[Immune tolerance induction in a severe hemophilia A patient with inhibitor].
OBJECTIVE
To explore the immune tolerance induction (ITI) in a severe hemophilia A patient with inhibitor, and to improve the therapeutic efficacy for patient.


METHODS
The FVIII:C was assayed by one-stage method and FVIII antibody by Bethesda method. Mutation screening of FVIII gene intron 22 inversion was performed using LD-PCR.


RESULTS
FVIII gene intron 22 inversion was detected in this patient. Clinical tolerance to FVIII was successfully induced after administration of the ITI regimen combined with immunosuppression. A fall of inhibitor titer from 8 BU to 0 BU after treatment for 3 months, and in vivo FVIII recovery (> 66%) was normalized. The patient had no bleeding episode in the following 6 months.


CONCLUSIONS
This is the first case report on successful immune tolerance induction therapy in Chinese hemophilia A patient. ITI is the most effective therapy for hemophilia A with inhibitor.
State Key Laboratory of Experimental Hematology, Institute of Hematology & Blood Diseases Hospital, CAMS & PUMC, Tianjin 300020, China.
Lei
L
Zhang
Feng
F
Xue
Xiao-Fan
XF
Liu
Ze-Ping
ZP
Zhou
Yong-Ze
YZ
Liu
Meng-Su
MS
Tian
Lin
L
Shen
Xian-Hui
XH
Xu
Hong-Li
HL
Zhang
Ren-Chi
RC
Yang
D001323:Autoantibodies / Q000276:immunology; D005169:Factor VIII / Q000235:genetics; D006467:Hemophilia A / Q000235:genetics; D006801:Humans; D007108:Immune Tolerance / Q000187:drug effects; D007165:Immunosuppression Therapy
D001323:Autoantibodies; D005169:Factor VIII