PROPHYLAXIS FOR INHERITED DEFICIENCY OF FACTOR X (PROTECT-X): A SYSTEMATIC REVIEW
Bibliographic record
Abstract
Hereditary factor X (FX) deficiency (FXD) is a rare autosomal recessive bleeding disorder. The reduced or absent plasma FX clotting activity leads to spontaneous hemorrhages or bleeds after minor trauma. FXD severity varies among mild (FX 6%‒10%), moderate (FX 1%‒5%), and severe (FX < 1%), and the bleeding risk is inversely proportional to the residual plasma FX clotting activity. Therapy is based on treating (episodic) or avoiding (prophylaxis) bleeds with clotting agents, such as fresh frozen plasma, Prothrombin Complex Concentrate (PCC) or partially activated PCC, and exogenous FX replacement. We performed a literature systematic review to evaluate the efficacy/effectiveness and safety of prophylaxis against bleeds in people with FXD (PwFXD) compared to exclusive episodic treatment or prophylaxis with a different clotting agent. The systematic review was registered in PROSPERO (CRD42024535021) and is under conduction based on the PRISMA 2020 statement. Literature assessment was performed on 06/05/2024 based on strategic search directed to specific databases (PubMed, Embase, and LILACS). No language nor publication period were used as restrictions. Randomized controlled trials, non-randomized controlled trials, observational studies (prospective cohort, retrospective cohort, and cross-sectional), and case reports that evaluated the prophylaxis in PwFXD were included. Any publication reporting on prophylaxis in PwFXD was included. Reports on prophylaxis during surgical procedures were excluded. In addition, prophylaxis-referenced citations from the included publications and from the excluded reviews published within the previous 10 years were evaluated for eligibility. In addition to metadata, we will extract data on bleeds, clotting agent consumption, quality of life, and adverse events (e.g., death, discontinuation of treatment, and development of anti-FX antibodies). Meta-analysis will be performed when feasible. Study quality and risk of bias evaluation will be performed using the Newcastle-Ottawa Scale. The literature database search and the manual citations search retrieved 4,409 and 134 records, respectively. After selection according to the inclusion and exclusion criteria, 26 publications were included. The included publications were published from 1992 to 2024, only in English. The studies were performed mainly in Europe and North America. Designs comprised interventional (6, 23.1%) studies, observational (9, 34.6%), and case reports/series (11, 42.3%). Of 413 participants described, 395 (96%) were PwFXD, of whom 117 (29%) received prophylaxis. Final data will be reported in the congress. It is expected that our findings contribute to the management of PwFXD.
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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 | 0.005 | 0.019 |
| Meta-epidemiology (narrow) | 0.001 | 0.001 |
| Meta-epidemiology (broad) | 0.007 | 0.005 |
| Bibliometrics | 0.007 | 0.008 |
| Science and technology studies | 0.000 | 0.001 |
| Scholarly communication | 0.002 | 0.002 |
| Open science | 0.002 | 0.001 |
| Research integrity | 0.002 | 0.001 |
| Insufficient payload (model declined to judge) | 0.005 | 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".