Evidence map›Paper›PMID 39692401›Full record

Trial reportHaemophilia : the official journal of the World Federation of Hemophilia2025

Pain-Related Quality of Life Outcomes in People With Haemophilia A Receiving Emicizumab: A Post Hoc Analysis of the HAVEN 1, 3 and 4 and STASEY Studies.

Cedric Hermans, Mark W Skinner, Brittany Gentile, Elise Lim, Miranda Minhas, Katya Moreno, Eunice Tzeng, Tyler W Buckner

3 registry-linked trialsAbstract readClinical Trial, Phase III
In one paragraph

Trial report in Haemophilia : the official journal of the World Federation of Hemophilia, 2025. 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 5 papers.

0numbers the graph read from it
0cells of the map it votes in
5citing 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
3 · Its place in the literature

Who cites it

5 citing papers in PubMed.

  1. Article
  2. Article
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  5. 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.

Cedric HermansHaemostasis and Thrombosis Unit, Division of Haematology, Cliniques Universitaires Saint-Luc, Catholic University of Louvain (UCLouvain), Brussels, Belgium.ORCID https://orcid.org/0000-0001-5429-8437
Mark W SkinnerDepartment of Health Research Methods, Evidence and Impact, McMaster University, Hamilton, Ontario, Canada.ORCID https://orcid.org/0000-0002-0934-0680
Brittany GentileProduct Development, Genentech, Inc., South San Francisco, California, USA.
Elise LimData & Statistical Sciences OMNI, Genentech, Inc., South San Francisco, California, USA.
Miranda MinhasUS Medical Affairs, Genentech, Inc., South San Francisco, California, USA.
Katya MorenoGlobal Product Development/Medical Affairs (PDMAO), F. Hoffmann-La Roche Ltd, Basel, Switzerland.
Eunice TzengRare Blood Disease, US Medical Affairs, Genentech, Inc., South San Francisco, California, USA.
Tyler W BucknerDepartment of Medicine, University of Colorado School of Medicine, Aurora, Colorado, USA.ORCID https://orcid.org/0000-0003-1954-4385

Funding

F. Hoffmann-La Roche Ltd.
6 · The paper itself

Abstract

introductionPeople with haemophilia A (PwHA) experience acute and chronic pain associated with reduced quality of life (QoL).

aimsThis post hoc analysis of pooled data from the HAVEN 1 (NCT02622321), 3 (NCT02847637), 4 (NCT03020160) and STASEY (NCT0319179) studies assessed the impact of emicizumab prophylaxis on pain-related QoL in PwHA.

methodsPwHA received emicizumab during the four studies. In this analysis, pain was assessed using patient-reported responses to pain-specific questions from the Haem-A-QoL/Haemo-QoL-SF and the pain/discomfort dimension of the EQ-5D-5L. Responses were recorded at baseline and at regular intervals for up to 78 weeks following treatment initiation. Additional analyses evaluated the population with target joints at baseline, and the overall population stratified by age, factor (F)VIII inhibitor status and prior treatment.

resultsAt the data cut-off, 504 PwHA had been treated across the four studies; 464 and 470 completed the Haem-A-QoL/Haemo-QoL-SF and the EQ-5D-5L, respectively. Improvements in pain-related QoL were observed by Week 13 of emicizumab prophylaxis and maintained through Week 78. In the overall population, responses of 'never/rarely' for 'my swellings hurt' and 'pain in joints' increased from 37.0% and 30.0% at baseline to 84.0% and 61.0% by Week 13, suggesting reductions in acute and chronic pain, respectively. Similar improvements were seen in the target joint population, and across all strata. Greater improvements were observed in younger versus older PwHA.

conclusionPain-related QoL improved with emicizumab prophylaxis regardless of target joints, age, FVIII inhibitor status or prior treatment. Haemophilia-specific assessments are needed to accurately capture and characterize pain in PwHA.

Indexed as

Antibodies, BispecificAntibodies, Monoclonal, HumanizedHemophilia APainQuality of LifeAdolescentAdultAgedFemaleHumansMaleMiddle AgedYoung AdultAntibodies, BispecificAntibodies, Monoclonal, Humanizedemicizumabarthralgiaemicizumabhaemarthrosishaemophiliapainprophylaxisquality of life

Identifiers

PMID39692401
PMCPMC11780222

What OpenQuestion holds

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LicenceCC BY-NC
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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.