The second phase of the project “National registry of patients with thalassemia major in Bulgaria” was successfully completed in October 2010. The aim was to update the information on patients who were registered during the first phase of the project and to register newly diagnosed and not yet registered patients with thalassemia major. A total of 241 questionnaires (18 for primary epidemiological information about newly diagnosed patients and 223 for data update) was collected with the active assistance of medical specialists from blood transfusion centers and thalassemia patient association. After statistical processing of the submitted data, it was found that the number of men is 126 (52.28%) and 115 (47.72%) for women respectively. The average age of patients with thalassemia major is 19.5 ± 11.9 years. Their treatment is conducted in 12 hospitals in the country and chelation therapy takes place in the blood transfusion specialized centers (Sofia – three centers, Plovdiv – two centers, Varna, Stara Zagora, Pleven, Burgas, Ruse, Yambol and Silistra). These results were discussed and adopted as official for the country at a workshop of the Expert group on thalassemia, held in Varna in November. A subsequent update and collection of new epidemiological data in will be organized in March-April 2011.
BURLQOL-RD Project, funded by the EC, officially started on 20 April 2010. Its main outcome will be an integrated and harmonized set of instruments to assess and monitor socio-economic burden and health-related quality of life (HRQOL) of patients affected by rare diseases and their caregivers in EU. The Information Centre for Rare Diseases and Orphan Drugs through BAPES is an associated partner in this project. For further information – click here or visit the project official website.
Updated information from the National thalassaemia major registry was obtained in March 2010. Results show that there are 223 thalassaemia major patients in Bulgaria, who are treated in eight centres – Sofia (90), Plovdiv (54), Varna (26), Stara Zagora (23), Burgas (15), Pleven (9), Gabrovo (2). The crude prevalence for Bulgaria is 2.93/100 000 persons. The average age of the registered patients is 19.5 years. By the end of the year a process of updating and registration of new patients will be conducted too. More information about the results of the register can be found here.
The project is implemented as a result of the common work and cooperation between BAPES, ICRDOD, Medical Centre „RareDis“, Bulgarian Scientific Society of Clinical and Transfusion Hematology and regional transfusion hematology centres in Bulgaria. Its main purpose is to create an epidemiological tool for identifying and tracking each patient. The registry will help doctors, researchers and health authorities to determine prevalence, morbidity, long-term outcomes and quality of life of the Bulgarian patients with thalassaemia major. Moreover, this experience can be used as a model for creation of registries for other rare diseases.
Project Duration – 20.04.2010 – 20.04.2013 (36 months)
12 Partners
Fundación Canaria de Investigación y Salud (FUNCIS), Spain
Instituto de Salud Carlos III (ISCIII), Spain
Instituto Superiore di Sanita (ISS), Italy
Federación Española de Enfermedades Raras (FEDER), Spain
Bulgarian Association for Promotion of Education and Science (BAPES), Bulgaria
London School of Economics and Political Science (LSE-Health), United Kingdom
University Paris Val de Marne (UPVM), France
Leibniz University Hannover (LUH), Germany
The Swedish Institute for Health Economics (IHE), Sweden
Universita Commerciale “Luigi Bocconi” (Bocconi), Italy
Centre for Public Affairs Studies Foundation (CPASF), Hungary
Mario Negri Institute for Pharmacological Research (IRFMN), Italy
7 Collaborating Partners from EU
Total Budget: 1 175 044 euros
EAHC Co-Funding: 705 022 euros
Project Coordinator: Prof. Julio López Bastida, Fundación Canaria de Investigación y Salud (FUNCIS), Spain
Official Website – www.burqol-rd.com
Project Objectives:
– To generate a methodological framework to measure the socio-economic burden of RD
– To define a methodological framework to measure the HRQOL of RD
– To develop unified instruments to gather information on the socio-economic burden and HRQOL of RD throughout Europe
– To perform a pilot study measuring the socio-economic burden and HRQOL for selected RD
– To refine and package the tools developed for continued and more extensive costs and HRQOL studies of RD
Project Outcomes:
An integrated and harmonized set of instruments to assess and monitor socio-economic burden and HRQOL of patients affected by RD and their caregivers.
1. A detailed analysis of the services (health and social care) received by people with specific RD in different EU countries, including the identification of formal and informal care.
2. A report on the current socioeconomic and HRQOL status of RD patients and caregivers for the selected RD and EU countries.
3. The results and deliverables that emerge from this project will stimulate the future comparability and monitoring of RD in Europe as well as anticipate future information needs.
