Hepatocellular Cancer Screening and Gene Therapy Preparedness the Focus of New Recommendations

Two new documents have been issued by the NBDF’s Medical and Scientific Advisory Council (MASAC), which periodically writes recommendations and advisories on treatment, research, and other general health concerns of the inheritable bleeding disorders community. These new recommendations center on topics most relevant to adults with hemophilia.

Document 281 lays out criteria for hepatocellular cancer (HCC) screening and surveillance in people with hepatitis B and hepatitis C (HCV), including those who have developed cirrhosis associated with advanced liver disease. Guidelines for utilizing proven HCC screening tools such as ultrasound are also described. The document concludes with a series of specific recommendations relevant to screening individuals with hemophilia, taking into consideration factors such as current viral status (e.g., HCV infected vs cleared) and fibrosis severity. View and download MASAC Recommendations on Screening for Development of Hepatocellular Cancer in Persons with Hepatitis B and C to learn more.

Document 282 is focused on helping hemophilia treatment centers prepare for commercial gene therapy, appreciating the complexities and unique considerations inherent in delivering such a novel treatment. It describes a “roadmap” for achieving preparedness, encompassing five primary components: 1) Education and Institutional Training 2) Patient Eligibility 3) Insurance Authorization and Support 4) Procurement and Administration 5) Patient Monitoring and Follow-up. The document includes a list of existing educational resources from various entities including NBDF, Medscape, the American Society of Gene, and Cell Therapy, and the IHTC Partners Program. Also incorporated are recommendations on fostering clear communication between potentially eligible patients and their providers, with shared decision making as a guiding principle. View and download MASAC Recommendations on Hemophilia Treatment Center Preparedness for Delivering Gene Therapy for Hemophilia to learn more.

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

Source: National Bleeding Disorders Foundation, November 2023