Evidence map›Paper›PMID 35646171›Full record

ArticleJournal of medicine and life2022

Expert opinion on current and future prophylaxis therapies aimed at improving protection for people with hemophilia A.

Angelika Batorova, Ana Boban, Melen Brinza, Toshiko Lissitchkov, Laszlo Nemes, Irena Zupan Preložnik, Petr Smejkal, Nadezhda Zozulya, Jerzy Windyga

Open access · diamondAbstract read
In one paragraph

Article in Journal of medicine and life, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 6 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
6citing papers in PubMed, 1 pooled it
1.8field-weighted citation impact, top 14% 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

6 citing papers in PubMed, 1 synthesis or guideline pooled it, 11 citations in OpenAlex.

  1. Pooled it
  2. Article
  3. Article
  4. Review
  5. Sexual activity and the adolescent with haemophilia.Acta bio-medica : Atenei Parmensis · 2023
    Review
  6. 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

9 authors at 6 institutions in 9 countries.

Angelika BatorovaDepartment of Hematology and Transfusion Medicine, National Hemophilia Center, Faculty of Medicine of Comenius University and University Hospital, Bratislava, Slovakia.ORCID https://orcid.org/0000-0003-0567-4250
Ana BobanHaemophilia Centre, University Hospital Centre Zagreb, School of Medicine, University of Zagreb, Zagreb, Croatia.ORCID https://orcid.org/0000-0003-3532-2336
Melen BrinzaCentre of Hematology and Bone Marrow Transplant, European Haemophilia Comprehensive Care Centre, Fundeni Clinical Institute, Bucharest, Romania.
Toshiko LissitchkovExpert Center for Rare Haematological Disorders, Sofia, Bulgaria.
Laszlo NemesNational Haemophilia Centre and Haemostasis Department, Medical Centre of Hungarian Defence Forces, Budapest, Hungary.
Irena Zupan PreložnikDepartment of Haematology, University Medical Centre Ljubljana, Faculty of Medicine, University of Ljubljana, Ljubljana, Slovenia.ORCID https://orcid.org/0000-0001-9933-549X
Petr SmejkalDepartment of Clinical Haematology, University Hospital Brno, Brno, Czech Republic.
Nadezhda ZozulyaNational Research Center for Hematology, Moscow, Russia.ORCID https://orcid.org/0000-0001-7074-0926
Jerzy WindygaDepartment of Hemostasis Disorders and Internal Medicine, Institute of Hematology and Transfusion Medicine, Warsaw, Poland.
Pediatrics and Genetics · USInstytut Hematologii i Transfuzjologi · PLMasaryk University · CZNational Medical Research Center for Hematology · RUUniversity Hospital Bratislava · SKUniversity Hospital Centre Zagreb · HR

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

The next frontier in hemophilia A management has arrived. However, questions remain regarding the broader applicability of new and emerging hemophilia A therapies, such as the long-term safety and efficacy of non-factor therapies and optimal regimens for individual patients. With an ever-evolving clinical landscape, it is imperative for physicians to understand how available and future hemophilia A therapies could potentially be integrated into real-life clinical practice to improve patient outcomes. Against this background, nine hemophilia experts from Central European countries participated in a pre-advisory board meeting survey. The survey comprised 11 multiple-choice questions about current treatment practices and future factor and non-factor replacement therapies. The survey questions were developed to reflect current unmet needs in hemophilia management reflected in the literature. The experts also took part in a follow-up advisory board meeting to discuss the most important unmet needs for hemophilia management as well as the pre-meeting survey results. All experts highlighted the challenge of maintaining optimal trough levels with prophylaxis as their most pressing concern. Targeting trough levels of ≥30-50 IU/L or even higher to achieve less bleeding was highlighted as their preferred strategy. However, the experts had an equal opinion on how this could be achieved (

Indexed as

Hemophilia AEuropeHemorrhageHumansABR – annualized bleed rateaPCC – activated prothrombin complex concentratesBPAs – bypassing agentsBU – Bethesda unitsEHL – extended half-lifeextended half-lifeFVIII/IX – factor VIII/IXhemophiliaITI – immune tolerance inductionmHJHS – modified Haemophilia Joint Health Scorepd – plasma-derivedPPX – prophylaxisprophylaxisQoL – quality of liferFVIIIFc – recombinant FVIII Fc fusion proteinrFVIIIpeg – recombinant pegylated FVIIIr – recombinantSHL – standard half-lifesiRNA – small interfering RNATFPI – tissue factor pathway inhibitorTGA – thrombin generation assaysWFH – World Federation of Hemophilia

Identifiers

PMID35646171
PMCPMC9126455
OpenAlexW4282940665

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.