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.