Figure 1
Figure 2
![Factors affecting response to prophylaxis for haemophilia [25,56]](https://sciendo-parsed.s3.eu-central-1.amazonaws.com/647217e9215d2f6c89dbbb4c/j_jhp00178_fig_002.jpg?X-Amz-Algorithm=AWS4-HMAC-SHA256&X-Amz-Content-Sha256=UNSIGNED-PAYLOAD&X-Amz-Credential=ASIA6AP2G7AKL33A6BUB%2F20251212%2Feu-central-1%2Fs3%2Faws4_request&X-Amz-Date=20251212T124319Z&X-Amz-Expires=3600&X-Amz-Security-Token=IQoJb3JpZ2luX2VjEDsaDGV1LWNlbnRyYWwtMSJHMEUCIFdA6vGFBlk5nltuG56BTAaoKZMEP%2BbnAJPe33ikb54cAiEA5rgdtY3rCYV6RfOsjKlLCOp%2BZzD04qBdAn3g4s6YALUqvQUIBBACGgw5NjMxMzQyODk5NDAiDOj%2BXjBHiaHd3dIMxCqaBdIY8b%2FaRxu4DJcXCkZdN4ZOPDSTbE3jGUOlaJvKQcj8YlKPYJ0GQBqTluSpAo5MhHlfuoBed0fdMcXCfvRAB1cjO6RnzbNdfZCXekM7HC7XmRFb3RLfG5kjII6%2BRiZXpbmrR4MYaHClHwiiSTbr9LtC6F4bs6UQsJCXNsGbFJAQw1%2FiFIqIvqv7uqJkuS21C5s5rE5ttDJDLQgZPXIlflCSxPOz0HqYu8b%2BGi%2BruKODjD9%2BKoaIK3cvypW9IpR03TtwE1s%2Ft2xDv%2Bip%2Bc%2BBHEbDw0w37q2lxsPSNNXtHccqIDjz1ixyKDOOVO50xqvEU67hIBSZMQyK1tn7lyjove8ZjDskEbvCkaUDmOT3qtht4gY8jKQMMB1ezD0OYDpqBkOVB%2Fb9Fb3Z73SjHWrFQnC2bBzP8WJZaAemBSph30%2F6gMVPnu7L7693AQUIL1rvMvyrHq%2BrSoxyHOI%2BwHBrAxBFGDl6AKi3a0A1tsvr6GKpLQzQxOGZVN731twtvN%2Ffwc9SUp1lJCL%2BNaSsSttO8xxVdHhVDWJHMsbKJXxeXbjJDkshdWOjTHO6OHvUgNTeA3%2FbVleDS4WXOu5u6Qzy3s6iWRzMguuxcn%2FWNDdkLZWjGRtGVh%2BjOd72hS4zyPnrN7cOww%2BMLJgS%2BLePnMzco0%2FglzzFgEjYNtkitSJb4Cd2%2Blq3Y1GgIMvOUtDeLl9eqbHbRJIz2ouIQ91MvQpr0N%2FsxyX0SGsPoKDt541wurDuTC9JsmSD%2BzMWyxn3bYFQ1OuF8jImHHVsxmGi6MGEeQiXI6eFzLdlq8U7ZR%2BG39Wg4OLMVJB8%2BMpusUkq1wNV%2BIhbNenN4GlQeU8FZ23l3LwQdj9W5XCMEnsnVFCcu3GzXLiLoePv2LzTCDC84%2B%2FJBjqxATTSFQ5kMAdQkmp1qEWjvLPGL2sYmw9ZgRDjiDGgpeeCPWMdDRtTcZwQ0MwGx7Awem7%2Fok7VAqr%2F8ycpZZm8CQMb9%2BqztGbsHcqBpIYTUL0794aKkVmpdBLbSxHjNet%2B%2FdVdfFvGeohddQqZwlmKWfjN1B2dFSC19fqjbP1RL6xqDFVVqQCS9P7q2VFZpl0J0%2B%2FdRc2vVlKYslyDGsgSCBC4YB11DrEnYxeYoxIKVqBA1w%3D%3D&X-Amz-Signature=7b0d05f025e16be7d478657e77992659ec2c14cbcc59cf3659751efead213faa&X-Amz-SignedHeaders=host&x-amz-checksum-mode=ENABLED&x-id=GetObject)
Figure 3
Figure 4
![Peaks and troughs of factor levels and haemostasis differ between modalities [51]](https://sciendo-parsed.s3.eu-central-1.amazonaws.com/647217e9215d2f6c89dbbb4c/j_jhp00178_fig_004.jpg?X-Amz-Algorithm=AWS4-HMAC-SHA256&X-Amz-Content-Sha256=UNSIGNED-PAYLOAD&X-Amz-Credential=ASIA6AP2G7AKL33A6BUB%2F20251212%2Feu-central-1%2Fs3%2Faws4_request&X-Amz-Date=20251212T124319Z&X-Amz-Expires=3600&X-Amz-Security-Token=IQoJb3JpZ2luX2VjEDsaDGV1LWNlbnRyYWwtMSJHMEUCIFdA6vGFBlk5nltuG56BTAaoKZMEP%2BbnAJPe33ikb54cAiEA5rgdtY3rCYV6RfOsjKlLCOp%2BZzD04qBdAn3g4s6YALUqvQUIBBACGgw5NjMxMzQyODk5NDAiDOj%2BXjBHiaHd3dIMxCqaBdIY8b%2FaRxu4DJcXCkZdN4ZOPDSTbE3jGUOlaJvKQcj8YlKPYJ0GQBqTluSpAo5MhHlfuoBed0fdMcXCfvRAB1cjO6RnzbNdfZCXekM7HC7XmRFb3RLfG5kjII6%2BRiZXpbmrR4MYaHClHwiiSTbr9LtC6F4bs6UQsJCXNsGbFJAQw1%2FiFIqIvqv7uqJkuS21C5s5rE5ttDJDLQgZPXIlflCSxPOz0HqYu8b%2BGi%2BruKODjD9%2BKoaIK3cvypW9IpR03TtwE1s%2Ft2xDv%2Bip%2Bc%2BBHEbDw0w37q2lxsPSNNXtHccqIDjz1ixyKDOOVO50xqvEU67hIBSZMQyK1tn7lyjove8ZjDskEbvCkaUDmOT3qtht4gY8jKQMMB1ezD0OYDpqBkOVB%2Fb9Fb3Z73SjHWrFQnC2bBzP8WJZaAemBSph30%2F6gMVPnu7L7693AQUIL1rvMvyrHq%2BrSoxyHOI%2BwHBrAxBFGDl6AKi3a0A1tsvr6GKpLQzQxOGZVN731twtvN%2Ffwc9SUp1lJCL%2BNaSsSttO8xxVdHhVDWJHMsbKJXxeXbjJDkshdWOjTHO6OHvUgNTeA3%2FbVleDS4WXOu5u6Qzy3s6iWRzMguuxcn%2FWNDdkLZWjGRtGVh%2BjOd72hS4zyPnrN7cOww%2BMLJgS%2BLePnMzco0%2FglzzFgEjYNtkitSJb4Cd2%2Blq3Y1GgIMvOUtDeLl9eqbHbRJIz2ouIQ91MvQpr0N%2FsxyX0SGsPoKDt541wurDuTC9JsmSD%2BzMWyxn3bYFQ1OuF8jImHHVsxmGi6MGEeQiXI6eFzLdlq8U7ZR%2BG39Wg4OLMVJB8%2BMpusUkq1wNV%2BIhbNenN4GlQeU8FZ23l3LwQdj9W5XCMEnsnVFCcu3GzXLiLoePv2LzTCDC84%2B%2FJBjqxATTSFQ5kMAdQkmp1qEWjvLPGL2sYmw9ZgRDjiDGgpeeCPWMdDRtTcZwQ0MwGx7Awem7%2Fok7VAqr%2F8ycpZZm8CQMb9%2BqztGbsHcqBpIYTUL0794aKkVmpdBLbSxHjNet%2B%2FdVdfFvGeohddQqZwlmKWfjN1B2dFSC19fqjbP1RL6xqDFVVqQCS9P7q2VFZpl0J0%2B%2FdRc2vVlKYslyDGsgSCBC4YB11DrEnYxeYoxIKVqBA1w%3D%3D&X-Amz-Signature=48232b0d6ee06485f756969e3f750a6abb3c5fb38813eebda3858de4988d8ea0&X-Amz-SignedHeaders=host&x-amz-checksum-mode=ENABLED&x-id=GetObject)
Available examples and resources for shared decision making
| NAME | DESCRIPTION | URL |
|---|---|---|
| Ottawa Personal Decision Guides | Designed to help people identify their decision making needs, plan the next steps, track their progress, and share their views about any health-related or social decisions | https://decisionaid.ohri.ca/decguide.html |
| Laval University and McMaster University | Prepares the clinician to discuss scientific evidence with the patient (or caregiver) so they can make an informed decision together | https://www.boitedecision.ulaval.ca/fileadmin/documents/Boites_PDF/Prophylaxis/Dbox_prophylaxis_treatment_options_AN.pdf |
| The Mayo Clinic Shared Decision Making National Resource Center | Advances patient-centred medical care by promoting shared decision making through the development, implementation, and assessment of patient decision aids and shared decision making techniques | https://shareddecisions.mayoclinic.org/ |
| Dartmouth-Hitchcock and the Dartmouth Institute Center for Shared Decision Making | Provides patient decision aids, decision support counselling, and facilitation of advance care planning discussions | https://www.dartmouth-hitchcock.org/shared-decision-making/resources |
| The National Learning Consortium | SDM fact sheet with an overview of the process and links to other resources | https://www.healthit.gov/sites/default/files/nlc_shared_decision_making_fact_sheet.pdf |
Potential benefit and risk considerations for gene therapy for haemophilia [29,35,51,55]
| POTENTIAL BENEFIT CONSIDERATIONS | POTENTIAL RISK CONSIDERATIONS |
|---|---|
| One-time treatment modality | Novel treatment approach with limited clinical experience to date |
| Evidence of potential clinical efficacy in clinical trials | Limitations due to patient ineligibility |
| May decrease cost of treatment over time vs. prophylaxis or standard of care | Potential immune response to treatment |
| May improve quality of life vs. other treatment modalities | Limited clinical experience precludes availability of long-term safety data and durability |