Portal de Periódicos da CAPES

World Federation of Hemophilia: 50 years of advancing treatment for all

2013; Wiley; Volume: 19; Issue: 4 Linguagem: Inglês

10.1111/hae.12200

1365-2516

Mark W. Skinner, E. Myles,

Cancer-related gene regulation

For 50 years, the World Federation of Hemophilia (WFH) has been committed to improving care for people with bleeding disorders regardless of where they live. The WFH has developed successful programmes and activities to share knowledge through information exchange, education and training; improve diagnosis and treatment in developing countries; build capacity of National Member Organizations (NMO) to effectively serve the bleeding disorder community; and monitor and promote safety and supply of products used to treat bleeding disorders. The WFH has also established a global network of knowledgeable and committed volunteers, both medical and lay, who support the work of the WFH. Thanks to these programmes and volunteers, the WFH has become the leading global organization representing the bleeding disorder community and the cornerstone for global development. Due to the work of the WFH, substantial progress to close the global gap in care has been realized. However, our work is not complete and there remains vast unmet needs. It is well established that, with proper treatment, people with haemophilia can live perfectly healthy lives. Without treatment, the reality is that many will die young or, if they survive, will suffer joint damage that leaves them with permanent disabilities. Too many patients remain undiagnosed and too few receive adequate treatment. The WFH remains committed to its vision of achieving Treatment for All. On June 25, 1963, the WFH was founded when Frank Schnabel, then president of the Canadian Hemophilia Society, along with leaders from other national patient associations organized a global meeting, in Copenhagen, Denmark, to establish an international haemophilia organization. There were representatives from 12 countries: Argentina, Australia, Belgium, Canada, Denmark, France, Germany, Japan, the Netherlands, Sweden, the United Kingdom and the United States. From these original 12 countries, the WFH has grown to 122 NMOs covering 95% of the world's population (Fig. 1). Schnabel had a dream to 'alleviate the pain and plight of the world's haemophiliacs' 1. His opening words to those assembled still ring true. 'The threat to the life of just one haemophiliac would be sufficient reason for us to travel to this meeting. We are here however to help the hundreds of thousands of haemophiliacs by adding another organization which can be instrumental, in liaison with national societies' 1. Schnabel had severe haemophilia and, as for so many people around the world today, he grew up facing an unsure future with the prospect of a life of pain, disability and early death. Inspired by the difficulties he faced, he began a long crusade to change this reality. There were many others involved in the early and formative years of the WFH who served either as the interim (1963) or first (1964) officers or led the medical advisory board including: Sir Weldon Dalrymple-Champneys (UK), Prof. Kenneth Brinkhous (US), Henri Chaigneau (France), Cecil Harris, MD (Canada), E. Neumark, MD (UK), Knut-Eric Sjolin, MD (Denmark), Prof. J.P. Soulier (France), John Walsh (US), S. Van Creveld, MD (The Netherlands). Since its inception, the WFH held world congresses regularly and created a global network of health care providers, national haemophilia associations and people with haemophilia and their families. The WFH congress was first held in conjunction with other meetings, such as the International Society for the Rehabilitation of the Disabled 2. Peter Jones, MD, wrote: 'Congresses brought together members of the entire haemophilia community: health care providers, representatives of national haemophilia organizations, people with haemophilia and their families, pharmaceutical companies and researchers. Through these meetings, a truly global haemophilia network emerged that has continued to grow in size and influence. Schnabel explained that, "because of its scope, the Federation is able to provide certain services and facilities which enhance the activities of the national organizations. One of the most important aspects of the WFH is that it provides a mechanism for the exchange of information on a global scale", 2. Over the years, the WFH World Congress has evolved to become the world's largest scientific meeting dedicated to bleeding disorders. In 2012, the WFH held its 30th congress in Paris, France, attracting over 5400 participants from over 115 countries. The WFH reached a turning point in 1969 when, thanks to the work of Chaigneau and others, the WHO established official relations with the WFH. This recognition was instrumental in advancing its international reputation and attracting other national patient organizations. In 1970, the WFH launched its first global development programme, the International Hemophilia Treatment Centre (IHTC) Program, conceived by medical secretary Anthony Britten, MD, a doctor with severe haemophilia 3. In 1972, Pier Mannucci, MD, took over as IHTC chair and the programme vision refocused on training. The programme was renamed the International Hemophilia Training Centre Program. Through the 1970s, the IHTC Program provided intensive specialized training to members of the multidisciplinary team from the developing world through fellowships and workshops. In an IHTC history by Kevin Rickard, MD, (IHTC chair, 1986–96), he attributed much of the programme's early success to the 'enterprising, imaginative, productive, and forceful leadership' of Mannucci, who served as IHTC chair for 14 years (1972–86) 4. IHTC was often referred to by Rickard as the 'Jewel in the Crown' of the WFH 2. One of the key IHTC learnings has been that training is most effective when carried out in an environment similar to that of the trainees. In Thailand, the WFH worked with Prof. Parttraporn Isarangkura to promote progress in national haemophilia care. Under her direction, the Bangkok centre became a major venue for training