“In the next few years we will probably have personalization, at least to a large extent, of patient management. We are carrying out studies that go towards a combination of different targets to target myelofibrosis.” Thus Francesco Passamonti, full professor of Hematology, University of Milan and director of Complex Structure, department of Oncology and Onco-Hematology of the Milan Polyclinic, together with Alessandro Maria Vannucchi, full professor of Hematology, University of Florence and director of the Structure complex of Hematology, Careggi University Hospital, speaking today in Verona at a meeting with the press organized by GSK, in which the latest therapeutic innovations for the treatment of this bone marrow neoplasm characterized by the proliferation of abnormal red blood cells and accumulation of fibrous tissue.
Myelofibrosis – the experts explained – affects approximately 20 thousand patients in the United States and, globally, approximately 1 patient in 500 thousand. Although the cause is not entirely known, several factors influence the incidence of the disease which can arise due to dysregulation of the Jak-Stat pathway caused by driver mutations of 3 genes: Jak2, Calr and Mpl. The disease is characterized by disabling symptoms such as tiredness, splenomegaly (enlargement of the spleen), systemic symptoms and anemia. The latter, which has a great impact on the quality of life, can also worsen due to some Jak inhibitor drugs used. A new Jak inhibitor, momelotinib, has recently been approved in Europe, which also improves the symptom of anemia, thus making the patient less subject to transfusions, which can reach up to 2 per week.
“We are designing clinical studies against increasingly specific targets to increasingly personalize the treatment – underlines Passamonti – therefore to think about a combination therapy or just Jak inhibitors based on the characteristics of the individual patient”, such as the presence or absence “of the anemic form”.
Currently “we have no evidence that a pharmacological therapy today or in 3 years' time could have the ability to modify the natural evolution of myelofibrosis – specifies Vannucchi – but these approaches, even better if personalized, have certainly improved the quality of the patients' lives and their survival. The disease remains there and above all the risk of progression and the risk of evolution into acute leukemia remains, which is what really represents the next commitment for the future difficult to glimpse because, despite all our knowledge on the molecular mechanisms of the disease, we are still missing something, above all, given the genetic complexity of these diseases, it is difficult to imagine that a single agent can modify their natural history. hematologist oncology – what awaits us is an important and very demanding job.”
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