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Br J Haematol ; 204(5): 1977-1985, 2024 May.
Artigo em Inglês | MEDLINE | ID: mdl-38566598

RESUMO

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.


Assuntos
Aminopiridinas , Morfolinas , Púrpura Trombocitopênica Idiopática , Piridinas , Humanos , Pessoa de Meia-Idade , Feminino , Púrpura Trombocitopênica Idiopática/tratamento farmacológico , Masculino , Espanha , Aminopiridinas/uso terapêutico , Aminopiridinas/efeitos adversos , Idoso , Morfolinas/uso terapêutico , Morfolinas/efeitos adversos , Estudos Retrospectivos , Adulto , Piridinas/uso terapêutico , Piridinas/efeitos adversos , Oxazinas/uso terapêutico , Oxazinas/efeitos adversos , Pirimidinas/uso terapêutico , Pirimidinas/efeitos adversos , Resultado do Tratamento , Inibidores de Proteínas Quinases/uso terapêutico , Inibidores de Proteínas Quinases/efeitos adversos , Idoso de 80 Anos ou mais
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