9937 ISTH NHF Eval

Codon optimization of transgene cDNA constructs results in: *This question is required.

• Change in nucleotide sequence but NOT amino acids
• Change in both nucleotide sequence AND amino acids
• Change in amino acids, but NOT nucleotide sequence
• Change in promoter AND amino acids
• I’m not sure

In the schematic of the adeno-associated virus genome below, which element(s) is/are replaced to make an AAV therapeutic vector for gene therapy? *This question is required.

• Rep (replication gene)
• Cap (capsid gene)
• Rep and Cap genes
• The entire genome (ITRs, Rep, and Cap)
• I’m not sure

Which of the products currently in phase 3 clinical trials for hemophilia gene therapy included patients with AAV antibodies? *This question is required.

• Hemophilia B using vector AAV5
• Hemophilia B using vector AAV-Spark100
• Hemophilia A using vector AAV-Spark 200
• Hemophilia A using vector AAV5
• I’m not sure

Which of the following statements best describes the efficacy of gene therapy in the clinical trials for hemophilia A utilizing the AAV5 vector? *This question is required.

• Data for efficacy and safety are available for up to 156 weeks
• FVIII activity levels peaked at 12 weeks post injection
• For the highest dose, 6E13, mean FVIII activity levels remained above 20 IU/dL at week 156
• By year 2, annualized bleed rates and factor utilization were reduced by up to 85%
• I’m not sure

1. Health Care Professional Category *This question is required.

• Physician
• PhD/Researcher
• Pharmacist
• Technician/Scientist/Technologist
• RN, APRN, or Physician Assistant
• Student/Resident
• Post-doctoral Fellow
• Social Worker
• Genetic Counselor
• Physical Therapist/Occupational Therapist
• Health Business/Medical Administration Professional
• Health Care Regulator
• Other - specify Please enter an 'other' value for this selection.

2. Area of Primary Clinical/Laboratory Focus    *This question is required.

• Thrombosis/Clotting
• Bleeding Disorders Including Hemophilia
• Thrombosis and Hemophilia
• Hematology
• Hematology/Oncology
• Internal Medicine
• Genetics
• Rehabilitation
• Other clinical, specify Please enter an 'other' value for this selection.
• Other non-clinical, specify Please enter an 'other' value for this selection.

3. What is your role in the care of individuals with hemophilia? Select all that apply *This question is required.

• Physician involved in direct patient care (management and monitoring of bleeding risk)
• Investigator in clinical trials of patients with hemophilia
• Perform assays/assessments in a specialized coagulation laboratory
• Physical, occupational, and/or mental health therapy
• Basic research related to hemophilia, but not related to patient care
• Patient education
• Other - specify Please enter an 'other' value for this selection.

6. Please rate your ability BEFORE the activity *This question is required.

Space Cell	0 - Not Relevant	1 - Low	2	3	4	5 - High
KNOWLEDGE of gene therapy approaches in development for hemophilia A and B:	KNOWLEDGE of gene therapy approaches in development for hemophilia A and B:: 0 - Not Relevant	KNOWLEDGE of gene therapy approaches in development for hemophilia A and B:: 1 - Low	KNOWLEDGE of gene therapy approaches in development for hemophilia A and B:: 2	KNOWLEDGE of gene therapy approaches in development for hemophilia A and B:: 3	KNOWLEDGE of gene therapy approaches in development for hemophilia A and B:: 4	KNOWLEDGE of gene therapy approaches in development for hemophilia A and B:: 5 - High
ABILITY to describe the recent clinical trial results of gene therapy for patients with hemophilia A and hemophilia B	ABILITY to describe the recent clinical trial results of gene therapy for patients with hemophilia A and hemophilia B: 0 - Not Relevant	ABILITY to describe the recent clinical trial results of gene therapy for patients with hemophilia A and hemophilia B: 1 - Low	ABILITY to describe the recent clinical trial results of gene therapy for patients with hemophilia A and hemophilia B: 2	ABILITY to describe the recent clinical trial results of gene therapy for patients with hemophilia A and hemophilia B: 3	ABILITY to describe the recent clinical trial results of gene therapy for patients with hemophilia A and hemophilia B: 4	ABILITY to describe the recent clinical trial results of gene therapy for patients with hemophilia A and hemophilia B: 5 - High
KNOWLEDGE of the changes/modifications/additions to current hemophilia treatment protocols in order to ensure readiness for gene therapy:	KNOWLEDGE of the changes/modifications/additions to current hemophilia treatment protocols in order to ensure readiness for gene therapy:: 0 - Not Relevant	KNOWLEDGE of the changes/modifications/additions to current hemophilia treatment protocols in order to ensure readiness for gene therapy:: 1 - Low	KNOWLEDGE of the changes/modifications/additions to current hemophilia treatment protocols in order to ensure readiness for gene therapy:: 2	KNOWLEDGE of the changes/modifications/additions to current hemophilia treatment protocols in order to ensure readiness for gene therapy:: 3	KNOWLEDGE of the changes/modifications/additions to current hemophilia treatment protocols in order to ensure readiness for gene therapy:: 4	KNOWLEDGE of the changes/modifications/additions to current hemophilia treatment protocols in order to ensure readiness for gene therapy:: 5 - High

