Are Women Welcome in Haemophilia Trials?
Bibliographic record
Abstract
We read with interest the letter from Fedewa et al., focused on the inclusion of female participants in interventional clinical trial for haemophilia [1]. The specific needs of women and girls with haemophilia have long been overlooked, with recognition of women as affected by, rather than just carriers of, haemophilia only introduced in 2021 [2]. Care for women with haemophilia and carriers in our treatment centres has historically focused on pregnancy management, primarily to ensure early detection and management of male offspring. The specific needs of women and girls with haemophilia are often overlooked in clinical trials and treatment guidelines and therapeutic equivalency is often assumed between males and females in the absence of evidence. Indeed, even in the recent 2024 ISTH guidelines, the only reference to women with haemophilia is to state twice ‘Since haemophilia can also affect women, all the recommendations in this guideline, whether strong or conditional, also apply to women who have low plasma levels of FVIII or FIX and a propensity toward bleeding’ [3]. These two identical statements comprise only 0.5% of the total guideline word count (68/12,483 words), with no further tailoring of approaches to women and girls with haemophilia [3]. While the role of prophylaxis for males with haemophilia is now established, data on the use of prophylaxis for heavy menstrual bleeding (HMB) in women with haemophilia are lacking. As part of our work in the European Association for Haemophilia and Allied Disorders (EAHAD) we have recently undertaken a project similar to that described by Fedewa et al. Our review focused on examining the evidence base, if any, for the use of prophylaxis in women with haemophilia for the management of HMB (periodic prophylaxis). Our project outcomes complement that of Fedewa et al., highlighting not only the failure to enrol women in clinical trials in haemophilia but also the lack of female-specific entry criteria and outcomes in haemophilia trial design.
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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 | 0.124 | 0.492 |
| Meta-epidemiology (narrow) | 0.001 | 0.002 |
| Meta-epidemiology (broad) | 0.005 | 0.002 |
| Bibliometrics | 0.002 | 0.003 |
| Science and technology studies | 0.004 | 0.011 |
| Scholarly communication | 0.016 | 0.023 |
| Open science | 0.005 | 0.006 |
| Research integrity | 0.043 | 0.032 |
| Insufficient payload (model declined to judge) | 0.028 | 0.010 |
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".