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The Global Gap in the Hemophilia Paradigm Shift: Disparities in Research, Care, and Musculoskeletal Health
Felipe Querol-Giner1, Magdalena Querol-Giner2, Ana Chimeno-Hernández3,4
1PTinMotion Research Group, Department of Physiotherapy, University of Valencia, 46010 Valencia, Spain.
Background: Hemophilia care has undergone a major therapeutic transformation with the introduction of extended half-life products, non-replacement therapies, and gene therapy. However, the benefits of these advances are not equally distributed worldwide, and their impact on long-term musculoskeletal outcomes remains uncertain. Objective: To analyze global disparities in hemophilia care and research production in the context of recent therapeutic advances, with particular attention to musculoskeletal management, physiotherapy, and scalable strategies for resource-limited settings. Methods: A narrative review with a structured literature search was conducted. Two conceptual blocks were explored: global disparities and access to care in hemophilia, and recent therapeutic advances, including non-replacement therapies, extended half-life products, and gene therapy. Retrieved records were screened using Rayyan, and a structured workflow diagram was used to summarize the literature identification and selection process. A descriptive analysis was also performed to identify representative authors, institutions, and geographic patterns in hemophilia research. Results: The evidence shows substantial global disparities in diagnosis, access to treatment, healthcare infrastructure, and research production. Scientific output remains concentrated in high-income countries, while low- and middle-income regions are underrepresented. Advanced therapies consistently reduce bleeding rates and treatment burden, but concerns remain regarding access, affordability, durability, breakthrough bleeding, and long-term structural joint outcomes. Musculoskeletal complications, including subclinical bleeding and hemophilic arthropathy, remain clinically relevant despite improved hematologic control. Conclusions: The current paradigm shift in hemophilia care is not uniformly experienced worldwide. Addressing global disparities requires not only expanding access to advanced therapies, but also strengthening research capacity, implementing multidisciplinary care models, and integrating scalable interventions such as physiotherapy, patient education, and simplified diagnostic tools. Accessible musculoskeletal assessment strategies may help improve early detection, functional outcomes, and equity of care in resource-limited settings.
Background: Hemophilia care has undergone a major therapeutic transformation with the introduction of extended half-life products, non-replacement therapies, and gene therapy. However, the benefits of these advances are not equally distributed worldwide, and their impact on long-term musculoskeletal outcomes remains uncertain. Objective: To analyze global disparities in hemophilia care and research production in the context of recent therapeutic advances, with particular attention to musculoskeletal management, physiotherapy, and scalable strategies for resource-limited settings. Methods: A narrative review with a structured literature search was conducted. Two conceptual blocks were explored: global disparities and access to care in hemophilia, and recent therapeutic advances, including non-replacement therapies, extended half-life products, and gene therapy. Retrieved records were screened using Rayyan, and a structured workflow diagram was used to summarize the literature identification and selection process. A descriptive analysis was also performed to identify representative authors, institutions, and geographic patterns in hemophilia research. Results: The evidence shows substantial global disparities in diagnosis, access to treatment, healthcare infrastructure, and research production. Scientific output remains concentrated in high-income countries, while low- and middle-income regions are underrepresented. Advanced therapies consistently reduce bleeding rates and treatment burden, but concerns remain regarding access, affordability, durability, breakthrough bleeding, and long-term structural joint outcomes. Musculoskeletal complications, including subclinical bleeding and hemophilic arthropathy, remain clinically relevant despite improved hematologic control. Conclusions: The current paradigm shift in hemophilia care is not uniformly experienced worldwide. Addressing global disparities requires not only expanding access to advanced therapies, but also strengthening research capacity, implementing multidisciplinary care models, and integrating scalable interventions such as physiotherapy, patient education, and simplified diagnostic tools. Accessible musculoskeletal assessment strategies may help improve early detection, functional outcomes, and equity of care in resource-limited settings.
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