P133 Impact of elexacaftor/tezacaftor/ivacaftor on clinical outcomes and quality of life in children with cystic fibrosis aged 6–11 years in the real-world setting
Bibliographic record
Abstract
Introduction The TRAJECTORY Study, an ongoing real-world observational study, previously showed that elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) improves clinical outcomes and quality of life (QoL) in people with CF aged ≥12 years. We examine the effect of ELX/TEZ/IVA on health outcomes among children aged 6–11 years after 12 months of treatment. Methods Medical record data was collected from 42 sites (United Kingdom, Canada, Germany, Italy, Portugal, Spain) for 12 months prior to (baseline) and up to 5 years after ELX/TEZ/IVA initiation (follow-up). Outcomes included body mass index (BMI) BMI z-score, percent predicted forced expiratory volume in one second (ppFEV1), annualized event rates of pulmonary exacerbations (PEx) and hospitalizations, and QoL as measured by the Cystic Fibrosis Questionnaire-Revised (CFQ-R; Children Ages 6–11 [Interviewer-administered], Parents/Caregivers [proxy for children ages 6–11]). Descriptive analyses were stratified by prior CF transmembrane conductance regulator modulator (CFTRm) use and baseline ppFEV1. Results Among the 163 children aged 6–11 years who initiated ELX/TEZ/IVA between August 2019–June 2023 and had 12 months of follow-up, 49.7% (81/163) were female, 49.1% (80/163) were homozygous for F508del-CFTR, and 23.3% (38/163) had prior CFTRm use. At ELX/TEZ/IVA initiation, the mean (SD) age, BMI z-score, and ppFEV1 were 8.2 (1.6) years, -0.25 (0.85), and 95.2 (14.9). Most had ppFEV1 >90 (62.0%, 101/163) and ppFEV1 ≥70 to ≤90 (31.9%, 52/163). At 12 months, ELZ/TEZ/IVA treatment led to mean (SD) increases in BMI z-score (+0.14 [0.45]) and ppFEV1 (+8.7 percentage points [10.8]), and decreased annualized event rates of PEx (1.1 to 0.6) and hospitalizations (0.6 to 0.2). All CFQ-R domains showed improvement through 12 months regardless of prior CFTR modulator use. Domains with the most improvement observed in mean (SD) change for parents/caregivers were Weight (14.3 [36.0]), Respiratory Symptoms (11.7 [20.6]), Digestive Symptoms (10.0 [8.9]), Eating Problems (9.4 [25.6]), and Treatment Burden (9.4 [21.0]) (table 1); consistent improvements were reported for children ages 6–11 in all domains. Conclusions ELX/TEZ/IVA treatment showed improvement in function and nutritional status, and respiratory and non-respiratory CFQ-R domains for children aged 6–11 years after 12 months of treatment.
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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 | 0.001 | 0.004 |
| Meta-epidemiology (narrow) | 0.000 | 0.000 |
| Meta-epidemiology (broad) | 0.000 | 0.001 |
| Bibliometrics | 0.000 | 0.001 |
| Science and technology studies | 0.000 | 0.000 |
| Scholarly communication | 0.001 | 0.000 |
| Open science | 0.000 | 0.000 |
| Research integrity | 0.000 | 0.001 |
| Insufficient payload (model declined to judge) | 0.002 | 0.000 |
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".