Abstract Number: PB0633
Meeting: ISTH 2021 Congress
Background: WFH guideline recommend any type of prophylaxis regimen– standard, intermediate-dose or low-dose prophylaxis- is better than on-demand treatment for hemophilia patients. The latter two regimens are generally applied in adult patients in China.
Aims: To compare the efficacy and safety of low-dose and intermediate-dose prophylaxis in adult patients with severe hemophilia A.
Methods: Sixteen adult patients with severe hemophilia A who received low-dose prophylaxis treatment then transited to intermediate-dose prophylaxis under regular follow-up in hemophilia clinic of Chengdu third people’s Hospital were included. The data of annual bleeding rate(ABR)、the annual joint bleeding rate(AJBR)、the total ultrasonic score (HEAD-US-C) and HJHS score of bilateral elbow joint, knee joint and ankle joint were collected retrospectively. We conduct paired Wilcoxon test with these data by SPSS 22.0.
Results: Sixteen adult patients with severe hemophilia A had a median age of 30 years (20-37 years). The average FVIII consumption per year is 1182 IU/kg during low-dose prophylaxis period and 1767 IU/kg during intermediate-dose prophylaxis period respectively. The average ABR reduced from 21 to 12 (p=0.001) and the average AJBR declined from 18 to 10(p=0.002) after regimen adjustment. The average total ultrasound score showed no significantly difference between low-dose(26.9) and intermediate-dose (31.81) prophylaxis. The average HJHS score demonstrate a significant reduction from 43. 69 points during the low-dose prophylactic treatment period to 37. 31points during the intermediate-dose prophylactic treatment(p=0.001). No FVIII inhibitors occurred after 1 year of intermediate-dose prophylaxis.
Conclusions: Intermediate-dose prophylaxis can significantly reduce the ABR and AJBR in adult patients with severe hemophilia A. Although the joint structure damage is not reversable, the joint function can be improved. Medium dose prophylaxis does not increase the risk of FVIII inhibitors in patients with severe hemophilia A.
To cite this abstract in AMA style:高华 h, Chen R, Zhou S. Compare the Efficacy and Safety of Low-dose and Intermediate-dose Prophylactic Therapy in Adult Patients with Severe Hemophilia A, a Self-control Study [abstract]. Res Pract Thromb Haemost. 2021; 5 (Suppl 2). https://abstracts.isth.org/abstract/compare-the-efficacy-and-safety-of-low-dose-and-intermediate-dose-prophylactic-therapy-in-adult-patients-with-severe-hemophilia-a-a-self-control-study/. Accessed November 28, 2023.
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