acceptable adverse-event profile. These findings support the cited statement of broad consistency in efficacy and safety, without establishing consistency for every endpoint of the later trial. (LLM, conf=0.90)
Strength:plain· not yet calibratedCausal:correlational· not yet calibratedTests✓ Asserted: passes✓ Checkable: passes? Load-bearing: unclear✓ Based: passes- Supported
Every source the paper cites for this claim was checked and supports it.
In the paper
“The improvement in the primary end point of absolute change in percentage of predicted FEV1 was 10.6 points in patients with the Gly551Asp allele.10”
§ Elexacaftor–Tezacaftor–Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele > DISCUSSION
What the sources say
- Supportedcites [10] Ramsey BW, Davies J, McElvaney NG, A CFTR potentiator in patients with cystic fibrosis and the G551D mutation. N Engl J Med 2011; 365: 1663– 72. 22047557 PMC3230303 source
The Results (Clinical Efficacy) report that the primary endpoint, change in percent of predicted FEV1 through week 24, improved by 10.6 percentage points with ivacaftor relative to placebo in patients with the G551D mutation (Gly551Asp). The 10.6-point figure is the between-group treatment effect, not the change within the ivacaftor group alone. (LLM, conf=0.93)
Strength:plain· not yet calibratedCausal:direct· not yet calibratedTests✓ Asserted: passes✓ Checkable: passes? Load-bearing: unclear✓ Based: passes- Supported
The cited sources support only part of this claim — read each source’s verdict below.
In the paper
“In proof-of-concept trials that evaluated this therapeutic approach,16,18 elexacaftor–tezacaftor–ivacaftor led to improvements in spirometry, patient-reported respiratory symptoms, and sweat chloride concentration, a marker of CFTR activity, in patients with cystic fibrosis with a single Phe508del allele.18”
§ Elexacaftor–Tezacaftor–Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele > Abstract > CONCLUSIONS
What the sources say
- Partly supportedcites [16] Davies JC, Moskowitz SM, Brown C, VX-659-tezacaftor–ivacaftor in patients with cystic fibrosis and one or two Phe508del alleles. N Engl J Med 2018; 379: 1599– 611. 30334693 PMC6277022 sourceExperimental — accuracy not yet measured
The Results (Phase 2 Efficacy; page not provided) report improved FEV1, sweat chloride concentration, and CFQ-R respiratory scores in patients with a single Phe508del allele. But the Methods (Clinical Development; page not provided) identify the treatment as VX-659–tezacaftor–ivacaftor, not elexacaftor–tezacaftor–ivacaftor. This source therefore supports the outcomes and patient group, but not the named regimen. (LLM, conf=0.88)
- Supportedcites [18] Keating D, Marigowda G, Burr L, VX-445-tezacaftor–ivacaftor in patients with cystic fibrosis and one or two Phe508del alleles. N Engl J Med 2018; 379: 1612– 20. 30334692 PMC6289290 source
The Methods describe a proof-of-concept phase 2 trial that included patients with one Phe508del allele and a minimal-function mutation. The Results report improved FEV1, sweat chloride concentration, and CFQ-R respiratory-domain scores in that group; the Methods identify the CFQ-R domain as a measure of respiratory symptoms. (LLM, conf=0.93)
Tests✓ Asserted: passes✓ Checkable: passes✓ Load-bearing: passes✓ Based: passes- Rests on an assumption: Sweat chloride concentration is a marker of CFTR activity.
- Partly supported
Every source the paper cites for this claim was checked and supports it.
In the paper
“The restoration of Phe508del CFTR by elexacaftor–tezacaftor–ivacaftor was further confirmed by a concurrent phase 3 trial involving patients with two Phe508del alleles, which showed substantially improved outcomes, including increased lung function and decreased patient-reported respiratory symptoms, as compared with the dual combination of tezacaftor–ivacaftor.32”
§ Elexacaftor–Tezacaftor–Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele > DISCUSSION
What the sources say
- Supportedcites [32] Heijerman HG, McKone EF, Downey DG, Efficacy and safety of the elexacaftor plus tezacaftor plus ivacaftor combination regimen in people with cystic fibrosis homozygous for the F508del mutation: a double-blind, randomised, phase 3 trial. Lancet 2019 10 31 (Epub ahead of print). PMC7571408 31679946 source
Split into 2 atoms — 2 confirmed, 0 partially supported, 0 disputed, 0 misattributed, 0 unverifiable. Expand the claim to see per-atom verdicts.
Strength:plain· not yet calibratedCausal:direct· not yet calibratedTests✓ Asserted: passes✓ Checkable: passes? Load-bearing: unclear✓ Based: passes- Supported
A result the paper reports from its own data — it cites no other source for it, so there is none to check. The quote shows it is in the paper.
In the paper
“Rash occurred in 22 patients (10.9%) in the elexacaftor–tezacaftor–ivacaftor group and 13 patients (6.5%) in the placebo group.”
§ Elexacaftor–Tezacaftor–Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele > RESULTS > SAFETY
Strength:plain· not yet calibratedn = 22Tests✓ Asserted: passes✓ Checkable: passes? Load-bearing: unclear✓ Based: passesA result the paper reports from its own data — it cites no other source for it, so there is none to check. The quote shows it is in the paper.
In the paper
“The majority of patients in the elexacaftor–tezacaftor–ivacaftor group had adverse events that were mild (33.2%) or moderate (50.5%) in severity.”
§ Elexacaftor–Tezacaftor–Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele > RESULTS > SAFETY
Strength:plain· not yet calibratedTests✓ Asserted: passes✓ Checkable: passes? Load-bearing: unclear✓ Based: passesA result the paper reports from its own data — it cites no other source for it, so there is none to check. The quote shows it is in the paper.
In the paper
“There were no relevant safety findings in other clinical or laboratory assessments.”
§ Elexacaftor–Tezacaftor–Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele > RESULTS > SAFETY
Strength:plain· not yet calibratedTests✓ Asserted: passes✓ Checkable: passes? Load-bearing: unclear✓ Based: passesA result the paper reports from its own data — it cites no other source for it, so there is none to check. The quote shows it is in the paper.
