Recent studies have shown that pharmacokinetic (PK)-guided extended half-life (EHL) factor VIII can improve joint health in patients with hemophilia A. However, some patients experience suboptimal joint outcomes despite optimized PK-guided therapy. Low-dose emicizumab has emerged as a potential option for improving bleeding control and joint health in patients who do not respond adequately to PK-guided EHL factor VIII. The objectives of the study is to compare clinical bleeding outcomes, joint health, cartilage biomarkers, and musculoskeletal ultrasound findings (HEAD-US) in hemophilia A patients in Thailand during PK-guided EHL factor VIII therapy and after switching to low-dose emicizumab. Eighteen patients with hemophilia A from King Chulalongkorn Memorial Hospital, Thailand, aged 8-28 years, who were receiving PK-guided EHL factor VIII were enrolled. Patients with a Hemophilia Joint Health Score (HJHS) ≥ 12 were switched to low-dose emicizumab. A loading dose of 2 mcg/kg was administered, followed by dosing every 2 weeks during the first month and every 4 weeks thereafter. Clinical bleeding, annual bleeding rate (ABR), annual joint bleeding rate (AJBR), HJHS, cartilage biomarkers, and musculoskeletal ultrasound findings (HEAD-US) were evaluated every 4 months for up to 8 months after switching.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
18
All Hemophilia A patients in the study had received treatment with PK-guided EHL factor VIII. The patients who had HJHS more than 12 were switched to treatment with low dose emicizumab
Chulalongkorn University
Bangkok, Thailand
Annual bleeding rate
Annual bleeding rate report as times per year
Time frame: 8 months
Annual joint bleeding rate
Annual joint bleeding rate report as times per year
Time frame: 8 months
Hemophilia Joint Health Score (HJHS)
HJHS is the physical examination assessment tool for hemophilia patients evaluated most commonly 6 joints affected by bleeding (both elbows, both knees and both ankles). The scores range from 0 (minimum) to 125 (maximum). The lowest score means the best joint health.
Time frame: 8 months
The quality of life assessment instrument for children and adolescents with haemophilia (Hae-QoL)
The assessment tools evaluate physical, psychological, family, social aspect of Hemophilia patients. There are 35 questions. Each questions has multiple choices (never, seldom, somtimes, often, always) represent score 1 (never) to 5 (always). The total raw score range from 35 to 175 and then must be transform to "transformed score" ranged from 0 to 100. 0 is the best and 100 is the worst score.
Time frame: 8 months
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