Researchers UCBM coordinated by Prof. Migliaccio in a multicenter study with 11 research centers

A group of researchers coordinated by Professor Anna Rita Migliaccio, in collaboration with 11 US research centers and theUniversity of Florence, Careggi University Hospital el 'Istituto Superiore di Sanita, contributed to one clinical study on 21 patients with advanced myelofibrosis. Myelofibrosis is a rare tumor that causes the appearance of fibrous tissue in the bone marrow, which modifies its composition, in addition toingredness of the spleen and liver and produces symptoms such as tiredness, muscle pain, fever, night sweats, itching and even thrombosis.

The results, published in the prestigious journal Clinical Cancer Research, have shown that i 21 patients undergoing the clinical trial phase I with a transform inhibitor druging growth factor TGF-β 1/3 were no longer dependent on platelet transfusion during the treatment period and, in some cases they were able to suspend the transfusions, with a noticeable improvement in their quality of life. The period of treatment has indeed induced a increased platelet count in 81% of patients, three of whom achieved normal blood platelet levels. The treatment also revealed no significant toxicities.

Researchers fromUniversità Campus Bio-Medico di Roma have played a fundamental role in identifying the mechanism through which the platelet count was restored in treated patients by verifying the maturation of megariocytes, highly specialized hematopoietic cells responsible for the production of platelets. Previous studies by the same researchers at the Bio-Medico Campus had highlighted a high level of transformationing Growth factor β1 (TGFβ1) in the marrow plays a primary role in the evolution of myeloproliferative diseases into myelofibrosis. This study therefore highlighted how the action of the treatment, by interrupting the effects of TGFβ1, promotes the production of platelets in patients with myelofibrosis, at least partially curing their complex symptoms.

"This is an outstanding result – explained Anna Rita Migliaccio, adjunct professor of Histology and Embryology UCBM, Senior Investigator at the Altius Institute for Biomedical Sciences in Seattle – because none of the experimental treatments evaluated thus far for this disease have had an effect on platelet counts."

"This is an important result also for the world of scientific research – continues Professor Migliaccio - The collaboration between researchers atUniversità Campus Bio-Medico di Roma and the clinical research is innovative in its method because in this study the basic research continued to collaborate actively even during the analysis phase of the clinical results".

Myelofibrosis is a rare tumor belonging to the group of so-called myeloproliferative neoplasms: it represents the final stage of pre-leukemic forms called ph negative myeloproliferative diseases. Its incidence in the population, according to Orphanet, is equal in Europe to 0,1-1 per 100.00 people and a prevalence of 2,7 people per 100.000; the average age at diagnosis is 65 years, but one patient out of 4 arrives at diagnosis under the age of 56 and 11% are under 46 years of age. Pre-leukemic diseases are generally benign, easily treated and allow a good quality of life for patients until, due to a still poorly understood mechanism, they progress to myelofibrosis. It is a condition characterized by insufficient production of blood cells in the marrow and development of hematopoiesis in the spleen, which can evolve into leukemia, for which the only therapy currently is marrow transplantation.

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