Management of haemophilia A with inhibitors: A regional crosstalk

Authors: Peyvandi, F; Kavakli, K; El-Beshlawy, A; Rangarajan, S

Affiliations: Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, Milan, Italy. Department of Pathophysiology and Transplantation, Università degli Studi di Milanoo, Milan, Italy. Department of Pediatric Hematology, Ege University Children’s Hospital, Izmir, Turkey. Department of Pediatric Hematology, Faculty of Medicine, Cairo University, Cairo, Egypt. Department of Haematology, University Hospital Southampton NHS Foundation Trust, Southampton, UK. Clinical Trials and Research Unit, KJ Somaiya Super Specialty Hospital and Research Centre, Mumbai, India.

Publication: Haemophilia; 2022

Abstract: INTRODUCTION: The development of inhibitors with factor VIII (FVIII) replacement therapy is one of the most common and challenging complications of haemophilia A (HA) treatment, jeopardizing treatment efficacy and predisposing patients to high risks of morbidity and mortality. The management of patients with inhibitors is particularly challenging in countries where resources are limited. AIM: To provide a comprehensive summary of the management of HA with inhibitors while focusing on differences in practice between Western and non-Western countries and how resource scarcity can impact HA management, leading to suboptimal outcomes in patients with inhibitors. METHODS: Summary of key evidence and regional expert opinion. RESULTS: We address, particularly, the diagnosis of and testing for inhibitors, as well as the epidemiology of inhibitors, including incidence, prevalence, and disease burden. Secondly, we provide an overview of the current treatment landscape in HA with inhibitors regarding the eradication of inhibitors with immune tolerance induction and the treatment and prevention of bleeding with bypassing agents, non-factor replacement agents and other experimental therapies. This is complemented with insights from the authors around the applicability of, and challenges associated with, such therapies in their settings of practice. CONCLUSIONS: We conclude by proposing some key steps towards bridging the gaps in the management of HA with inhibitors in resource-limited countries, including: (1) the collection of quality data that can inform healthcare reforms and policies; (2) improving disease knowledge among healthcare practitioners and patients with the aim of standardising disease management across centres and (3) working towards promoting equal access to HA care and therapies for everyone.