EBMT

A Report from the Paediatric Diseases Working Party

Giannina Gaslini Institute, venue of the First ESH-EBMT Training Course on Blood and Marrow Transplantation in Children

The EBMT Paediatric Diseases Working Party and the First ESH-EBMT Training Course on Blood and Marrow Transplantation in Children

In 1995, during the yearly meeting of the European Group for Blood and Marrow Transplantation (EBMT) held in Davos, Switzerland, the issues related to the differences underlying treatment approaches towards adult and paediatric patients were discussed in-depth. An outcome of this exchange was the creation by a small group of EBMT Members of the Paediatric Diseases Working Party (PDWP), whose overriding mission was to design specific transplantation programmes for paediatric patients. The following year, 1996, the Working Party members came together in Erice, Italy, for their first Meeting.

During the last few years our Working Party has grown, and currently 19 European countries and at least 3 non-European ones participate in its activities. This international profile feeds the background against which most of the actual debates on stem cell transplantation in children take place. For example, we are now tackling issues related to the current JACIE standards for haematopoietic stem cell transplantation (HSCT) in patients under 18 years of age. The PDsWP firmly believes that children can only be treated by a specially trained and experienced paediatric HSCT team, and that, to ensure quality and effectiveness of delivered care, a centre must perform at least ten allo-HSCT procedures per year. Possible exceptions could be reserved for areas or countries that lack dedicated HSCT Units, but that nonetheless have active government support for paediatric stem cell transplantation programmes. In addition, the PDsWP recently analysed the risk factors for transplant-related mortality observed in the more than 80,000 patients below 18 years of age given allo-HSCT over the past 30 years included in the EBMT Registry. The aim was to collect hard evidence and reliable data to bear out our claims. We came to the conclusion that children and adolescents have different demands before, during and after stem cell transplantation compared to adults, and that they require special care by experienced staff. Treatment at highly skilled paediatric centres that are actively involved in cooperative studies has been shown to improve survival, which is why authorities should carefully plan transplant activities and concentrate resources on experienced Institutions. Competent care for highly specialised procedures, such as HSCT in children, is cost effective only when enough procedures are performed at the same centre. The yearly minimum of ten allogeneic transplants must be respected.

Keeping in mind the main goals of our WP, we decided to organise the first ESH-EBMT Training Course on Blood and Marrow Transplantation in children in order to promote and facilitate access to cutting edge knowledge in blood and marrow transplantation at a European level. For this purpose, the leading European and international experts operating in the field of HSCT in children gathered in Genova at the Badia Benedettina complex, a magnificent training facility that comprises a former convent, the historic Villa Quartara, and a large park sloping down towards the sea. The course explored the main issues impacting paediatric HSCT. During 3 days of intensive discussions, a broad range of issues concerning the field were covered. Keynote speeches on the changing role of HSCT in children, on the role of umbilical cord blood transplantation in children, and on NK cell activity and HSCT were presented, respectively, by Franco Locatelli, Eliane Gluckman and Lorenzo Moretta. Ten plenary sessions with 24 up-front lectures focussed on issues related to conditioning regimens, data registration, early and late complications, acute leukaemia, non malignant diseases, solid tumours and infectious diseases. Furthermore, no fewer than 18 participants delivered brief presentations on interesting case reports pertinent to course topics. Finally, to enhance exchange between attendees and course faculty, nine “Meet the Expert Breakfast Sessions” were held, and a “Meet the Expert Dinner” closed the proceedings on the last evening of the course.

The faculty included 36 speakers: 23 from Italy, 12 from eight other European countries, and one from a non-European country. One hundred and two young physicians joined us in Genova: 27 from Italy, 51 from 19 other European countries, 22 from 13 countries outside Europe (North America 4, South America 6, Africa 2, Australia 2, Asia 8). Finally, 49 grants for free registration and free accommodation were assigned on a competitive basis to young physicians, thanks to the generosity of several sponsors whom I wish once again to acknowledge with deep and sincere thanks.

Giorgio Dini, MD
Course Director
Chairman, EBMT Paediatric Diseases Working Party