In the paper
“Adverse events of elevated aminotransferase levels occurred in 22 patients (10.9%) in the elexacaftor–tezacaftor–ivacaftor group and 8 patients (4.0%) in the placebo group.”
§ Elexacaftor–Tezacaftor–Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele > RESULTS > SAFETY
Strength:plain· not yet calibratedn = 22Tests✓ Asserted: passes✓ Checkable: passes? Load-bearing: unclear✓ Based: passes
- Probably not a substantial claim. 14
- It may state two separate claims as one. 22
The sources with a verdict support this claim; the rest weren’t checked yet, have no full text we could read, or their verdicts aren’t rated yet.
In the paper
“Pulmonary exacerbations are important clinical events associated with disease progression21-24; elexacaftor–tezacaftor–ivacaftor resulted in a lower rate of pulmonary exacerbations, including severe events leading to hospitalization or treatment with intravenous antibiotics, than placebo.”
§ Elexacaftor–Tezacaftor–Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele > DISCUSSION
What the sources say
- Not checked — no full text of the sourcecites [21] Amadori A, Antonelli A, Balteri I, Schreiber A, Bugiani M, De Rose V. Recurrent exacerbations affect FEV(1) decline in adult patients with cystic fibrosis. Respir Med 2009; 103: 407– 13. 19027279 source
- Not checked — no full text of the sourcecites [22] De Boer K, Vandemheen KL, Tullis E, Exacerbation frequency and clinical outcomes in adult patients with cystic fibrosis. Thorax 2011; 66: 680– 5. 21680566 source
- Supportedcites [23] Konstan MW, Wagener JS, Vandevanter DR, Risk factors for rate of decline in FEV1 in adults with cystic fibrosis. J Cyst Fibros 2012; 11: 405– 11. 22561369 PMC4086189 source
The Introduction identifies pulmonary exacerbations treated with IV antibiotics as a risk factor for FEV1 decline in children with cystic fibrosis. The Discussion says exacerbation frequency has been associated with increased risk of lung transplantation or death, although this study did not find it to be a statistically significant predictor of FEV1 decline in adults. (LLM, conf=0.88)
- Supportedcites [24] Sanders DB, Zhao Q, Li Z, Farrell PM. Poor recovery from cystic fibrosis pulmonary exacerbations is associated with poor long-term outcomes. Pediatr Pulmonol 2017; 52: 1268– 75. 28881091 PMC5639928 source
The Discussion describes pulmonary exacerbations treated with IV antibiotics as important clinical endpoints associated with progression of lung disease. The Introduction also links these events to worsening lung function, supporting the citing sentence’s background claim. (LLM, conf=0.91)
Strength:plain· not yet calibratedCausal:correlational· not yet calibratedTests✓ Asserted: passes✓ Checkable: passes✗ Load-bearing: fails✓ Based: passesProbably not a substantial claim. The paper’s conclusions may not depend on it.
The sources with a verdict support this claim; the rest weren’t checked yet, have no full text we could read, or their verdicts aren’t rated yet.
In the paper
“CFTR modulators treat the underlying cause of disease and have improved clinical outcomes in persons with specific CFTR mutations.9-12”
§ Elexacaftor–Tezacaftor–Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele > Abstract > CONCLUSIONS
What the sources say
- Supportedcites [9] Wainwright CE, Elborn JS, Ramsey BW, Lumacaftor–ivacaftor in patients with cystic fibrosis homozygous for Phe508del CFTR. N Engl J Med 2015; 373: 220– 31. 25981758 PMC4764353 source
The Discussion reports that lumacaftor–ivacaftor, designed to address the underlying cause by targeting CFTR, improved FEV1 and reduced pulmonary exacerbations in patients homozygous for Phe508del. The paper also reports improved clinical outcomes with ivacaftor in patients with certain gating mutations. (LLM, conf=0.92)
- Supportedcites [10] Ramsey BW, Davies J, McElvaney NG, A CFTR potentiator in patients with cystic fibrosis and the G551D mutation. N Engl J Med 2011; 365: 1663– 72. 22047557 PMC3230303 source
The Discussion reports that ivacaftor, a CFTR potentiator targeting CFTR dysfunction, significantly improved clinical outcomes in patients with at least one G551D-CFTR mutation. This paper supports the claim through that specific modulator and mutation. (LLM, conf=0.90)
- Not checked — no full text of the sourcecites [11] Taylor-Cousar JL, Munck A, McKone EF, Tezacaftor–ivacaftor in patients with cystic fibrosis homozygous for Phe508del. N Engl J Med 2017; 377: 2013– 23. 29099344 source
- Supportedcites [12] Rowe SM, Daines C, Ringshausen FC, Tezacaftor–ivacaftor in residual-function heterozygotes with cystic fibrosis. N Engl J Med 2017; 377: 2024– 35. 29099333 PMC6472479 source
The Introduction describes CFTR modulators as acting on the defective protein’s gating or processing. The Discussion reports improved clinical outcomes with tezacaftor–ivacaftor and ivacaftor in patients with specific CFTR mutations, supporting both parts of the claim. (LLM, conf=0.91)
Tests✓ Asserted: passes✓ Checkable: passes✓ Load-bearing: passes✓ Based: passesIt may state two separate claims as one.
- Supported
The cited sources support only part of this claim — read each source’s verdict below.
In the paper
“The efficacy outcomes confirm the hypothesis that elexacaftor–tezacaftor–ivacaftor effectively modulates the function of Phe508del CFTR from a single allele, providing pronounced benefits in a population of patients in whom previous CFTR modulator therapies were not effective.15,19”
§ Elexacaftor–Tezacaftor–Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele > DISCUSSION
What the sources say
- Not checked — no full text of the sourcecites [15] Rowe SM, McColley SA, Rietschel E, Lumacaftor/ivacaftor treatment of patients with cystic fibrosis heterozygous for F508del-CFTR. Ann Am Thorac Soc 2017; 14: 213– 9. 27898234 PMC5461999 source
- Partly supportedcites [19] Clancy JP. Rapid therapeutic advances in CFTR modulator science. Pediatr Pulmonol 2018; 53: Suppl 3: S4– S11. PMC6585954 30289627 sourceExperimental — accuracy not yet measured
Split into 2 atoms — 0 confirmed, 2 partially supported, 0 disputed, 0 misattributed, 0 unverifiable. Expand the claim to see per-atom verdicts.
