New Recommendations Issued on Bleeding Disorders Treatment, Bone Health and Mental Health
The National Bleeding Disorders Foundation (NBDF) has announced that three new documents have been issued by the Medical and Scientific Advisory Council (MASAC).
MASAC creates recommendations and advisories on treatment, research and other general health concerns of the inheritable bleeding disorders community.
Mental Health and Substance Use Disorder Treatment
Document 289 lays out the reasons why individuals with inheritable bleeding disorders should have equal access to residential/inpatient mental health and substance use disorder facilities. The authors maintain that this access should not be contingent upon factors such as their particular bleeding disorder (BD) diagnosis, the medications they take, or the geographic location where they reside. The document provides useful background information on the treatment/management of BDs, emphasizing the independence associated with self-administration of therapies. It also debunks the position that an adult BD patient is medically “unstable” or “unfit” for admission to these facilities.
Treatment of Inherited Bleeding Disorders
Document 290 is the latest version of the council’s most enduring and broad-based treatment document. It offers MASAC’s guidance on 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, and 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 details on the technologies employed in manufacturing these therapies, plus context for their indications. Included in this update are the two gene therapies recently approved for hemophilia A and B in the U.S.
Bone Health and Physical Therapy
Recognizing that a strong skeletal system is vital for overall health and quality of life, Document 291 includes comprehensive recommendations for the assessment and treatment of bone health in people with BDs. The authors explain that patients with certain BDs such as FVIII deficiency are at a higher risk of bone health complications, therefore early risk mitigation efforts are critical. These recommendations were informed by a medical literature review, the input of a multidisciplinary panel, and a review by various key stakeholders.
View Physical Therapy Management for Bone Health in People with Bleeding Disorders.
Visit NBDF to access a searchable list of all current MASAC documents.
Source: National Bleeding Disorders Foundation, November 2024