Recombinant porcine sequence factor <scp>VIII</scp> (rp<scp>FVIII</scp>) for acquired haemophilia A: practical clinical experience of its use in seven patients
Bibliographic record
Abstract
Introduction A recombinant porcine factor VIII B‐domain‐deleted product (rp FVIII ; OBIZUR , Baxalta Incorporated, Deerfield, IL 60015, USA ) was recently approved for treatment of bleeding episodes in adults with acquired haemophilia A ( AHA ) in the United States. To date, no clinical experience outside the registration study has been reported. Aim To describe early clinical experience using rp FVIII for AHA . Methods A retrospective chart review of seven patients with AHA treated with rp FVIII at four institutions from November 2014 to October 2015. Results The time to diagnosis of AHA ranged from 5 days to 6 weeks. Six major and one other bleed were treated with rp FVIII following unsatisfactory bypassing agent ( BPA ) therapy. Good haemostatic efficacy was seen in five of seven cases. rp FVIII loading doses of 100 ( n = 6) or 200 U kg −1 ( n = 1) increased FVIII activity from <1 to 9% at baseline to 109–650% within 0.25–7 h in six of seven cases. Subsequent median doses ranged from 30 to 100 U kg −1 for 3–26 days. No rp FVIII ‐related adverse events were reported. Three patients survived with inhibitor eradication, one with persistent inhibitor, two died with inhibitors present and one was discharged and later died from unrelated causes. Conclusions rp FVIII showed good haemostatic efficacy with no recurrences in most cases, with consumption substantially less than in the registration study. Treatment decisions were based on FVIII activity levels and clinical assessment. The ability to titrate rp FVIII dose using FVIII activity was considered advantageous compared with BPA therapy. Notable delays in diagnosis were observed.
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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 | 0.026 |
| Meta-epidemiology (narrow) | 0.001 | 0.000 |
| Meta-epidemiology (broad) | 0.002 | 0.001 |
| Bibliometrics | 0.001 | 0.001 |
| Science and technology studies | 0.000 | 0.001 |
| Scholarly communication | 0.000 | 0.001 |
| Open science | 0.001 | 0.000 |
| Research integrity | 0.001 | 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".