Thalassaemia International Federation
By the late 1980s, the prevalence of thalassaemia - a disorder of the haemoglobin molecule inside red blood cells - had decreased in Europe and in Mediterranean countries (Northern Africa and the Middle East). The success of prevention programs in Cyprus and Italy, for example, resulted by the mid-1980s in the dramatic reduction of the number of affected births, plummeting to almost zero in Cyprus by 1986. In addition, thalassaemia patients in these countries enjoyed a reasonably good quality of life with the medical care that was provided to them. National thalassaemia patient groups played a vital role in the initiation and implementation of the control programs, which combined prevention and medical care. But they had another important issue to tackle. ‘Scientists had lost interest in thalassaemia, because there were very few new affected births every year in these countries. It was believed that gradually there would be no patients to treat,' says Dr Androulla Eleftheriou, Executive Director of the umbrella organisation. ‘We had to find a way to encourage and motivate them again to promote research programs to improve the lives of our children living with thalassaemia. This is one of the reasons why patient groups from different parts of the world got together in 1986 to create the Thalassaemia International Federation (TIF).'
Treatment for thalassaemia patients consists of blood transfusion and iron regulation drugs and is a life-long process. Without adequate treatment, the life expectancy of a thalassaemia patient is short, and their quality of life very poor. ‘TIF promotes and supports research that leads to the continuous improvement of treatment and to a final cure for the disorder,' says Androulla Eleftheriou. In fact, to this date, the only validated cure for thalassaemia is bone marrow transplant, but as Dr Eleftheriou explains, ‘even under the best conditions and after fulfilling all criteria and pre-requisites, it can only address the needs of about 30% of the patients. In addition, it is still a very expensive procedure, not accessible to the great majority of our patients.'
The second motive behind the creation of TIF was the high prevalence of the disease in the Middle East, the Indian sub-continent, and throughout South East Asia (Southern China, Thailand, Malaysia). ‘In these countries, patients are still dying at a young age as a result of inadequate treatment and very often remain without diagnosis. TIF founding members believed that the experience of patient groups in the West could be transferred to those countries, where there were no patient groups and where control programs did not exist or were poorly implemented,' states Dr Androulla Eleftheriou. TIF has established official collaboration with the World Health Organisation (WHO) since 1996 to support its work towards promoting control programmes worldwide. Since its creation, the Federation has developed a wide range of activities in some 60 countries; it has now more than 90 national thalassaemia associations as members.
One of TIF's most important and successful activities is its educational programme. The programme prepares, publishes, translates and distributes free-of-charge updated and validated information on the disease in the form of leaflets, books and CDs to national health authorities, national patient associations and health professionals worldwide. ‘TIF also organises field trips and delegation visits to individual countries or regions to compile information on the status of control programs in an effort to tailor our support and guidance, according to specific country or regional needs,' explains Dr Eleftheriou.
Since the beginning of the 21st century, there has been an unexpected need to re-address control programmes in Northern Europe. ‘Due to the migration of populations from highly affected countries, thalassaemia now constitutes a very serious public health issue in all European countries. Their national health services had not developed control programmes because the disorder was non-existent or extremely rare in the native population. The result is that in some Northern European countries, despite the existence of high quality public health services, patients with thalassaemia have a very poor quality of life,' exclaims Dr Eleftheriou. Thus, after working hard to control thalassaemia in Mediterranean countries and the developing world, TIF is now returning to all of Europe to raise awareness on the need for national screening programs and protocols for medical care throughout the European continent. ‘We will be holding a workshop on thalassaemia during the 4th European Conference on Rare Diseases (ECRD) in Lisbon in November 2007. The UK has only last year set up a national screening programme for haemoglobin disorders and we hope that many other European countries will follow suit. Results of our work show that TIF has had a positive impact on national health authorities and we are very pleased to become members of Eurordis and start collaborating. We are confident that we can rely on Eurordis' expertise and network to help us have a greater impact on patients, and medical and pharmaceutical societies in Europe,' says Dr Androulla Eleftheriou.
This article was previously published in the May 2007 issue of our newsletter.
Author: Nathacha Appanah
Photos: All photos except blood cells © TIF