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Forerunners in the field of HSCT, the EBMT gives us a window on its work

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Per Ljungman
MD PhD

EBMT Secretary

European Group for Blood and Marrow Transplantation
Maastricht
The Netherlands

E: [email protected]

Stem-cell transplantation (SCT) has emerged as a major treatment option for patients with haematological malignancies. It has experienced explosive development over the last 25 years, from its early role as an exclusive procedure to treat a small number of young patients mainly suffering from leukaemia, to become an established procedure that knows no age restrictions and is used for many different disorders, both malignant and nonmalignant. SCT is now used to treat a wide range of diseases, ranging from acute leukaemia through haematological malignancies and solid tumours to autoimmune diseases and congenital disorders.

  • Landmarks in the development of SCT include:
  • The creation of unrelated donor registries, which now list more than 10 million volunteer donors from all over the world.
  • The discovery of new stem-cell sources such as peripheral blood stem cells, cord blood stem cells and mesenchymal stem cells.
  • The introduction of reduced intensity conditioning regimens with the knowledge that allogeneic SCT cures patients also through immunotherapeutic mechanisms.
  • Better management of transplant-related severe complications, such as graft-versus-host disease and infections.

These developments have been achieved by a longstanding collaboration between transplant ­centres in Europe and across the world through the creation of both prospective and retrospective joint studies drawing on data from very large patient ­registries.

European Group for Blood and Marrow Transplantation
The European Group for Blood and Marrow ­Transplantation (EBMT) was established in 1974 and is a non-profit-making organisation that aims to promote all aspects associated with transplantation of haematopoietic stem cells from all donor sources and donor types including basic and clinical research, education, standardisation, quality control and accreditation for transplant procedures.

The EBMT unites 3,728 bone marrow transplantation (BMT) professionals from 527 centres located in 57 different countries across the world and the ­numbers are growing steadily every year as more BMT teams become interested in the work of the EBMT. The organisation is led by a board elected by the members and consists of 11 working parties, eight committees, a nurses’ group, a data management group, a central registry, 13 national SCT ­registries and the local organising committee for the annual meeting. The work of the EBMT is generously supported by 20 corporate patrons who collaborate closely with the EBMT to further the organisation’s educational and research activities.

The EBMT Registry employs a central data ­management system to collect transplant data on 31 disease classifications from its members. The ­registry currently contains data from more than 272,000 transplants, with 84% of the EBMT’s ­member centres actively reporting transplant data to the EBMT Registry. This percentage continues to increase annually. Data is used for both clinical ­studies and quality control, and access to data is one of the benefits of EBMT membership. New ­registries are created as the development of SCT continues. An example is the Cell Therapy Registry (CTR), recently set up with a view to collecting data on foetal or adult stem cells, or progenitor cells used for treatments other than haematopoietic stem-cell transplantation, or donor lymphocyte infusion, as well as data on the clinical characteristics of and outcomes for the patients treated with these new modalities. Based on the EBMT registry data, a total of 48 scientific reports were published in peer-reviewed journals in 2006, and more than 100 retrospective studies are currently under way.

Since 1990 the EBMT’s activity survey office has carried out an annual survey organised by ­Professor Alois Gratwohl and Helen Baldomero to collect data on numbers of patients treated with a

haematopoietic stem cell transplant (HSCT) ­according to indication, donor type and stem cell source. The survey has become a useful tool for assessing trends in HSCT across Europe and providing essential data for counselling and planning.

The combination of scientific expertise and access to the wealth of data in the registry database has also provided the EBMT with an excellent ­scientific foundation for the development of study ­protocols. To complement the organisation’s ­scientific strengths, significant investment in the clinical trials infrastructure has now provided a robust operational support structure. Eleven prospective clinical trials are ­currently open for recruitment, with 16 already completed.

Most of the EBMT’s key scientific and educational activities are channelled through its 11 ­working parties. The nature of the WPs is varied, but the majority deal with specific diseases, including acute ­leukaemia, aplastic anaemia, autoimmune diseases, chronic leukaemias, inborn errors, lymphoma, ­paediatric diseases and solid tumours, while the remainder work in other fields of major importance to SCT such as immunobiology, infectious diseases and late effects.

