To this aim, TEDDY (the European Network of Excellence for Paediatric Clinical Research that includes many DEEP members) has forwarded to EnprEMA an extensive recently-published monograph deriving from a large collaborative work developed by the International Network of Clinicians for Endocrinopathies in Thalassemia and Adolescent Medicine (ICET-A). The publication, written by Vincenzo De Sanctis, Ashraf T. Soliman et al., includes both a literature review on general principles of good transition care for patients with chronic diseases and/or disabilities and a specific insight on medical care and transition phase for patients with thalassemia in different countries (Turkey, Iran, Saudi Arabia, Italy, Romania, Greece, Qatar, India, Oman, Egypt and Cyprus). In particular, the article “Medical care and transition phase of thalassemia in different countries: the ICET-A experience” has been prepared with the contribution of Prof. Adriana Ceci, Bonifazi Fedele and Felisi Maria Grazia of the DEEP Consortium and is aimed to discuss the best solution to provide always optimal treatment for all patients with thalassemia.

Considering that the management of thalassaemia patients requires multidisciplinary and collaborative interventions, the article highlights the need to identify the appropriate dimensions of Centres of Expertise (CEs), based both on the number and age of patients, and on their specific clinical, therapeutic and research needs. Legislation is required to endorse and organise the individual units and CEs. The article also underlines that an efficient schedule for medical care and transition phase for patients with thalassemia should be carefully adjusted to the national health system structure and capabilities as well as to the socio-economic status and epidemiology of thalassemia of the individual country.

A copy of the article (not available online) can be friendly provided by request to authors involved in the DEEP project (Prof. Adriana Ceci, Bonifazi Fedele and Felisi Maria Grazia) or writing at [email protected].

The initiative is organised by the H. H. Sheikh Sultan Bin Khalifa Bin Zayed Al Nahyan Humanitarian & Scientific Foundation, a non-profit non-governmental organization dedicated to value substantive efforts and contributions that have been made towards improving quality of life of patients with Thalassemia & other Hemoglobinopathies, by Individuals & Institutions, at National, Regional or International level.

This 2015 edition has been established by H. H. Sheikh Sultan Bin Khalifa Al Nahyan Humanitarian & Scientific Foundation in collaboration with ENERCA partner, Thalassemia International Federation (TIF), and included three categories: International Awards, Arab Awards and National Awards.

The Higher Scientific Committee Members for the International Awards was composed by Dr. Androulla Eleftheriou, Head of Committee, Executive Director of Thalassaemia International Federation (Cyprus), together with Professor Dimitris Loukopoulos (Greece), Professor Suthat Fuscharoen (Thailand), Professor John Porter (UK) and Professor Maria D. Capellini (Italy).

Prof. Amal el-Beshlawy, Professor of Pediatric Hematology at Cairo University (Egypt) and President of the Egyptian Thalassemia Association won the Best Published Research award in the 2015 Arab Awards Category.

The DEEP Consortium is proud of this award received by one of the project partner and congratulated her for the high level of her research and the recognized excellence in the field of Thalassemia.

The DEEP project supports this initiative and will strengthen the collaboration with the Sheikh Sultan bin Khalifa Al Nahyan Humanitarian and Science Foundation, linked by the common interest in employing scientific research to improve the quality of the lives of patients and their families.

In such a scenario, “the development of a tailored oral chelator for children”- as explained by Prof. Ceci – “responds to a still unmet need in the treatment of haemoglobinopathies, including thalassaemia, which require chronic blood transfusions and, therefore, iron chelation”.

Prof. Della Pasqua clarified the innovative approaches, implemented to overcome sensitive issues and ethical concerns about the conduct of trials in small children, which have led to the development of a Pharmacokinetic study aimed at determining the appropriate dosing requirements in children below six years of age.

The achievement of the project objectives, as explained by Dr. Bonifazi, was made possible thanks to the joint collaboration of experts in the field of thalassaemia, haematology, paediatric clinical trial design and management, regulatory and ethics, who gathered together to form a network of research institutions, clinical centres and patients associations from both European and non-European countries.

In conclusion, Prof. El Beshlawy highlighted that, as reported by a recent cost-utility analysis “deferiprone still represents the most cost-effective treatment available for managing chronic iron overload patients”, if compared to the other available chelators, thus resulting in a more easily accessible, safe and efficacious treatment with substantial cost savings for health services.

The 6th DEEP General Assembly will take place from the 18th to the 20th of May, 2015 in Tirana (Albania), with the participation of the whole DEEP Consortium as well as of external experts. The General Assembly will also host  the fourth Ethics Board meeting and two open meeting sessions (Clinical Research in Albania and Networks and Projects in a Global Context) with the participation of international prominent personalities in the field of clinical research. The DEEP-2 Investigators’ Meeting, a specific CRAs’ training session and individual training sessions on GCP, e-CRF and drug management will be available during the Meeting.

DEEP is the first clinical trial in which the University Hospital Centre “Mother Teresa” of Tirana (which represents the main and most important Albanian Hospital) is officially involved, after the entry into force of the new Albanian law concerning clinical trials (which took place in July 2014), and forthcoming implementation decrees.

The inclusion of Albania in the DEEP study represents an important milestone for a country that is currently managing its first non-profit paediatric trials. This will pave the way towards a new era for clinical research in Albania to the heightening of the quality of the clinical research in Albanian public institutes.

More details on the event and the related agenda will be soon available.

What started off as an European event, first launched by EURORDIS and its Council of National Alliances in 2008 with USA joining in 2009, is now a worldwide “mobilization”, inspiring thousands of other related events on a global scale.

The theme of Rare Disease Day 2015 is “Living with a rare disease” – paying tribute to the millions of people whose lives are affected by a rare disease and also to their families, friends and loved ones, who stand by them and support them every step of the way. Indeed, the slogan Day-by-day, hand-in-hand of the Official Rare Disease Day 2015 Video evokes the solidarity between families, patient organisations and communities.

DEEP welcomes and meets the concepts of this campaign by becoming a friend of Rare Disease Day and showing its support on its official website. This year Rare Disease Day encourages us to continue finding ways to work together to provide the different kinds of care to people living with thalassaemia and to push the boarders of research slightly further from its already encouraging progresses.

The DEEP project’s publishable summary has been approved by the European Commission and published on CORDIS, the Community Research and Development Information Service of the European Commission, in its role to disseminate the outcomes of research projects funded by the European Union.

On CORDIS the publishable summary is referred to as a “Report Summary” and can be found under the “Results” section of the  DEEP project’s permanent link.

Based on the project’s “Report Summary”, CORDIS science editors have in addition drafted a “Result in Brief“, that is, a short text written in a language more easily understandable by a broader public, thereby promoting the dissemination of knowledge from European research activities and supporting the exploitation of EU-funded research results. It is available in 6 languages (English, French, German, Spanish, Polish and Italian).