Addressing the Unmet Needs of Patients with Acute Hepatic Porphyria


Brendan M. McGuire, MD, MS
Professor of Medicine, Medical Director of Liver Transplantation, University of Alabama at Birmingham (UAB), Birmingham, AL
Brendan M. McGuire, MD, MS

Brendan M. McGuire, MD, MS, is Liver Center Director and Medical Director of Liver Transplantation at the University of Alabama at Birmingham (UAB). He received his undergraduate degree in chemical engineering at the University of Notre Dame, master’s degree in biomedical engineering at Pennsylvania State University, and medical degree at the University of Pittsburgh. He completed his internal medicine residency and fellowship in gastroenterology and hepatology and nutrition at the University of Minnesota. In 1996, Dr. McGuire joined the faculty at UAB where he worked alongside the late Joseph R. Bloomer, MD, a pioneer in the research and treatment of porphyria. As medical director of liver transplantation at UAB, Dr. McGuire has participated in the management of over 2,500 transplant recipients and has devoted his career to the care of patients with liver disease, liver transplant recipients, and patients with porphyria.

Dr. McGuire is an active member of the American Board of Internal Medicine Transplant Hepatology Committee, American Association for the Study of Liver Diseases, American Society of Transplantation, American Porphyrias Expert Collaborative, United Porphyria Association, and the Porphyria Consortium Network. The Porphyria Consortium Network has been active in advancing the diagnosis, management, and treatment of patients with porphyria. These treatments have improved patient lives and decreased the need for liver transplantation.

Manish Thapar, MD, FACG, FAASLD
Associate Professor of Medicine
Division of Gastroenterology and Hepatology, Sidney Kimmel Medical College at Thomas Jefferson University, Philadelphia, PA
Manish Thapar, MD, FACG, FAASLD

Dr. Thapar is a clinically trained Hepatologist who takes care of patients with viral hepatitis, fatty liver disease, cirrhosis, hepatocellular cancer, and liver transplant recipients. He has a special interest in hereditary and genetic disorders of the liver such as Porphyrias, Hemochromatosis and Wilson’s disease. Dr. Thapar has trained with Porphyria experts and works closely with patient care advocacy groups to take care of patients with Porphyrias. He has authored articles and book chapters on Porphyria and is also involved in clinical research.

Statement of Need

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.

Learning Objectives

At the end of this CME/CE activity, participants should be able to:

  • Relate disease etiology and pathophysiology to clinical manifestations of AHP.
  • Apply diagnostic resources to identify and confirm a diagnosis of AHP.
  • Integrate novel therapies in the management of AHP.
  • Implement long-term strategies to manage and minimize disease burden in patients with AHP.

Financial Support

Supported by an educational grant from Alnylam Pharmaceuticals, Inc.

Target Audience

Physicians, physician associates (PAs), nurse practitioners (NPs), nurses and pharmacists specializing in hematology, oncology and/or primary care

Credit Information

Jointly Accredited Provider

In support of improving patient care, CME Outfitters, LLC, is jointly accredited by the Accreditation Council for Continuing Medical Education (ACCME), the Accreditation Council for Pharmacy Education (ACPE), and the American Nurses Credentialing Center (ANCC), to provide continuing education for the healthcare team.

Interprofessional (IPCE) 1.5

This activity was planned by and for the healthcare team, and learners will receive 1.5 Interprofessional Continuing Education (IPCE) credit for learning and change.

Physicians (ACCME) 1.5

CME Outfitters, LLC, is accredited by the Accreditation Council for Continuing Medical Education (ACCME) to provide continuing medical education for physicians. CME Outfitters, LLC, designates this enduring activity for a maximum of 1.5 AMA PRA Category 1 Credit(s). Physicians should claim only the credit commensurate with the extent of their participation in the activity.

Nurses (ANCC) 1.5

This activity is designated for 1.5 contact hours. Nurse practitioners can apply for AMA PRA Category 1 Credit(s)™ through the American Academy of Nurse Practitioners (AANP). AANP will accept AMA PRA Category 1 Credit(s)™ from Jointly Accredited Organizations. Nurse practitioners can also apply for credit through their state boards.

Note for California Nurses

This continuing nursing education activity was approved by the California Board of Registered Nursing. CME Outfitters, LLC’s provider number is CEP15510.

Pharmacists (ACPE) 1.5

This application-based activity is approved for 1.5 contact hours (0.15 CEUs) of continuing pharmacy credit.
Activity UAN: JA0007185-0000-22-107-H01-P

PAs (AAPA) 1.5

CME Outfitters, LLC, has been authorized by the American Academy of PAs (AAPA) to award AAPA Category 1 CME credit for activities planned in accordance with AAPA CME Criteria. This activity is designated for 1.5 AAPA Category 1 CME credits. Approval is valid until expiration date listed above. PAs should only claim credit commensurate with the extent of their participation.


Successful completion of this CME activity, which includes participation in the evaluation component, enables the participant to earn up to 1.50 medical knowledge MOC points in the American Board of Internal Medicine's (ABIM) Maintenance of Certification (MOC) program. Participants will earn MOC points equivalent to the amount of CME credits claimed for the activity. It is the CME activity provider's responsibility to submit participant completion information to ACCME for the purpose of granting ABIM MOC credit.

MIPS Improvement Activity

Completion of this accredited CME activity meets the expectations of an Accredited Safety or Quality Improvement Program (IA_PSPA_28) for the Merit-based Incentive Payment Program (MIPS). Clinicians should submit their improvement activities by attestation via the CMS Quality Payment Program website.

Royal College MOC

Through an agreement between the Accreditation Council for Continuing Medical Education and the Royal College of Physicians and Surgeons of Canada, medical practitioners participating in the Royal College MOC Program may record completion of accredited activities registered under the ACCME’s “CME in Support of MOC” program in Section 3 of the Royal College’s MOC Program.

Disclosure Declaration

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.

The following peer reviewer and CME Outfitters staff have no financial relationships:

  • Shirley Michelle Franks, MSN, APRN, FNP-BC (peer reviewer)
  • Thomas Mitchell (planning committee)
  • Warren Beckman (planning committee)
  • Susan H. Yarbrough, CHCP (planning committee)
  • Sandra Caballero, PharmD (planning committee)
  • Sharon Tordoff (planning committee)

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.

Obtaining Credits

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Addressing the Unmet Needs of Patients with Acute Hepatic Porphyria
Event Date: 10/03/2022