Long-term efficacy of A <sub>1</sub> -PI therapy in RAPID and its extension study
Bibliographic record
Abstract
Background RAPID ( NCT00261833 ) showed that augmentation therapy with alpha-1 proteinase inhibitor (A 1 -PI Z, Zemaira®, CSL Behring) slows lung density decline in A 1 -PI-deficient patients. Aim To assess clinical benefit over 48 months in subjects who completed RAPID and have data from ≥2 CT scans from the extension study to date ( NCT00670007 ). Methods In a randomized, controlled design, RAPID subjects received A 1 -PI Z or placebo for 24 months, after which non-US subjects entered the 24-month extension study and all received A 1 -PI Z. Changes in adjusted lung density P15 at total lung capacity were assessed by CT. Results Rate of lung density decline was significantly reduced with A 1 -PI Z vs. placebo and was similar (1–1.5 g/L/y) among A 1 -PI Z subjects in the blinded and open-label phases. In the ongoing extension study, the delayed-start cohort (placebo–A 1 -PI Z) had a reduced rate of decline very similar to the early-start cohort. Changes in adjusted lung density P15 in RAPID and its ongoing extension study Study Point estimate g/L/y (standard error) Group difference (g/L/y) p-value Early-start cohort Delayed-start cohort RAPID A1-PI Z (N=75) Placebo (N=64) Completed RAPID and enrolled in extension -1.51 (0.25) -2.26 (0.27) 0.75 0.021 RAPID A1-PI Z (N=53) Placebo (N=53) Completed RAPID and ≥2 CT scans from extension -1.36 (0.30) -1.94 (0.30) 0.58 0.086 Extension A1-PI Z (N=53) A1-PI Z (N=53) Completed RAPID and ≥2 CT scans from extension -1.06 (0.35) -0.97 (0.35) -0.10 0.576 P15=lung volume-adjusted 15th percentile of lung density Conclusions A 1 -PI Z showed clinical benefit over 4 years and at different intervention points (early or delayed). Early treatment may reduce overall progression of emphysema in A 1 -PI-deficient patients.
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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 | 0.004 | 0.002 |
| Meta-epidemiology (narrow) | 0.001 | 0.000 |
| Meta-epidemiology (broad) | 0.002 | 0.001 |
| Bibliometrics | 0.000 | 0.000 |
| Science and technology studies | 0.000 | 0.000 |
| Scholarly communication | 0.001 | 0.001 |
| Open science | 0.001 | 0.001 |
| Research integrity | 0.001 | 0.001 |
| Insufficient payload (model declined to judge) | 0.004 | 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".