Background:
Because of their characteristics, rare diseases require the combined efforts of health and social care professionals, politicians, managers and researchers to increase the availability of effective disease management tools to improve care and to extend both life expectancy and Health Related Quality of Life (HRQOL). Given the nature and the goals of the BURQOL-RD project, it is clear that a fundamental beneficiary of the results of this project will be the families and caregivers of those affected by RD, a group that is often overlooked when considering such devastating diseases.
Updated information from the National thalassaemia major registry was obtained in March 2010. Results show that there are 223 thalassaemia major patients in Bulgaria, who are treated in eight centres – Sofia (90), Plovdiv (54), Varna (26), Stara Zagora (23), Burgas (15), Pleven (9), Gabrovo (2). The crude prevalence for Bulgaria is 2.93/100 000 persons. The average age of the registered patients is 19.5 years. By the end of the year a process of updating and registration of new patients will be conducted too. More information about the results of the register can be found here.
The project is implemented as a result of the common work and cooperation between BAPES, ICRDOD, Medical Centre „RareDis“, Bulgarian Scientific Society of Clinical and Transfusion Hematology and regional transfusion hematology centres in Bulgaria. Its main purpose is to create an epidemiological tool for identifying and tracking each patient. The registry will help doctors, researchers and health authorities to determine prevalence, morbidity, long-term outcomes and quality of life of the Bulgarian patients with thalassaemia major. Moreover, this experience can be used as a model for creation of registries for other rare diseases.
On 28 October 2009, BAPES was officially given the status of data privacy administrator of rare diseases registries by the Commission for Protection of Data Privacy. After that, the collection of epidemiological data for the project “National registry of thalassaemia major patients in Bulgaria” started. Initial information shows that by January 2010 there are 208 thalassaemia major patients in Bulgaria, who are treated in seven centres – Sofia (95), Plovdiv (36), Varna (26), Burgas (14), Stara Zagora (19), Pleven (18). Currently, the data is been analyzed and the results will be published in March 2010. The project is implemented as a result of the common work and cooperation between BAPES, ICRDOD, Medical Centre „RareDis“, Bulgarian Scientific Society of Clinical and Transfusion Hematology and regional transfusion hematology centres in Bulgaria. Its main purpose is to create an epidemiological tool for identifying and tracking each patient. The registry will help doctors, researchers and health authorities to determine prevalence, morbidity, long-term outcomes and quality of life of the Bulgarian patients with thalassemia major. Moreover, this experience can be used as a model for creation of registries for other rare diseases.
On 28 October 2009, BAPES was officially given the status of data privacy administrator of rare diseases registries by the Commission for Protection of Data Privacy. After that, the collection of epidemiological data for the project “National registry of thalassaemia major patients in Bulgaria” started. Initial information shows that by January 2010 there are 208 thalassaemia major patients in Bulgaria, who are treated in seven centres – Sofia (95), Plovdiv (36), Varna (26), Burgas (14), Stara Zagora (19), Pleven (18). Currently, the data is been analyzed and the results will be published in March 2010. The project is implemented as a result of the common work and cooperation between BAPES, ICRDOD, Medical Centre „RareDis“, Bulgarian Scientific Society of Clinical and Transfusion Hematology and regional transfusion hematology centres in Bulgaria. Its main purpose is to create an epidemiological tool for identifying and tracking each patient. The registry will help doctors, researchers and health authorities to determine prevalence, morbidity, long-term outcomes and quality of life of the Bulgarian patients with thalassemia major. Moreover, this experience can be used as a model for creation of registries for other rare diseases.
А pilot epidemiological study of morbidity and incidence of chronic myeloid leukemia (CML) in Bulgaria has been completed. Information for 205 patients with this disease in Bulgaria has been collected. The final data will be published soon on our site and the results will help to launch a national registry for this disease. The pilot study was organized and conducted in close collaboration between ICRDOD, Scientific Society of Clinical and Transfusion Hematology and Medical Center “Raredis”.
National registry of patients with thalassemia major in Bulgaria is a project in the field of rare diseases which BAPES started in August 2008. Its aim is to establish an epidemiological tool to identify and follow up regularly the medical history of each patient. The registry will help doctors, scientists and health authorities to determine the incidence, prevalence, long-term clinical outcome and quality of life of Bulgarian patients with thalassemia major. They will also be able to compare overall management of thalassemia patients in Bulgaria with other countries. Moreover, this experience could be used as a model for starting registries for other rare diseases in the country. Initial data, based on consensus of the national expert group for thalassemia major show that currently there are 211 patients treated at 7 regional centres – Sofia (72), Plovdiv (54), Stara Zagora (37), Varna (23), Burgas (14), Pleven (11). This project is currently awaiting its final approval through the Commission for personal data collection and expected to start effectively in September 2009. For updated information, please click here.