Immunogenicity, efficacy and safety of Nuwiq<sup>®</sup> (human‐cl rh<scp>FVIII</scp>) in previously untreated patients with severe haemophilia A—Interim results from the NuProtect Study
Bibliographic record
Abstract
Introduction Nuwiq ® (Human‐cl rh FVIII ) is a fourth generation recombinant FVIII , produced in a human cell line, without chemical modification or protein fusion. No inhibitors developed in studies with Nuwiq ® in 201 previously treated patients with haemophilia A ( HA ). The immunogenicity, efficacy and safety of Nuwiq ® in previously untreated patients ( PUP s) with severe HA are being assessed in the ongoing NuProtect study. Methods The study, conducted across 38 centres worldwide, is evaluating 110 true PUP s of all ages and ethnicities enrolled for study up to 100 exposure days ( ED s) or 5 years maximum. The primary objective is to assess the immunogenicity of Nuwiq ® (inhibitor activity ≥0.6 BU ) using the Nijmegen‐modified Bethesda assay at a central laboratory. Results Data for 66 PUP s with ≥20 ED s from a preplanned interim analysis were analysed. High‐titre ( HT ) inhibitors developed in 8 of 66 patients after a median of 11.5 ED s (range 6‐24). Five patients developed low‐titre inhibitors (4 transient). The cumulative incidence (95% confidence interval) was 12.8% (4.5%, 21.2%) for HT inhibitors and 20.8% (10.7%, 31.0%) for all inhibitors. During inhibitor‐free periods, median annualized bleeding rates during prophylaxis were 0 for spontaneous bleeds and 2.40 for all bleeds. Efficacy was rated as “excellent” or “good” in treating 91.8% of bleeds. Efficacy of surgical prophylaxis was “excellent” or “good” for 8 (89%) procedures and “moderate” for 1 (11%). No tolerability concerns were evident. Conclusion These interim data show a cumulative incidence of 12.8% for HT inhibitors and convincing efficacy and tolerability in PUP s treated with Nuwiq ® .
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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 | 0.001 | 0.002 |
| Meta-epidemiology (narrow) | 0.001 | 0.000 |
| Meta-epidemiology (broad) | 0.001 | 0.000 |
| Bibliometrics | 0.000 | 0.000 |
| Science and technology studies | 0.001 | 0.001 |
| Scholarly communication | 0.000 | 0.000 |
| Open science | 0.001 | 0.001 |
| Research integrity | 0.000 | 0.001 |
| 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".