Normalisation of Haemostasis in Haemophilia A

Abstract

Haemophilia A (Factor VIII [FVIII] levels ≤40 IU/dL) is a chronic condition with consequences beyond bleeding complications. Many people with haemophilia A (PwHA) experience pain, joint damage, psychosocial impacts, restrictions in daily activities, and limitations in physical activities. Cédric Hermans, Professor at the Cliniques Universitaires Saint-Luc, Brussels, Belgium, outlined how ambitious treatment goals, beyond converting severe haemophilia A into a more moderate or mild form of the condition, are required. With new treatments, it will be possible to target FVIII activity levels in the non-haemophilia range (>40 IU/dL), allowing PwHA to reach freedom from bleeds, leading to a haemophilia-free mindset, and comparable quality of life (QoL) with their peers. Maria Elisa Mancuso, Senior Haematology Consultant at IRCCS Humanitas Research Hospital, Milan, Italy, highlighted the evolution of haemophilia A treatments; she showed clinical evidence that a zero-bleed goal may require sustained FVIII activity levels >40 IU/dL for complete protection against all types of bleeds and joint damage. Rubén Berrueco, Paediatric Haematologist at the Sant Joan de Déu Barcelona Children’s Hospital, Spain, described the haemophilia paediatric patient journey, and how uncertainties related to bleeds and treatment burden pose unique challenges for children and their caregivers. He presented his perspectives on challenges with current treatments (e.g., delayed inhibitor development, subclinical bleeds, and lack of skills for intravenous administration) and the need to improve self-autonomy and decrease hospital dependency. New treatments to achieve the non-haemophilia range of FVIII could address current unmet needs. The experts discussed that treatments for many diseases (e.g., diabetes, hypertension) aim to restore normal values (blood sugar, blood pressure), which was not the case until now for haemophilia. A more patient-centred approach with treatments targeting normal values of FVIII could allow all PwHA to become mentally and physically liberated from the constraints of their condition, and to live with optimised health and well-being.