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ArticleResearch and practice in thrombosis and haemostasis2024

Emicizumab is efficacious in people with hemophilia A with comorbidities aged ≥50 years: analysis of 4 phase III trials.

Víctor Jiménez-Yuste, Johannes Oldenburg, Eunice Tzeng, Elise Lim, Fabian Sanabria, Johnny Mahlangu

4 registry-linked trialsAbstract read
In one paragraph

Article in Research and practice in thrombosis and haemostasis, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 4 registered trials, which are not on this 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.

NCT02622321 phase3completednot on this map

A Randomized, Multicenter, Open-Label, Phase III Clinical Trial to Evaluate the Efficacy, Safety, and Pharmacokinetics of Prophylactic Emicizumab Versus no Prophylaxis in Hemophilia A Patients With Inhibitors

TypeinterventionalSponsorHoffmann-La RocheRan2015 to 2020Enrolled113ConditionsHemophilia AArmsEmicizumab, rFVIIa, aPCC
NCT02847637 phase3completednot on this map

A Randomized, Multicenter, Open-Label, Phase III Clinical Trial to Evaluate the Efficacy, Safety, and Pharmacokinetics of Prophylactic Emicizumab Versus no Prophylaxis in Hemophilia A Patients Without Inhibitors

TypeinterventionalSponsorHoffmann-La RocheRan2016 to 2022Enrolled152ConditionsHemophilia AArmsEmicizumab, Factor VIII (FVIII)
NCT03020160 phase3completednot on this map

A Multicenter, Open-Label, Phase III Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Emicizumab Given Every 4 Weeks (Q4W) in Patients With Hemophilia A

TypeinterventionalSponsorHoffmann-La RocheRan2017 to 2022Enrolled48ConditionsHemophilia AArmsEmicizumab
NCT03191799 phase3completednot on this map

A Single-Arm, Multicenter Phase IIIB Clinical Trial to Evaluate the Safety and Tolerability of Prophylactic Emicizumab in Hemophilia A Patients With Inhibitors

TypeinterventionalSponsorHoffmann-La RocheRan2017 to 2020Enrolled195ConditionsHemophilia AArmsEmicizumab
3 · Its place in the literature

Who cites it

1 citing paper in PubMed.

  1. Haemophilia Prophylaxis in the Age of Innovation: Exploring Opportunities for Personalized Treatment.Haemophilia : the official journal of the World Federation of Hemophilia · 2025
    Review
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.

Víctor Jiménez-YusteHematology Department, Hospital Universitario La Paz, IdiPAZ, Autónoma University, Madrid, Spain.
Johannes OldenburgInstitute of Experimental Haematology and Transfusion Medicine, University of Bonn, Bonn, Germany.
Eunice TzengGenentech, Inc., South San Francisco, California, USA.
Elise LimGenentech, Inc., South San Francisco, California, USA.
Fabian SanabriaF. Hoffmann-La Roche Ltd, Basel, Switzerland.
Johnny MahlanguFaculty of Health Sciences, University of the Witwatersrand and National Health Laboratory Service, Johannesburg, South Africa.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Background: The treatment of older people with hemophilia A (HA) can be complicated by comorbidities. Objectives: This post hoc analysis evaluates the efficacy and safety of emicizumab in people with HA aged ≥50 years with cardiovascular (CV) risk factors or HIV and/or hepatitis C virus (HCV) infection. Methods: The HAVEN 1 (NCT02622321), HAVEN 3 (NCT02847637), HAVEN 4 (NCT03020160), and STASEY (NCT03191799) studies enrolled adults/adolescents with severe HA. Participants were categorized as having a comorbidity if they had any CV risk factors (including history of CV disease, hypertension, diabetes, hyperlipidemia, prior stroke, or obesity), HIV, and/or HCV infection. Efficacy and safety outcomes were compared by age (<50 vs ≥50 years). Results: Of 504 participants at data cutoff, 408 were aged <50 years and 96 were aged ≥50 years. In people with HA aged <50 years, 26.7% had ≥1 CV risk factor and 29.4% had HIV and/or HCV infection. In people with HA aged ≥50 years, 72.9% had ≥1 CV risk factor and 74.0% had HIV and/or HCV infection. The mean (95% CI) annualized bleed rate for treated bleeds was 1.29 (0.07-6.06) for people with HA aged <50 years and 1.82 (0.19-6.93) for people with HA aged ≥50 years. No significant differences in annualized bleed rates were observed for those with comorbidities compared with those without. Safety outcomes were similar regardless of age. Conclusion: This pooled analysis suggests that emicizumab efficacy and safety in people with HA aged ≥50 years with CV and HIV/HCV comorbidities were consistent with those in people with HA aged <50 years enrolled in the HAVEN 1, 3, and 4 and STASEY studies.

Indexed as

comorbiditiescomorbidityemicizumabhemophilia Aprophylaxis

Identifiers

PMID38783987
PMCPMC11112372

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.