Evidence map›Paper›PMID 38193069›Full record

ArticleResearch and practice in thrombosis and haemostasis2023

Emicizumab use in females with moderate or mild hemophilia A without factor VIII inhibitors who warrant prophylaxis.

Cedric Hermans, Giuliana Ventriglia, Samya Obaji, Benjamin M Beckermann, Michaela Lehle, Olivier Catalani, Roseline d'Oiron, Laurent Frenzel

Registry-linked trialOpen access · goldAbstract read
In one paragraph

Article in Research and practice in thrombosis and haemostasis, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT04158648 (A Multicenter, Open-Label Study to Evaluate the Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of Emicizumab in Patients With Mild or Moderate Hemophilia A Without FVIII Inhibitors), which is not on this map. Cited by 3 papers.

0numbers the graph read from it
0cells of the map it votes in
3citing papers in PubMed
1.2field-weighted citation impact, top 18% 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.

NCT04158648 phase3completednot on this map

A Multicenter, Open-Label Study to Evaluate the Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of Emicizumab in Patients With Mild or Moderate Hemophilia A Without FVIII Inhibitors

TypeinterventionalSponsorHoffmann-La RocheRan2020 to 2025Enrolled73ConditionsMild Hereditary Factor VIII Deficiency Disease Without Inhibitor, Moderate Hereditary Factor VIII Deficiency Disease Without Inhibitor, Hemophilia AArmsEmicizumab
3 · Its place in the literature

Who cites it

3 citing papers in PubMed, 4 citations in OpenAlex.

  1. Observational
  2. Article
  3. 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

8 authors at 5 institutions in 4 countries.

Cedric HermansDivision of Haematology, Cliniques Universitaires Saint-Luc, Université Catholique de Louvain (UCLouvain), Brussels, Belgium.
Giuliana VentrigliaProduct Development Clinical Science Hematology, F. Hoffmann-La Roche Ltd, Basel, Switzerland.
Samya ObajiCardiff Haemophilia Centre, University Hospital of Wales, Cardiff, United Kingdom.
Benjamin M BeckermannProduct Development Clinical Science Oncology & Hematology, F. Hoffmann-La Roche Ltd, Basel, Switzerland.
Michaela LehleProduct Development Clinical Science Oncology & Hematology, F. Hoffmann-La Roche Ltd, Basel, Switzerland.
Olivier CatalaniStatistics, F. Hoffmann-La Roche Ltd, Basel, Switzerland.
Roseline d'OironReference centre for Haemophilia and Rare Congenital Bleeding Disorders, Bicêtre Hospital AP-HP, University of Paris-Saclay and UMR-S1176 INSERM, Le Kremlin-Bicêtre, France.
Laurent FrenzelCentre de Traitement de l'Hémophilie, AP-HP, Hôpital Necker Enfants Malades, Paris, France.
Roche (Switzerland) · CHCliniques Universitaires Saint-Luc · BEHôpital Necker-Enfants Malades · FRInserm · FRUniversity Hospital of Wales · GB

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Background: Hemophilia A (HA) is predominantly associated with males due to X-linked inheritance. Males and females with HA have shared unmet medical needs, highlighting the necessity for comprehensive care irrespective of sex. Objectives: This analysis investigated the efficacy and safety of emicizumab prophylaxis in 3 females with HA. Methods: HAVEN 6 (NCT04158648) is a phase III study of emicizumab in people with non-severe HA without factor (F)VIII inhibitors warranting prophylaxis per investigator assessment, and the study methodology has been reported previously. Female-specific endpoints included menstruation-related quality of life and menstruation heaviness. Results: HAVEN 6 enrolled 3 females aged ≥18 years and within reproductive age ( Conclusion: Overall, results were consistent with those reported in the male population enrolled in the HAVEN 6 study, suggesting efficacy and a favorable safety profile for emicizumab in females with non-severe HA warranting prophylaxis.

Indexed as

emicizumabfemalehemophilia Aprophylaxis

Identifiers

PMID38193069
PMCPMC10772889
OpenAlexW4387844360

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC-ND
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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.