Tests✓ Asserted: passes✓ Checkable: passes✓ Load-bearing: passes✓ Based: passesIt may state two separate claims as one.
The cited sources support only part of this claim — read each source’s verdict below.
In the paper
“The current benchmark for highly effective CFTR modulator therapy is ivacaftor for patients with the Gly551Asp CFTR mutation,10 in whom disease modification has been shown with long-term use, including decreased lung-function decline and decreased mortality.25-28”
§ Elexacaftor–Tezacaftor–Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele > DISCUSSION
What the sources say
- Supportedcites [10] Ramsey BW, Davies J, McElvaney NG, A CFTR potentiator in patients with cystic fibrosis and the G551D mutation. N Engl J Med 2011; 365: 1663– 72. 22047557 PMC3230303 source
Split into 2 atoms — 2 confirmed, 0 partially supported, 0 disputed, 0 misattributed, 0 unverifiable. Expand the claim to see per-atom verdicts.
- Not checked — no full text of the sourcecites [25] Sawicki GS, McKone EF, Pasta DJ, Sustained benefit from ivacaftor demonstrated by combining clinical trial and cystic fibrosis patient registry data. Am J Respir Crit Care Med 2015; 192: 836– 42. 26132840 source
- Not checked — no full text of the sourcecites [26] Volkova N, Moy K, Evans J, Disease progression in patients with cystic fibrosis treated with ivacaftor: data from national US and UK registries. J Cyst Fibros 2019 6 10 (Epub ahead of print). 31196670 source
- Not checked — no full text of the sourcecites [27] Rowe SM, Heltshe SL, Gonska T, Clinical mechanism of the cystic fibrosis transmembrane conductance regulator potentiator ivacaftor in G551D-mediated cystic fibrosis. Am J Respir Crit Care Med 2014; 190: 175– 84. 24927234 PMC4226057 source
- Partly supportedcites [28] Bessonova L, Volkova N, Higgins M, Data from the US and UK cystic fibrosis registries support disease modification by CFTR modulation with ivacaftor. Thorax 2018; 73: 731– 40. 29748252 PMC6204955 sourceExperimental — accuracy not yet measured
Split into 2 atoms — 1 confirmed, 1 partially supported, 0 disputed, 0 misattributed, 0 unverifiable. Expand the claim to see per-atom verdicts.
Tests✓ Asserted: passes✓ Checkable: passes✓ Load-bearing: passes✓ Based: passesIt may state two separate claims as one.
- Supported
The sources with a verdict support this claim; the rest weren’t checked yet, have no full text we could read, or their verdicts aren’t rated yet.
In the paper
“Cystic fibrosis is a lethal, inherited, autosomal recessive disorder that affects approximately 80,000 people worldwide and is caused by mutations in the gene encoding the cystic fibrosis transmembrane conductance regulator (CFTR) protein that lead to reduced CFTR function.1-3”
§ Elexacaftor–Tezacaftor–Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele > Abstract > CONCLUSIONS
What the sources say
- Not checked — no full text of the sourcecites [1] O’Sullivan BP, Freedman SD. Cystic fibrosis. Lancet 2009; 373: 1891– 904. 19403164 source
- Not checked — no full text of the sourcecites [2] Elborn JS. Cystic fibrosis. Lancet 2016; 388: 2519– 31. 27140670 source
- Supportedcites [3] Sanders DB, Fink AK. Background and epidemiology. Pediatr Clin North Am 2016; 63: 567– 84. 27469176 PMC4967225 source
The Introduction identifies mutations of the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) gene as causing errors that therapies for people with cystic fibrosis seek to correct. This supports the attributed claim about the genetic cause of CF. (LLM, conf=0.92)
Strength:plain· not yet calibratedCausal:direct· not yet calibratedTests✓ Asserted: passes✓ Checkable: passes✗ Load-bearing: fails✓ Based: passesProbably not a substantial claim. The paper’s conclusions may not depend on it.
The cited sources support only part of this claim — read each source’s verdict below.
In the paper
“Ivacaftor and tezacaftor–ivacaftor, two components of this triple combination, are approved therapies, with well-characterized safety profiles.10-12,30”
§ Elexacaftor–Tezacaftor–Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele > DISCUSSION
What the sources say
- Partly supportedcites [10] Ramsey BW, Davies J, McElvaney NG, A CFTR potentiator in patients with cystic fibrosis and the G551D mutation. N Engl J Med 2011; 365: 1663– 72. 22047557 PMC3230303 sourceExperimental — accuracy not yet measured
The Discussion reports that 48 weeks of ivacaftor was not associated with greater safety risk than placebo, supporting the ivacaftor safety portion. This paper does not establish approval status or address the safety of tezacaftor–ivacaftor; it describes ivacaftor as investigational at the time of the study. Page numbers were not supplied for these passages. (LLM, conf=0.88)
- Not checked — no full text of the sourcecites [11] Taylor-Cousar JL, Munck A, McKone EF, Tezacaftor–ivacaftor in patients with cystic fibrosis homozygous for Phe508del. N Engl J Med 2017; 377: 2013– 23. 29099344 source
- Partly supportedcites [12] Rowe SM, Daines C, Ringshausen FC, Tezacaftor–ivacaftor in residual-function heterozygotes with cystic fibrosis. N Engl J Med 2017; 377: 2024– 35. 29099333 PMC6472479 sourceExperimental — accuracy not yet measured
The Discussion reports that tezacaftor–ivacaftor was safe and well tolerated in this trial and that ivacaftor recapitulated its well-established safety profile; the Results > Safety section provides supporting adverse-event data. Our copy does not state that both treatments were approved therapies, so it does not support that part of the claim. (LLM, conf=0.85)
- Partly supportedcites [30] Habib AR, Kajbafzadeh M, Desai S, Yang CL, Skolnik K, Quon BS. A systematic review of the clinical efficacy and safety of CFTR modulators in cystic fibrosis. Sci Rep 2019; 9: 7234. 31076617 PMC6510767 sourceExperimental — accuracy not yet measured
The Discussion (page not provided) concludes that ivacaftor is safe for people with a G551D mutation and that tezacaftor–ivacaftor is safer than lumacaftor–ivacaftor in F508del-homozygous individuals. It also says long-term placebo-controlled effects remain unclear. Our copy supports safety findings in those studied groups, but does not establish that both therapies are approved or that their safety profiles are well characterized without those limits. (LLM, conf=0.85)
Strength:boosted· not yet calibratedTests✓ Asserted: passes✓ Checkable: passes✗ Load-bearing: fails✓ Based: passesProbably not a substantial claim. The paper’s conclusions may not depend on it. It may state two separate claims as one.
