Evidence map›Paper›PMID 34163136›Full record

ReviewBiologics : targets & therapy2021

Recent Advances in the Treatment of Hemophilia: A Review.

Emanuela Marchesini, Massimo Morfini, Leonard Valentino

Registry-linked trialAbstract readReview
In one paragraph

Review in Biologics : targets & therapy, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT05920512 (PLA General Hospital), which is not on this map. Cited by 42 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
42citing papers in PubMed, 1 pooled it
–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.

NCT05920512 phase1 / phase2unknown statusnot on this mapstarted 2022, after this paper: background citation

PLA General Hospital

TypeinterventionalSponsorXue-chun LuRan2022 to 2025Enrolled10ConditionsHemophiliaArmsSodium valproate extended-release tablets
3 · Its place in the literature

Who cites it

42 citing papers in PubMed, 1 synthesis or guideline pooled it.

  1. Surgery for chronic arthropathy in people with haemophilia.The Cochrane database of systematic reviews · 2022
    Pooled it
  2. Review
  3. Article
  4. Review
  5. Exploring gene editing as a potential therapeutic strategy for hemophilia.Frontiers in bioengineering and biotechnology · 2026
    Review
  6. Article
  7. Review
  8. Article
  9. Article
  10. Article
  11. Observational
  12. Article
  13. Burden of Haemophilia A in South Korea: A Serial Cross-Sectional Study From 2008 to 2021.Haemophilia : the official journal of the World Federation of Hemophilia · 2025
    Article
  14. Clinical perspective: Advancing hemophilia treatment through gene therapy approaches.Molecular therapy : the journal of the American Society of Gene Therapy · 2025
    Review
  15. Review
  16. Article
  17. Review
  18. Development of a novel gene editing lexicon for hemophilia: methodology and results.Research and practice in thrombosis and haemostasis · 2025
    Article
  19. Review
  20. 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.

Emanuela MarchesiniHemophilia Centre, SC Vascular and Emergency Department, University of Perugia, Perugia, Italy.
Massimo MorfiniItalian Association of Haemophilia Centres (AICE), Naples, Italy.ORCID 0000-0001-6565-4943
Leonard ValentinoNational Hemophilia Foundation, New York, NY, USA.ORCID 0000-0002-7927-6418

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Progress in hemophilia therapy has been remarkable in the first 20 years of the third millennium, but the innovation began with the description the fractionation of plasma in 1946. The first concentrates followed the discovery of FVIII in the cryoprecipitate of frozen plasma and FIX in the supernatant in the early 1960s, which led to the initial attempts at replacement therapy. Unfortunately, the lack of screening methods for viral pathogens resulted in people with hemophilia (PWH) receiving concentrates contaminated by hepatitis A virus, hepatitis C virus, and human immunodeficiency virus, as these concentrates were made from large industrial pools of plasma derived from thousands of donors. Fortunately, by 1985, viral screening methods and proper virucidal techniques were developed that made concentrates safe. Increasingly pure products followed the introduction of chromatography steps with monoclonal antibodies in the production process. The problem of immunogenicity of exogenously administered concentrates has not yet had a complete solution. The development of alloantibodies against FVIII in about 25-35% of PWH is the most serious adverse effect of replacement therapy. The next major advance followed the cloning of the

Indexed as

adverse eventsextended half-life concentratesgene therapynon-replacement therapypharmacokineticsreplacement therapy

Identifiers

PMID34163136
PMCPMC8214539

What OpenQuestion holds

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Registered trials

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.