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

C.M. da Silva Pinto, L. Martins da Silva, F. Ferreira de Carvalho Jr

• 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 sev-eral 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.