How effective is fostamatinib?

Fostamatinib (Fostamatinib) is a newly approved drug for the treatment of primary immune thrombocytopenia (ITP) in adults. Its mechanism is mainly through inhibiting splenic tyrosine kinase (Syk). Current clinical studies have confirmed that fotantinib has good safety and efficacy in patients with ITP, but it should be noted that clinical trial results may differ in actual application.

In a prospective and retrospective study involving 138 ITP patients from42 Spanish medical centers, the efficacy of fotantinib was comprehensively evaluated. These patients include primary and secondary ITP. The overall median age is 66 years old, and 55.8% of them are women. After patients were diagnosed with ITP, the median duration of treatment with fotantinib was 51 months. Before starting treatment, these patients had undergone a median of 4 other treatments, including eltrombopag (76.1%), romistin (57.2%), and intravenous immunoglobulin (44.2%). It is noteworthy that about 42% of patients had symptoms or signs of bleeding within one month before treatment.

The therapeutic response to fotantinib showed significant results.79% of patients had a positive response to the drug, and the complete response rate reached 53.6%, that is, the platelet count exceeded 100*10^9/L. In 83 patients treated with fotantinib alone, a response rate of up to 85.4% was observed. During the 27-month observation period, the proportion of duration of response was 83.3%. In addition, the median time to platelet response was only 11 days, indicating that fotantinib can quickly increase patients' platelet levels.

This real-world study demonstrates that fotantinib demonstrates promising efficacy and safety in patients with primary and secondary ITP across a broad age range. However, some adverse events were reported during treatment, the most common of which included diarrhea (28 cases) and hypertension (21 cases). Some patients even develop more serious complications, such as deep vein thrombosis and acute myocardial infarction.

Reference materials:https://pubmed.ncbi.nlm.nih.gov/38843478/