ArticleHaemophilia : the official journal of the World Federation of Hemophilia2022
Individualised prophylaxis based on personalised target trough FVIII level optimised clinical outcomes in paediatric patients with severe haemophilia A.
Article in Haemophilia : the official journal of the World Federation of Hemophilia, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 6 papers.
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Who cites it
6 citing papers in PubMed.
- Emicizumab prophylaxis vs immune tolerance induction in children with severe hemophilia A and inhibitors: a retrospective comparison of bleeding control, quality of life, and cost.Research and practice in thrombosis and haemostasis · 2026Article
- From pharmacokinetic-guided FVIII prophylaxis to low-dose Emicizumab prophylaxis: a pilot study revealed a flush of hope.Annals of hematology · 2025Article
- The Need for a Bleed Type-Specific Annual Bleeding Rate in Hemophilia Studies.JMIR public health and surveillance · 2024Article
- Real pandemic world results of pharmacokinetic-tailored personalized prophylaxis of bleeds in Polish children and adolescents with severe hemophilia A.Frontiers in pediatrics · 2023Article
- Individualised prophylaxis based on personalised target trough FVIII level optimised clinical outcomes in paediatric patients with severe haemophilia A.Haemophilia : the official journal of the World Federation of Hemophilia · 2022Article
- Exploration of the minimum necessary FVIII level at different physical activity levels in pediatric patients with hemophilia A.Frontiers in pediatrics · 2022Article
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Authors and funding
10 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
introductionAs standard care of severe haemophilia A (SHA), prophylaxis should be individualised.
aimThis study aimed to investigate the effectiveness of this new-proposed individualised prophylaxis protocol.
methodsBoys with SHA were enrolled and followed a PK-guided, trough-level escalating protocol of prophylaxis after a six-month observational period. In the next 2 years, clinical assessments including joint bleeds, ultrasound (US) scores and Haemophilia Joint Health Score (HJHS) in both sides of ankles, knees and elbows were conducted every 6 months as a scoring system, which determined whether the trough level's escalation. Adjustment of dosing regimen was based on WAPPS-Hemo.
resultsFifty-eight SHA boys were finally analysed. Their age and bodyweight were 5.3(2.8,6.9) years and 21.5(16,25) kg. During the study, 47 escalations were conducted. At study exit, the patient number and proportion of different trough level groups were: < 1 IU/dl, 17.2% (10/58); 1-3 IU/dl, 53.5% (31/58); 3-5 IU/dl, 15.5% (9/58); > 5 IU/dl, 13.8% (8/58). Significantly reduced annualised bleeding rate [4(0,8) to 0(0,2), p < .0001] and annualised joint bleeding rate [2(0,4) to 0(0,.25), p < .0001] was observed at study exit as well as the continuous trend of increased zero bleeding proportion (ZBP) (27.6%-69.0%) and zero joint bleeding proportion (46.5%-81.3%). Besides, 85% (6/7) of the target joints vanished. Statistical improvements of US scores (p = .04) and HJHS (p = .02) were also reported at study exit.
conclusionOur results showed the effectiveness of our protocol based on individualised target trough level and emphasise the importance of personalised prophylaxis.
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