Successful immune tolerance induction with a plasma-derived FVIII concentrate and intravenous immunoglobulins in a pediatric patient with congenital severe hemophilia A and poor prognostic factors

摘要

We present the case of a pediatric patient born in July 1991, diagnosed with severe hemophilia A at 8 months of life after a hemarthrosis. He was treated with regular factor replacement therapy on-demand until an inhibitor was detected (1.75-2.5 BU) at the age of 6. The patient started an immunotolerance induction (ITI) program, which was discontinued 3 months later because of parental decision based on inhibitor persistence (3.75-6.75 BU). On-demand treatment with recombinant activated FVII in bleeding episodes was applied. Titer peaked 13 months later (37 BU). On May 2003 (age 11), rescue ITI with plasma-derived FVIII (Fanhdi, 100IU/kg per 24h daily) and intravenous immunoglobulin (IVIg) (Flebogamma, 1g/kg per 24h for 2 days every 3 weeks) was started. Inhibitor eradication was achieved after 16 months of ITI. The patient continued with FVIII+IVIg treatment for 3 additional months when he was switched to FVIII prophylaxis (40IU/kg 3 times a week). At present, the patient is inhibitor-free.