Evidence map›Paper›PMID 35905294›Full record

Trial reportHaemophilia : the official journal of the World Federation of Hemophilia2022

Effect of emicizumab prophylaxis on bone and joint health markers in people with haemophilia A without factor VIII inhibitors in the HAVEN 3 study.

Anna Kiialainen, Markus Niggli, Christine L Kempton, Giancarlo Castaman, Tiffany Chang, Ido Paz-Priel, Joanne I Adamkewicz, Gallia G Levy

Registry-linked trialOpen access · hybridAbstract readRandomized Controlled TrialClinical Trial, Phase III
In one paragraph

Trial report in Haemophilia : the official journal of the World Federation of Hemophilia, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT02847637 (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), which is not on this map. Cited by 21 papers, 2 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
21citing papers in PubMed, 2 pooled it
4.7field-weighted citation impact, top 4% 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.

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)
3 · Its place in the literature

Who cites it

21 citing papers in PubMed, 2 syntheses or guidelines pooled it, 34 citations in OpenAlex.

  1. Pooled it
  2. Pooled it
  3. Trial
  4. Trial
  5. Trial
  6. Observational
  7. Review
  8. Article
  9. Review
  10. The haemophilia joint health score for the assessment of joint health in patients with haemophilia.Haemophilia : the official journal of the World Federation of Hemophilia · 2024
    Review
  11. Article
  12. Review
  13. Review
  14. Article
  15. Article
  16. Article
  17. Article
  18. Review
  19. Gene therapy for hemophilia: looking beyond factor expression.Experimental biology and medicine (Maywood, N.J.) · 2022
    Review
  20. Emicizumab Prophylaxis in Patients with Severe Hemophilia A: Insights from A Resource Limited Country.Clinical and applied thrombosis/hemostasis : official journal of the International Academy of Clinical and Applied Thrombosis/Hemostasis
    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 3 countries.

Anna KiialainenF. Hoffmann-La Roche Ltd, Basel, Switzerland.ORCID https://orcid.org/0000-0002-3273-9649
Markus NiggliF. Hoffmann-La Roche Ltd, Basel, Switzerland.
Christine L KemptonHemophilia of Georgia Center for Bleeding & Clotting Disorders of Emory, Emory University School of Medicine, Atlanta, Georgia, USA.ORCID https://orcid.org/0000-0002-2391-2016
Giancarlo CastamanCenter for Bleeding Disorders and Coagulation, Careggi University Hospital, Florence, Italy.
Tiffany ChangGenentech, Inc., South San Francisco, California, USA.
Ido Paz-PrielGenentech, Inc., South San Francisco, California, USA.
Joanne I AdamkewiczGenentech, Inc., South San Francisco, California, USA.
Gallia G LevyGenentech, Inc., South San Francisco, California, USA.
Fondazione GraphiTech · ITRoche (Switzerland) · CHAzienda Ospedaliero-Universitaria Careggi · ITEmory University · USSpark Therapeutics (United States) · US

Funding

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

Abstract

introductionEmicizumab prophylaxis significantly reduces bleeding events; however, the associated impact on bone/joint health is unknown.

aimTo explore the effect of emicizumab prophylaxis on bone/joint health in people with haemophilia A (PwHA) without FVIII inhibitors enrolled in HAVEN 3 (NCT02847637).

methodsHaemophilia joint health scores (HJHS; v2.1) were evaluated at baseline and Weeks 49 and 97 in PwHA receiving emicizumab (n = 134), and at baseline and Weeks 49, 73 and 97 in PwHA who switched to emicizumab after 24 weeks of no prophylaxis (n = 17). Bone and joint biomarkers were measured in 117 PwHA at baseline and at Weeks 13, 25, 49 and 73.

resultsHJHS was lower for PwHA who were previously on FVIII prophylaxis, aged <40 years or had no target joints at baseline compared with PwHA who were receiving no prophylaxis, aged ≥40 years or with target joints. Clinically significant mean (95% confidence interval) improvements from baseline of -2.13 (-3.96, -.29) in HJHS joint-specific domains were observed at Week 49 in PwHA with at least one target joint at study entry (n = 71); these changes were maintained through Week 97. Improvements in HJHS from baseline were also observed for PwHA aged 12-39 years. Biomarkers of bone resorption/formation, cartilage degradation/synthesis, and inflammation did not change significantly during emicizumab prophylaxis.

conclusionsClinically relevant improvements in HJHS were observed in younger PwHA and those with target joints after 48 weeks of emicizumab in HAVEN 3. Biomarkers of bone/joint health did not show significant changes during 72 weeks of emicizumab prophylaxis.

Indexed as

Antibodies, BispecificHemophilia AAntibodies, Monoclonal, HumanizedBiomarkersFactor VIIIHumansAntibodies, BispecificAntibodies, Monoclonal, HumanizedBiomarkersemicizumabFactor VIIIbiomarkersboneemicizumabfactor VIIIhaemophilia Ajointsprophylaxis

Identifiers

PMID35905294
PMCPMC9796488
OpenAlexW4288707385

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC
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.