Hemophilia A Patient
Conditions
Keywords
Hemophilia A, Hemarthrosis, Extended half life factor VIII, Low dose Emicizumab, Cartilage biomarker, HEAD-US
Brief summary
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.
Interventions
DRUGSwitching to low dose emicizumab
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
Sponsors
Chulalongkorn University
Study design
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
MALE
Age
3 Years to 30 Years
Healthy volunteers
No
Inclusion criteria
* hemophilia A patients aged 3-30 years who received treatment with PK-guided EHL Factor VIII for more than 8 months
Exclusion criteria
* hemophilia A patients with presence of factor VIII inhibitors
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Annual bleeding rate | 8 months | Annual bleeding rate report as times per year |
| Annual joint bleeding rate | 8 months | Annual joint bleeding rate report as times per year |
| Hemophilia Joint Health Score (HJHS) | 8 months | 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. |
| The quality of life assessment instrument for children and adolescents with haemophilia (Hae-QoL) | 8 months | 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. |
Countries
Thailand
Outcome results
None listed