1. Objectives and expected achievements
Myelofibrosis with myeloid metaplasia (MMM), is a disorder in which a somatic mutation leads a multipotent hematopoietic progenitor cell to acquire a clonal proliferative advantage. The functional derangement of the hematopoietic cell clone determines the phenotype of the disease, which has constantly been desccribed as myelofibrosis, immature myeloid and erythroid cells in the peripheral blood, anisopoikilocytosis with teardrop erythrocytes, and progressive splenomegaly due to myeloid metaplasia.
MMM is associated with substantial morbidity and mortality and its treatment is still largely unsatisfactory. Data regarding the safety and efficacy of new therapies in this diseases are anecdotal and based on small, uncontrolled, non comparable case series. Indeed, in this disease, controlled therapeutic trials are difficult to perform for two main reasons:
a) the rarity of the disorder which make hard to collect a suitable number of patients;
b) the absence of reliable, standardized and internationally recognized definitions of improvement and therefore the inability to measure the success or failure of interventions as well as subtle or even major diffe differences in the evolution of the diseases...
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