Evidence map›Paper›PMID 41288435›Full record

ArticleHaemophilia : the official journal of the World Federation of Hemophilia

In Silico Assessment of Limited Blood Sampling Strategies for Individualised Pharmacokinetic-guided Dosing of Efanesoctocog Alfa in Haemophilia A Patients.

Jelien den Hollander, Marjon H Cnossen, Ron A A Mathôt

Abstract read
In one paragraph

Article in Haemophilia : the official journal of the World Federation of Hemophilia. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

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.

2 · The registry

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.

3 · Its place in the literature

Who cites it

1 citing paper in PubMed.

  1. Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

3 authors.

Jelien den HollanderHospital Pharmacy-Clinical Pharmacology, Academic Medical Centre Amsterdam, Amsterdam, the Netherlands.ORCID https://orcid.org/0009-0001-5883-0475
Marjon H CnossenDepartment of Pediatric Hematology and Oncology, Erasmus MC Sophia Children's Hospital, University Medical Centre Rotterdam, Rotterdam, the Netherlands.ORCID https://orcid.org/0000-0003-1557-2995
Ron A A MathôtHospital Pharmacy-Clinical Pharmacology, Academic Medical Centre Amsterdam, Amsterdam, the Netherlands.ORCID https://orcid.org/0000-0001-8401-0879

Funding

BayerCSL BehringNetherlands Organisation for Scientific ResearchSwedish Orphan Biovitrum
6 · The paper itself

Abstract

introductionEfanesoctocog alfa is a novel factor VIII (FVIII) concentrate with a unique molecular design that enables Von Willebrand Factor-independent clearance in patients with haemophilia A. Limited sampling strategies (LSSs) are necessary to implement accurate pharmacokinetic (PK)-guided dosing for efanesoctocog alfa in clinical practice.

aimThis in silico study aims to evaluate the predictive performance of 10 LSSs with one to three samples for estimating individual PK profiles of efanesoctocog alfa.

methodsMonte Carlo simulations based on a published population PK model generated individual FVIII activity-time profiles for a virtual population. LSSs were applied to sample from these profiles, and PK parameters, FVIII activity peak level at 0.5 h (C

resultsAll LSSs complied with our requirements of a relative mean prediction error of <±5% and a relative root mean square error of <25%. For prophylactic dosing advice, a LSS was considered clinically suitable if at least 80% of predicted C

conclusionsSeveral LSSs demonstrated adequate predictive performance for PK-guided dosing of efanesoctocog alfa.

Indexed as

Blood Specimen CollectionComputer SimulationFactor VIIIHemophilia ADrug CombinationsHumansMonte Carlo MethodPrecision Medicinevon Willebrand FactorDrug CombinationsFactor VIIIfactor VIII, von Willebrand factor drug combinationvon Willebrand Factorefanesoctocog alfafactor VIIIhaemophilia Alimited sampling strategypharmacokinetics

Identifiers

PMID41288435
PMCPMC12904192

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Registered trials

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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.