From Theory to Therapy: Evaluating Canadian Hemophilia Treatment Centres' Readiness for Gene Therapy Implementation
Notice bibliographique
Résumé
Introduction: Gene therapies (GT) present a transformative promise for persons with hemophilia, potentially offering a long-term solution to improve patient care and experiences. However, in publicly funded systems, GT's promise will fall short if stakeholders' preparedness, capacity, and perceptions are not proactively examined to address barriers that might delay and limit its equitable access. Therefore, this study aims to examine healthcare professionals' (HCPs) perspectives on their readiness, motivation, and capacity to implement and use GT within Canadian Hemophilia Treatment Centres (HTCs). Methods: We conducted a cross-sectional online survey of HCPs across all Canadian HTCs, developed using the R=MC2 framework for organizational change. In this framework, organizational readiness is described as a function of the motivation and collective capacity to adopt and sustain an innovation, which in this context refers to hemophilia GT. We conducted cognitive pretesting and pilot testing to examine validity. We distributed the survey through each Canadian professional organizations involved in hemophilia care. Data were analyzed using descriptive statistics for quantitative data and coding into categories for qualitative data. Results: A total of 45 HCPs completed the survey from 80.1% (21/26) of Canadian HTCs, with a median experience in hemophilia care of 8 years (interquartile range [IQR] 5-15). Respondents included nurses/nurse practitioners (47.7%), physicians (37.8%), physiotherapists (8.9%), social workers (4.4%), and pharmacists (2.2%). All provinces were represented with the highest participation from Ontario (40%). Perceived Need, Benefits and Acceptance: Most respondents (76%) supported the integration of GT into the Canadian healthcare system, with a more favourable outlook for hemophilia B. Respondents identified a greater perceived need for GT in hemophilia B (76%) compared to hemophilia A (64%) and expressed higher confidence in the sufficiency of clinical evidence for hemophilia B GT (76%) versus hemophilia A (47%). Additionally, patient acceptance was perceived to be higher for hemophilia B given the current treatment landscape. Preparedness and Resources for GT Adoption: Despite their support, respondents raised concerns about HTCs' readiness, including insufficient human resources, patient education and support mechanisms, and clinical capacity for intensive post-GT monitoring. Only 20% felt their HTC had sufficient human resources, stressing needs for more nursing and psychosocial support. Respondents felt unprepared for GT implementation, highlighting low confidence in their HTC expertise and considerable educational gaps and needs, particularly from non-physicians. Careful Candidate Selection for GT: Suitable candidates were characterized by adherence to monitoring and treatment, minimal comorbidities specific to the risks of hepatotoxicity and/or corticosteroids related toxicities, and strong psychosocial stability or support system. Conversely, candidates with significant liver disease or metabolic health issues, poor adherence, psychological distress, and logistical barriers-such as difficulty in traveling to HTC or frequent clinic visits-were deemed less or not suitable. HTCs Coordination and Collaboration: Traditional hub-and-spoke models might not fit Canadian context. Varying opinions were noted regarding shared and individual responsibilities between HTCs. To enhance collaboration and workflow processes, respondents highlighted the need for national guidelines on patient selection, monitoring, and adverse event management. Establishing a national GT advisory group was supported by 67% of the 14 HTC directors, reflecting the need for centralized expertise and guidance. Conclusion: The successful integration of GT into hemophilia care in Canada requires addressing significant logistical, educational, and system-level barriers, enhancing support mechanisms and HTC resources, and ensuring careful candidate selection and coordination. Enhanced training for HCPs, clear clinical guidelines and protocols, and improved patient support mechanisms are essential to fully realize the potential of GT. Establishing a national GT advisory group and leveraging patient registries for long-term monitoring could facilitate the effective and equitable delivery of GT across Canada.
Récupéré en direct depuis OpenAlex et désinversé. Les résumés ne sont pas conservés dans cette base de données : les index inversés représentent 8,6 Go des 9,3 Go de texte de la base, et le serveur dispose de 13 Go libres.
Comment cette classification a été obtenuedéplier
Prédiction distillée sur la base complète
Imitation des enseignantsNi prévalence calibrée, ni vérité terrain. Validation humaine à venir. Apprise à partir de 10 348 étiquettes directes de Codex et de 10 348 étiquettes directes de Gemma. Le mode candidate est l'union des têtes enseignantes seuillées; le consensus est leur intersection. Ces sorties portent le statut machine_predicted_unvalidated et ne sont ni des étiquettes humaines ni des étiquettes directes de modèles de pointe.
Scores Codex et Gemma par catégorie
| Catégorie | Codex | Gemma |
|---|---|---|
| Métarecherche | 0,000 | 0,000 |
| Méta-épidémiologie (sens strict) | 0,000 | 0,000 |
| Méta-épidémiologie (sens large) | 0,000 | 0,000 |
| Bibliométrie | 0,000 | 0,000 |
| Études des sciences et des technologies | 0,000 | 0,000 |
| Communication savante | 0,000 | 0,000 |
| Science ouverte | 0,000 | 0,000 |
| Intégrité de la recherche | 0,000 | 0,000 |
| Charge utile insuffisante (le modèle a refusé de juger) | 0,000 | 0,000 |
Scores machine (provisoires)
Les deux têtes enseignantes du modèle étudiant, lues sur ce travail. Un score ordonne la base pour la relecture; il n'affirme jamais une catégorie, et le statut de validation accompagne chaque rangée tel quel.
Scores de référence d'un modèle non mature (critères de maturité non atteints, 7 itérations). Un score ordonne; il n'affirme jamais une catégorie.
score_only:v0-immature-baseline · tel quel depuis la passe de notation : score_only signifie que le nombre peut ordonner les travaux, et qu'aucune étiquette de catégorie n'en découleClassification
machine, non validéePrédiction automatique; un appel candidat d’une seule tête enseignante, pas un consensus.
Le détail, modèle par modèle et score par score, se trouve en fin de page sous « Comment cette classification a été obtenue ».