Evidence map›Paper›PMID 40242991›Full record

ReviewHaemophilia : the official journal of the World Federation of Hemophilia2025

Haemophilia Prophylaxis in the Age of Innovation: Exploring Opportunities for Personalized Treatment.

Yesim Dargaud, Fabienne Volot, Stephanie Desage, Claire Pouplard, Valerie Chamouard, Anne Lienhart

Abstract readReview
In one paragraph

Review in Haemophilia : the official journal of the World Federation of Hemophilia, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 5 papers.

0numbers the graph read from it
0cells of the map it votes in
5citing 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

5 citing papers in PubMed.

  1. Article
  2. Review
  3. NXT007 enhancesResearch and practice in thrombosis and haemostasis · 2025
    Article
  4. Review
  5. 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

6 authors.

Yesim DargaudNational Reference Centre of Haemophilia, Clinical Haemostasis Unit, Hospices Civils de Lyon and Université Claude Bernard Lyon 1, Lyon, France.ORCID https://orcid.org/0000-0002-4888-1463
Fabienne VolotHaemophilia Comprehensive Care Centre, Dijon Bourgogne University Hospital, Dijon, France.
Stephanie DesageNational Reference Centre of Haemophilia, Clinical Haemostasis Unit, Hospices Civils de Lyon and Université Claude Bernard Lyon 1, Lyon, France.ORCID https://orcid.org/0000-0003-0455-0248
Claire PouplardDepartment of hemostasis, University Hospital of Tours, Tours, France.
Valerie ChamouardNational Reference Centre of Haemophilia, Clinical Haemostasis Unit, Hospices Civils de Lyon and Université Claude Bernard Lyon 1, Lyon, France.
Anne LienhartNational Reference Centre of Haemophilia, Clinical Haemostasis Unit, Hospices Civils de Lyon and Université Claude Bernard Lyon 1, Lyon, France.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

introductionRecent advancements in haemophilia treatment have introduced a range of innovative therapies, including activated FVIII mimetics, ultra-extended half-life recombinant FVIII, rebalancing agents and gene therapy. These developments have transformed treatment options and expanded prophylaxis strategies.

aimThis article reviews key criteria and personalization strategies for the optimization of prophylaxis, tailored to the unique needs of each haemophilia patient on an individual basis. DISCUSSION AND

conclusionAlthough these new therapies offer significant promise in controlling bleeding and enhancing quality of life, challenges such as variable efficacy, potential long-term risks and high costs remain. Additionally, the absence of validated surrogate markers for non-factor therapies limits their optimal use in clinical settings. The primary goal of haemophilia care is to provide optimal personalized treatment that enables patients to lead unrestricted lives. However, the introduction of these novel treatments necessitates a shift in treatment paradigms and the development of improved evaluation tools for better personalization. Choosing the most appropriate treatment requires careful clinical follow-up, taking into account patient preferences, disease burden, and individual and environmental factors that affect patients' lives. Ongoing research and real-world studies are essential to assess long-term efficacy and safety. In addition, improving educational resources-such as digital platforms, virtual reality tools and personalized educational materials tailored to patients' life goals-and effective patient engagement strategies will be critical to achieving successful treatment personalization and adherence.

Indexed as

Hemophilia APrecision MedicineFactor VIIIGenetic TherapyHumansQuality of LifeFactor VIIIefanesoctocog alfagene therapyhaemophilianon‐factor therapiespersonalized prophylaxis

Identifiers

PMID40242991
PMCPMC12306857

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC-ND
Read underepoch 390

Registered trials

None linked

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.