Lung Volume Recruitment and Quality of Life in Duchenne Muscular Dystrophy: Secondary Analysis of the STEADFAST Randomized Controlled Trial
Bibliographic record
Abstract
Abstract Rationale Lung volume recruitment (LVR) is prescribed for children with Duchenne muscular dystrophy (DMD) to maintain chest wall compliance and assist airway clearance. Objective We aimed to determine if twice-daily LVR compared with standard treatment in children with DMD improved health-related quality of life (HRQOL) over 2 years. Methods A multicenter, assessor-blinded randomized controlled trial was conducted among boys aged 6–16 years with DMD with forced vital capacity >30% predicted to undergo conventional treatment or conventional treatment and manual LVR twice daily for 2 years. The outcome was HRQOL measured by two validated instruments (Pediatric Quality of Life Inventory [PedsQL] generic version 4.0 and PedsQL DMD version 3.0) every 6 months. Results Sixty-two boys (33 in the LVR group and 29 controls) with a median (IQR) age of 11.4 (9.4–13.4) years completed the study. Median (IQR) baseline HRQOL scores assessed by parent/caregiver PedsQL were 58.0 (41.9–67.0) and 55.2 (47.8–63.5) for the LVR and control groups. HRQOL trajectories were stable during the 2-year study period except in terms of parent communication, which improved. No statistically significant interactions were detected between visit and treatment group for any PedsQL outcomes. DMD PedsQL communication scores in the LVR group showed more positive change compared with controls. Treatment scores in the LVR group showed less positive change over time compared with controls; however, after correcting for multiple testing, the changes were no longer significant. Respiratory symptom rate did not differ between groups and was not associated with any HRQOL outcome. Conclusions There was no difference in HRQOL between the LVR and control groups after 2 years. The burden of LVR did not appear to adversely affect HRQOL.
Fetched live from OpenAlex and de-inverted. Abstracts are not stored in this database: the inverted indexes are 8.6 GB of the frame’s 9.3 GB of text, and the host has 13 GB free.
How this classification was reachedexpand
Full frame distilled prediction
Teacher imitationNot calibrated prevalence, not ground truth. Human validation pending. Learned from the 10,348 direct Codex labels and 10,348 direct Gemma labels. Candidate is the union of thresholded teacher heads; consensus is their intersection. These outputs are machine_predicted_unvalidated and are not human labels or direct frontier model labels.
Codex and Gemma teacher scores by category
| Category | Codex | Gemma |
|---|---|---|
| Metaresearch | 0.006 | 0.005 |
| Meta-epidemiology (narrow) | 0.000 | 0.000 |
| Meta-epidemiology (broad) | 0.003 | 0.002 |
| Bibliometrics | 0.000 | 0.002 |
| Science and technology studies | 0.000 | 0.002 |
| Scholarly communication | 0.000 | 0.000 |
| Open science | 0.000 | 0.000 |
| Research integrity | 0.000 | 0.000 |
| Insufficient payload (model declined to judge) | 0.000 | 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 teacher head, 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".