Περίληψη:
The development of antibodies to factor VIII (FVIII) in severely
affected haemophilia A patients is a serious complication associated
with increased morbidity and mortality. Bypassing agents are used to
treat acute bleeding episodes; however, elimination of the inhibitors
can only be achieved with immune tolerance therapy (ITT) in 60-80% of
cases. High responding (HR) inhibitors are more likely to respond to ITT
if the titre is decreased to < 5 BU over time or in selected cases after
the administration of immunosuppressive drugs, plasmapheresis or
immunoabsorption, techniques difficult to apply in children. Anti-CD20
(rituximab), a monoclonal antibody, was given as an alternative
treatment in two haemophilic children with HR inhibitors and impaired
quality of life, due to recurrent haemarthrosis. Rituximab was given at
the dose of 375 mg m(-2), once weekly for four consecutive weeks. Both
patients showed a partial response to rituximab reducing the inhibitor
titre to < 5 BU, thus facilitating ITT initiation; however, only the
older patient eradicated the inhibitor within 21 days after application
of ITT. The second patient, despite depletion of B cells, did not
respond to ITT. No long-term side effects have been observed in both
patients for a follow-up period of 20 and 18 months respectively. In
conclusion, rituximab appears to be an alternative effective therapy to
rapidly reduce or eliminate the inhibitor in selected cases of severely
affected haemophiliacs before further proceeding to ITT. However, the
dose and appropriate schedule, as well as long-term side effects need
further investigation.
Συγγραφείς:
Moschovi, M
Aronis, S
Trimis, G
Platokouki, H
Salavoura,
K
Tzortzatou-Stathopoulou, F