on how to provide maximum treatment benefits with limited resources, and her centre eventually became an IHTC. Since the beginning of the programme, 550 individuals from 80 countries covering all medical disciplines have been awarded fellowships to train at one of the recognized training centres 5. In 2011, a long-term evaluation of the impact of the IHTC programme was conducted of 135 fellows during 2006–10. The study found that 93% of the 128 respondents continue to work in haemophilia care at the time of the survey and 98% planned to continue for the next 5 years demonstrating the sustainable value of the programme 6. Postfellowship, the network of fellows also provides an important base for many other WFH development programmes. Although the 1970s saw a revolution in treatment with the availability of plasma-derived clotting factor concentrates (CFC), global access to skilled care was still lagging. Thus, the WFH organized an international conference in Bonn, Germany, to develop a blueprint for haemophilia care in the 1980s to 'expand contemporary comprehensive care of hemophiliacs' for the world. 7. However, when AIDS hit the haemophilia community in 1982, the sense of hopefulness that marked the beginning of the decade quickly turned to darkness and despair. 'AIDS was totally unexpected,' wrote Mannucci. 'And a vision of progress and optimism was overtaken by one of gloom and despair. There was uncertainty and confusion' 8. In 1983, at the WFH Congress in Stockholm, Sweden, Bruce Evatt, MD, presented data connecting HIV infection in haemophilia patients and CFCs. In response, the WFH set up the World Hemophilia AIDS Center with the Los Angeles Orthopaedic Hospital, under the direction of Shelby Dietrich, MD, to provide rapid access to vital information about the disease. Worldwide, tens of thousands of people with haemophilia contracted HIV and hepatitis from their treatment products. Among the victims was Frank Schnabel, who died in 1987. Until the end, he reaffirmed his vision with the words: 'We are going to emerge victorious' 9. Having witnessed what blood-borne viruses did to a generation of boys, men, their wives and families with haemophilia, the community also took action to make sure such a thing would never happen again. 'I saw people die, friends of mine who died,' said David Page, chair of the WFH Blood Safety, Supply and Availability Committee. 'We said, never again on our watch. We will do everything we can to make sure that doesn't happen' 10. Since 2000, the WFH has hosted a biannual global forum on the safety, supply and availability of treatment products to discuss and debate issues with all stakeholders together and in 2002 launched a global series of workshops to train regulators how to evaluate product safety. Charles Carman, a US business professional, was elected WFH president in 1988. During his tenure, he introduced important management structures and broadened the WFH's funding base. Under his leadership, medical experts and leaders of national haemophilia associations met in Paris, France, in April 1990, to develop The Decade Plan, a strategic plan launched in 1992, designed to carry the WFH into the next millennium 11. The Plan identified critical issues and necessary steps to advance the development of the comprehensive care model globally. Unfortunately, Charles Carman did not see the fruition of his work. He stepped down as president in 1993, and died in 1995. Rev. Alan Tanner (UK) served as acting president until the next WFH General Assembly in 1994, at which Brian O'Mahony (Ireland) was elected president. O'Mahony unified the governance of WFH bringing together the WFH's Executive Committee and Council, into one body, composed equally of doctors and people with a bleeding disorder. Greater access to improved products, self-treatment and prophylaxis in developed countries highlighted the stark differences with developing countries. Under O'Mahony, along with WFH Executive Director Line Robillard, VP Medical Carol Kasper, MD and Evatt the WFH focused its efforts more on the developing world, designing programmes to help countries help themselves leading to sustainable national care programmes. WFH activities also expanded to include safety and supply, data and demographics, laboratory training, humanitarian aid and capacity building for its NMOs. One major step was the introduction of the WFH Twinning Programs in 1994–95, pairing up haemophilia organizations and treatment centres in developed countries with those in developing countries. 'Dr. Guglielmo Mariani of Italy had the idea of 'twinning' a well-established haemophilia [treatment centre] programme with a new or struggling one,' wrote Kasper. 'It worked so well that twinning of national haemophilia organizations was added' 12. Operation Access, a health care development project in Chile, represented the WFH's first major success in achieving rapid and significant improvement in haemophilia care. The WFH brought together what came to be called the 'winning coalition' wherein the national patient organization carried out an educational and advocacy role, the Ministry of Health agreed to establish a national haemophilia programme, a key treater coordinated the project's implementation, others received specialized training and manufacturers donated treatment products. The WFH served as a catalyst and adviser. The lives of Chileans with haemophilia changed dramatically in 5 years and the 'winning coalition' was adopted as part of the WFH development strategy. Based on these early health care development projects, the essential elements for a systemic integrated model to introduce and develop sustainable national care emerged. The WFH Development Model (WFH Model) was created by Evatt, Kasper, O'Mahony, Robillard and WFH Programs Director Claudia Black. These elements, which are interdependent, comprise (i) ensuring accurate laboratory diagnosis; (ii) achieving government support for a national programme; (iii) improving the care delivery system; (iv) increasing the availability of treatment products; and (v) building a strong national patient organization 13. A sixth element, the