ABSTRACT
Immune thrombocytopenia (ITP) is characterized by low platelet counts (PLTs) and an increased risk of bleeding. Fostamatinib, a spleen tyrosine kinase inhibitor, has been approved as a second-line treatment for ITP. Real-world data on fostamatinib are lacking. This observational, retrospective, multicentre study, conducted in the Andalusia region of Spain, evaluated 44 adult primary ITP patients (47.7% female; median age 58 years; newly diagnosed ITP 6.8%; persistent 13.6%; chronic 79.5%; median four prior treatments) after ≥ 4 weeks of fostamatinib therapy. The median PLT at the initiation of fostamatinib was 15 × 109/L. Common reasons for starting fostamatinib were refractoriness or intolerance to prior therapy, oral medication preference, history of thrombosis and cardiovascular risk. Dosing was individualized based on efficacy and tolerance. After 2 weeks, global response rate was 56.8% (response and complete response). Response rates were 70.5%, 62.5% and 64% at 4 weeks, 12 weeks and at the end of the study respectively. Adverse events were mild, and no patients discontinued as a result. This real-world study demonstrated a response rate similar to fostamatinib as seen in the pivotal clinical trials while including newly diagnosed patients and allowing for individualized dosing.
Subject(s)
Aminopyridines , Morpholines , Purpura, Thrombocytopenic, Idiopathic , Pyridines , Humans , Middle Aged , Female , Purpura, Thrombocytopenic, Idiopathic/drug therapy , Male , Spain , Aminopyridines/therapeutic use , Aminopyridines/adverse effects , Aged , Morpholines/therapeutic use , Morpholines/adverse effects , Retrospective Studies , Adult , Pyridines/therapeutic use , Pyridines/adverse effects , Oxazines/therapeutic use , Oxazines/adverse effects , Pyrimidines/therapeutic use , Pyrimidines/adverse effects , Treatment Outcome , Protein Kinase Inhibitors/therapeutic use , Protein Kinase Inhibitors/adverse effects , Aged, 80 and overSubject(s)
Antibodies, Monoclonal/therapeutic use , Antineoplastic Agents/therapeutic use , Leukemia, Lymphocytic, Chronic, B-Cell/complications , Leukemia, Lymphocytic, Chronic, B-Cell/drug therapy , Red-Cell Aplasia, Pure/complications , Aged , Antibodies, Monoclonal, Murine-Derived , Humans , Male , RituximabABSTRACT
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