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1.
J Pediatr Hematol Oncol ; 46(3): e220-e222, 2024 Apr 01.
Artigo em Inglês | MEDLINE | ID: mdl-38447071

RESUMO

Acquired thrombotic thrombocytopenic (aTTP) purpura is a life-threatening condition that can lead to devastating thromboembolic events. Recently, caplacizumab has been shown to rapidly restore platelet numbers and reduce the risk of severe end-organ damage when added to plasma exchanges (PEXs) and immunosuppression (IST). Here, we report the outcomes in 3 children with aTTP who were treated with caplacizumab in combination with PEXs and IST. In all 3 patients, platelet count increased to >15,000/mm 3 in 24 h and normalized on day 4, whereas normalization of ADAMTS13 activity >50% and elimination of the inhibitor was achieved after 18 to 89 days. Epistaxis was observed in 2 patients and was the only side effect related to caplacizumab. Caplacizumab is a promising agent for first-line treatment of children with aTTP.


Assuntos
Púrpura Trombocitopênica Trombótica , Anticorpos de Domínio Único , Criança , Humanos , Púrpura Trombocitopênica Trombótica/tratamento farmacológico , Troca Plasmática , Fator de von Willebrand , Terapia de Imunossupressão , Proteína ADAMTS13
2.
Sci Rep ; 11(1): 9401, 2021 04 30.
Artigo em Inglês | MEDLINE | ID: mdl-33931737

RESUMO

Immune thrombocytopenia (ITP) is believed to be associated with platelet function defects. However, their mechanisms are poorly understood, in particular with regard to differences between ITP phases, patient age, and therapy. We investigated platelet function and bleeding in children with either persistent or chronic ITP, with or without romiplostim therapy. The study included 151 children with ITP, of whom 56 had disease duration less than 12 months (grouped together as acute/persistent) and 95 were chronic. Samples of 57 healthy children were used as controls, while 5 patients with leukemia, 5 with aplastic anemia, 4 with MYH9-associated thrombocytopenia, and 7 with Wiskott-Aldrich syndrome were used as non-ITP thrombocytopenia controls. Whole blood flow cytometry revealed that platelets in both acute/persistent and chronic ITP were increased in size compared with healthy donors. They were also pre-activated as assessed by PAC1, CD62p, cytosolic calcium, and procoagulant platelet levels. This pattern was not observed in other childhood thrombocytopenias. Pre-activation by CD62p was higher in the bleeding group in the chronic ITP cohort only. Romiplostim treatment decreased size and pre-activation of the patient platelets, but not calcium. Our data suggest that increased size, pre-activation, and cytosolic calcium are common for all ITP platelets, but their association with bleeding could depend on the disease phase.


Assuntos
Plaquetas/efeitos dos fármacos , Sinalização do Cálcio , Hemorragia/etiologia , Púrpura Trombocitopênica Idiopática/sangue , Receptores Fc/uso terapêutico , Proteínas Recombinantes de Fusão/uso terapêutico , Trombopoetina/uso terapêutico , Adolescente , Estudos de Casos e Controles , Criança , Pré-Escolar , Feminino , Humanos , Lactente , Masculino , Testes de Função Plaquetária , Púrpura Trombocitopênica Idiopática/complicações , Púrpura Trombocitopênica Idiopática/tratamento farmacológico , Proteínas Recombinantes de Fusão/farmacologia , Trombopoetina/farmacologia
3.
Int J Hematol ; 105(6): 841-848, 2017 Jun.
Artigo em Inglês | MEDLINE | ID: mdl-28271416

RESUMO

It has been suggested that platelet function in chronic immune thrombocytopenic purpura (ITP) may be abnormal. Thrombopoietin mimetics used for treatment can affect it, but the data remain limited. We investigated platelet function of 20 children diagnosed with severe ITP (aged 1-16 years, 12 females and eight males). Platelet functional activity in whole blood was characterized by flow cytometry before and after stimulation with SFLLRN plus collagen-related peptide. Levels of CD42b, PAC1, and CD62P, but not CD61 or annexin V, were significantly increased (P < 0.05) in resting platelets of patients before treatment compared with healthy donors. On average, PAC1 and CD62P in patients after activation were also significantly elevated, although some patients failed to activate integrins. Romiplostim (1-15 µg/kg/week s.c.) was prescribed to seven patients, with clinical improvement in six. Interestingly, one patient had clinical improvement without platelet count increase. Eltrombopag (25-75 mg/day p.o.) was given to four patients, with positive response in one. Others switched to romiplostim, with one stable positive response, one unstable positive response, and one non-responding. Platelet quality improved with romiplostim treatment, and their parameters approached the normal values. Our results suggest that platelets in children with severe ITP are pre-activated and abnormal, but improve with treatment.


Assuntos
Benzoatos/administração & dosagem , Plaquetas/metabolismo , Hemorragia , Hidrazinas/administração & dosagem , Púrpura Trombocitopênica Idiopática , Pirazóis/administração & dosagem , Receptores Fc , Proteínas Recombinantes de Fusão , Trombopoetina , Adolescente , Anexina A5/sangue , Antígenos CD/sangue , Criança , Pré-Escolar , Feminino , Hemorragia/sangue , Hemorragia/induzido quimicamente , Hemorragia/tratamento farmacológico , Humanos , Lactente , Masculino , Contagem de Plaquetas , Púrpura Trombocitopênica Idiopática/sangue , Púrpura Trombocitopênica Idiopática/tratamento farmacológico , Receptores Fc/administração & dosagem , Receptores de Polipeptídeo Hipofisário Ativador de Adenilato Ciclase/sangue , Proteínas Recombinantes de Fusão/administração & dosagem , Proteínas Recombinantes de Fusão/efeitos adversos , Trombopoetina/administração & dosagem , Trombopoetina/efeitos adversos
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