Management Strategies in Persons with Inherited Hemophilia Requiring Antithrombotic Therapy: A Scoping Review
Notice bibliographique
Résumé
Abstract Background: Cardiovascular disorders (CVD) and venous thromboembolism (VTE) are increasingly reported in people with hemophilia (PWH) whose life expectancy has improved dramatically over the past decades. While management of thrombotic events among PWH requires navigating the delicate balance between recurrent ischemic events and hemorrhage, current practices are highly variable and largely informed by expert opinion. Objective: We sought to examine and summarize what is known about the management, practice variation and outcomes of PWH requiring antithrombotic therapy (ATT) for cardiovascular disorders (CVD) or venous thromboembolism (VTE). We also aimed to identify current knowledge and practice gaps in the management of CVD and VTE in PWH. Methods: We conducted a scoping review guided by Arksey and O'Malley (2002), and Levac et al.'s (2010) methodological frameworks. We also adhered to the Preferred Reporting Items for Systematic reviews and Meta-Analyses extension for Scoping Reviews (PRISMA-ScR). A search strategy, developed iteratively in collaboration with a medical librarian, was performed on MEDLINE, EMBASE, and Scopus from inception to May 3, 2021. Studies of any design, published in English or French, in full-text or abstract, were included if they reported on more than two patients with inherited hemophilia A or B requiring ATT for CVD or VTE. Following eligibility assessment in duplicate, data from each study was charted by two independent reviewers to report the type of research designs, population, geographical location, interventions, and outcomes. Conflicts were resolved by consensus. We conducted a descriptive analysis and narrative synthesis to identify common themes and knowledge gaps. Results: We screened 3929 articles and selected 38 studies for inclusion (23 full-text and 15 abstracts). Published studies were largely retrospective (28/38, 73.7%), single center (25/38, 65.8%), and from Europe (25/38, 65.8%). A total of 567 patients with hemophilia A or B were reported. Common indications for ATT use included: coronary artery disease (26/38, 68.4%), including a history of percutaneous coronary intervention or coronary artery bypass procedures (14/38, 36.8%), atrial fibrillation (13/38, 34.2%), VTE treatment or prevention (10/38, 26.3%), cerebrovascular disease (9/38, 23.7%) and valvular heart disease (7/38, 18.4%). ATT included single antiplatelet therapy (28/38, 73.7%), heparins (21/38, 55.3%), dual antiplatelet therapy (19/38, 50%), vitamin K antagonists (10/38, 26.3%), and direct oral anticoagulants (5/38, 13.2%). The use of hemostatic therapies, either on-demand or prophylactically, was reported in 36 studies (94.7%). Clotting factors concentrates were used in 35 studies (92.1%). Other agents included desmopressin (2/38, 5.2%) and emicizumab (1/38, 2.6%). Only 19 studies (50%) reported target factor levels (peak and/or trough) while on ATT. Outcomes related to bleeding while on ATT were described in 36 studies (94.7%). Bleeding events resulting in a change in hemostatic regimen occurred in 9 studies (25%), while bleeding resulting in a change or discontinuation of ATT occurred in 10 studies (27.8%). In 10 studies (26.3%), no pathologic bleeding was observed in PWH on ATT. Four key themes were generated iteratively to describe the observed wide practice variation in the management of PWH requiring ATT: (1) Deviation from established and evolving CVD/VTE-related standards of care; (2) Difficulty weighing competing bleeding and thrombotic risks; (3) Advocacy for individualized strategies and multidisciplinary care; and (4) Absence of high-quality and long-term data for PWH. Discussion and Conclusion: Our scoping review highlights unmet needs in the management of an aging population of PWH with increasing prevalence of CVD and VTE. Management patterns are inconsistent and diverge from those of non-hemophilic patients. Current literature provides limited information on key issues such as hemostatic regimen, target factor levels, bleeding events and long-term outcomes. Prospective, clinically actionable data are needed to inform optimal and evidence-based management strategies of CVD and VTE in PWH. Higher quality data should culminate in much needed guidelines co-produced with patient partners and medical experts in hemophilia, CVD, and VTE. Disclosures Khalife: Pfizer Canada: Honoraria, Research Funding; Canadian Hemophilia Society: Research Funding.
Récupéré en direct depuis OpenAlex et désinversé. Les résumés ne sont pas conservés dans cette base de données : les index inversés représentent 8,6 Go des 9,3 Go de texte de la base, et le serveur dispose de 13 Go libres.
Comment cette classification a été obtenuedéplier
Prédiction machine sur la base complète
Imitation des enseignantsNi prévalence calibrée, ni vérité terrain. Validation humaine à venir. Le volet Gemma est une étiquette directe du modèle pour chaque travail de la base, lue sur la notice réduite au titre. Le volet Codex est un classifieur appris des 10 348 étiquettes directes de Codex et calibré sur les taux pondérés de l'échantillon; les champs sans appui suffisant ne portent aucun appel Codex. Le mode candidate est l'union des deux volets; le consensus est leur intersection. Ces sorties portent le statut machine_predicted_unvalidated et ne sont pas des étiquettes humaines.
Scores du classifieur distillé par catégorie (deux têtes)
| Catégorie | Codex | Gemma |
|---|---|---|
| Métarecherche | 0,010 | 0,054 |
| Méta-épidémiologie (sens strict) | 0,001 | 0,001 |
| Méta-épidémiologie (sens large) | 0,005 | 0,006 |
| Bibliométrie | 0,013 | 0,011 |
| Études des sciences et des technologies | 0,001 | 0,001 |
| Communication savante | 0,003 | 0,003 |
| Science ouverte | 0,002 | 0,002 |
| Intégrité de la recherche | 0,002 | 0,001 |
| Charge utile insuffisante (le modèle a refusé de juger) | 0,003 | 0,000 |
Scores machine (provisoires)
Les deux têtes enseignantes du modèle étudiant, lues sur ce travail. Un score ordonne la base pour la relecture; il n'affirme jamais une catégorie, et le statut de validation accompagne chaque rangée tel quel.
Scores de référence d'un modèle non mature (critères de maturité non atteints, 7 itérations). Un score ordonne; il n'affirme jamais une catégorie.
score_only:v0-immature-baseline · tel quel depuis la passe de notation : score_only signifie que le nombre peut ordonner les travaux, et qu'aucune étiquette de catégorie n'en découleClassification
machine, non validéePrédiction automatique; un appel candidat d’une seule source (Gemma direct ou Codex distillé), pas un consensus.
Le détail, modèle par modèle et score par score, se trouve en fin de page sous « Comment cette classification a été obtenue ».