- Partly supported
A result the paper reports from its own data — it cites no other source for it, so there is none to check. The quote shows it is in the paper.
In the paper
“In the elexacaftor–tezacaftor–ivacaftor group, elevated levels of alanine aminotransferase or aspartate aminotransferase that were greater than three times, greater than five times, and greater than eight times the upper limit of the normal range occurred in 16 patients (7.9%), 5 patients (2.5%), and 3 patients (1.5%), respectively, as compared with 11 patients (5.5%), 3 patients (1.5%), and 2”
§ Elexacaftor–Tezacaftor–Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele > RESULTS > SAFETY
Strength:plain· not yet calibratedn = 16Tests✓ Asserted: passes✓ Checkable: passes? Load-bearing: unclear✓ Based: passesIt may state two separate claims as one.
A result the paper reports from its own data — it cites no other source for it, so there is none to check. The quote shows it is in the paper.
In the paper
“Sustained improvement in percentage of predicted FEV1 was seen through week 24 (final analysis), with a mean treatment difference of 14.3 points relative to placebo (P<0.001) (Table 2 and Fig. 1A).”
§ Elexacaftor–Tezacaftor–Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele > RESULTS > EFFICACY
Tests✓ Asserted: passes✓ Checkable: passes✓ Load-bearing: passes✓ Based: passesIt may state two separate claims as one.
A result the paper reports from its own data — it cites no other source for it, so there is none to check. The quote shows it is in the paper.
In the paper
“Serious adverse events occurred in 28 patients (13.9%) in the elexacaftor–tezacaftor–ivacaftor group and 42 patients (20.9%) in the placebo group (Table 3 and Table S6); excluding serious adverse events of pulmonary exacerbation, serious adverse events occurred in 20 patients (9.9%) in the elexacaftor–tezacaftor–ivacaftor group and 16 patients (8.0%) in the placebo group.”
§ Elexacaftor–Tezacaftor–Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele > RESULTS > SAFETY
Tests✓ Asserted: passes✓ Checkable: passes✓ Load-bearing: passes✓ Based: passesIt may state two separate claims as one.
A result the paper reports from its own data — it cites no other source for it, so there is none to check. The quote shows it is in the paper.
In the paper
“Two patients (1.0%) in the elexacaftor–tezacaftor–ivacaftor group discontinued the trial regimen because of adverse events: rash in 1 patient and portal hypertension in a patient with preexisting cirrhosis.”
§ Elexacaftor–Tezacaftor–Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele > RESULTS > SAFETY
Strength:plain· not yet calibratedn = 2Tests✓ Asserted: passes✓ Checkable: passes? Load-bearing: unclear✓ Based: passesIt may state two separate claims as one.
A result the paper reports from its own data — it cites no other source for it, so there is none to check. The quote shows it is in the paper.
In the paper
“The baseline mean systolic and diastolic blood pressures in the elexacaftor–tezacaftor–ivacaftor group were 113.4 mm Hg and 69.4 mm Hg, and they increased by 3.1 mm Hg and 1.9 mm Hg, respectively, at week 24 (Table S9).”
§ Elexacaftor–Tezacaftor–Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele > RESULTS > SAFETY
Strength:plain· not yet calibratedCausal:direct· not yet calibratedTests✓ Asserted: passes✓ Checkable: passes? Load-bearing: unclear✓ Based: passesIt may state two separate claims as one.
A result the paper reports from its own data — it cites no other source for it, so there is none to check. The quote shows it is in the paper.
In the paper
“In both trial groups, rash was more common in female patients than in male patients and more common in female patients who used hormonal contraceptives than in those who did not (Table S7).”
§ Elexacaftor–Tezacaftor–Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele > RESULTS > SAFETY
Strength:plain· not yet calibratedCausal:correlational· not yet calibratedTests✓ Asserted: passes✓ Checkable: passes✗ Load-bearing: fails✓ Based: passesProbably not a substantial claim. The paper’s conclusions may not depend on it. It may state two separate claims as one.
A result the paper reports from its own data — it cites no other source for it, so there is none to check. The quote shows it is in the paper.
In the paper
“The percentage of patients with at least one adverse event was 93.1% in the elexacaftor–tezacaftor–ivacaftor group and 96.0% in the placebo group; excluding adverse events of pulmonary exacerbation, the percentage was 92.6% in the elexacaftor–tezacaftor–ivacaftor group and 93.0% in the placebo group.”
§ Elexacaftor–Tezacaftor–Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele > RESULTS > SAFETY
Strength:plain· not yet calibratedTests✓ Asserted: passes✓ Checkable: passes? Load-bearing: unclear✓ Based: passesIt may state two separate claims as one.
A result the paper reports from its own data — it cites no other source for it, so there is none to check. The quote shows it is in the paper.
In the paper
“Elevated levels of creatine kinase were often associated with exercise, and no elevations of creatine kinase led to discontinuation of the trial regimen (Table S8).”
§ Elexacaftor–Tezacaftor–Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele > RESULTS > SAFETY
Strength:plain· not yet calibratedCausal:correlational· not yet calibratedTests✓ Asserted: passes✓ Checkable: passes? Load-bearing: unclear✓ Based: passesIt may state two separate claims as one.
A result the paper reports from its own data — it cites no other source for it, so there is none to check. The quote shows it is in the paper.
In the paper
“Additional observations included elevated levels of creatine kinase and blood-pressure changes in the elexacaftor–tezacaftor–ivacaftor group.”
