How effective is Fostamatinib?

Fostamatinib (Fostamatinib) is a newly approved drug for the treatment of adults with primary immune thrombocytopenia (ITP). It is a spleen tyrosine kinase (Syk) inhibitor. Clinical studies have demonstrated the safety and efficacy of fotantinib in patients with ITP, but it is important to note that the results of clinical trials may differ in actual applications.

In a prospective and retrospective study of 138 ITP patients from 42 Spanish medical centers, the efficacy of fotantinib was evaluated in depth. Among these patients, including those with primary and secondary ITP, the overall median age was 66 years old, and 55.8% of them were women. Patients received fotantinib for a median of 51 months after being diagnosed with ITP. Before starting fotantinib treatment, the median number of treatments for patients was 4, which mainly included other treatments such as eltrombopag (76.1%), romistin (57.2%) and intravenous immunoglobulin (44.2%). In addition, 58 patients (approximately 42.0%) had symptoms or signs of bleeding within one month before starting treatment.

The therapeutic response to fotantinib showed good results.79% of patients responded to the drug, of which the complete response rate reached 53.6%, that is, the platelet count exceeded 100*10^9/L. Among 83 patients who received fotantinib monotherapy, the high response rate reached 85.4%. Over the 27-month observation period, the proportion of duration of response was 83.3%. It is worth mentioning that the median time to platelet response was 11 days, indicating that fotantinib can quickly increase patients' platelet levels.

In this real-world study, fotantinib was shown to be effective and safe in patients with primary and secondary ITP across a broad age range, but some adverse events occurred during treatment, the most common of which included diarrhea (28 cases) and hypertension (21 cases). In addition, some patients developed more serious complications, such as deep vein thrombosis and acute myocardial infarction.

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