Three new documents have been issued by NBDF’s Medical and Scientific Advisory Council (MASAC), which creates recommendations and advisories on current treatment, research matters, and other general health concerns for the inheritable bleeding disorders community.
Off-Label Use of Emicizumab in Acquired Hemophilia A
Document 292 is focused on acquired hemophilia A (AHA), a rare autoimmune disease that causes the human body to produce antibodies against factor VIII. It primarily affects older individuals and is often associated with other autoimmune diseases, malignancies, and/or the use of certain medications. AHA can cause severe, even life-threatening bleeding complications. While certain factor replacement therapies (e.g. bypassing agents) may be used to treat AHA, they come with drawbacks, including the burden of frequent intravenous infusions and a risk of excessive clotting. MASAC therefore recommends that given the high morbidity and mortality observed in individuals with due to bleeding complications and other adverse events, the subcutaneously administered therapy emicizumab should be considered for bleeding prophylaxis from the time of AHA diagnosis. Emicizumab is a humanized antibody that mimics activated FVIII to allow continuation of the coagulation cascade. This recommendation also includes additional guidance specific to dosing emicizumab in AHA patients.
Use of Anti-Tissue Factor Pathway Inhibitors in Hemophilia
Document 293 includes several recommendations on the clinical use of concizumab-mtci, which is indicated for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients 12 years of age and older with hemophilia A or B with or without factor VIII or IX inhibitors. Concizumab-mtci is a subcutaneous therapy developed with a synthetically produced (recombinant) antibody derived from a single cell clone and designed to be equally effective in individuals with hemophilia A and B, irrespective of inhibitor status. It targets an anticoagulant protein known as tissue factor pathway inhibitor (TFPI). Also known as an anti-tissue factor pathway inhibitor (anti-TFPI), concizumab-mtci works by blocking and effectively preventing TFPI from performing the anticoagulant function that it naturally carries out in the human body. This type of therapy provides an alternative to traditional prophylaxis with intravenously delivered factor replacement products. The recommendation includes guidance on dosing, administration, usage around surgery, concomitant use with other therapies, and the importance of patient education on the risk of thrombosis.
The Role of HTC Social Workers
Document 294 encompasses a comprehensive set of broad-based recommendations for each of the primary roles provided by clinical social workers (SWs) practicing at the hemophilia treatment centers that serve the bleeding disorders community. Informed by the work of NBDF’s Social Work Working Group and two important studies, these recommendations are meant to help establish clarity on the multifaceted roles and responsibilities of HTC SWs. This document was created with an eye towards the considerable barriers faced by SWs including limited resources, time constraints, and competing priorities, plus the disparities between actual and ideal roles identified by SWs themselves. The recommendations home in on six key roles of social workers, including: patient counseling; case management; financial/insurance; outreach/programs; administrative; and grants/research.
Visit NBDF to access a searchable list of all current MASAC documents.
