Multicenter study with 11 US research centers, the National Institute of Health and the Careggi University Hospital of Florence. The researchers ofUniversità Campus Bio-Medico di Roma identified the mechanism by which platelet counts were restored in patients undergoing treatment.
Rome, 20 July 2023 - A group of researchers coordinated by the professor Anna Rita Migliaccio, in collaboration with 11 US research centers and with the University of Florence, the Careggi University Hospital and the Istituto Superiore di Sanità, contributed to a clinical study on 21 patients suffering from advanced myelofibrosis.
Myelofibrosis is a rare cancer which causes the appearance of fibrous tissue in the bone marrow, which changes its composition, as well as the enlargement 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 the 21 patients undergoing the phase I clinical trial with a drug inhibiting the transforming growth factor TGF-β 1/3 they were no longer dependent on platelet transfusion during the treatment period and, in some cases, they were able to suspend transfusions, with a clear improvement in their quality of life. The treatment period has in fact induced a increased platelet count in 81% of patients, three of whom achieved normal blood platelet levels. The treatment also revealed no significant toxicities.
The researchers ofUniversità Campus Bio-Medico di Roma 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 Campus Bio-Medico had highlighted how a high level of transforming growth factor β1 (TGFβ1) in the marrow plays a primary role in the evolution of myeloproliferative diseases into myelofibrosis. This study has therefore highlighted how the action of the treatment, interrupting the effects of TGFβ1, promotes the production of platelets in patients with myelofibrosis curing at least in part their complex symptoms.
"This is an exceptional achievement - has explained Anna Rita Migliaccio, adjunct professor of Histology and Embryology at theUniversità Campus Bio-Medico di Roma, Senior Investigator at the Altius Institute for Biomedical Sciences in Seattle – because none of the experimental treatments evaluated so far for this disease have had an effect on the number of platelets".
“This is an important result also for the world of scientific research – continues Professor Migliaccio - Collaboration between researchers ofUniversità 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: represents the final stage of pre-leukemic forms called pH negative myeloproliferative diseases. Its incidence in the population, according to Orphanet, is 0,1-1 in 100.00 people in Europe and a prevalence of 2,7 people in 100.000; the average age at diagnosis is 65 years, but one in 4 patients is diagnosed under 56 years of age and 11% are under 46 years of age.
Pre-leukemic diseases they are generally benign, easily treatable and allow a good quality of life for patients, until, due to a mechanism that is still poorly understood, 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 progress to leukemia, for which the only therapy currently is marrow transplant.
