CHAPEL HILL, N.C., June 21, 2024 /PRNewswire/ -- 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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