PERCEPTIONS

How would you rate your ability to explain the science of adeno-associated virus [AAV] mediated liver-directed gene therapy for hemophilia to a colleague?

How would you rate your ability to answer patient questions about the safety and efficacy of gene therapy for hemophilia based on clinical trial results to date?

How would you rate your ability to describe how gene therapy may impact an individual’s current treatment for hemophilia

KNOWLEDGE-BASED QUESTIONS

Which of the following was a methodological challenge associated with early gene transfer studies for hemophilia A (relative to hemophilia B)?

Gene therapy for patients with hemophilia has used a viral vector transfer approach that results in:

Successful gene therapy for patients with hemophilia has affected which cell populations

In early phase clinical trials using AA5-FVIII gene transfer in men with severe hemophilia A, what was the protein produced by this method?

In some studies of AAV-mediated FIX gene transfer in patients with hemophilia B, a codon-optimized FIX Padua (FIX-R338L) transgene is being utilized. What is the rationale for this approach?

Which statement describes safety outcomes from recent investigation of AAV5-gene therapy with expression of wild-type FIX in adults with hemophilia B

Of the following patients with hemophilia A, who would you consider potentially eligible for participation in current clinical trials of gene therapy?

Which of the following has been a common adverse event noted in clinical trials of gene therapy for patients with hemophilia?

Based on currently available data, what is the appropriate duration of monitoring for patients following gene therapy for hemophilia?

9085 ISTH TTT Post-Assessment Survey