Editorial: Modern approaches to hemophilia management in children and adolescents
Bibliographic record
Abstract
Editorial on the Research Topic Modern approaches to hemophilia management in children and adolescentsHemophilia A and B are the most common severe bleeding disorders which are due to partial or total deficiency of coagulation factor VIII (FVIII) or factor IX (FIX), respectively.Depending on the severity of coagulation factor deficiency, the affected individuals may experience bleeding only with severe trauma or during major surgery (mild hemophilia) to spontaneous hemarthrosis, hematomas, and intracranial hemorrhages in the absence of any identifiable trauma (severe hemophilia) (1).Until recently, coagulation factor replacement therapy via plasma-derived factor concentrates or recombinant factor products has been the mainstay of hemophilia treatment, provided either prophylactically or on-demand to treat bleeding episodes.Despite the widespread availability of safe and effective replacement therapy, many individuals with hemophilia continue to experience significant burden from their treatment, breakthrough bleeding, and debilitating arthropathy.In addition, the relatively high rate of inhibitor development to factor products can cause major morbidity and mortality, leading to a considerable burden on patients, families, and healthcare systems (2).Over the past two decades, however, there has been remarkable progress in the management of hemophilia.Cloning of the genes encoding factor VIII and factor IX, together with advances in bioengineering have led to novel therapies that offer easier modes of administration, extended half-lives, and improved patient outcomes and quality of life.In addition, gene therapy is under investigation as the next frontier in hemophilia treatment, especially for hemophilia B (3, 4).While these advances are associated with improvement of patients' outcomes, they also result in many new observations and questions that were not previously encountered.Furthermore, access to these treatments remains limited to the minority of people with hemophilia worldwide.In this Research Topic, Urasiński et al. review the Polish national experience of switching prophylaxis factor replacement therapy from standard-dose prophylaxis with a plasma-derived FVIII to recombinant FVIII in previously-treated patients (PTP), as the result of the national tender.Authors aimed to address two important questions: safety of switching from a plasmaderived product to a recombinant product, and the effectiveness of PK-tailored dosing vs. standard-dose approach.While the national tender mandated the switching of the products, children and adolescents had the choice of standard-dose or PK-tailored approach.Following the switch, no patient developed high-titer or persistent inhibitor (two patients had low-titer but transient inhibitors).Curiously, patients who chose standard-dose prophylaxis had a lower
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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 | 0.002 | 0.012 |
| Meta-epidemiology (narrow) | 0.003 | 0.001 |
| Meta-epidemiology (broad) | 0.003 | 0.002 |
| Bibliometrics | 0.002 | 0.001 |
| Science and technology studies | 0.002 | 0.001 |
| Scholarly communication | 0.004 | 0.004 |
| Open science | 0.003 | 0.001 |
| Research integrity | 0.008 | 0.009 |
| Insufficient payload (model declined to judge) | 0.038 | 0.024 |
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".