Hemofilia A congénita con inhibidores e inducción de inmunotolerancia en niños y adolescentes jóvenes: ¿podría Brasil ser un ejemplo para otros países?

Palabras clave

inmuno tolerancia

Cómo citar

De Paula, R., Silva Pinto, C. M. ., Martins da Silva, L., & Ferreira de Carvalho Jr, F. . (2022). Hemofilia A congénita con inhibidores e inducción de inmunotolerancia en niños y adolescentes jóvenes: ¿podría Brasil ser un ejemplo para otros países? ITI in children and adolescents: Brazil as example. Revista Hematología, 26(1). Recuperado a partir de https://revistahematologia.com.ar/index.php/Revista/article/view/413


Brazil has the third highest population of patients diagnosed with hemophilia A in the world and the first in Latin America. Factor VIII (FVIII) replacement prophylaxis is the standard of care currently recommended worldwide. However, about 30% of all patients with severe hemophilia A will develop neutralizing antibodies against FVIII, called inhibitors. The proposed aim of immune tolerance induction (ITI) therapy is to eradicate inhibitors, and several protocol variations are available. In Brazil, ITI treatment follows an escalating rationale, initiating with a low-dose scheme using FVIII 50 IU/kg, three times a week. When the absence of a decline in inhibitor titer with a low-dose regimen of at least 20%, in every six-month period after the beginning of ITI, is observed, it is recommended to initiate high-dose scheme using FVIII 100 IU/kg every day. About one third of all patients with hemophilia A are children and adolescents. Disease management in this population prompts different challenges, potentially leading to chronic and lifelong disabilities, and the age at first treatment also seems to act as a risk factor for inhibitor development. In this narrative review, the authors’ conclusion provides comprehensive knowledge regarding severe congenital hemophilia A with inhibitors (CHAWI) and ITI in children and adolescents, discussing different ITI protocols, with different outcomes, focusing on comparing Brazil's with other global guidelines.



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