Table 3.
Guide for Physicians for Discussing Gene Therapy with Persons with Hemophilia
| Topic | Sub-topics |
|---|---|
| Fundamentals of gene therapy | What is a vector? |
| What is a functional gene? | |
| How is the vector delivered? | |
| How does the body react to the vector? | |
| Anti-vector antibodies | |
| Expectations after gene therapy | |
| Efficacy | Treatment of bleeding events |
| Frequency of bleeding events | |
| Pain management | |
| Factor expression (level and duration) | |
| Safety (short- term) | Side effects (ie, transaminitis, inhibitor development, thrombosis, anti-vector antibodies) |
| Treatment of side effects | |
| Safety (long-term) | Risk of developing cancer or other disorders |
| Possibility of unknown side effects | |
| Long-term monitoring and annual follow-up for many years | |
| Eligibility for gene therapy | Severity of disease (factor level ≤2%) |
| Adults or young adults | |
| Patients without inhibitors | |
| Patients without neutralizing antibodies to AAV capsid | |
| Current clinical trials | Status of ongoing clinical trials |
| Major findings of clinical trials | |
| Other things to consider | Psychological functioning and emotional maturity |
| Social support | |
| Family planning |