Trial reportJournal of blood medicine2025
Effectiveness of PK-Guided Personalized Recombinant FVIII Treatment in Patients with Hemophilia A: Clinical Case Experiences Based on an Observational Study.
Trial report in Journal of blood medicine, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
1 citing paper in PubMed.
- Can Model-Informed Precision Dosing of Beta-Lactams in Critical Care Improve Clinical Outcomes?Chemotherapy · 2026Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
5 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Purpose: Prophylaxis with recombinant factor VIII (rFVIII) products is the gold-standard treatment for hemophilia A. However, interindividual differences affect the half-life and clearance of rFVIII products. The myPKFiT is a web-based medical-device software program for population pharmacokinetic (PK) simulation of FVIII products to guide accurate FVIII doses and dosing intervals. In this Japanese multicenter observational study, the efficacy of regimen adjustment using myPKFiT was examined. Patients and Methods: Male patients with hemophilia A undergoing personalized treatment with myPKFiT using either octocog alfa or rurioctocog alfa pegol were included. Patients were aged <18 years. Primary endpoint was annualized bleeding rate (ABR). Secondary endpoints were ABR by type of bleeding, rFVIII product consumption, physical activity level, quality of life, and frequency of rFVIII administrations. Results are presented descriptively; however, for exploratory analysis, data before and after regimen adjustment were compared using the Wilcoxon signed-rank test. Results: Seven patients aged 3-17 years (median age 13 years) participated in the study. Mean ABR for all bleeds decreased by 0.86 after PK-guided regimen adjustment. Four patients showed zero ABR before and after regimen adjustment using myPKFiT. No significant differences were noted in the consumption of rFVIII products. However, mean rFVIII consumption decreased in two patients after PK-guided regimen adjustment. Three patients increased physical activity and, according to the treatment based on the PK-guided regimen adjustment, this resulted in no increased bleeding. Conclusion: The results from this study in a small number of patients suggest that PK-guided regimen adjustment with myPKFiT may support optimization of the individual prophylactic administration of the FVIII products octocog alfa and rurioctocog alfa pegol. Study Registration: UMIN000044800.
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