ability to track and report patient health outcomes, was added in 2013. When the WFH first began meeting with governments, they were asked to provide supportive data; for example, governments wanted to know how many people were affected, what treatment and care cost and how many had complications. In response, in 1998, the WFH developed a survey to collect basic demographic information as well as data on care delivery, treatment products and complications such as HIV and hepatitis C. Over time, these data have also allowed WFH to track programme progress, target resources and identify development needs 14. Historically, WFH country development programmes focused on one or two elements of the WFH Model. To advance care further and faster, O'Mahony had an idea for a new intensive programme that would work on all five development elements at once to close the gap in diagnosis, access to treatment products and mortality that existed between developed and developing countries. On World Hemophilia Day (April 17) in 2003, the WFH launched the Global Alliance for Progress (GAP) Program, which was a culmination of all that the WFH had learned about building sustainable care. This 10-year health care development initiative aimed to greatly increase the diagnosis and treatment of people with haemophilia in 20 developing countries. An overarching goal was to identify 50 000 people with haemophilia globally. In 2013, GAP celebrated its tenth anniversary and a decade of success in achieving demonstrable change in each of the 20 participating countries (Algeria, Armenia, Azerbaijan, Belarus, China, Ecuador, Egypt, Georgia, Jordan, Lebanon, Mexico, Moldova, Morocco, Peru, Philippines, Syria, Thailand, Tunisia, Russia and South Africa). In these countries, to date 26 381 patients with haemophilia were identified and national care programmes were established in 16 countries. Globally, the number people diagnosed with haemophilia increased from 105 971 in 2003 15 to 167 110 in 2011 16. Although the success of health care development projects like GAP depend on a winning coalition it is often the patient leaders, those who have the most to gain or lose, who are the local drivers for change. To better prepare the WFH NMOs in the late 1990s, the WFH began developing workshops to help patient leaders develop the skills to lobby their government for improved care. This grew into a global advocacy in action training programme launched in 2006. So far, over 1000 patient organization leaders have been trained and gone on to improve care in their own countries. Soon after being elected WFH president in 2004, Mark Skinner (US) led the WFH to adopt the vision of Treatment for All, which is the foundation upon which the WFH's global development strategy is built today 17. Treatment for All means that one day treatment will be available for all patients with inherited bleeding disorders regardless of where they live. It also means more than simply access to treatment products. It means: It is well established that, with proper treatment, people with haemophilia can live perfectly healthy lives. Without treatment, the reality is that many will die young or, if they survive, suffer joint damage that leaves them with permanent disabilities. The WFH works closely with government agencies, industry, clinicians and patient groups to achieve both the quality and desired quantity of treatment products. Access to treatment has been steadily increasing since the WFH first began collecting data on clotting factor concentrate usage in 2001 (Fig. 2) 18. Over the last 50 years, diagnosis and care for people with haemophilia have evolved greatly, but for other bleeding disorders, recognition and the level of care have not developed at the same rate. Innovative strategies and tools are needed to reach these vulnerable and underserved populations. Traditional outreach techniques may not be optimal approaches to identify women with bleeding disorders. To address this need, the WFH piloted a VWD outreach model suitable for developing countries in Egypt, Lebanon and Mexico. Targeted multilingual educational resources have been developed on VWD, rare factor deficiencies and inherited platelet disorders 19. The WFH work is not done, the gap in care still exists, and treatment for all is not yet a reality. Therefore, to mark the WFH's 50th anniversary, under the new leadership of WFH President Alain Weill (France) and WFH CEO John Bournas, the WFH has launched three new key initiatives, which are being funded through our 50th anniversary Close the Gap campaign. They are: the continuation of GAP (2013–22), a new initiative to address underserved countries and regions (The Cornerstone Initiative) and the WFH research programme 20, 21. Over the past 50 years, we have seen enormous advances in the treatment and care of bleeding disorders. Even though the reality of the past remains the reality of the present for many, the future for all is indeed bright. The WFH has played a critical role in bringing treatment and care to many parts of the world (Tables 1 and 2) and is well positioned to continue the quest to achieve Treatment for All in the years ahead. Working together as a global family, each year, we will move one step closer to closing the gap in care and achieving Treatment for All. Many people ask how WFH achieves as much as it has, and it is thanks to the hundreds of volunteers and WFH professional staff in our Montreal headquarters. WFH volunteers are leaders from haemophilia treatment centres, representatives from national haemophilia associations, specialists from government agencies, all of whom bring their skills and expertise to advance Treatment for All. Our thanks go as well to the many donors, supporters and partners who provide financial support. Including most notably Jan-Willem André de la Porte, an active sportsman, entrepreneur, businessman and generous supporter of the bleeding disorder community for many years who was invited in 2001 to become WFH Patron. The authors have no relevant conflicts to disclose. Mark Skinner is a former president of the WFH and Elizabeth Myles is the WFH Chief Operating Officer.