Improvements in health-related quality of life in people with cystic fibrosis ≥6 years of age treated with vanzacaftor/tezacaftor/deutivacaftor
Bibliographic record
Abstract
BACKGROUND: Vanzacaftor/tezacaftor/deutivacaftor (VNZ/TEZ/D-IVA) was shown to provide greater restoration of cystic fibrosis transmembrane conductance regulator (CFTR) function than prior CFTR modulators in people with cystic fibrosis (CF) aged ≥6 years. Here, we assessed the impact of VNZ/TEZ/D-IVA on health-related quality of life (HRQoL) in children, adolescents, and adults with CF. METHODS: Post-hoc analyses were conducted using VNZ/TEZ/D-IVA Phase 3 studies in adolescents and adults with F508del/minimal function variant (F/MF) genotypes (SKYLINE 102) or F/F and other responsive genotypes (SKYLINE 103) and children aged 6-11 years across all responsive genotypes (RIDGELINE 105). For participants aged ≥14 years, CF Questionnaire-Revised-8 Dimensions (CFQ-R-8D) utility scores, a CF-specific, preference-based scoring algorithm based on CFQ-R data, were compared for VNZ/TEZ/D-IVA and ELX/TEZ/IVA treatment through Week 52. For children aged 6-11 years, absolute changes in CFQ-R non-respiratory domain (CFQ-R non-RD) scores from baseline, following an ELX/TEZ/IVA run-in period, were assessed through Week 24. RESULTS: CFQ-R-8D utility scores were higher in adolescents and adults given VNZ/TEZ/D-IVA compared to ELX/TEZ/IVA (pooled 0.017; nominal P = 0.01); participants with F/MF genotypes had larger changes (0.031; nominal P = 0.004) compared to non-F/MF genotypes (0.008; nominal P = 0.35). Children treated with VNZ/TEZ/D-IVA had improvements in 6 out of 7 CFQ-R non-RD scores, ranging from 3.7 [95 % CI: 1.0, 6.3] points in emotional functioning to 7.2 [95 % CI: 3.8, 10.5] points in social functioning. CONCLUSIONS: VNZ/TEZ/D-IVA was associated with higher HRQoL in adolescents and adults aged ≥14 years compared to ELX/TEZ/IVA, with largest changes in participants with F/MF genotypes, and in children aged 6-11 years compared to ELX/TEZ/IVA baseline. These results highlight potential HRQoL benefits from greater CFTR function restoration with VNZ/TEZ/D-IVA.
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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 | 0.001 |
| Meta-epidemiology (narrow) | 0.000 | 0.000 |
| Meta-epidemiology (broad) | 0.001 | 0.001 |
| Bibliometrics | 0.000 | 0.000 |
| Science and technology studies | 0.000 | 0.000 |
| Scholarly communication | 0.000 | 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".