World Haemophilia Day 2023: What patients need to know about #WHD2023

On Monday, 17 April, World Haemophilia Day (#WHD2023) is commemorated globally. The initiative is driven by the World Federation of Haemophilia (WFH), the South African Haemophilia Foundation (SAHF) and supported by global healthcare organisations. These include Roche Pharma Southern Africa, a leader in haemophilia care and a global advocate for patient access and support. The #WHD2023 theme is “Access for All: Prevention of bleeds as the global standard of care”. To highlight the theme, Dr Nick Mangeya, Country Medical Director & Africa Genomics Programme Partnerships Lead at Roche South Africa, and Clerment Masoabi Sefojane, a young adult living with severe haemophilia and Chairman of the SAHF, outline some of the realities of this lesser-known disease in 2023.Haemophilia is a genetic disorder that affects the blood's ability to clot. This results in excessive bleeding from even minor injuries and even spontaneous bleeding in certain cases. The condition is caused by a deficiency in one of the clotting factors in the blood – typically factor 8 (haemophilia A – Classic haemophilia) or factor 9 (haemophilia B).1The most common form of haemophilia is haemophilia A, caused by factor 8 deficiency.2 Haemophilia is usually inherited. The mutation that causes the condition is passed down from parent to child, and it primarily affects males. Females can also carry the gene and pass it on to their children, but they are typically not affected by the disorder themselves – though there are exceptions.1

Speaking about the importance of World Haemophilia Day, Mr. Masoabi Clerment Sefojane, Chairperson of the South African Haemophilia Foundation said, “World Haemophilia Day serves as an opportunity to raise awareness about bleeding disorders, advocate for access to care, and mobilize resources towards addressing the challenges faced by patients with bleeding disorders, particularly in rural and underdeveloped communities.”

Says Dr Mangeya, “We are always looking for new ways to collaborate with key stakeholders including healthcare professionals, patient organisations and national healthcare systems, to understand barriers to access and to identify and deliver novel, tailored solutions to overcome local challenges and bridge current gaps in care for people with haemophilia all over the world.”Most people with bleeding disorders in developing countries have no access to diagnosis, treatment and care. Because of this, children and adults who have haemophilia face an uncertain future.3

On World Haemophilia Day and beyond, generating widespread awareness is critical. At Roche, we aim to strengthen connections in communities to deliver effective haemophilia A treatments for all patients.“We are collaborating with the haemophilia community to facilitate discussions and to increase understanding and awareness of the need for and benefit of effective haemophilia A therapy for all affected individuals.”

While haemophilia cannot be cured, people with haemophilia can live normal lives. Treatment involves replacing the missing blood clotting factor with a concentrate administered by injection so that the blood can clot properly.4

As a result of treatment, however, up to one in five people with haemophilia develop an antibody (inhibitor) that prevents the clotting factor from working to stop bleeding. This inhibitor makes treatment for bleeding episodes much more challenging.4

Sefojane elaborates on barriers to haemophilia care in Africa: “It is essential to understand that haemophilia is a condition that requires a multi-disciplinary treatment approach, including physiotherapy, rehabilitation, and mental health support. However, due to the lack of education and access to care, many people in underdeveloped communities are unable to get the proper treatment they require.

Mangeya concludes, “Education and access are the most powerful tools in addressing haemophilia. As healthcare providers, we are working to support patients’ understanding of their condition by spreading improved, useful and accessible information about the disease.“Patients with haemophilia can live better lives through improved management of their condition. Let’s support more patients along their journey – building partnerships to understand and provide solutions for real, everyday hurdles – physical, psychological and social – that people with haemophilia A, and their caregivers, face.”The Humanitarian Aid Programme – an initiative by the WFH – is working in partnership with Roche and other strategic partners to help address and improve access to haemophilia treatment in developing countries.For decades, the only treatment for people living with haemophilia A was blood transfusions during or after bleeding events.5 In the current era, patients have more innovative choices in treatment, and partnerships of this nature are finding innovative vehicles to ensure that all patients everywhere have the same access to innovation.By working toward a shared treatment vision for all, the WFH and all of its partners, including Roche, aim to address gaps in care and enable people with a high need for treatment to experience an improved quality of life and longer-term protection against bleeding episodes.To find out more about haemophilia, visit https://www.roche.com/stories/a-to-z-in-haemophilia/ or  https://haemophilia.org.za/.  

About Roche:

With a uniquely broad spectrum of innovative solutions, Roche is a leading healthcare company that has for over 100 years been active in the discovery, development, manufacture, and marketing of healthcare solutions. Our products and services address prevention, diagnosis, and treatment of diseases, thus enhancing well-being and quality of life.

Headquartered in Basel Switzerland, Roche employs over 85 000 people globally and sells its products in over 150 countries. Our multinational presence reinforces our ability to offer our healthcare solutions worldwide and to anticipate needs in all regions of the world. Roche South Africa employs over 400 people within the pharmaceutical and diagnostics divisions. We are committed to maintaining high ethical standards in our business dealings and in our efforts to protect the environment.

 

About SAHF:

The South African Haemophilia Foundation (SAHF) is a non-profit organization that was established in 1970. The foundation is dedicated to advocating for the needs of the bleeding disorders community in South Africa. SAHF's mission is to ensure that people with bleeding disorders have access to appropriate and quality care, education, and support. The organization also promotes research into bleeding disorders and encourages patient empowerment through education and community outreach.

The SAHF works closely with healthcare professionals (HCPs) through its Medical and Scientific Advisory Council (MASAC). The council is comprised of experts in the field of bleeding disorders who provide guidance and support to the foundation's efforts. Together, SAHF and MASAC strive to improve the lives of people with bleeding disorders in South Africa through education, research, and advocacy. Through its various programs and initiatives, SAHF continues to make a significant impact in the bleeding disorders community in South Africa.

References:

1. CDC (2022) What is Hemophilia, Centers for Disease Control and Prevention. Centers for Disease Control and Prevention. Available at: CDC_What is Haemophilia (Accessed: April 4, 2023).

2.  Guidelines: Treatment Guidelines for Haemophilia in South Africa \

3. World Federation of Hemophilia. Achieving Global Impact – Strategic plan: 2021-2025 [Internet; cited May 2022]. Available from: https://wfh.org/wp-content/uploads/2021/12/WFH-Strategic-Plan-2021-2025.pdf.  [Last accessed April 2021]

4. CDC (2022) Stories from people with hemophilia, Centers for Disease Control and Prevention. Centers for Disease Control and Prevention. Available at: https://www.cdc.gov/ncbddd/hemophilia/features/hemophiliaday.html# (Accessed: April 4, 2023).

5. National Haemophilia Foundation (no date) History of Haemophilia, National Hemophilia Foundation. National Haemophilia Foundation. Available at: https://www.hemophilia.org/bleeding-disorders-a-z/overview/history (Accessed: April 5, 2023).

Cassien Tribunal Aungane, Editor