Optimizing bleed prevention throughout the lifespan: Womb to Tomb

Abstract

The focus of care providers, patients and families is the ability to tailor care for persons with haemophilia (PWH) across the lifespan. Care requires knowledge of the bleeding disorder and age-related complications, risk of therapeutic interventions, and evaluation of individual characteristics that contribute to outcomes. The ultimate goal is to live a normal life without the burden of bleeding, for PWH and carriers. A wide range of therapeutic options is required to achieve personalized care. Over the last decade, substantial therapeutic advantages have been achieved in the treatment of haemophilia that include the development of a robust array of factor concentrates, novel haemostatic agents, and increased knowledge and awareness of disease associated outcomes and risk factors. Significant strides on the road to accessible gene therapy have been realized. This increased range of therapeutic modalities provides options for development and implementation of care plans for each patient at each stage of life that are more flexible compared to prior care regimens. Paradigms for management of haemophilia are changing. As a community, we must work together to use these resources wisely, to learn from outcomes with new therapies and diagnostic tools, to assure all patients can achieve improved care and outcomes regardless of disease state or country of origin.

Patient‐centred care in haemophilia: Patient perspectives on visualization and participation in decision‐making

“…9 Our results, based on the perspectives of patients, suggest that visualization techniques could be a helpful patient-centred care strategy. 9 Our results, based on the perspectives of patients, suggest that visualization techniques could be a helpful patient-centred care strategy.…”

“…Patient‐centred care is a widely recommended practice in haemophilia . Our results, based on the perspectives of patients, suggest that visualization techniques could be a helpful patient‐centred care strategy.…”

“…9 Dimensions of patient-centred care include respect for patients' preferences; coordination and integration of care; information and education; physical comfort; emotional support; involvement of family or friends; continuity and transition and access to care. 9 Dimensions of patient-centred care include respect for patients' preferences; coordination and integration of care; information and education; physical comfort; emotional support; involvement of family or friends; continuity and transition and access to care.…”

“…Patient‐centred care is increasingly being promoted in order to deliver high‐quality care, including in haemophilia . Dimensions of patient‐centred care include respect for patients’ preferences; coordination and integration of care; information and education; physical comfort; emotional support; involvement of family or friends; continuity and transition and access to care .…”

How I approach: Previously untreated patients with severe congenital hemophilia A

“…The review also discusses the approach to diagnosis, education, and comprehensive care within the hemophilia treatment center (HTC). References addressing the history of hemophilia, long‐term management, pharmacokinetic testing for personalized factor replacement, and novel therapies are listed in Table …”