Abstract Number: PB1008
Meeting: ISTH 2020 Congress
Theme: Hemophilia and Rare Bleeding Disorders » Hemophilia - Clinical
Background: Hemophilic arthropathy is the most serious complication of severe hemophilia A.
Aims: This is a interim research report for prospective comparative analysis of joint structure and function in Chinese children with severe hemophilia A under different doses FⅧ.(China registration number:ChiCTR-ONC-17011186)
Methods: The study included 47 children(4-17 years old) with no inhibitor in severe hemophilia A.There were 9 cases in on-demand treatment group,18 in low-dose prophylaxis(FⅧ10-15IU/kg,2-3/week,and≤30IU/kg/week) group,and 20 in medium-dose prophylaxis(FⅧ15-20IU/kg,2-3/week,and 45-60IU/kg/week) group.We investigated the changes of joints in children during the two-year period from bleeding phenotype(Annual bleeding rate,ABR/Annual target joint bleeding rate,ATJBR),HJHS(Hemophilia Joint Health Score),FISH(Functional Independence Score),ultrasound and MRI score.Median observation period was 22 months(658 days).
Results: There were no significant differences in median age,body weight,and BMI in each group(P>0.05).Children´s ABR(12.34/year/patient) in medium-dose group was reduced by 238.6% and 49.4% compared with on-demand group(41.79/year/patient) and low-dose group(18.44/year/patient).The ATJBR from the most severe joint:the difference between on-demand group(7.07/year/patient),low-dose group(2.85/year/patient) and medium-dose group(0.94/year/patient) was statistically significant(P1=0.015,P2=0.003,P3=0.000).The HJHS from the most severe target joint:on-demand group was degraded by 2.14±2.41 points,compared with low-dose group(-0.35±2.15) and medium-dose group(-0.69±1.99) were statistically different(P1=0.020,P2=0.008);but there was no statistical difference between low-dose and medium-dose groups(P=0.650).FISH score:there was a statistical difference between the on-demand and low-dose groups(P=0.034),while no difference between middle-dose and on-demand group,neither between low-dose and middle-dose group.The ultrasound score from the most severe target joint:85.7%(6/7) cases in on-demand group worsened,47.1%(8/17) in low-dose group and 36.8%(7/19) in middle-dose group, with no statistical difference between the groups.The MRI score from the most severe target joint:57.1%(4/7) progress in the on-demand group,which are statistically significant compared with 35.3%(6/17) in low-dose group and 23.5%(4/17) in middle-dose group(P1=0.026,P2=0.042),however,no difference between low and middle dose groups(P=0.201).
Conclusions: Preliminary interim results show that medium-dose prophylaxis in children with severe hemophilia A in China is slightly better than low-dose prophylaxis in protecting joint structure and function,and significantly better than on-demand therapy.
To cite this abstract in AMA style:
Meng L, Zhuang JM, Sun XY, Zhou X, Liu ZQ, Zhou F, Liu H, Sun J. Prospective Comparative Analysis of Joint Structure and Function in Chinese Children with Severe Hemophilia A under Different Doses Factor Ⅷ [abstract]. Res Pract Thromb Haemost. 2020; 4 (Suppl 1). https://abstracts.isth.org/abstract/prospective-comparative-analysis-of-joint-structure-and-function-in-chinese-children-with-severe-hemophilia-a-under-different-doses-factor-%e2%85%a7/. Accessed September 27, 2023.« Back to ISTH 2020 Congress
ISTH Congress Abstracts - https://abstracts.isth.org/abstract/prospective-comparative-analysis-of-joint-structure-and-function-in-chinese-children-with-severe-hemophilia-a-under-different-doses-factor-%e2%85%a7/