Open AccessPK‐tailored tertiary prophylaxis in patients with severe hemophilia A at Beijing Children's Hospital
Author(s) -
Li Peijing,
Chen Zhenping,
Cheng Xiaoling,
Wang Yan,
Zhang Ningning,
Zhen Yingzi,
Wu Xinyi,
Wu Runhui
Publication year - 2019
Publication title -
pediatric investigation
Language(s) - English
Resource type - Journals
ISSN - 2574-2272
DOI - 10.1002/ped4.12122
Subject(s) - medicine , regimen , dosing , pediatrics , pharmacokinetics , incidence (geometry) , surgery , optics , physics
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.