Adnkronos Rome, June 13 (Adnkronos Salute) – 66,5% of patients with myelofibrosis are transfusion independent after 24 weeks of treatment with momelotinib, an oral Jak inhibitor, recently available in Italy: this is the key data that emerged from the Simplify-1 clinical study, of which a post hoc analysis is being presented at the European Congress of Haematology (EHA) underway in Milan, confirming the role of momelotinib in the treatment of anaemia, one of the most serious manifestations of the disease.
Even in patients already treated with other Jak-inhibitors, as highlighted in the Momentum study, the drug – a note reports – showed significant benefits on anemia, splenomegaly and symptoms. Myelofibrosis is a blood cancer that affects about 350 people a year in Italy, with a higher incidence between the ages of 60 and 70.
"Myelofibrosis - explains Francesco Passamonti, director of the Complex Structure of Hematology at the Policlinico di Milano and full professor of Hematology at the University of Milan - can worsen more or less slowly over several years in ways that vary depending on the patient. Generally, the initial phase consists of damage to the structure of the bone marrow. It is called the early or pre-fibrotic phase, since bone marrow fibrosis is not yet present". In the advanced phase, however, marrow fibrosis appears and an escape of immature stem cells from the bone marrow is observed. "These cells, through the blood - continues Passamonti - reach the spleen and the liver, where they accumulate. Usually, when the disease manifests itself, the typical alterations are already present: in addition to fibrosis, among others, anemia and enlargement of the spleen. In some cases (10-15 out of 100) myelofibrosis can evolve into a more severe pathology: acute myeloid leukemia".
About 40% of patients have moderate to severe anemia at diagnosis, but it is estimated that nearly all will develop it over time. This condition requires additional supportive care, primarily transfusions. And, unfortunately, patients who depend on transfusions have a poor quality of life and reduced survival. In cases where severe fatigue or significant splenomegaly is found, myelofibrosis can prevent a number of "normal" daily activities: walking, climbing stairs, making the bed, showering, cooking.
"The only therapy currently potentially capable of curing - Passamonti underlines - is a bone marrow transplant, but it is reserved for a small percentage of patients, generally under 70, due to the complexity and risks associated with it. Patients who are not eligible for a transplant are generally treated with a Jak inhibitor. Compared to other Jak inhibitors already used, momelotinib, administered orally once a day, has been shown to have a favorable impact on splenomegaly, symptoms and anemia, significantly reducing the transfusion load".
The new data presented at the Eha congress – the note concludes – reinforce the role of momelotinib as an effective therapeutic option for patients with myelofibrosis and anemia, highlighting the importance of early intervention on anemia to maximize clinical benefits. In particular, achieving hemoglobin levels above 10 g/dL was associated with increased overall survival. Furthermore, the data confirm a benefit on patient prognosis, for those who achieve transfusion independence associated or not with splenomegaly control.