§ Elexacaftor–Tezacaftor–Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele > RESULTS > SAFETY
Strength:plain· not yet calibratedTests✓ Asserted: passes✓ Checkable: passes? Load-bearing: unclear✓ Based: passesIt may state two separate claims as one.
The paper states this without a source or data behind it here, so there is nothing to check it against. The quote shows it is in the paper.
In the paper
“The elevated serum levels of creatine kinase that were observed were generally asymptomatic and often associated with exercise.”
§ Elexacaftor–Tezacaftor–Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele > DISCUSSION
Strength:plain· not yet calibratedCausal:correlational· not yet calibratedTests✓ Asserted: passes✓ Checkable: passes✗ Load-bearing: fails✓ Based: passesProbably not a substantial claim. The paper’s conclusions may not depend on it. It may state two separate claims as one.
A result the paper reports from its own data — it cites no other source for it, so there is none to check. The quote shows it is in the paper.
In the paper
“An increase in the incidence of elevated aminotransferase levels, which occur sporadically in many persons with cystic fibrosis, was observed with elexacaftor–tezacaftor–ivacaftor treatment; these adverse events were low grade (i.e., mild or moderate) in 20 of 22 patients (91%) and were not treatment-limiting.”
§ Elexacaftor–Tezacaftor–Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele > DISCUSSION
Tests✓ Asserted: passes✓ Checkable: passes? Load-bearing: unclear✓ Based: passesIt may state two separate claims as one.
The paper states this without a source or data behind it here, so there is nothing to check it against. The quote shows it is in the paper.
In the paper
“In general, cases of rash were mild to moderate and did not lead to alteration of treatment administration.”
§ Elexacaftor–Tezacaftor–Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele > DISCUSSION
Strength:plain· not yet calibratedTests✓ Asserted: passes✓ Checkable: passes? Load-bearing: unclear✓ Based: passesIt may state two separate claims as one.
What the paper did — it cites no source for this, so there is none to check. The quote shows it is in the paper.
In the paper
“A Lan–DeMets alpha-spending function was applied to control the overall type I error rate of 0.05 for the primary end point.”
§ Elexacaftor–Tezacaftor–Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele > METHODS > STATISTICAL ANALYSIS
Load-bearingStrength:plain· not yet calibratedTests✓ Asserted: passes✓ Checkable: passes✓ Load-bearing: passes✓ Based: passesProbably not a substantial claim.
In the paper
“FEV1 is a strong predictor of clinical status in cystic fibrosis,20 and elexacaftor–tezacaftor–ivacaftor resulted in sustained improvements in this end point.”
§ Elexacaftor–Tezacaftor–Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele > DISCUSSION
What the sources say
- Not checked — no full text of the sourcecites [20] Kerem E, Reisman J, Corey M, Canny GJ, Levison H. Prediction of mortality in patients with cystic fibrosis. N Engl J Med 1992; 326: 1187– 91. 1285737 source
Tests✓ Asserted: passes✓ Checkable: passes✓ Load-bearing: passes✓ Based: passesProbably not a substantial claim.
What the paper did — it cites no source for this, so there is none to check. The quote shows it is in the paper.
In the paper
“Patients were randomly assigned in a 1:1 ratio to receive elexacaftor (200 mg once daily) in triple combination with tezacaftor (100 mg once daily) and ivacaftor (150 mg every 12 hours) or matched placebos.”
§ Elexacaftor–Tezacaftor–Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele > METHODS > PARTICIPANTS, TRIAL DESIGN, AND OVERSIGHT
Load-bearingStrength:plain· not yet calibratedTests✓ Asserted: passes✓ Checkable: passes✓ Load-bearing: passes✓ Based: passesProbably not a substantial claim.
What the paper did — it cites no source for this, so there is none to check. The quote shows it is in the paper.
In the paper
“A prespecified interim analysis was conducted for the primary end point when at least 140 patients had completed the week 4 visit and at least 100 patients had completed the week 12 visit.”
§ Elexacaftor–Tezacaftor–Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele > METHODS > STATISTICAL ANALYSIS
Strength:plain· not yet calibratedn = 140Tests✓ Asserted: passes✓ Checkable: passes✗ Load-bearing: fails✓ Based: passesProbably not a substantial claim. The paper’s conclusions may not depend on it.
What the paper did — it cites no source for this, so there is none to check. The quote shows it is in the paper.
In the paper
“Overall, 405 patients underwent randomization, and 403 received at least one dose of the trial regimen (200 in the elexacaftor–tezacaftor–ivacaftor group and 203 in the placebo group) (Fig. S2).”
§ Elexacaftor–Tezacaftor–Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele > RESULTS > POPULATION
Strength:plain· not yet calibratedn = 405Tests✓ Asserted: passes✓ Checkable: passes? Load-bearing: unclear✓ Based: passesIt may state two separate claims as one.
What the paper did — it cites no source for this, so there is none to check. The quote shows it is in the paper.
In the paper
“This phase 3, multicenter, randomized, double-blind, placebo-controlled trial of elexacaftor–tezacaftor–ivacaftor involved patients 12 years of age or older with cystic fibrosis and Phe508del-minimal function genotypes.”
§ Elexacaftor–Tezacaftor–Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele > METHODS > PARTICIPANTS, TRIAL DESIGN, AND OVERSIGHT
Load-bearingStrength:plain· not yet calibratedTests✓ Asserted: passes✓ Checkable: passes✓ Load-bearing: passes✓ Based: passesIt may state two separate claims as one.
What the paper did — it cites no source for this, so there is none to check. The quote shows it is in the paper.
In the paper
“Assuming a 5% dropout rate at week 4 and a within-group standard deviation of 7 percentage points, we estimated that an interim-analysis sample size of 70 patients per trial group would provide approximately 98% power to detect a between-group difference of 5.0 points for the mean absolute change from baseline in percentage of predicted FEV1 at week 4, using a two-sided, two-sample t-test at a”
§ Elexacaftor–Tezacaftor–Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele > METHODS > STATISTICAL ANALYSIS
Strength:hedged· not yet calibratedn = 70Tests✓ Asserted: passes✓ Checkable: passes✗ Load-bearing: fails✓ Based: passesProbably not a substantial claim. The paper’s conclusions may not depend on it.
