(BPT) - When parents first learn of a child’s hemophilia diagnosis, they often feel scared and frustrated. However, by becoming more educated on the rare bleeding disorder, they often realize how fortunate they are to have access to therapy, a great team of healthcare professionals at their side and the support needed for their child to live a healthy life.
In the United States, no other genetic disease has seen the progress that has been made for hemophilia over the past 40 years.1 Hemophilia is a rare type of bleeding disorder that causes the blood to take a long time to clot as a result of a deficiency in one of several blood clotting factors. 2 In 1970, the life expectancy for someone born with hemophilia was about 20 years old, and today, with access to comprehensive care and treatment with factor replacement therapy, which can treat bleeding episodes or prevent bleeds, those with hemophilia have essentially a normal life expectancy.1
That’s not the case for all people in other countries. “Hemophilia treatment and prevention of bleeding is the goal for patients all over the world,” said Christopher Walsh, M.D., Associate Professor of Medicine, Hematology and Medical Oncology at Mount Sinai Hospital. Unfortunately, 75 percent of people with bleeding disorders around the world still do not have access to adequate treatment.3
World Hemophilia Day gives the community a chance to raise public awareness of progress made to date in hemophilia and reflect on how to best address the challenges that remain.
“The theme of this year’s World Hemophilia Day, ‘Speak out: Create Change,’ encourages the bleeding disorders community toward the goal of raising awareness and improving the treatment of inherited bleeding disorders,” said Patrick F. Fogarty, M.D., Director, Penn Comprehensive Hemophilia and Thrombosis Center and Assistant Professor of Medicine at the Hospital of the University of Pennsylvania. “For instance, the World Health Organization and the World Hemophilia Federation (WHF) have recommended a minimum of one IU of clotting factor concentrate per capita, yet the lack of access to factor concentrate remains a critical issue in many developing countries. To address this, the WHF’s Humanitarian Aid Program distributes donated factor concentrate to areas in need, which is an important step toward this goal.”
Drs. Walsh and Fogarty shared their perspectives as part of two roundtable discussions, hosted by Pfizer, a global leader in hemophilia care, to mark the day and discuss how lessons learned in treatment and grassroots advocacy in the United States can translate elsewhere. Joe Hopcraft, Senior Director, Global Innovative Pharma Business explained, “Pfizer recognizes that the lack of treatment options in developing countries is a significant challenge, and will continue to work with patient advocacy groups to address this.”
Len Azzarano, a parent of a child with hemophilia, also participated in the Pfizer events and said that he left feeling inspired by what is possible when different members of the community partner to create change. “My hope is that, by sharing our stories and experiences and working together, we’ll be able to help families around the world live better lives with hemophilia.”
Follow these tips to help advocate for people with hemophilia all year long:
- Educate yourself about hemophilia. Visit http://www.hemophiliavillage.com/ for tools, resources and information.
- Learn how you can get involved in the National Hemophilia Foundation’s advocacy efforts at advocacy.hemophilia.org
- Support the work being done around the world by the World Federation of Hemophilia (WFH). For example, the WFH Twinning Program, of which Pfizer has been a longtime sponsor, pairs emerging hemophilia treatment centers and patient organizations with more established ones around the globe to help improve diagnosis and medical attention for people with hemophilia.
Tips for Effective Grassroots Advocacy
- Identify your issue
- Know your facts
- Build your base of support
- Know your opposition
- Know the legislative process
- Use the media to raise awareness of your issue
1 Kaufman RJ. Powell JS. Molecular approaches for improved clotting factors for hemophilia. blood. 2013; September 24: 1-8. doi: 10.1182/blood-2013-07-498261
2 National Heart Lung and Blood Institute. “What is Hemophilia.” Accessed 22 April 2014. Available at http://www.nhlbi.nih.gov/health/health-topics/topics/hemophilia/
3 World Federation of Hemophilia. “World Federation of Hemophilia: Cornerstone of global development.” Accessed 22 April 2014. Available at http://www.wfh.org/en/about-us