Evidence map›Paper›PMID 31697801›Full record

Trial reportBlood2019

A multicenter, open-label phase 3 study of emicizumab prophylaxis in children with hemophilia A with inhibitors.

Guy Young, Ri Liesner, Tiffany Chang, Robert Sidonio, Johannes Oldenburg, Victor Jiménez-Yuste, Johnny Mahlangu, Rebecca Kruse-Jarres, Michael Wang, Marianne Uguen and 6 more

3 registry-linked trialsOpen access · bronzeAbstract readClinical Trial, Phase IIIMulticenter Study
PubMed Full text at PMC Publisher
In one paragraph

Trial report in Blood, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 3 registered trials, which are not on this map. Cited by 176 papers, 5 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
176citing papers in PubMed, 5 pooled it
30.6field-weighted citation impact, top 1% 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.

NCT02795767 phase3completednot on this map

A Multicenter, Open-Label, Phase III Clinical Trial to Evaluate the Efficacy, Safety, and Pharmacokinetics of Subcutaneous Administration of Emicizumab in Hemophilia A Pediatric Patients With Inhibitors

TypeinterventionalSponsorHoffmann-La RocheRan2016 to 2020Enrolled88ConditionsHemophilia AArmsEmicizumab
NCT04567511 phase4recruitingnot on this mapstarted 2022, after this paper: background citation

Prospective, Single-Arm, Open-Label Use of Hemlibra (Emicizumab) in the Treatment of Mild Hemophilia A

TypeinterventionalSponsorIndiana Hemophilia &Thrombosis Center, Inc.Ran2022 to 2027Enrolled20ConditionsFactor VIII Deficiency, CongenitalArmsEmicizumab
NCT06938659 phase2 / phase3not yet recruitingnot on this mapstarted 2025, after this paper: background citation

Comparison of Low Dose Emicizumab and Low Dose Factor VIII as a Prophylaxis in Hemophilia A Patients- a Randomized Control Trial'

TypeinterventionalSponsorDhaka Medical CollegeRan2025 to 2026Enrolled20ConditionsHemophilia AArmsEmicizumab Injection, Factor VIII (FVIII)
3 · Its place in the literature

Who cites it

176 citing papers in PubMed, 5 syntheses or guidelines pooled it, 367 citations in OpenAlex.

  1. [Chinese guidelines on the treatment of hemophilia (2025)].Zhonghua xue ye xue za zhi = Zhonghua xueyexue zazhi · 2025
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  11. Emicizumab dose up-titration in case of suboptimal bleeding control in people with haemophilia A.Haemophilia : the official journal of the World Federation of Hemophilia · 2023
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116 more citing papers are in PubMed but not listed here.

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

16 authors at 11 institutions in 8 countries.

Guy YoungChildren's Hospital Los Angeles, Keck School of Medicine, University of Southern California, Los Angeles, CA.
Ri LiesnerGreat Ormond Street Hospital, London, United Kingdom.
Tiffany ChangGenentech, Inc, South San Francisco, CA.
Robert SidonioChildren's Healthcare of Atlanta, Emory University, Atlanta, GA.
Johannes OldenburgInstitute of Experimental Hematology and Transfusion Medicine, University of Bonn, Bonn, Germany.
Victor Jiménez-YusteHospital Universitario La Paz, Autónoma University, Madrid, Spain.
Johnny MahlanguNational Health Laboratory Service (NHLS), Charlotte Maxeke Johannesburg Academic Hospital, University of the Witwatersrand, Johannesburg, South Africa.
Rebecca Kruse-JarresBloodworks Northwest, Seattle, WA.
Michael WangSchool of Medicine Hemophilia and Thrombosis Center, Anschutz Medical Campus, University of Colorado, Aurora, CO.
Marianne UguenF. Hoffmann-La Roche Ltd, Basel, Switzerland.
Michelle Y DoralGenentech, Inc, South San Francisco, CA.
Lilyan Y WrightGenentech, Inc, South San Francisco, CA.
Christophe SchmittF. Hoffmann-La Roche Ltd, Basel, Switzerland.
Gallia G LevyGenentech, Inc, South San Francisco, CA.
Midori ShimaDepartment of Pediatrics, Nara Medical University, Kashihara, Japan; and.
Maria Elisa MancusoAngelo Bianchi Bonomi Hemophilia and Thrombosis Centre, Ospedale Maggiore Policlinico, Fondazione Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Ca' Granda, Milan, Italy.
Roche (Switzerland) · CHBloodworks Northwest · USEmory University · USFondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico · ITGreat Ormond Street Hospital · GBHospital Universitario La Paz · ESNara Medical University · JPNational Health Laboratory Service · ZAUniversity of Bonn · DEUniversity of Colorado Anschutz Medical Campus · USUniversity of Southern California · US

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Emicizumab, a bispecific humanized monoclonal antibody, bridges activated factor IX (FIX) and FX to restore the function of missing activated FVIII in hemophilia A. Emicizumab prophylaxis in children with hemophilia A and FVIII inhibitors was investigated in a phase 3 trial (HAVEN 2). Participants, previously receiving episodic/prophylactic bypassing agents (BPAs), were treated with subcutaneous emicizumab: 1.5 mg/kg weekly (group A), 3 mg/kg every 2 weeks (group B), or 6 mg/kg every 4 weeks (group C). Pharmacokinetics, safety, and efficacy (including an intraindividual comparison of participants from a noninterventional study) were evaluated. Eighty-five participants aged <12 years were enrolled. In group A (n = 65), the annualized rate of treated bleeding events (ABRs) was 0.3 (95% confidence interval [CI], 0.17-0.50), and 77% had no treated bleeding events. Intraindividual comparison of 15 participants who previously took BPA prophylaxis showed that emicizumab prophylaxis reduced the ABR by 99% (95% CI, 97.4-99.4). In groups B (n = 10) and C (n = 10), ABRs were 0.2 (95% CI, 0.03-1.72) and 2.2 (95% CI, 0.69-6.81), respectively. The most frequent adverse events were nasopharyngitis and injection-site reactions; no thrombotic events occurred. Two of 88 participants developed antidrug antibodies (ADAs) with neutralizing potential, that is, associated with decreased emicizumab plasma concentrations: 1 experienced loss of efficacy, and, in the other, ADAs disappeared over time without intervention or breakthrough bleeding. All other participants achieved effective emicizumab plasma concentrations, regardless of the treatment regimen. Emicizumab prophylaxis has been shown to be a highly effective novel medication for children with hemophilia A and inhibitors. This trial was registered at www.clinicaltrials.gov as #NCT02795767.

Indexed as

AdolescentAntibodies, BispecificAntibodies, Monoclonal, HumanizedChildChild, PreschoolFactor VIIIFemaleHemophilia AHemorrhageHumansInfantIsoantibodiesMaleQuality of LifeTreatment OutcomeAntibodies, BispecificAntibodies, Monoclonal, HumanizedemicizumabFactor VIIIIsoantibodies

Identifiers

PMID31697801
PMCPMC6908828
OpenAlexW2980900975

What OpenQuestion holds

Textmetadata
Read underepoch 390

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.