“Between the pain, the lack of sleep and the cries of the children, we become very tired. So if there is someone who wants to help us, we really do need that help,” says a young mother from Thiès, Senegal. The story plays out over and over again around the world. The reality is that a majority of people with bleeding disorders still receive inadequate treatment or none at all.
The absence of clotting factor concentrate (CFC) in many developing countries has long meant that children there face a lifetime of physical pain and disability. The long-term effects can be seen in many communities. This is a tragedy—especially because treatment exists and should be made available to those most in need.
During the World Federation of Hemophilia (WFH) 2018 World Congress in May in Glasgow, Scotland, many attendees indicated how this can change. Patients told their stories about how receiving donated treatment products through the WFH Humanitarian Aid Program has drastically changed their lives and communities.
Addressing the Treatment Gap
Since its creation in 1996, the WFH Humanitarian Aid Program has addressed gaps in care. As the volume of donations it facilitates around the world increases, the number of people treated has started to steadily climb. In 2015, Bioverativ, a Sanofi company, and Sobi helped to make the WFH Humanitarian Aid Program more sustainable and predictable. The companies made significant donations of CFCs and committed substantial financial support for the program’s operational needs.
In 2017, the program helped to distribute more than 160 million IUs (international units) of CFC to 60 developing countries where people with bleeding disorders still receive inadequate or no treatment. These donations reached more than 16,000 patients and treated more than 45,000 bleeding episodes. The program also helped make possible 781 major and minor surgeries and allowed 1,210 patients to be put on prophylactic treatment.
Bioverativ’s and Sobi’s contributions translate into 500 million IUs over five years (2015-2020). These companies have been joined by others: Grifols made an eight-year commitment (2014-2021) totaling 200 million IUs; CSL Behring made a 10-year commitment (2009-2018) for 22 million IUs; and Green Cross’ three-year commitment (2017-2019) is for 6 million IUs.
However, more contributors are needed to help increase multiyear donations and ensure a steady flow of treatment products to the WFH network. Donations make it possible for people with bleeding disorders in the developing world to receive treatment for emergency situations, acute bleeds and corrective surgeries and for young children to receive prophylaxis.
Improving Patient Outreach
Working with its national member organizations around the world, the WFH helps to expand capacity in developing countries to achieve accurate laboratory diagnoses, to train healthcare professionals and to assist national patient organizations in furthering their outreach. In tandem with these efforts, the WFH Humanitarian Aid Program provides treatment to the most vulnerable.
“We have seen in the past a certain amount of hesitation on the part of healthcare professionals and patient organizations to conduct active outreach programs, as there was no available treatment in parts of the developing world for newly diagnosed patients,” says Assad Haffar, MD, WFH director of humanitarian aid. “With treatment now available through the WFH Humanitarian Aid Program for those most in need in these countries, healthcare professionals and patient organizations can rely on these donations to treat new patients and conduct outreach programs with more confidence.”
In one poignant case from El Salvador, a family was told by doctors that their son was going to die after he was hit on the head and suffered severe bleeding. Fortunately, WFH-donated factor was available, and it saved the child’s life. Since that incident, the boy has begun receiving prophylaxis, which has allowed him to live a more normal life, like so many other children in El Salvador and around the world. “We are seeing a new generation of patients with hemophilia,” says Armando Estrada, MD, a pediatric hematologist in San Salvador, El Salvador.
The effect of the WFH Humanitarian Aid Program on the lives of patients has been nothing short of transformative. This can only continue with the support of contributors, increased awareness within healthcare systems and the full support of the global bleeding disorders community. Together we can indeed achieve our shared vision of Treatment for All.
Learn more about the WFH Humanitarian Aid Program: treatmentforall.org