New documents issued by NBDF’s Medical and Scientific Advisory Council (MASAC) cover a wide range of topics relevant to bleeding disorder patients in the U.S. and abroad. They encompass recommendations for treatment with licensed therapies, chronic pain management and physical therapy, gene therapy terminology, and bleeding disorders diagnosis in women and girls. Also included are two new resolutions challenging international recommendations that could contribute to negative health and quality of life implications for patients. 


Chronic Pain Management and Physical Therapy

Document 283 features a series of recommendations for the assessment and treatment of chronic pain in individuals with hemophilia and other bleeding disorders. It stresses the importance of customized patient education on core subjects such as musculoskeletal anatomy, biomechanics, joint health, and the science behind pain. Also included are guidelines for therapeutic exercise (strengthening, stretching, aerobic), aquatic exercise, and manual therapy. Unique considerations for the use of electrotherapy, cryotherapy and heat therapy are also discussed.

View Physical Therapy Management of People with Bleeding Disorders and Chronic Pain.


Treatment of Inherited Bleeding Disorders

Document 284 is the latest version of the council’s most enduring and broad-based treatment document. It offers descriptions of, and recommendations for, the use of all therapies currently approved by the U.S. Food and Drug Administration to treat inheritable bleeding disorders such hemophilia, von Willebrand disease (VWD), rare factor deficiencies. Treatments outlined in the document include factor replacement therapies, non-factor therapies, gene therapies, and ancillary medications such as antifibrinolytics. It also provides background on the manufacturing of these therapies and context for their indications. Included in this update are the two gene therapies recently approved for hemophilia A and B in the U.S.

View MASAC Recommendations Concerning Products Licensed for the Treatment of Hemophilia and Selected Disorders of the Coagulation System.


New Gene Therapy Lexicon 

Document 285 explains the need for an agreed upon set of terms to help aid discussions about gene therapy amongst bleeding disorders patients, caregivers, and healthcare providers. It stresses the importance of a new gene therapy lexicon that is clear, objective, unbiased, and developed collaboratively with various community stakeholders.

View MASAC Recommendations Regarding the Need for Bleeding Disorders Gene Therapy Lexicon Development.


Bleeding Disorders in Women and Girls 

Document 286 includes a series of comprehensive recommendations focused on screening for bleeding symptoms in women and girls. It lays out specific guidelines for diagnostic evaluation, including special considerations relevant to testing for VWD. The document also provides over-arching guidance for treating and managing patients in a variety of scenarios, such as pregnancy, surgery, and in cases of emergency. It also includes recommendations for future research, national public outreach efforts, and education for healthcare providers.

View MASAC Recommendations Regarding Diagnosis and Management of Inherited Bleeding Disorders in Girls and Women with Personal and Family History of Bleeding.


Cryoprecipitate and the WHO Essential Medicines List

In Document 287, MASAC disagrees with the World Health Organization (WHO) decision to recommend cryoprecipitate (Cryo) in their 23rd Essential Medicines List (EML) for the treatment of hemophilia – this includes both pathogen-reduced Cryo (PR Cryo) and non-pathogen-reduced Cryo (non-PR Cryo). The decision adds a product to the EML that carries a viral infection risk, while leaving safer factor replacement products on the complementary medicine list. The result would unnecessarily expose patients to viral infections. 


Excerpt from 287-

“MASAC recommends the WHO follow their previously well-established principles for accessibility, efficacy, and safety for many drugs, remove non-PR Cryo from the list entirely, switch PR Cryo to the complementary list and place CFCs on the core list to promote prevention of infectious disease and bleeding resulting in deaths in countries that rely upon the WHO EML for accurate information,“ urges the council.


“Furthermore, given the urgency and irreversible nature of many pathogen infections we call upon the WHO to take immediate action and not wait to correct this egregious action until the next scheduled revision of the WHO-EML in 2025.”

Read the full MASAC Resolution on the World Health Organization Essential Medicine List.


Resolution on Proposed ISTH Treatment Guidelines 

In Document 288, MASAC responds to recent draft hemophilia treatment guidelines of the International Society on Thrombosis and Haemostasis (ISTH). The authors are concerned that the guidelines, as currently written, do not fully consider the current treatment landscape where newer therapies have enabled improved patient-centered outcomes, including increased daily living participation, economic activity, and quality of life for bleeding disorder patients. MASAC asserts that the draft recommendations are antithetical to achievable 21st century treatment goals (e.g. zero bleeds) and to efforts at achieving patient-centered outcomes through shared decision making. 

Read the complete MASAC Resolution on Proposed Hemophilia Treatment Guidelines from the International Society on Thrombosis and Haemostasis.


Visit NBDF to access a searchable list of all current MASAC documents.