Clinical benefits of a Bayesian model for plasma-derived factor VIII/VWF after one year of pharmacokinetic-guided prophylaxis in severe/moderate hemophilia A patients

Megías‐Vericat, Juan Eduardo; Bonanad, S.; Haya, Saturnino; Cid, Ana Rosa; Marqués, María Remedios; Ferrada, Alejandra; Monte-Boquet, Emilio; Pérez‐Alenda, Sofía; Bosch, Pau; Querol‐Giner, Felipe; Poveda, José Luís

doi:10.1016/j.thromres.2021.07.009

Cited by 4 publications

(3 citation statements)

References 38 publications

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“…Urgent request of a higher trough level was also revealed by the low zero bleeding proportions of different trough level groups at study entry. This was in accordance with other well conducted studies before 20,21 . During the individualised prophylaxis period, the most escalations (59.6%) were due to the joint bleeds alone.…”

Section: Discussionsupporting

confidence: 92%

“…This was in accordance with other well conducted studies before. 20 , 21 During the individualised prophylaxis period, the most escalations (59.6%) were due to the joint bleeds alone. But if joint bleed is the only criteria for escalation, dose adjustment would not have been performed in the remaining 40.6% in lack of systematical evaluation.…”

Section: Discussionmentioning

confidence: 99%

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Individualised prophylaxis based on personalised target trough FVIII level optimised clinical outcomes in paediatric patients with severe haemophilia A

Huang

et al. 2022

Haemophilia

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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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Section: Discussionsupporting

confidence: 92%

Section: Discussionmentioning

confidence: 99%

Individualised prophylaxis based on personalised target trough FVIII level optimised clinical outcomes in paediatric patients with severe haemophilia A

Huang

et al. 2022

Haemophilia

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“…The incidence is about 1/1000. Clinical manifestations are mainly bleeding of different degrees, such as nasal bleeding, gingival bleeding, skin ecchymosis, bleeding after skin abrasion, excessive menstrual blood volume, postpartum bleeding, and postoperative bleeding [ 7 ].…”

Section: Introductionmentioning

confidence: 99%

[Retracted] Correlation Analysis of DNA Methylation in the von Willebrand Factor Promoter Region and the Risk of Unexplained Recurrent Hemophilia: Systematic Review and Meta‐Analysis

Jing

Yang

et al. 2022

Contrast Media & Molecular Imaging

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This study systematically reviewed the effect of DNA methylation in the promoter region of the coagulation factor vWF gene on the risk of unexplained recurrent hemophilia. PubMed, Medline, Web of Science, and other computers were used to search the database, and the statistical randomized controlled trials of coagulation factor vWF in the risk analysis of unknown recurrent hemophilia were collected. The Cochrane systematic evaluation method was used to evaluate the quality of the included kinds of literature, and Revman5 software was used to sort out and analyze the kinds of literature. Meta-analysis showed that there was a statistical difference between the experimental group and the control group in case fatality rate (OR = 1.76, 95% CI (1.29, 2.39), P = 0.0003 , I2 = 0%, Z = 3.58), adverse events (OR = 2.38, 95% CI (1.65, 3.45), P < 0.00001 , I2 = 0%, Z = 4.60), incidence of joint hemorrhage (OR = 2.52, 95% CI (1.62, 3.91), P < 0.00001 , I2 = 0%, Z = 4.12), incidence of subcutaneous stasis (OR = 1.76, 95% CI (1.26, 2.45), P = 0.0009 , I2 = 5%, Z = 3.33), and hematoma volume (OR = 1.78, 95% CI (1.32, 2.40), P = 0.0001 , I2 = 23%, Z = 3.80). DNA methylation in the promoter region of the coagulation factor vWF gene was significantly associated with the risk of unexplained recurrent hemophilia. Whether demethylation can improve the bleeding index of patients with recurrent hemophilia remains to be further explored.

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The road to implementation of pharmacokinetic-guided dosing of factor replacement therapy in hemophilia and allied bleeding disorders. Identifying knowledge gaps by mapping barriers and facilitators

et al. 2023

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Clinical benefits of a Bayesian model for plasma-derived factor VIII/VWF after one year of pharmacokinetic-guided prophylaxis in severe/moderate hemophilia A patients

Cited by 4 publications

References 38 publications

Individualised prophylaxis based on personalised target trough FVIII level optimised clinical outcomes in paediatric patients with severe haemophilia A

Individualised prophylaxis based on personalised target trough FVIII level optimised clinical outcomes in paediatric patients with severe haemophilia A

[Retracted] Correlation Analysis of DNA Methylation in the von Willebrand Factor Promoter Region and the Risk of Unexplained Recurrent Hemophilia: Systematic Review and Meta‐Analysis

The road to implementation of pharmacokinetic-guided dosing of factor replacement therapy in hemophilia and allied bleeding disorders. Identifying knowledge gaps by mapping barriers and facilitators

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