Health-related quality of life in adults with hemophilia B after gene therapy with fidanacogene elaparvovec: results from the BENEGENE-2 trial
Bibliographic record
Abstract
BACKGROUND: BENEGENE-2 is an ongoing phase 3 trial of fidanacogene elaparvovec, an adeno-associated virus gene therapy vector delivering a high-activity factor (F)IX variant, FIX-R338L (FIX-Padua). OBJECTIVES: To assess patient-reported outcome measures in BENEGENE-2. METHODS: Eligible males (N = 45) with hemophilia B received a single fidanacogene elaparvovec infusion after ≥6 months of FIX prophylaxis. Participants completed assessments pre- and post-gene therapy, including the Hemophilia Quality of Life Questionnaire for Adults (Haem-A-QoL), Hemophilia Activities List (HAL), Patient Global Impression of Change-Hemophilia, Hemophilia Life Impacts Questionnaire, and European Quality of Life 5-Dimensions 5-levels. RESULTS: Post-gene therapy, more participants (64.4% vs 28.9%) were free from bleeds compared with pre-gene therapy. Overall, 64.4% of participants did not require exogenous FIX infusions post-gene therapy. Changes from pre- to week 52 post-gene therapy suggested improved health-related quality of life in the key secondary endpoints of the Haem-A-QoL Physical Health domain (mean [SD] score change, -7.7 [15.8]; P = .005) and the HAL Complex Lower Extremity Activities score (mean [SD] score change, 7.6 [19.6]; P = .024). Additionally, there was a mean (SD) improvement of -11.2 (9.1), P < .001, in the Haem-A-QoL total score, and 6.7 (10.9), P < .001, in the HAL total score. In the exploratory endpoints, changes in the European Quality of Life 5-Dimensions 5-levels index score (mean [SD] score change, 0.05 [0.10]; P = .003) and visual analog scale (mean [SD] score change, 5.8 [15.6]; P = .030) suggested improved health status. Overall, 56.1% of participants reported that their overall impression of life with hemophilia (Patient Global Impression of Change-Hemophilia) had "greatly improved." Improvements were observed in Hemophilia Life Impacts Questionnaire domains related to living with and treating hemophilia. CONCLUSION: Fidanacogene elaparvovec improved health-related quality of life and health and functional status in adults with hemophilia B.
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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) | 0.001 | 0.000 |
| Meta-epidemiology (broad) | 0.001 | 0.002 |
| Bibliometrics | 0.000 | 0.000 |
| Science and technology studies | 0.000 | 0.001 |
| Scholarly communication | 0.001 | 0.001 |
| Open science | 0.000 | 0.000 |
| Research integrity | 0.001 | 0.002 |
| Insufficient payload (model declined to judge) | 0.001 | 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".