Evidence map›Paper›PMID 31033112›Full record

ArticleHaemophilia : the official journal of the World Federation of Hemophilia2019

The changing face of immune tolerance induction in haemophilia A with the advent of emicizumab.

Manuel Carcao, Carmen Escuriola-Ettingshausen, Elena Santagostino, Johannes Oldenburg, Ri Liesner, Beatrice Nolan, Angelika Bátorová, Saturnino Haya, Guy Young, Future of Immunotolerance Treatment Group

Open access · hybridAbstract read
In one paragraph

Article in Haemophilia : the official journal of the World Federation of Hemophilia, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 45 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
45citing papers in PubMed, 1 pooled it
9.7field-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.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

45 citing papers in PubMed, 1 synthesis or guideline pooled it, 93 citations in OpenAlex.

  1. Pooled it
  2. Extension Study With rVIII-SingleChain in Previously Untreated Patients (PUPs) With Severe Haemophilia A.Haemophilia : the official journal of the World Federation of Hemophilia · 2025
    Trial
  3. Trial
  4. Trial
  5. Article
  6. Article
  7. Article
  8. Race, Ethnicity, and Hemophilia: A Scoping Review.Journal of racial and ethnic health disparities · 2026
    Review
  9. Article
  10. Article
  11. Review
  12. Article
  13. Article
  14. Article
  15. Simoctocog alfa (NuwiqTherapeutic advances in hematology · 2024
    Review
  16. Article
  17. Article
  18. Article
  19. Article
  20. 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

10 authors at 7 institutions in 7 countries.

Manuel CarcaoDivision of Haematology/Oncology, Department of Paediatrics and Child Health Evaluative Sciences, Research Institute, The Hospital for Sick Children, University of Toronto, Toronto, Ontario, Canada.ORCID https://orcid.org/0000-0001-5350-1763
Carmen Escuriola-EttingshausenHaemophilia Centre Rhine Main, Möerfelden-Walldorf, Germany.
Elena SantagostinoMaggiore Hospital Policlinico, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Milan, Italy.
Johannes OldenburgInstitute of Experimental Haematology and Transfusion Medicine, University of Bonn, Bonn, Germany.ORCID https://orcid.org/0000-0003-3038-2145
Ri LiesnerHaemophilia Centre, Great Ormond Street Hospital for Children, NHS Trust Haemophilia Centre, London, UK.
Beatrice NolanOur Lady's Children's Hospital Crumlin, Dublin, Ireland.
Angelika BátorováDepartment of Haematology and Transfusion Medicine and National Haemophilia Centre, University Hospital, Comenius University, Bratislava, Slovakia.
Saturnino HayaUnit for Congenital Bleeding Disorders, Hospital Universitario y Politécnico La Fe, Valencia, Spain.
Guy YoungChildren's Hospital Los Angeles, University of Southern California Keck School of Medicine, Los Angeles, California.ORCID https://orcid.org/0000-0001-6013-1254
Future of Immunotolerance Treatment Group
Children's Health Ireland at Crumlin · IEComenius University Bratislava · SKGreat Ormond Street Hospital · GBHospital Universitari i Politècnic La Fe · ESUniversity of Bonn · DEUniversity of Southern California · USUniversity of Toronto · CA

Funding

Grifols, S.A., Barcelona, Spain
6 · The paper itself

Abstract

introductionAs a result of the new treatment paradigm that the haemophilia community will face with the availability of novel (non-factor) therapies, an updated consensus on ITI recommendations and inhibitor management strategies is needed.

aimThe Future of Immunotolerance Treatment (FIT) group was established to contemplate, determine and recommend the best management options for patients with haemophilia A and inhibitors. DISCUSSION AND

conclusionsDespite the considerable success of emicizumab in the management of inhibitor patients, the FIT group still sees the importance of eradicating inhibitors. However, the availability of emicizumab and other non-factor therapies in the future might impact greatly on how ITI is undertaken. Theoretically, concomitant use of emicizumab and FVIII might allow emicizumab to effectively prevent bleeding with lower dose ITI regimens. This might allow for the greater adoption of low-dose/low-frequency FVIII ITI regimens, which may result in a reduced need for central venous access devices while still maintaining a reasonable likelihood of ITI success. The FIT group proposes a new management algorithm for current ITI (without emicizumab) and a hypothetical new approach with the availability of emicizumab. As there are no published data regarding the concomitant use of emicizumab and FVIII for ITI, the FIT Expert group encourages the undertaking of properly conducted prospective studies to explore these approaches further.

Indexed as

Antibodies, BispecificAntibodies, Monoclonal, HumanizedAntibodies, NeutralizingDose-Response Relationship, DrugFactor VIIIHemophilia AHumansImmune ToleranceRisk AssessmentAntibodies, BispecificAntibodies, Monoclonal, HumanizedAntibodies, NeutralizingemicizumabFactor VIIIbypassing agentsemicizumabfactor VIIIhaemophilia Aimmune tolerance inductioninhibitor

Identifiers

PMID31033112
PMCPMC6850066
OpenAlexW2943226571

What OpenQuestion holds

Textmetadata
LicenceCC BY
Read underepoch 390

Registered trials

None linked

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.