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.

Hematology Reports
|June 25, 2026
PubMed

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.

Related Concept Videos

Issues And Trends In Healthcare Delivery System01:29

Issues And Trends In Healthcare Delivery System

The issues and trends in healthcare delivery are constantly changing. The COVID-19 pandemic is one recent issue that wreaked havoc on healthcare systems, causing a shortage of healthcare workers, high demand for medicines and supplies, and increased medical expenditure due to a lack of insurance. Other issues include rising healthcare costs and care fragmentation.
Cost Containment
Payment for healthcare services has historically promoted adoption of costly and often unnecessary or inefficient...
Disorders of Hemostasis01:24

Disorders of Hemostasis

Hemostasis, the process that stops bleeding after a blood vessel injury, is crucial for maintaining the integrity of the circulatory system. However, disorders of hemostasis can disrupt this delicate balance, leading to either excessive clotting or bleeding. These disorders can be broadly classified into thromboembolic disorders and bleeding disorders.
Thromboembolic Disorders
Two factors primarily cause thromboembolic conditions.
Dimensions of Health and Illness01:21

Dimensions of Health and Illness

The factors influencing the health-illness continuum can be internal or external and may or may not be under conscious control. They are related to the following eight human dimensions, and each dimension is interrelated to one other.
Peripheral Artery Disease III: Interprofessional Care01:27

Peripheral Artery Disease III: Interprofessional Care

Peripheral Artery Disease (PAD) is characterized by narrowed arteries that diminish blood flow to the extremities. Effective management of PAD requires an interprofessional approach involving various healthcare professionals. The critical aspects of interprofessional care for PAD patients focus on risk factor modification, drug therapy, exercise therapy, nutrition therapy, critical limb ischemia care, and interventional radiology and surgical procedures.The primary treatment goal for PAD...
Bone Disorders01:29

Bone Disorders

Aging and its effect on bone remodeling is the most common cause of bone disorders. In young and healthy people, bone deposition and resorption happen at an equal rate to maintain optimal bone health.
Bone deposition is also affected by the levels of sex hormones like estrogen and testosterone that promote osteoblast activity and bone matrix synthesis. When the level of these hormones decreases due to aging, it causes a reduction in bone deposition. As a result, bone resorption by osteoclasts...
Bias in Epidemiological Studies01:29

Bias in Epidemiological Studies

Biases can arise at various stages of research, from study design and data collection to analysis and interpretation. Recognizing and addressing these biases is essential to ensure the validity and reliability of epidemiological findings.Broadly speaking, biases in epidemiology fall into three main categories: selection bias, information bias, and confounding. A more detailed description of possible biases is: