Immunotolerance induction effectivity in hemophilia A children and neutralizing alloantibodies.

Abstract

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.

• Authors: Verónica Soto A, Daniela Cortez S, Macarena González S
• Journal: Revista chilena de pediatria (Rev Chil Pediatr) Vol. 91 Issue 2 Pg. 232-238 (Apr 2020) ISSN: 0717-6228 [Electronic] Chile
• Vernacular Title: Efectividad de inducción de tolerancia inmune en niños con hemofilia A y aloanticuerpos neutralizantes.
• PMID: 32730542 (Publication Type: Journal Article)

Chemical References

• Isoantibodies
• F8 protein, human
• Factor VIII

Topics

• Child
• Child, Preschool
• Factor VIII (immunology, therapeutic use)
• Female
• Follow-Up Studies
• Hemophilia A (immunology, therapy)
• Humans
• Immune Tolerance (immunology)
• Immunotherapy (methods)
• Infant
• Isoantibodies (immunology)
• Male
• Retrospective Studies
• Treatment Outcome