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Individualised prophylaxis based on personalised target trough FVIII level optimised clinical outcomes in paediatric
1Hematology Center, Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, Beijing, China.
Insights
Individualised prophylaxis for severe haemophilia A (SHA) significantly reduced bleeding events. This personalized approach, guided by pharmacokinetic (PK) levels, improved joint health and zero bleeding rates in boys with SHA.
Area of Science:
- Hematology
- Pediatric Medicine
- Pharmacokinetics
Background:
- Standard care for severe haemophilia A (SHA) necessitates individualized prophylaxis strategies.
- Previous approaches often lacked personalized dosing, potentially impacting treatment efficacy.
Purpose of the Study:
- To evaluate the effectiveness of a novel, individualized prophylaxis protocol for boys with severe haemophilia A.
- To assess the impact of a pharmacokinetic (PK)-guided, trough-level escalating regimen on clinical outcomes.
Main Methods:
- Boys with SHA underwent a six-month observational period followed by a 2-year PK-guided, trough-level escalating prophylaxis protocol.
- Clinical assessments, including joint bleeds, ultrasound (US) scores, and Haemophilia Joint Health Score (HJHS), were conducted every six months.
- Dosing adjustments were managed using the WAPPS-Hemo system based on achieved trough levels.
Main Results:
- The study analyzed 58 boys with SHA, with significant reductions in annualised bleeding rates (4 to 0) and annualised joint bleeding rates (2 to 0).
- Zero bleeding proportion increased from 27.6% to 69.0%, and zero joint bleeding proportion rose from 46.5% to 81.3%.
- Statistically significant improvements were observed in US scores (p=0.04) and HJHS (p=0.02), with 85% of target joints resolving.
Conclusions:
- The individualized prophylaxis protocol, based on target trough levels, demonstrated significant effectiveness in managing severe haemophilia A.
- These findings underscore the critical importance of personalized prophylaxis strategies in optimizing patient outcomes.
Introduction:
As standard care of severe haemophilia A (SHA), prophylaxis should be individualised.
Aim:
This study aimed to investigate the effectiveness of this new-proposed individualised prophylaxis protocol.
Methods:
Boys 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.
Results:
Fifty-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.
Conclusion:
Our results showed the effectiveness of our protocol based on individualised target trough level and emphasise the importance of personalised prophylaxis.
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