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 maisRESUMO
Catastrophic antiphospholipid syndrome is an infrequent disease in children, but of major relevance because of its high morbidity and mortality. We report the case of a child with digital ischaemia in whom, after aetiological screening, the diagnosis of catastrophic antiphospholipid syndrome was made.
Assuntos
Síndrome Antifosfolipídica , Síndrome Antifosfolipídica/complicações , Síndrome Antifosfolipídica/diagnóstico , Criança , Humanos , Isquemia/diagnóstico , Isquemia/etiologiaRESUMO
El síndrome antifosfolípido catastrófico es una entidad infrecuente en Pediatría, pero con importante relevancia dada la elevada morbimortalidad. Se expone el caso de un niño con isquemia digital en el que, tras realizar despistaje etiológico de diferentes entidades infecciosas e inflamatorias, se llegó al diagnóstico de síndrome antifosfolípido catastrófico primario.(AU)
Catastrophic antiphospholipid syndrome is an infrequent disease in children, but of major relevance because of its high morbidity and mortality. We report the case of a child with digital ischaemia in whom, after aetiological screening, the diagnosis of catastrophic antiphospholipid syndrome was made.(AU)
Assuntos
Humanos , Masculino , Criança , Isquemia , Síndrome Antifosfolipídica , Indicadores de Morbimortalidade , Microangiopatias Trombóticas , Anticorpos Antifosfolipídeos , Reumatologia , PediatriaRESUMO
Catastrophic antiphospholipid syndrome is an infrequent disease in children, but of major relevance because of its high morbidity and mortality. We report the case of a child with digital ischaemia in whom, after aetiological screening, the diagnosis of catastrophic antiphospholipid syndrome was made.
