Evidence map›Paper›PMID 37916312›Full record

ReviewHaematologica2024

Emicizumab: the hemophilia A game-changer.

Pedro E Alcedo Andrade, Pier Mannuccio Mannucci, Craig M Kessler

Open access · goldAbstract readReview
In one paragraph

Review in Haematologica, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 27 papers.

0numbers the graph read from it
0cells of the map it votes in
27citing papers in PubMed
7.8field-weighted citation impact, top 2% of its field
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

27 citing papers in PubMed, 27 citations in OpenAlex.

  1. Safety and Use of Eptacog Beta 225 µg/kg in Patients With Haemophilia A or B With Inhibitors.Haemophilia : the official journal of the World Federation of Hemophilia · 2025
    Trial
  2. Review
  3. Observational
  4. Non-Factor Therapies in Haemophilia: The Era of Factor VIII Mimetics and Targeted Rebalancing Agents.Transfusion medicine and hemotherapy : offizielles Organ der Deutschen Gesellschaft fur Transfusionsmedizin und Immunhamatologie · 2026
    Review
  5. Article
  6. Review
  7. Article
  8. Article
  9. Article
  10. Article
  11. Review
  12. Article
  13. Factor VIIIResearch and practice in thrombosis and haemostasis · 2026
    Article
  14. Anti-emicizumab antibodies and their relevance in clinical practice.Research and practice in thrombosis and haemostasis · 2026
    Article
  15. Review
  16. Article
  17. Normalization in hemophilia: conceptual foundations and clinical implications.Research and practice in thrombosis and haemostasis · 2025
    Review
  18. Review
  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 at 2 institutions in 2 countries.

Pedro E Alcedo AndradeGeorgetown University Medical Center, Lombardi Comprehensive Cancer Center, Washington, DC,.
Pier Mannuccio MannucciFondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi, Hemophilia and Thrombosis Center, Milan.
Craig M KesslerGeorgetown University Medical Center, Lombardi Comprehensive Cancer Center, Washington, District of Columbia. kesslerc@gunet.georgetown.edu.
Georgetown University Medical Center · USFondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico · IT

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

In hemophilia, the unmet needs regarding adherence to prophylaxis and lack of effective long-term prophylaxis regimens, especially in patients with inhibitors, led to the production of emicizumab, the first non-factor medicine for subcutaneous administration in patients with severe and moderate hemophilia A with or without factor VIII inhibitors. This review describes the research steps behind the development of this game-changing medication as well as its success in the prophylaxis of bleeding episodes, as witnessed by the results of pivotal clinical trials but also by real-life use in the frame of a still expanding global market. We also discuss potential and actual adverse events and the nuances related to clinical use, such as laboratory monitoring, development of neutralizing antidrug antibodies, risk of thrombosis/hypercoagulability and role in the management of surgical operations. The potential of emicizumab to prevent bleeding in other congenital and acquired coagulation disorders is also outlined.

Indexed as

Antibodies, BispecificAntibodies, Monoclonal, HumanizedHemophilia AFactor VIIIHemorrhageHumansAntibodies, BispecificAntibodies, Monoclonal, HumanizedemicizumabFactor VIII

Identifiers

PMID37916312
PMCPMC11063855
OpenAlexW4388226471

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC
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.