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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und Mark W Skinner
Institute for Policy Advancement Ltd, McMaster UniversityWashington,
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18. Juni 2021
Über diesen Artikel
Artikel-Kategorie: Clinical Practice
Online veröffentlicht: 18. Juni 2021
Seitenbereich: 69 - 79
DOI: https://doi.org/10.17225/jhp00178
Schlüsselwörter
© 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%2F20250913%2Feu-central-1%2Fs3%2Faws4_request&X-Amz-Date=20250913T010115Z&X-Amz-Expires=3600&X-Amz-Signature=abc10d34e04332f32dbc4c549f297db896e372d3417f0a739bf7459abe847bcf&X-Amz-SignedHeaders=host&x-amz-checksum-mode=ENABLED&x-id=GetObject)
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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%2F20250913%2Feu-central-1%2Fs3%2Faws4_request&X-Amz-Date=20250913T010115Z&X-Amz-Expires=3600&X-Amz-Signature=8ec6bd101cc3c6fd270c8f3a603e6e9783843bab10f1207ff47197cf2ed27daa&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 |