CHAPEL HILL,
N.C., June 21, 2024 /CNW/ -- The International
Society on Thrombosis and Haemostasis (ISTH), the leading
international professional medical-scientific organization
dedicated to advancing the understanding, prevention, diagnosis,
and treatment of conditions related to thrombosis and hemostasis,
has published the first clinical practice guideline utilizing
strict GRADE methodology for the treatment of congenital hemophilia
A and B.
Hemophilia, a rare bleeding disorder resulting
from deficiencies in blood coagulation factors, affects hundreds of
thousands individuals worldwide and demands intricate management
strategies. As the first comprehensive guideline based on the
Grading of Recommendations Assessment, Development, and Evaluation
(GRADE) approach it provides a rigorous and structured
decision-making framework for clinicians, healthcare professionals,
patients and their caregivers.
The objective of this guideline is to offer a
comprehensive overview of evidence in making informed
treatment decisions. Developed over several years, the ISTH
assembled a multidisciplinary panel comprising physicians and
patient representatives with global representation, balanced to
mitigate potential biases. This diverse panel prioritized key
clinical questions and outcomes crucial for both clinicians and
patients.
The recommendations underwent rigorous analyses,
including public commentary, ensuring comprehensiveness,
transparency and inclusivity.
The culmination of this endeavor is a set of 13
recommendations, addressing critical aspects of hemophilia A and B
care. They are accompanied by extensive remarks providing
additional context and guidance for interpretation and
implementation.
Among these recommendations, strong endorsements
advocate for prophylactic treatment over episodic interventions for
severe and moderately severe hemophilia A and B, underscoring a
paradigm shift in clinical practice.
Recognizing that hemophilia is a rare and complex
disease that poses challenges for guideline development and
implementation, the new guideline contributes valuable insights
into the evolving hemophilia treatment landscape. It illustrates
and emphasizes the importance of patient involvement and shared
decision-making in treatment decisions and advocates for individual
and situational therapeutic choices. Moreover, the guideline
addresses important gaps in scientific data and identifies critical
research priorities.
"With hemophilia management being complex, our
guideline provides clarity, guiding treatment decisions with
evidence-based insight, yet highlighting the importance of making
treatment decisions consistent with patients' individual risks,
values, and preferences," stated Suely M.
Rezende, M.D., Ph.D., Chair of the ISTH Hemophilia Guideline
Panel. "Our collaborative effort has yielded a roadmap for
optimizing patient care, setting a new standard in hemophilia
treatment guidelines."
To provide important context, commentaries have
been published alongside the guideline to provide further guidance
on its interpretation and implementation. The commentaries,
authored by preeminent experts also address challenges for clinical
practice and policy, discuss strengths and limitations of the GRADE
methodology for rare diseases, share how observational data can add
highly valuable information in the absence of RCT data and comment
on the pros and cons of the guideline.
Looking ahead as new and quality data becomes
available in the dynamic and ever-evolving landscape of new
hemophilia treatments that offer unprecedented improvements in
treatment choices for clinicians and patients, the ISTH remains
committed to advancing hemophilia research and ultimately improving
the lives of people with hemophilia.
Future updates to the guideline are expected to
encompass emerging treatments such as novel high-sustained Factor
VIII as well as new non-factor therapies and gene therapy. By
continuously updating and complementing this guideline, the ISTH
aims to drive innovation and enhance outcomes for individuals
living with hemophilia worldwide. Additionally, it will provide
further opportunities for working and collaborating with
stakeholders globally to ensure that the latest advancements and
best clinical practice guidance is provided and effectively
disseminated and implemented, ultimately benefiting innovation in
the global hemophilia community.
For more information, to access the full
guideline and read all three corresponding commentaries, please
visit https://www.isth.org/page/hemophiliaguideline.
About the International Society on Thrombosis
and Haemostasis (ISTH)
Founded in 1969, the ISTH is the
leading worldwide not-for-profit organization dedicated to
advancing the understanding, prevention, diagnosis, and treatment
of conditions related to thrombosis and hemostasis. ISTH is an
international medical-scientific professional membership
organization with more than 7,000 clinicians, researchers, and
educators working together to improve the lives of patients in more
than 110 countries around the world. Among its highly regarded
activities and initiatives are education and standardization
programs, clinical and laboratory practice guidelines and guidance,
research activities, meetings and congresses, peer-reviewed
publications, expert committees, and World Thrombosis Day on 13
October. Visit ISTH online at www.isth.org.
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SOURCE International Society on Thrombosis and Haemostasis