Rare diseases have recently been the objective of a great deal of studies aimed at elucidating pathways and individuating effective, possibly targeted therapies. The meeting on Ph negative Myeloproliferative and Myelodysplastic Syndromes will cover aspects of these disorders from the bench to the bed. In 1951, Dameshek recognised the close interrelation between Polycythemia Vera (PV), Essential Thrombocythaemia (ET), and Idiopathic Myelofibrosis (IMF) and coined the term “Myeloproliferative Disorders” (MPDs). These conditions were regarded as the result of the uncontrolled proliferation of one or more bone marrow lineage, triggered by unknown stimuli, which in some cases could also lead to myeloid metaplasia of the spleen. Milestones in the history of MPDs were, in the 1970s, the recognition of the cellular independence from growth factors as a critical mechanism for the disease, and second, the identification of the stem cell nature and clonal origin of PV and ET. From 1980s onwards, new insights in the molecular pathogenesis have been increasingly achieved and have peaked in the last decade. Particularly, in 2005, an acquired single point mutation in the gene of the cytoplasmic tyrosine kinase JAK2 has been simultaneously described in MPD by five different groups. Subsequent studies have focused on the frequency of this mutation and its correlation with the clinical phenotype. On the basis of these recent developments, a revision of the current WHO diagnostic criteria has been proposed. Other scientific achievements of the last decade include the results of randomised clinical trials carried out in Europe in patients with MPD. As a consequence of these studies, therapy has been optimised and today PV and ET patients are stratified according to their vascular risk and to new prognostic factors, including JAK2 burden and leukocytosis. Studies of IMF are less impressive. However, the efficacy of haematopoietic stem cell transplantation is being currently explored in this disorder by well designed controlled clinical trials. Myelodysplastic Sindromes (MDS) encompass a spectrum of haematopoietic stem cell malignancies, but specific defects responsible for these diseases remain unknown. With the advent of new discoveries of the pathobiology of these disorders, new drugs have been developed, particularly for patients with specific cytogenetic abnormalities. In the recent years, a place for bone marrow transplantation in high risk and young patients with MDS has been defined. The haematologists from the Haematology/Oncology Department of the Ospedali Riuniti Bergamo, Italy, have long been involved in both clinical and basic research in MPDs, providing original contributions to the advances in the management of these disorders and the related thrombotic complications. It is therefore a great honour to host in the city of Bergamo this International symposium on ‘Myeloproliferative and Myelodysplastic Syndromes’. The most distinguished scientists in the field are invited to talk and will focus on the most recent clinical advances and new mechanisms for these diseases. Ample space for the discussion and for sharing ideas with experts of undoubted experience is allowed during the meeting. A round table to discuss on the new WHO criteria for the diagnosis of MPDs will outline the future direction in the field. It is our hope that this meeting will provide a favourable atmosphere to debate hot issues between scientists and will represent a valuable opportunity to generate new ideas and start future collaborations.
Tiziano Barbui and Giuseppe Remuzzi Co-Presidents of the Meeting