Acute hepatic porphyria (AHP) is a group of rare metabolic defects caused by inherited deficiencies in the heme biosynthetic pathway, leading to accumulations of porphyrin precursors. AHP is characterized by intensely painful attacks that can be life-threatening if incorrectly diagnosed. Nearly 80% of patients with AHP are females between the ages of 20 and 50. Treatment choices in acute attacks are limited, and historically, liver transplantation was the only curative option. Standard therapy includes supportive measures and intravenous hemin administration. Recently, subcutaneous administration of small interfering RNA (siRNA) targeting ALA synthase 1 (ALAS1) has been approved to treat AHP. This novel approach suppresses acute attacks and hemin usage.
Because of their key role in managing AHP, it is critical that clinicians involved in hematology stay current with best practices for managing this complex and often confusing disease. In this activity, experts in AHP provide practical and patient-centered guidance for optimizing screening, diagnosis, and treatment.
At the end of this CME/CE activity, participants should be able to:
Supported by an educational grant from Alnylam Pharmaceuticals, Inc.
Physicians, physician associates (PAs), nurse practitioners (NPs), nurses and pharmacists specializing in hematology, oncology and/or primary care
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Dr. McGuire reports the following financial relationships: Advisory Board: Mitsubishi Tanabe Pharma. Research Support: Alnylam Pharmaceuticals, Inc.; Arrowhead Pharmaceuticals, Inc.; Disc Medicine; and Grifols, S.A.
Dr. Thapar reports the following financial relationships: Advisory Board: Alnylam Pharmaceuticals, Inc.; Disc Medicine; Mitsubishi Tanabe Pharma; and Recordati Rare Diseases. Research Support: Alnylam Pharmaceuticals, Inc.; and Mitsubishi Tanabe Pharma. Speakers Bureau: Alnylam Pharmaceuticals, Inc.
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