Accreditation and quality control are further ­important focal points for the EBMT. In 1998, the EBMT and the International Society for ­Cellular ­Therapy (ISCT) set up the Joint Accreditation ­Committee (JACIE), a nonprofit body established for the purposes of assessment and accreditation in the field of haematopoietic stem-cell ­transplantation. JACIE’s primary aim is to promote high-­quality patient care and laboratory performance in haematopoietic stem-cell collection, processing and transplantation centres through an internationally recognised ­system of accreditation. JACIE, in ­collaboration with the US-based Foundation for the Accreditation of Cellular Therapy (FACT), has established international standards for the provision of quality ­medical and laboratory practice in HSC transplantation. Through its programme of inspection and accreditation, JACIE encourages health institutions and ­facilities ­performing HSC transplantation to ­voluntarily meet these standards and recognises compliance with standards by certification of accredited ­programmes. Currently 38 centres from 13 different European countries have achieved JACIE accreditation. Forty-nine centres are currently in the accreditation ­process and 121 centres have already registered for the ­programme.

An area of increasing importance is the ­regulatory environment, with pan-European initiatives such as the implementation of new EU directives – most recently, the Clinical Trials Directive (DIR 2001/20/EC) and the Human Tissues and Cells Directive (DIR 2004/23/EC). The EBMT has become increasingly active in this area with contacts in the European ­Commission, the ­European Parliament and EMEA to offer expert advice and inform developments in the field. Collaborative relationships have also been established with competent authorities across Europe to encourage harmonisation. It is important to facilitate the ongoing ­evolution of SCT in order to expand the treatment options available for the many patients with an increasing number of treatable diseases. The regulatory framework therefore needs to find a comfortable balance between safeguarding the safety of patient treatments by guaranteeing quality, without ­hindering the necessary research to allow the ­science to progress. The EBMT can assist in these fields thanks to its global network of bone marrow ­transplant ­professionals, transplant centres and registries.

Each year the EBMT organises an annual ­meeting to foster scientific exchange and ­networking between physicians, nurses and data managers. The EBMT annual congress takes place in a ­different European city each year and promotes an ­innovative and ­interesting scientific programme, which is accompanied by informative workshops, plenary sessions, poster sessions and oral presentations. Participation has been increasing by about 2% a year since the first meeting in 1972, and 2007 saw 3,618 delegates from more than 75 different ­countries attended that year’s meeting in Lyon, France. The next EBMT annual congress will take place in ­Florence, Italy, in March–April 2008 (see Resources). Last year also saw the very first EBMT Patient and Family Day, which proved a huge success with 345 people attending a series of plenary sessions and workshops. The next Patient and Family Day is on the day prior to the annual congress.

Education is a major objective of the EBMT. A number of cutting-edge educational courses are organised annually by the working parties and through collaboration with the European School of Haematology. Another important way of disseminating knowledge is through the outreach programme, which aims to provide assistance to new transplant centres setting up in countries where stem-cell transplantation has been less well established.

One of the EBMT’s key objectives over the next few years will be to continue increasing its scientific output and research in the area of haematopoietic stem-cell transplantation by cultivating its ­public ­relations and making the science more ­readily ­available to the general public. The EBMT is also actively involved in the establishment of a global ­network of outcome registries with the aim of developing a global ­activity survey and investigating the issues and technical solutions for the development of universal patient and donor IDs. The group is also working to broaden its base by making patients and families, as well as donor groups, an integral part of the EBMT. Increased involvement and collaboration with concerned parties will inform the future vision of the organisation and strengthen the EBMT’s ­contribution to the development of this exciting field.

Resources
European Group for Blood and Marrow Transplantation
W: www.ebmt.org

Forthcoming conference
European Group for Blood and Marrow Transplantation 34th Annual Meeting, Florence, Italy, 30 March–2 April 2008
W: www.akm.ch/ebmt2008

International Society for Cellular Therapy
W: www.celltherapysociety.org

Foundation for the Accreditation of Cellular Therapy
W: www.factwebsite.org

European School of Haematology
W: www.esh.org






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