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Myelofibrosis, is there an effective treatment to avoid the risk of it turning into acute leukemia?

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Myelofibrosis, is there an effective treatment to avoid the risk of it turning into acute leukemia?

by Francesco Passamonti

Therapy depends on the symptoms and today it is possible to extend the survival of patients and reduce debilitating disorders, allowing, in most cases, a return to daily and work activities and a normal social life.

I am 45 years old and four years ago I discovered that I have myelofibrosis, which for now does not require treatment, but I know that it could evolve into acute leukemia. I would like to know if there are any new treatments and, perhaps, if we can hope for something that will make us recover completely.

Francesco Passamonti, Director of Hematology, Ospedale Maggiore Policlinico, Milan, responds

Myelofibrosis is a chronic myeloproliferative neoplasm which every year is diagnosed in approximately one thousand Italians, mostly in their sixties, but many cases occur in young people. There are therapies capable of keeping the disease under control even for years, but two important problems remain open: preventing the disease from evolving into acute myeloid leukemia (this happens in approximately 10% of patients) and preventing excessive enlargement of the spleen, very frequent, which entails serious consequences for patients.
In the initial stages myelofibrosis is asymptomatic and, as in your case, does not require treatment. However, it is important that you monitor some symptoms (fever in the evening, night sweats and weight loss), the hemoglobin and platelet values, which can reduce over time, and the size of the spleen because its excessive enlargement, which is very frequent, leads to serious consequences for patients.

Available therapies

The therapy depends on the symptoms and, to establish how to proceed, an osteomedullary biopsy is needed (useful to repeat it in case of initial progression) and the study of the genes involved (such as JAK2, CALR or MPL) with a test called NGS (next generation sequencing ) because based on any mutation present in the patient it is possible to opt for certain targeted drugs or others. The therapies available today make it possible to extend the survival of patients and reduce debilitating symptoms, allowing, in most cases, a return to daily and work activities and a normal social life. The only treatment that allows for definitive healing is the allogeneic stem cell transplant (from a healthy donor), reserved for certain phases of the disease, for those who are in good general condition and are under 70-75 years of age. It is a procedure that has been in use for many years and has guaranteed recovery for many patients.

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Studies in progress

To keep splenomegaly (enlarged spleen) or other symptoms at bay we can use JAK inhibitor drugs (such as ruxolitinib and fedratinib), already reimbursed in Italy, and we await the arrival of pacritinib and momelotinib for patients with anemia or thrombocytopenia. To improve anemia, we currently do not have efficient drugs, but in Italy we are studying molecules such as luspatercept (already approved for some types of myelodysplastic syndromes and thalassemias) and others still in the early phase of testing. Scientific research has been working for years to improve the results achieved so far and during the last congress of the American Society of Hematology we presented the initial results of two combination studies in the first line of treatment: TRANFORM-1 (ruxolitinib and navitoclax) , MANIFEST-2 (ruxolitinib and pelabresib). By taking advantage of the different mechanisms of action of the drugs in the combination, we doubled the percentage of responses on splenomegaly compared to ruxolitinib alone.

Reduce symptoms

There are many new molecules that we are studying in myelofibrosis, with different targets. The hope is not only to reduce the spleen and the symptoms, which are also important for the patient, but also to turn off some biological aspects of the disease, such as the quantity of the JAK2/CALR/MPL genes with the aim of modifying the natural history of myelofibrosis, that is, blocking or delay its progression.

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March 1, 2024

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