Immunotolerance induction effectivity in hemophilia A children and neutralizing alloantibodies. / Efectividad de inducción de tolerancia inmune en niños con hemofilia A y aloanticuerpos neutralizantes.
Rev Chil Pediatr
; 91(2): 232-238, 2020 Apr.
Article
em En, Es
| MEDLINE
| ID: mdl-32730542
INTRODUCTION: The development of anti-factor VIII neutralizing antibodies in hemophilia A is the most severe com plication related to treatment. Immune tolerance induction (ITI) is the only known treatment for eradicating inhibitors. A successful ITI allows using factor VIII (FVIII) again for the treatment or prophylaxis of hemorrhagic events. OBJECTIVE: To report the experience of pediatric patients who underwent ITI in the country's public health care network. PATIENTS AND METHOD: Retrospective and descriptive analysis of 13 pediatric patients with severe Hemophilia A and high-titer inhibitors persis tence who underwent ITI and complete follow-up. Plasma-derived FVIII concentrate was used at 70 180 IU/kg/day doses. The success of the treatment is defined by achieving a negative titer and a half life recovery of the FVIII. The results were expressed in median (range). RESULTS: In 13 patients, the inhibitor was identified at an average age of 17.6 months, after 35.2 days of exposure to the FVIII. 11 patients (84.6%) recovered the half-life of FVIII after 49.6 months of treatment. In the patients who responded to treatment, the inhibitor titer was negative at 6 months on average. CONCLUSIONS: ITI is the treatment of choice for patients with hemophilia A and inhibitors persistence. ITI must be perso nalized since the time response is variable in each patient.
Texto completo:
1
Coleções:
01-internacional
Base de dados:
MEDLINE
Assunto principal:
Fator VIII
/
Hemofilia A
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Tolerância Imunológica
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Imunoterapia
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Isoanticorpos
Tipo de estudo:
Observational_studies
/
Prognostic_studies
Limite:
Child
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Child, preschool
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Female
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Humans
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Infant
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Male
Idioma:
En
/
Es
Revista:
Rev Chil Pediatr
Ano de publicação:
2020
Tipo de documento:
Article
País de afiliação:
Chile
País de publicação:
Chile