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. 2020 Mar 19;4(4):644–651. doi: 10.1002/rth2.12326

Table 2.

Self‐reported ability

Self‐reported ability Physicians directly involved in care of people with hemophilia (%) Other respondents (%)
Ability to explain the science of AAV‐mediated liver‐directed gene therapy for hemophilia to a colleague (n = 177)
1—I’ve never heard of gene therapy 3 3
2—I do not have a clear understanding of how gene therapy works 6 6
3—I’ve learned about gene therapy, but still don’t think that I could explain it well to someone else 26 41
4—I know enough about gene therapy to feel comfortable educating colleagues, patients, and caregivers 54 40
5—I consider myself an expert 11 10
Ability to answer patient questions about gene therapy for hemophilia based on clinical trial results to date (n = 143)
1—I would not be able to answer questions about clinical trials of gene therapy for hemophilia 2 7
2—I have read about some of the studies, but would not feel very comfortable answering questions 11 24
3—I could answer a few basic questions about the studies 27 28
4—I feel comfortable answering questions about clinical trial results in gene therapy 50 30
5—I consider myself an expert 10 11
Ability to describe how gene therapy may impact an individual’s current treatment for hemophilia (n = 143)
1—I am not able to describe how gene therapy may impact an individual’s current treatment 1 9
2—I have a limited understanding of how gene therapy may impact current treatment practice for patients 16 22
3—I am able to explain how gene therapy may impact current treatment practice for patients 35 21
4—I am very comfortable with my ability to explain to my patients and colleagues how gene therapy may impact current treatment practice 38 35
5—I consider myself an expert 10 13

Abbreviation: AAV, adeno‐associated viral.