7. Please rate your ability AFTER the activity *This question is required.

Space Cell	0 - Not Relevant	1 - Low	2	3	4	5 - High
KNOWLEDGE of gene therapy approaches in development for hemophilia A and B:	KNOWLEDGE of gene therapy approaches in development for hemophilia A and B:: 0 - Not Relevant	KNOWLEDGE of gene therapy approaches in development for hemophilia A and B:: 1 - Low	KNOWLEDGE of gene therapy approaches in development for hemophilia A and B:: 2	KNOWLEDGE of gene therapy approaches in development for hemophilia A and B:: 3	KNOWLEDGE of gene therapy approaches in development for hemophilia A and B:: 4	KNOWLEDGE of gene therapy approaches in development for hemophilia A and B:: 5 - High
ABILITY to describe the recent clinical trial results of gene therapy for patients with hemophilia A and hemophilia B:	ABILITY to describe the recent clinical trial results of gene therapy for patients with hemophilia A and hemophilia B:: 0 - Not Relevant	ABILITY to describe the recent clinical trial results of gene therapy for patients with hemophilia A and hemophilia B:: 1 - Low	ABILITY to describe the recent clinical trial results of gene therapy for patients with hemophilia A and hemophilia B:: 2	ABILITY to describe the recent clinical trial results of gene therapy for patients with hemophilia A and hemophilia B:: 3	ABILITY to describe the recent clinical trial results of gene therapy for patients with hemophilia A and hemophilia B:: 4	ABILITY to describe the recent clinical trial results of gene therapy for patients with hemophilia A and hemophilia B:: 5 - High
KNOWLEDGE of the changes/modifications/additions to current hemophilia treatment protocols in order to ensure readiness for gene therapy:	KNOWLEDGE of the changes/modifications/additions to current hemophilia treatment protocols in order to ensure readiness for gene therapy:: 0 - Not Relevant	KNOWLEDGE of the changes/modifications/additions to current hemophilia treatment protocols in order to ensure readiness for gene therapy:: 1 - Low	KNOWLEDGE of the changes/modifications/additions to current hemophilia treatment protocols in order to ensure readiness for gene therapy:: 2	KNOWLEDGE of the changes/modifications/additions to current hemophilia treatment protocols in order to ensure readiness for gene therapy:: 3	KNOWLEDGE of the changes/modifications/additions to current hemophilia treatment protocols in order to ensure readiness for gene therapy:: 4	KNOWLEDGE of the changes/modifications/additions to current hemophilia treatment protocols in order to ensure readiness for gene therapy:: 5 - High

8. Do you intend to make changes or apply new knowledge to your practice as a result of this activity? *This question is required.

• Yes*
• I’m possibly considering changes*
• This activity affirms what I already do in practice
• No, I do not intend to make any changes

If you answered yes or possibly considering changes, please check off what you intend to do differently or incorporate into your clinical management of patients as a result of this educational activity. *This question is required.

• Discuss gene therapy in the context of other therapies for hemophilia with peers and colleagues
• Stay up-to-date with new developments related to gene therapy for patients with hemophilia
• Review the results of clinical studies that have evaluated the safety and efficacy of gene therapy for hemophilia
• Increase efforts to identify patients with hemophilia who are candidates for gene therapy
• Initiate conversations about the pros and cons of gene therapy with patients and caregivers
• Expand my understanding of the concerns and unknowns associated with the future of gene therapy for hemophilia
• Other - please specify

10. Was the format of this educational activity appropriate for the content presented? *This question is required.

• Yes
• No

11. Did this educational activity meet your learning needs? *This question is required.

• Yes
• No

13. Was this activity scientifically sound and free of commercial bias or influence?  *This question is required.

• Yes
• No

14. FOR EUROPEAN UNION AND UNITED KINGDOM LEARNERS ONLY:
The France Foundation would like to email you educational opportunities relevant to your practice to the address you provided. May we have your consent?

• Yes
• No