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Importance  Tertiary prophylaxis using a low‐dose regimen is usually insufficient to prevent recurrent joint bleeding and deterioration in joint diseases in children with severe hemophilia A. Pharmacokinetic ( PK ) dosing is a useful approach to increase the precision and efficiency of prophylaxis.    Objective  To explore the efficacy of  PK ‐tailored tertiary prophylaxis in children with severe hemophilia A.    Methods  We implemented a  PK ‐tailored tertiary prophylaxis program for 15 boys with severe hemophilia A aged 5–16 years at Beijing Children's Hospital. Following  PK  testing and a 6‐month evaluation period (phase I), 15 patients were divided in two groups according to individual  PK  data and actual bleeding: (1) a  PK ‐tailored group [modified prophylaxis regimen according to  PK  data for the next 6 months (phase  II );  n  = 8] and (2) a maintenance group (continued the original regimen for the next 6 months;  n  = 7). We compared the bleeding rate, infusion frequency, and factor  VIII  ( FVIII ) consumption between the two groups.    Results  In the  PK ‐tailored group, the median annual joint bleeding rate was reduced from 7.8 in phase I to 1.4 in phase  II , mean annual total factor consumption increased from 1619.0  IU /kg in phase I to 2401.9  IU /kg in phase  II , and median infusion frequency for prophylaxis increased from 104 times/year in phase I to 156 times/year in phase  II  ( P  < 0.05). Although the  FVIII  consumption increased, it remained at approximately half of the standard method.    Interpretation   PK ‐tailored prophylaxis may represent a more efficient approach to individual prophylaxis in China, but further studies are required to verify this.

PK‐tailored tertiary prophylaxis in patients with severe hemophilia A at Beijing Children's Hospital