A claim is checked only against the cited paper’s full text, never its abstract, and none is available for its sources.
In the paper
“The Phe508del CFTR mutation causes defective intracellular processing and trafficking and decreased stability, which drastically reduces the quantity of CFTR protein at the apical surface of epithelial cells.4,7,8”
§ Elexacaftor–Tezacaftor–Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele > Abstract > CONCLUSIONS
What the sources say
- Not checked — no full text of the sourcecites [4] Rowe SM, Miller S, Sorscher EJ. Cystic fibrosis. N Engl J Med 2005; 352: 1992– 2001. 15888700 source
- Not checked — no full text of the sourcecites [7] Dalemans W, Barbry P, Champigny G, Altered chloride ion channel kinetics associated with the delta F508 cystic fibrosis mutation. Nature 1991; 354: 526– 8. 1722027 source
- Not checked — no full text of the sourcecites [8] Lukacs GL, Chang XB, Bear C, The delta F508 mutation decreases the stability of cystic fibrosis transmembrane conductance regulator in the plasma membrane: determination of functional half-lives on transfected cells. J Biol Chem 1993; 268: 21592– 8. 7691813 source
Tests✓ Asserted: passes✓ Checkable: passes✓ Load-bearing: passes✓ Based: passesIt may state two separate claims as one.
What the paper did — it cites no source for this, so there is none to check. The quote shows it is in the paper.
In the paper
“Randomization was performed in permuted blocks, with stratification according to percentage of predicted FEV1 at screening (<70% vs. ≥70%), age at screening (<18 years vs. ≥18 years), and sex.”
§ Elexacaftor–Tezacaftor–Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele > METHODS > PARTICIPANTS, TRIAL DESIGN, AND OVERSIGHT
Strength:plain· not yet calibratedTests✓ Asserted: passes✓ Checkable: passes✗ Load-bearing: fails✓ Based: passesProbably not a substantial claim. The paper’s conclusions may not depend on it.
What the paper did — it cites no source for this, so there is none to check. The quote shows it is in the paper.
In the paper
“The mean adherence to the trial regimen was more than 98% in both trial groups.”
§ Elexacaftor–Tezacaftor–Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele > RESULTS > POPULATION
Strength:plain· not yet calibratedTests✓ Asserted: passes✓ Checkable: passes✗ Load-bearing: fails✓ Based: passesProbably not a substantial claim. The paper’s conclusions may not depend on it.
In the paper
“Phe508del CFTR protein also exhibits defective channel gating, which further limits anion transport.7”
§ Elexacaftor–Tezacaftor–Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele > Abstract > CONCLUSIONS
What the sources say
- Not checked — no full text of the sourcecites [7] Dalemans W, Barbry P, Champigny G, Altered chloride ion channel kinetics associated with the delta F508 cystic fibrosis mutation. Nature 1991; 354: 526– 8. 1722027 source
Tests✓ Asserted: passes✓ Checkable: passes✓ Load-bearing: passes✓ Based: passesIt may state two separate claims as one.
In the paper
“Although there are hundreds of different disease-causing mutations, nearly 90% of persons with cystic fibrosis have at least one copy of the most common mutation, the Phe508del CFTR mutation.6”
§ Elexacaftor–Tezacaftor–Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele > Abstract > CONCLUSIONS
What the sources say
- Not checked yetcites [6] Marshall B, Faro A, Fink AK, Cystic fibrosis patient registry: 2017 annual data report. Bethesda, MD: Cystic Fibrosis Foundation, 2018.
Strength:plain· not yet calibratedTests✓ Asserted: passes✓ Checkable: passes✗ Load-bearing: fails✓ Based: passesProbably not a substantial claim. The paper’s conclusions may not depend on it.
What the paper did — it cites no source for this, so there is none to check. The quote shows it is in the paper.
In the paper
“Efficacy analyses included all patients who underwent randomization and received at least one dose of elexacaftor–tezacaftor–ivacaftor or placebo.”
§ Elexacaftor–Tezacaftor–Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele > METHODS > STATISTICAL ANALYSIS
Load-bearingStrength:plain· not yet calibratedTests✓ Asserted: passes✓ Checkable: passes✓ Load-bearing: passes✓ Based: passesProbably not a substantial claim.
A conclusion the paper draws from its own results — it cites no other source for it, so there is none to check. The quote shows it is in the paper.
In the paper
“Similar to these agents, elexacaftor–tezacaftor–ivacaftor therapy was associated with adverse events that were mostly mild to moderate, that generally represented common manifestations of cystic fibrosis, and that led to few treatment discontinuations.”
§ Elexacaftor–Tezacaftor–Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele > DISCUSSION
Tests✓ Asserted: passes✓ Checkable: passes✓ Load-bearing: passes✓ Based: passesIt may state two separate claims as one.
A conclusion the paper draws from its own results — it cites no other source for it, so there is none to check. The quote shows it is in the paper.
In the paper
“These results provide evidence that elexacaftor–tezacaftor–ivacaftor can modulate a single Phe508del allele in people with cystic fibrosis, thus addressing the underlying cause of disease in the large majority of patients.”
§ Elexacaftor–Tezacaftor–Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele > DISCUSSION
Tests✓ Asserted: passes✓ Checkable: passes✓ Load-bearing: passes✓ Based: passesIt may state two separate claims as one.
- Rests on an assumption: Sweat chloride concentration is a marker of CFTR activity.
The paper relies on this without support.
Sweat chloride concentration is a marker of CFTR activity.
“elexacaftor–tezacaftor–ivacaftor led to improvements in spirometry, patient-reported respiratory symptoms, and sweat chloride concentration, a marker of CFTR activity, in patients with cystic fibrosis with a single Phe508del allele.18”
Claims other papers make about this one
Each is checked against this paper’s own text: does it say what the citing paper says it does?
The source backs part of the claim, not all of it — or backs it for a different population, condition or comparison.
In the citing paper
“The clinical trials with Trikafta showed only partial normalization of the 60%–65% mean baseline ppFEV1 after 24 weeks of treatment in compound heterozygous F508del (16) or at 4 weeks in homozygous F508del patients (15).”
