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Background  Immune tolerance induction ( ITI ) therapy is currently unaffordable in China. Management of hemophilia A children with high‐titer inhibitor is therefore a challenge.    Aim  To describe the  ITI  strategy using plasma‐derived  factor VIII /von Willebrand factor concentrate (pd FVIII / VWF ) +/− immunosuppression and to report its efficacy in children with hemophilia A having poor‐risk status for  ITI  success.    Methods  A prospective pilot study on children with hemophilia A having poor‐risk status (all with at least inhibitor titer > 10  BU  pre‐ ITI  initiation). Patients received ~50  IU /kg  FVIII  every other day using domestic intermediate purity pd FVIII / VWF  products, either alone or in combination with rituximab +/− prednisone.    Results  Sixteen patients with median age 2.9 (range, 2.2‐13.2) years and median pre‐ ITI  inhibitor titer 30.7 (range, 10.4‐128)  BU  were enrolled. Analysis at median 14.7 (range, 12.4‐22.6) months’ follow‐up showed a total response rate of 87.5%. This included success (achieving inhibitor < 0.6  BU ) in 13 patients (81.3%) in a median of 8.8 (range, 3.2‐11.8) months, and partial success (achieving inhibitor < 5  BU  but > 0.6 BU ) in 1 (6.3%). Compared to the pre‐ ITI  period, the mean bleeds/month during  ITI  was 0.51 (64.0% reduction), and joint bleeds/month was 0.34 (64.3% reduction). This low‐dose  ITI  strategy cost less by 70% to 87% than that for the high‐dose  FVIII  regimen. No severe adverse events were observed.    Conclusion  This low‐dose  ITI  strategy of pd FVIII / VWF  +/− immunosuppression achieved relatively satisfactory outcomes in children with hemophilia A inhibitor having poor‐risk status. This low‐dose regimen showed economic advantages and is therefore suitable for using in China. However, further study in a larger cohort with a longer follow‐up time is needed.

Low‐dose immune tolerance induction for children with hemophilia A with poor‐risk high‐titer inhibitors: A pilot study in China