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, PAs, nurse practitioners, nurses and pharmacists specializing in hematology, oncology and/or primary care.
It is the policy of CME Outfitters, LLC, to ensure independence, balance, objectivity, and scientific rigor and integrity in all of their CE activities. Faculty must disclose to the participants any relationships with commercial companies whose products or devices may be mentioned in faculty presentations, or with the commercial supporter of this CE activity. CME Outfitters, LLC, has evaluated, identified, and mitigated any potential conflicts of interest through a rigorous content validation procedure, use of evidence-based data/research, and a multidisciplinary peer review process. The following information is for participant information only. It is not assumed that these relationships will have a negative impact on the presentations.
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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Faculty of this CE activity may include discussions of products or devices that are not currently labeled for use by the FDA. The faculty have been informed of their responsibility to disclose to the audience if they will be discussing off-label or investigational uses (any uses not approved by the FDA) of products or devices.
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