From Allosteric folding correction of F508del and rare CFTR mutants by elexacaftor-tezacaftor-ivacaftor (Trikafta) combination (2020) · cites this paper as [16]
Experimental — accuracy not yet measuredThe source backs part of the claim, not all of it — or backs it for a different population, condition or comparison.
In the citing paper
“The efficacy and safety of the triple combination of ELX, TEZ and IVA (ELX/TEZ/IVA) were established in two phase 3 pivotal trials in people with CF ≥12 years of age who were heterozygous for F508del and a minimal function mutation (F/MF) or had the F/F genotype [17–19].”
From Long-term safety and efficacy of elexacaftor/tezacaftor/ivacaftor in people with cystic fibrosis and at least one F508del allele: 144-week interim results from a 192-week open-label extension study (2023) · cites this paper as [18]
Experimental — accuracy not yet measuredThe source backs part of the claim, not all of it — or backs it for a different population, condition or comparison.
In the citing paper
“In both trials, ELX/TEZ/IVA treatment led to significant improvements in FEV1 % pred, CFQ-R respiratory domain score and sweat chloride concentration.”
From Long-term safety and efficacy of elexacaftor/tezacaftor/ivacaftor in people with cystic fibrosis and at least one F508del allele: 144-week interim results from a 192-week open-label extension study (2023) · cites this paper as [18]
Experimental — accuracy not yet measuredThe source backs part of the claim, not all of it — or backs it for a different population, condition or comparison.
In the citing paper
“Participants taking ELX/TEZ/IVA in the parent studies had steady and sustained increases in BMI [17, 18].”
From Long-term safety and efficacy of elexacaftor/tezacaftor/ivacaftor in people with cystic fibrosis and at least one F508del allele: 144-week interim results from a 192-week open-label extension study (2023) · cites this paper as [18]
Experimental — accuracy not yet measuredThe source backs part of the claim, not all of it — or backs it for a different population, condition or comparison.
In the citing paper
“During the first 144 weeks of this extension study, the estimated pulmonary exacerbation rate for patients with F/MF genotypes (0.20) was lower than in the ELX/TEZ/IVA arm of the 24-week parent study (0.37) [18] and the rate of pulmonary exacerbations for participants in both genotype groups was lower than in the previous 24-week interim analysis of this extension study (0.19 versus 0.30) [23].”
From Long-term safety and efficacy of elexacaftor/tezacaftor/ivacaftor in people with cystic fibrosis and at least one F508del allele: 144-week interim results from a 192-week open-label extension study (2023) · cites this paper as [18]
Experimental — accuracy not yet measured
The source backs part of the claim, not all of it — or backs it for a different population, condition or comparison.
In the citing paper
“Phase 3 clinical trials showed elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) was safe and efficacious in people with cystic fibrosis (CF) with ≥1 F508del-CFTR allele.”
From Real-world safety and effectiveness of elexacaftor/tezacaftor/ivacaftor in people with cystic fibrosis: Interim results of a long-term registry-based study (2023) · cites this paper as [8]
Experimental — accuracy not yet measuredThe cited source says what the claim attributes to it.
In the citing paper
“in which treatment with ELX/TEZ/IVA resulted in a 63% reduction in pulmonary exacerbations compared with placebo27”
The cited source says what the claim attributes to it.
In the citing paper
“In the concurrent phase 3 trial in pwCF in whom a single F508del was responsible for the treatment response, marked improvements in clinical outcomes substantiate the ability of ELX/TEZ/IVA to restore F508del-CFTR function.27”
The cited source says what the claim attributes to it.
In the citing paper
“In patients with the F/F genotype, these improvements were superior to those seen with TEZ/IVA.”
The cited source says what the claim attributes to it.
In the citing paper
“In the pivotal Studies 445-102 and 445-103, participants who received ELX/TEZ/IVA had rapid and statistically significant increases in FEV1 % pred and CFQ-R respiratory domain score along with significant decreases in sweat chloride concentration [17, 18].”
The cited source says what the claim attributes to it.
In the citing paper
“However, clinical studies of ELX/TEZ/IVA completed before the start of the pandemic reported similar robust decreases in the frequency of PEx [ 8-10 , 12 ].”
In the citing paper
“A triple combination regimen of elexacaftor (ELX)/TEZ/IVA was shown to be safe and efficacious in patients aged ≥12 years with CF and heterozygous for F508del-CFTR and a minimal function mutation ( F /MF genotypes; Study 445-102) or homozygous for F508del-CFTR ( F/F genotype; Study 445-103) [ 7 , 8 ].”
From EFFECT OF ELEXACAFTOR/TEZACAFTOR/IVACAFTOR ON ANNUAL RATE OF LUNG FUNCTION DECLINE IN PEOPLE WITH CYSTIC FIBROSIS (2023) · cites this paper as [7]
In the citing paper
“the improvements in ppFEV 1 by genotype observed in this study were broadly consistent with what has been reported in ELX/TEZ/IVA Phase 3 clinical trials (14.3, 10.0, and 3.7 percentage points in people with an F /MF genotype, F/F genotype, and F /G and F /RF genotypes, respectively) [ 8 , 9 , 17 ].”
