S68 Long-term effects of elexacaftor/tezacaftor/ivacaftor on clinical outcomes and quality of life among adolescents and adults aged 12+ with cystic fibrosis: Interim results from the TRAJECTORY Study
Bibliographic record
Abstract
Introduction TRAJECTORY is an ongoing, observational, medical record (MR) abstraction study in the United Kingdom, Canada, Germany, Italy, Portugal and Spain among people with CF (PwCF) who initiated elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) August 2019 – June 2023. We assessed long-term effects of ELX/TEZ/IVA on clinical outcomes and quality of life (QoL) in PwCF aged ≥12 years with a minimum of 12 months and up to 36 months of ELX/TEZ/IVA treatment. Methods MR data from 42 sites were collected for 12 months before ELX/TEZ/IVA initiation (baseline) and up to 5 years after (follow-up). Outcomes included body mass index (BMI), percent predicted forced expiratory volume in one second (ppFEV1), annualized event rates of PEx and hospitalizations, and QoL (self-report using the Cystic Fibrosis Questionnaire-Revised [CFQ-R; Child 12–13, Teen-Adult 14+]). Descriptive analyses were stratified by prior CF transmembrane conductance regulator modulator (CFTRm) use and baseline ppFEV1. Outcomes were evaluated at 12 months, 24 months, and 36 months post-ELX/TEZ/IVA initiation. Results Of 508 PwCF aged ≥12 years with ≥12 months of follow-up, ~50.0% were female (49.6%, 252/508), homozygous for F508del-CFTR (50.0%, 254/508), and had prior CFTRm use (48.4%, 246/508). At ELX/TEZ/IVA initiation, mean (SD) age, ppFEV1, and BMI were 30.4 (12.8) years, 63.2 (24.0) and 21.5 (3.6) kg/m2. At initiation, 58.5% had ppFEV1 between 40 and 90 (ppFEV1 ≥40 to <70: 36.8%, 187/508; ppFEV1 ≥70 to ≤90: 21.7%, 110/508). After 36 months, ELX/TEZ/IVA treatment led to mean (SD) increases in BMI (+1.9 kg/m2 [2.2]) and ppFEV1 (+9.3 percentage points [10.1]), and decreases in annualized event rates of PEx (0.4 to 0.3) and hospitalizations (1.0 to 0.5). Regardless of prior CFTRm use, all CFQ-R domain mean scores showed improvement at 12 months, 24 months, and 36 months. The largest domain increases in mean (SD) absolute change from baseline at 36 months were Respiratory Symptoms (26.3 [19.3]), Health Perceptions (15.3 [18.3]), Physical Functioning (12.2 [19.6]), School Functioning (+12.0 [16.2]), Vitality (9.7 [17.6]) and Treatment Burden (9.1 [14.4]) (table 1). Conclusions ELX/TEZ/IVA treatment showed substantial benefits in clinical outcomes and QoL in the real-world setting with sustained improvement up to at least 36 months for PwCF aged ≥12 years.
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How this classification was reachedexpand
Full frame machine prediction
Teacher imitationNot calibrated prevalence, not ground truth. Human validation pending. The Gemma side is a direct model label for every work in the frame, read from the title-only record. The Codex side is a classifier learned from the 10,348 direct Codex labels and calibrated to design-weighted sample rates; fields without enough sample support carry no Codex call. Candidate is the union of the two sides; consensus is their intersection. These outputs are machine_predicted_unvalidated and are not human labels.
Distilled classifier scores by category (both heads)
| Category | Codex | Gemma |
|---|---|---|
| Metaresearch | 0.002 | 0.006 |
| Meta-epidemiology (narrow) | 0.000 | 0.000 |
| Meta-epidemiology (broad) | 0.001 | 0.002 |
| Bibliometrics | 0.001 | 0.001 |
| Science and technology studies | 0.000 | 0.000 |
| Scholarly communication | 0.001 | 0.001 |
| Open science | 0.000 | 0.001 |
| Research integrity | 0.000 | 0.001 |
| Insufficient payload (model declined to judge) | 0.005 | 0.001 |
Machine scores (provisional)
The two teacher heads of the student model, read on this work. A score orders the frame for review; it never asserts a category, and the validation status ships verbatim with every row.
Baseline scores from an immature model (maturity gate not passed, 7 training rounds). Scores rank; they never assert a category.
score_only:v0-immature-baseline · verbatim from the scoring run: score_only means the number may rank works, and no category label ships from itClassification
machine, unvalidatedMachine predicted; a candidate call from one source (direct Gemma or distilled Codex), not a consensus.
How this classification was reached, model by model and score by score, is at the end of the page under "How this classification was reached".