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dc.contributor.author M Rezende, Suely
dc.contributor.author Neumann Burotto, Gonzalo Ignacio
dc.contributor.author Angchaisuksiri, Pantep
dc.contributor.author Awodu, Omolade
dc.contributor.author Boban, Ana
dc.contributor.author Cuker, Adam
dc.contributor.author A Curtin , Julie
dc.contributor.author Fijnvandraat, Karin
dc.contributor.author Gouw , Samantha C
dc.contributor.author Gualtierotti, Roberta
dc.contributor.author Makris, Michael
dc.contributor.author Nahuelhual, Paula
dc.contributor.author O'Connell , Niamh
dc.contributor.author Saxena, Renu
dc.contributor.author Shima, Midori
dc.contributor.author Wu, Runhui
dc.contributor.author R Rosendaal, Frits
dc.date.accessioned 2024-10-11T15:10:11Z
dc.date.available 2024-10-11T15:10:11Z
dc.date.issued 2024-06-20
dc.identifier.issn 1538-7933
dc.identifier.other Mendeley: b055e8b9-863b-3731-8bd0-86ae80e5ef76
dc.identifier.uri https://repositorio.uss.cl/handle/uss/14080
dc.description Publisher Copyright: © 2024
dc.description.abstract Abstract Background: Hemophilia is a rare congenital bleeding disorder that results from complete or partial deficiency of blood coagulation factor (F)VIII (hemophilia A) or FIX (hemophilia B) due to pathogenic variants in their coding genes. Hemophilia requires complex management. To date, there is no evidence-based clinical practice guideline on hemophilia treatment based on the Grading of Recommendations Assessment, Development, and Evaluation (GRADE) approach. Objectives: This evidence-based clinical practice guideline from the International Society on Thrombosis and Haemostasis aims to provide an overview of evidence and support patients, caregivers, hematologists, pediatricians, other clinicians, researchers, and stakeholders in treatment decisions about congenital hemophilia A and B. Methods: The International Society on Thrombosis and Haemostasis formed a multidisciplinary guideline panel of physicians and patients with global representation, balanced to minimize potential bias from conflicts of interest. The panel prioritized a set of clinical questions and outcomes according to their importance for clinicians and patients. A methodological team supported the guideline development process, including searching for evidence and performing systematic reviews. The GRADE approach was used, including GRADE Evidence to Decision frameworks. The recommendations were subject to public comment. Results: The panel selected 13 questions, of which 11 addressed the treatment of hemophilia A and 2 the treatment of hemophilia B. Specifically, the panel addressed questions on prophylactic and episodic treatment with FVIII concentrates, bypassing agents, and nonfactor therapy (emicizumab) for hemophilia A (with and without inhibitors) as well as immune tolerance induction for hemophilia A. For hemophilia B, the panel addressed questions on prophylactic and episodic treatment of bleeding events with FIX concentrates. Agreement was reached for all 13 recommendations, of which 7 (54%) were based on evidence from randomized clinical trials, 3 (23%) on observational studies, and 3 (23%) on indirect comparisons. Conclusion: Strong recommendations were issued for prophylactic over episodic treatment for severe and moderately severe hemophilia A and B. Only conditional recommendations were issued for the remaining questions. Future research should focus on direct treatment comparisons and the treatment of hemophilia B with and without inhibitors. Future updates of this guideline will provide an updated evidence synthesis on the current questions and focus on new FVIII and FIX concentrates, novel nonfactor therapies, and gene therapy for severe and nonsevere hemophilia A and B. Keywords: bleeding; clinical practice guideline; evidence-based practice; hemophilia A; hemophilia B. en
dc.language.iso eng
dc.relation.ispartof vol. 22 Issue: no. 9 Pages: 2629-2652
dc.source Journal of Thrombosis and Haemostasis
dc.title International Society on Thrombosis and Haemostasis clinical practice guideline for treatment of congenital hemophilia A and B based on the Grading of Recommendations Assessment, Development, and Evaluation methodology en
dc.type Artículo
dc.identifier.doi 10.1016/j.jtha.2024.05.026
dc.publisher.department Facultad de Medicina y Ciencia


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