Citations
Cites
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Update on Key Emerging Challenges in Cystic Fibrosis
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A CFTR Potentiator in Patients with Cystic Fibrosis and the G551D Mutation
Ramsey, B. W., Davies, J., McElvaney, N. G., Tullis, E., Bell, S. C., Dřevínek, P., Griese, M., McKone, E. F., Wainwright, C. E., Konstan, M. W., et al. (2011) · New England Journal of Medicine · doi:10.1056/nejmoa1105185 · Cited by 2,195
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Lumacaftor–Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del CFTR
Wainwright, C. E., Elborn, J. S., Ramsey, B. W., Marigowda, G., Huang, X., Cipolli, M., Colombo, C., Davies, J. C., De Boeck, K., Flume, P. A., et al. (2015) · New England Journal of Medicine · doi:10.1056/nejmoa1409547 · Cited by 1,622
Self-citationHeijerman, H. G. M., McKone, E. F., Downey, D. G., Van Braeckel, E., Rowe, S. M., Tullis, E., Mall, M. A., Welter, J. J., Ramsey, B. W., McKee, C. M., et al. (2019) · The Lancet · doi:10.1016/s0140-6736(19)32597-8 · Cited by 1,414
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Perkins, R. C., Shah, M. A., & Sawicki, G. S. (2021) · Pediatric Pulmonology · doi:10.1002/ppul.25251 · Cited by 13
Optical Measurements of Sweat for in Vivo Quantification of CFTR Function in Individual Sweat Glands
Treggiari, D., Kleinfelder, K., Bertini, M., Tridello, G., Fedrigo, A., Pintani, E., Iansa, P., Casiraghi, A., Minghetti, P., Cipolli, M., et al. (2021) · Journal of Cystic Fibrosis · doi:10.1016/j.jcf.2021.03.003 · Cited by 13
Goeckeler-Fried, J. L., Denny, R. A., Joshi, D., Hill, C., Larsen, M. B., Chiang, A., Frizzell, R. A., Wipf, P., Sorscher, E. J., & Brodsky, J. L. (2021) · Bioorganic & Medicinal Chemistry Letters · doi:10.1016/j.bmcl.2021.128243 · Cited by 13
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Thambuluru, S. R., Kyazimzade, S., Despotes, K. A., Kirk, D., & Goralski, J. L. (2021) · Journal of Cystic Fibrosis · doi:10.1016/j.jcf.2021.11.013 · Cited by 13
Beswick, D. M., Humphries, S. M., Miller, J. E., Balkissoon, C. D., Khatiwada, A., Vladar, E. K., Ramakrishnan, V. R., Lynch, D. A., & Taylor‐Cousar, J. L. (2022) · International Forum of Allergy & Rhinology · doi:10.1002/alr.23016 · Cited by 13
Self-citationLaselva, O., Criscione, M. L., Allegretta, C., Gioia, S. D., Liso, A., & Conese, M. (2022) · Frontiers in Molecular Biosciences · doi:10.3389/fmolb.2022.905468 · Cited by 13
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Metadata
- Authors
- Middleton, P. G., Mall, M. A., Dřevínek, P., Lands, L. C., McKone, E. F., Polineni, D., Ramsey, B. W., Taylor-Cousar, J. L., Tullis, E., Vermeulen, F., Marigowda, G., McKee, C. M., Moskowitz, S. M., Nair, N., Savage, J., Simard, C., Tian, S., Waltz, D., Xuan, F., Rowe, S. M., & Jain, R.
- Affiliations
- The University of Sydney (AU)
- From the Department of Respiratory and Sleep Medicine, Westmead Hospital and CF Research Group, Ludwig Engel Centre for Respiratory Research, Westmead Institute for Medical Research, University of Sydney, Westmead, NSW, Australia (P.G.M.); the Department of Pediatric Pulmonology, Immunology, and Intensive Care Medicine, Charité–Universitätsmedizin Berlin, the Berlin Institute of Health, and the German Center for Lung Research, Berlin (M.A.M.); the Department of Medical Microbiology, Department of...
- University College Dublin (IE)
- ORCID
- Venue
- New England Journal of Medicine
- Publisher
- Massachusetts Medical Society
- Published
- 2019-11-07
- Volume
- 381
- Issue
- 19
- Pages
- 1809-1819
- ISSN
- 0028-4793 (ISSN-L)
- 1533-4406
- PMC
- PMC7282384
- OpenAlex
- W2985405196
- Cited by
- 2,198
- References
- 38
- Citations by year
- 2026: 273 · 2025: 357 · 2024: 336 · 2023: 349 · 2022: 350 · 2021: 294 · 2020: 222 · 2019: 13
- FWCI
- 161.15
- Type
- journal article
- Language
- en
- Open access
- bronze
- doi.org
- Locations
- New England Journal of Medicine (publishedVersion)
- PubMed (publishedVersion)
- PubMed Central (acceptedVersion)
- Lirias (submittedVersion, other-oa)
- Data Archiving and Networked Services (DANS) (submittedVersion)
- Ghent University Academic Bibliography (Ghent University) (submittedVersion)
- VUBIR (Vrije Universiteit Brussel) (submittedVersion)
- Funders
- Vertex Pharmaceuticals Incorporated
- Topics
- Health Sciences › Medicine › Pulmonary and Respiratory Medicine › Cystic Fibrosis Research Advances
- Health Sciences › Medicine › Pulmonary and Respiratory Medicine › Inhalation and Respiratory Drug Delivery
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Genetic and Kidney Cyst Diseases
- Keywords
- elexacaftor/tezacaftor/ivacaftor, CFTR correctors, F508del mutation, CFTR function, FEV1, pulmonary exacerbations, sweat chloride, Cystic Fibrosis Questionnaire-Revised, respiratory quality of life, CFTR modulator therapy, drug safety
- MeSH terms
- Adolescent, Adult, Aminophenols, Child, Chlorides, Cystic Fibrosis, Double-Blind Method, Drug Combinations, Female, Forced Expiratory Volume, Genotype, Humans, Indoles, Male, Mutation, Pyrazoles, Pyridines, Pyrrolidines, Sweat, Quinolones, Cystic Fibrosis Transmembrane Conductance Regulator, Benzodioxoles, Young Adult, Chloride Channel Agonists
About these checks
Registry, retraction, Cites and statistics results are computed, not judged, with one exception: a printed reference that no index lists is read once by a model before it counts as a citation error. Claims are extracted by a model and kept only when their quote appears verbatim in the text; each cited source is then checked, with a model’s help, for whether it says what the claim relies on. Those verdicts can be wrong — read the claim’s quote and the cited source before relying on one.
A language model drew the claims from the paper on 2026-11-04, under version 3 of the claim definition (model deepseek-v4.1-flash, prompt extract-v3).
A second model labelled each claim on 2026-11-04 (model jev-1.13-20260917, definition revision gold-guideline-v0.2).
Cited sources are checked with a language model’s help (gpt-6-sol).
Statistics and arithmetic are recomputed, not judged.
Confidence bands are shown only for fields calibrated on our annotated test set. None is calibrated yet, so no band is shown.
What counts as a claim ›How we measure confidence ›How we check this ›
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