Online Education

Gene Therapy for Providers 101

July 01, 2022

The estimated time to complete this activity is 45 minutes.

The purpose of this activity is to enable learners to develop basic knowledge of gene therapy for hemophilia, thereby enhancing learner competence to discuss gene therapy with patients who may benefit from this emerging treatment.

This learning activity provides basic education on hemophilia gene therapy, including the aims of gene therapy; the packaging, delivery, and activity of the replacement genes; and the means by which the replacement genes lead to production and/or availability of normal clotting factor in persons with hemophilia. The module will also review information that may be particularly important when discussing hemophilia gene therapy with patients, such as knowledgeable informed consent, outcomes data, and potential risks and benefits.

This enduring material is intended for multidisciplinary providers at hemophilia treatment centers, including nurses, physicians, pharmacists, physical therapists, social workers, and genetic counselors providing care to persons with hemophilia A and hemophilia B.

At the conclusion of this enduring material, the participants should be better able to:

• Describe a pathway to preparedness for gene therapy from education of the multidisciplinary healthcare team to clinical delivery
• Provide an overview of the goal of gene therapy for hemophilia
• Explain the elements of gene therapy (i.e. packaging, administration, mechanism of action)
• Define the profile of gene therapy candidates with FVIII deficiency and FIX deficiency
• Review known adverse effects from completed and ongoing clinical trials
• Articulate critical considerations for the multidisciplinary healthcare team in providing care for patients receiving gene therapy
• Discuss issues related to obtaining knowledgeable informed consent and counseling patients

• Steven Pipe, MD
• Shelley Crary, MD, MSCS

If you have any questions about this activity, please contact Brooke Hernandez in Indianapolis, IN, 317-871-0011 ext. 813 or via email at bhernandez@ihtc.org. For technical support please email support@partnersprn.org or open a ticket in the Partners Support Center.

• 2 gigabytes of RAM
• High-speed internet connection
• Internet browser (current version) – Firefox, Safari, Chrome, or Edge
  • Cookies and JavaScript must be enabled in the browser

Gene Therapy for Providers 201

July 01, 2022

The estimated time to complete this activity is 60 minutes.

The purpose of this activity is to enable HTC multidisciplinary staff and institutional stakeholders to gain knowledge on the steps necessary to prepare an institution to administer AAV-mediated gene therapy for hemophilia.

This learning activity is a process-oriented intermediate course that will guide HTC staff through the steps an institution must take to prepare to administer hemophilia gene therapy upon commercial availability of these products.

This enduring material is intended for HTC multidisciplinary staff (physicians, physician assistants, nurses, nurse practitioners, physical therapists, social workers, pharmacists, geneticists, genetic counselors, infusion room staff) and institutional stakeholders (e.g., HTC administrators and directors, clinic and pharmacy billing staff, insurance coordinators).

At the conclusion of this enduring material, the participants should be better able to:

• Identify patients who are eligible for AAV-mediated gene therapy
• Discuss key concepts involving education and shared decision-making for gene therapy
• Identify logistical/infrastructure requirements to procure, reconstitute and administer gene therapy at a dosing site
• Articulate the regional approach of preparing for gene therapy administration
• Distinguish the roles and responsibilities within the HTC team involved in the safety and efficacy assessments during short-and long-term monitoring before and after gene therapy
• Describe approaches to prevention, diagnosis, and treatment of vector-induced transaminitis after gene therapy

• Rebecca Kruse-Jarres, MD, MPH
• Ulrike Margret Reiss, MD
• Courtney Dawn Thornburg, MD, MS

If you have any questions about this activity, please contact Brooke Hernandez in Indianapolis, IN, 317-871-0011 ext. 813 or via email at bhernandez@ihtc.org. For technical support please email support@partnersprn.org or open a ticket in the Partners Support Center.

• 2 gigabytes of RAM
• High-speed internet connection
• Internet browser (current version) – Firefox, Safari, Chrome, or Edge
  • Cookies and JavaScript must be enabled in the browser

Gene Therapy for Providers 301

July 01, 2022

The estimated time to complete this activity is 60 minutes.

The purpose of this activity is to enable HTC multidisciplinary staff and hospital staff to support patients and their families as they consider hemophilia gene therapy, including education of patients and families on basic concepts in hemophilia gene therapy; administrative preparedness for providers; and short- and long-term care demands.

This learning activity is a process-oriented intermediate course that will guide HTC staff through the steps an institution must take to prepare to administer hemophilia gene therapy upon commercial availability of these products.

This enduring material is intended for physicians, physician assistants, nurses, nurse practitioners, physical therapists, social workers, pharmacists, and infusion room staff.

At the conclusion of this enduring material, the participants should be better able to:

• Discuss introductory concepts in hemophilia gene therapy
• Outline the process of preparing patients, hemophilia treatment centers, and hospital systems for the administration of gene therapy
• Explain the steps necessary for multidisciplinary team members to engage in shared patient-centered and population focused problem solving when procuring a hemophilia gene therapy product, preparing the product, and infusing the patient safely
• Discuss short term monitoring, specifically immediately after infusion and during the first three months of follow up
• Discuss long term monitoring, specifically from 3 to 24 months post infusion and > 24 months post infusion

• Doris V. Quon, MD
• Lindsey A. George, MD

If you have any questions about this activity, please contact Brooke Hernandez in Indianapolis, IN, 317-871-0011 ext. 813 or via email at bhernandez@ihtc.org. For technical support please email support@partnersprn.org or open a ticket in the Partners Support Center.

• 2 gigabytes of RAM
• High-speed internet connection
• Internet browser (current version) – Firefox, Safari, Chrome, or Edge
  • Cookies and JavaScript must be enabled in the browser