Personalising haemophilia management with shared decision making
Leonard A Valentino
Rush University, National Hemophilia FoundationChicago, USA
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, Victor Blanchette
Department of Pediatrics, University of Toronto and Division of Hematology/Oncology, Hospital for Sick ChildrenToronto, Canada
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, Claude Negrier
Unité d’Hemostase Clinique, Hôpital, Louis Pradel, Hospices Civils de Lyon, Université Claude BernardLyon, France
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, , , Thomas Sannié
Association française des hémophilesParis, France
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and Mark W Skinner
Institute for Policy Advancement Ltd, McMaster UniversityWashington,
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Jun 18, 2021
About this article
Article Category: Clinical Practice
Published Online: Jun 18, 2021
Page range: 69 - 79
DOI: https://doi.org/10.17225/jhp00178
Keywords
© 2021 Leonard A Valentino et al., published by Sciendo
This work is licensed under the Creative Commons Attribution-NonCommercial-NoDerivatives 3.0 License.
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=AKIA6AP2G7AKOUXAVR44%2F20250912%2Feu-central-1%2Fs3%2Faws4_request&X-Amz-Date=20250912T232058Z&X-Amz-Expires=3600&X-Amz-Signature=994028fe70727a4cb8b8d07f5057f063fe9b106ef5dd19d064de6a1b6d25ef3e&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=AKIA6AP2G7AKOUXAVR44%2F20250912%2Feu-central-1%2Fs3%2Faws4_request&X-Amz-Date=20250912T232058Z&X-Amz-Expires=3600&X-Amz-Signature=f661bdbf49390b05570f4e54de6fefeb69b7e567ce55c0bab45c7b32019cb60f&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 | |
| 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 | |
| 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 | |
| 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 | |
| The National Learning Consortium | SDM fact sheet with an overview of the process and links to other resources |
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 |