Abstract Number: PB0322
Meeting: ISTH 2020 Congress
Background: Relationship between PK parameters and imaging examination scores were not reported before.
Aims: To investigate the relationship between individual PK profiles and bleeds or imaging scores in Chinese boys with severe hemophilia A.
Methods: Thirty-five boys with severe hemophilia A were enrolled in Beijing Children’s Hospital in 2018. After a wash-out period of 72 hours and a single FVIII infusion of 50IU/kg, blood samples were collected and tested at five time points for PK assessment. Trough level and time below 1IU/dL weekly were generated by WAPPS-Hemo. X-ray, ultrasound and MRI were used to check the both sides of knees, elbows and ankles in the form of Pettersson, HEAD-US and IPSG MRI scores. Baseline and bleeding details were collected.
Results: Twenty-one boys kept trough FVIII level above 1IU/dL in daily prophylaxis. Compared with those with lower trough level (< 1IU/dL), patients in higher trough FVIII level group (>1IU/dL) had fewer bleedings (ABR:3 vs 5, P< 0.05; AJBR: 1 vs 2, P< 0.05 ), lower Pettersson score
(0 vs 2, P< 0.05), HEAD-US score (1.5 vs. 3, P< 0.05) and HJHS scores (1.5 vs. 4, P< 0.05). Time below 1IU/dL weekly was correlated with ABR (r=0.69, P< 0.01), AJBR (r=0.64, P< 0.01),MRI scores (r=0.54, P< 0.05) US scores (r=0.50, P< 0.05), X-ray scores (r-0.49, P< 0.05) and HJHS scores (r=0.47, P< 0.05). In addition, US score had a good relationship with X-ray scores
(r=0.78, P< 0.001), MRI scores (r=0.92, P< 0.001) and HJHS score (r=0.57, P< 0.001).
Conclusions: Patients with longer time spent below a trough FVIII level of 1IU/dL bleed more and had higher imaging and HJHS scores. The US has good relationship with X-ray, MRI and HJHS scores and has potential to be used in hemophilia patients’ daily examinations.
To cite this abstract in AMA style:Kun H, Chen Z, Li G, Wu R, Zhen Y, Zhang N. Bleeds and Imaging Scores in Relation to Pharmacokinetics of Coagulation Factor VIII in Chinese Pediatric Patients with Severe Hemophilia A [abstract]. Res Pract Thromb Haemost. 2020; 4 (Suppl 1). https://abstracts.isth.org/abstract/bleeds-and-imaging-scores-in-relation-to-pharmacokinetics-of-coagulation-factor-viii-in-chinese-pediatric-patients-with-severe-hemophilia-a/. Accessed December 5, 2021.
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