Wang Youqun, Yang Qiao, Zheng Liangda, Wang Xianting, Jiang Wenhua, Lu Lijun, Yan Lixia, Zhang Miaomiao, Chen Lili
Department of Hematology, Taizhou First People's Hospital, Huangyan Hospital of Wenzhou Medical University, Taizhou, China.
Acta Haematol. 2022;145(4):354-361. doi: 10.1159/000521360. Epub 2021 Dec 8.
The objective of the study was to investigate the effect of multiple clinical parameters (age, weight, blood types, and bleeding types) on FVIII pharmacokinetic parameters (PK parameters) in adult patients with severe hemophilia A (SHA), draw up individualized preventive treatment plans, and observe clinical efficacy.
Forty SHA patients treated in our hospital from January 2018 to May 2019 were enrolled, with their age, weight, blood types, bleeding types, and PK parameters measured to analyze the effects of clinical parameters on PK parameters. Individualized preventive treatment was developed, and patients were followed up for 1 year. The annual bleeding rate (ABR), annual joint bleeding rate (AJBR), and annual FVIII dosage were observed and compared before and after treatment.
Weight, blood types, and bleeding types could affect the PK parameters of FVIII. A prophylaxis plan was formulated under the guidance of FVIII half-life. After 1 year of follow-up, the mean ABR dropped from 36.54 to 4.06, decreased by 88.9%, the mean AJBR dropped from 28.36 to 2.75, decreased by 90%, and annual FVIII dosage increased by 47%. The dosage of FVIII in 8 patients after was less than that before prophylaxis, and the average half-life time of these 8 patients was 13.32 h.
(1) Weight, blood types, and bleeding types of adult SHA patients could affect FVIII half-life. As body mass index increased, FVIII half-life was significantly prolonged. The FVIII half-life of patients with type O blood was significantly shorter than those with other blood types, and the FVIII half-life of knee joint bleeding was conspicuously shorter than those of elbow joint bleeding. (2) Individualized preventive treatment could markedly reduce the number of bleeds. For patients with a long half-life period, the total annual FVIII dosage could be reduced to achieve bleeding prevention.
本研究旨在探讨多种临床参数（年龄、体重、血型和出血类型）对重度A型血友病（SHA）成年患者FVIII药代动力学参数（PK参数）的影响，制定个体化预防治疗方案，并观察临床疗效。
选取2018年1月至2019年5月在我院治疗的40例SHA患者，测量其年龄、体重、血型、出血类型和PK参数，分析临床参数对PK参数的影响。制定个体化预防治疗方案，并对患者进行1年的随访。观察并比较治疗前后的年出血率（ABR）、年关节出血率（AJBR）和年FVIII用量。
体重、血型和出血类型可影响FVIII的PK参数。根据FVIII半衰期制定了预防方案。随访1年后，平均ABR从36.54降至4.06，下降了88.9%，平均AJBR从28.36降至2.75，下降了90%，年FVIII用量增加了47%。8例患者治疗后的FVIII用量低于预防前，这8例患者的平均半衰期为13.32小时。
（1）成年SHA患者的体重、血型和出血类型可影响FVIII半衰期。随着体重指数增加，FVIII半衰期显著延长。O型血患者的FVIII半衰期显著短于其他血型患者，膝关节出血患者的FVIII半衰期明显短于肘关节出血患者。（2）个体化预防治疗可显著减少出血次数。对于半衰期较长的患者，可减少每年FVIII的总用量以实现出血预防。
