Hemophilia: The Paradigm Shift and the Unresolved Challenges

Dear Colleagues,

Over the past decade, hemophilia care has experienced an unprecedented paradigm shift, driven by the advancement of extended half-life concentrates, bispecific antibodies, and, more recently, gene therapy. These advances have transformed the natural history of the disease, improving life expectancy and quality of life for patients with access to treatment. However, this therapeutic revolution coexists with persistent musculoskeletal complications, chronic pain, and significant disparities in global access to care. As people with hemophilia live to an older age, age-related comorbidities are emerging as new clinical challenges. Additionally, female carriers often experience bleeding symptoms and psychological distress associated with genetic transmission. Physiotherapy, rehabilitation, and musculoskeletal ultrasound remain essential components of multidisciplinary care.

This Special Issue invites readers to explore the challenges and opportunities of comprehensive care in hemophilia, under the clinical leadership of hematology, and to study aspects such as musculoskeletal complications, diagnostic tools, quality-of-life assessment, and new models of multidisciplinary care.

We aim to encourage reflection on how to consolidate this new therapeutic paradigm without losing sight of the problems that remain unresolved. Improvements in hemophilia care should not focus solely on innovation and access to emerging therapies but also on providing equitable care that includes physical rehabilitation, psychosocial support, and sustained collaboration among specialists and patient associations worldwide.

Dr. Felipe Querol
Guest Editor

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