Publications by authors named "E Beggiato"
Calreticulin (CALR) mutations are detected in around 20% of patients with primary and post-essential thrombocythemia myelofibrosis (MF). Regardless of driver mutations, patients with splenomegaly and symptoms are generally treated with JAK2-inhibitors, most commonly ruxolitinib. Recently, new therapies specifically targeting the CALR mutant clone have entered clinical investigation.
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- Ruxolitinib (RUX) is a treatment for myelofibrosis, mainly studied in high-risk patients, but is often given to intermediate-1 patients with limited data on its effects.
- In a study of 1,055 myelofibrosis patients, over half were classified as intermediate-1 risk, with notable symptoms and some having high-molecular-risk mutations.
- The study found that after 6 months of RUX treatment, a significant proportion of patients experienced improvements in spleen size and symptoms, with certain factors like the absence of high-molecular-risk mutations being linked to better treatment responses.
Article Synopsis
- People with essential thrombocythemia (ET) usually take low-dose aspirin once a day to avoid blood clots, but this isn't always effective because their platelet levels change quickly.
- A study tested if taking aspirin twice a day would work better and found that it did help lower certain blood markers and symptoms over 20 months.
- The results showed that twice-daily aspirin caused fewer major blood clots, didn't lead to significant bleeding problems, and made patients feel better overall compared to taking it once a day.
Article Synopsis
- Iptacopan, an oral factor B inhibitor, shows promise in treating paroxysmal nocturnal hemoglobinuria patients suffering from persistent hemolytic anemia, especially those not responding to anti-C5 therapy.
- In two phase 3 trials, iptacopan significantly improved hemoglobin levels in patients with low baseline hemoglobin (under 10 g/dL), with many experiencing increases of at least 2 g/dL without needing blood transfusions.
- The results revealed that 85% of patients in the first trial and nearly all in the second trial experienced a notable increase in hemoglobin levels, leading to reduced fatigue and dependency on transfusions.