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
Issued Date
2024-01-01
Resource Type
ISSN
15387933
eISSN
15387836
Scopus ID
2-s2.0-85196892587
Journal Title
Journal of Thrombosis and Haemostasis
Rights Holder(s)
SCOPUS
Bibliographic Citation
Journal of Thrombosis and Haemostasis (2024)
Suggested Citation
Rezende S.M., Neumann I., Angchaisuksiri P., Awodu O., Boban A., Cuker A., Curtin J.A., Fijnvandraat K., Gouw S.C., Gualtierotti R., Makris M., Nahuelhual P., O'Connell N., Saxena R., Shima M., Wu R., Rosendaal F.R. 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. Journal of Thrombosis and Haemostasis (2024). doi:10.1016/j.jtha.2024.05.026 Retrieved from: https://repository.li.mahidol.ac.th/handle/20.500.14594/100031
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
Author's Affiliation
School of Medicine, Trinity College Dublin
Beijing Children's Hospital, Capital Medical University
Emma Kinderziekenhuis
KBC Zagreb
Università degli Studi di Milano
Universidade Federal de Minas Gerais
The Children's Hospital at Westmead
Penn Medicine
University of Benin
Ministerio de Salud de Chile
Faculty of Medicine Ramathibodi Hospital, Mahidol University
Leids Universitair Medisch Centrum
Nara Medical University
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
Universidad del Desarrollo
University of Zagreb School of Medicine
St James's Hospital
Sanquin Research
The University of Sheffield
Universidad San Sebastián
Medanta Hospital
Beijing Children's Hospital, Capital Medical University
Emma Kinderziekenhuis
KBC Zagreb
Università degli Studi di Milano
Universidade Federal de Minas Gerais
The Children's Hospital at Westmead
Penn Medicine
University of Benin
Ministerio de Salud de Chile
Faculty of Medicine Ramathibodi Hospital, Mahidol University
Leids Universitair Medisch Centrum
Nara Medical University
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
Universidad del Desarrollo
University of Zagreb School of Medicine
St James's Hospital
Sanquin Research
The University of Sheffield
Universidad San Sebastián
Medanta Hospital
Corresponding Author(s)
Other Contributor(s)
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.