Evidence map›Paper›PMID 39381602›Full record

ReviewTherapeutic advances in hematology2024

Future needs for continuing innovation in hemophilia: improving outcomes for individuals of all severities, including women and those in resource-constrained regions.

Jan Blatný, Jan Astermark, Cristina Catarino, Gerry Dolan, Karin Fijnvandraat, Cédric Hermans, Katharina Holstein, Víctor Jiménez-Yuste, Robert Klamroth, Michelle Lavin and 6 more

Abstract readReview
In one paragraph

Review in Therapeutic advances in hematology, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing 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.

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

1 citing paper in PubMed.

  1. 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

16 authors.

Jan BlatnýHospital Bory, Nemocnica Bory, a.s., Ivana Kadlečíka 2, Bratislava 841 06, Slovakia.ORCID https://orcid.org/0000-0001-6261-9157
Jan AstermarkDepartment of Translational Medicine, Lund University, and Department of Hematology, Oncology and Radiation Physics, Skåne University Hospital, Malmö, Sweden.
Cristina CatarinoImmunochemotherapy Department, Congenital Coagulopathies Comprehensive Care Centre, Santa Maria University Hospital, Lisbon University, Lisbon, Portugal.
Gerry DolanCentre for Haemostasis and Thrombosis, St Thomas' Comprehensive Care Centre, London, UK.
Karin FijnvandraatAmsterdam UMC, University of Amsterdam, Emma Children's Hospital, Pediatric Hematology, Amsterdam, Netherlands.
Cédric HermansDivision of Haematology, Saint-Luc University Hospital, Catholic University of Louvain, Brussels, Belgium.ORCID https://orcid.org/0000-0001-5429-8437
Katharina HolsteinDepartment of Haematology and Oncology, University Medical Centre Hamburg-Eppendorf, Hamburg, Germany.ORCID https://orcid.org/0000-0003-3753-0972
Víctor Jiménez-YusteDepartment of Haematology, La Paz University Hospital-IdiPaz, Autónoma University of Madrid, Madrid, Spain.ORCID https://orcid.org/0000-0003-3937-3499
Robert KlamrothDepartment of Internal Medicine, Vivantes Hospital Friedrichshain, Berlin, Germany, and Institute of Experimental Haematology and Transfusion Medicine, University Hospital Bonn, Medical Faculty, University of Bonn, Bonn, Germany.
Michelle LavinIrish Centre for Vascular Biology, School of Pharmacy and Biomedical Sciences, Royal College of Surgeons in Ireland, Dublin, Ireland.
Peter J LentingUniversité Paris-Saclay, INSERM, Hémostase Inflammation Thrombose HITh U1176, 94276, Le Kremlin-Bicêtre, France.
Sébastien LobetService d'ergothérapie et de kinésithérapie, Cliniques universitaires Saint-Luc, Université catholique de Louvain (UCLouvain), Brussels, Belgium.ORCID https://orcid.org/0000-0002-3829-6850
Maria Elisa MancusoCenter for Thrombosis and Hemorrhagic Diseases, IRCCS Humanitas Research Hospital, Rozzano, Milan, Italy and Humanitas University, Pieve Emanuele, Milan, Italy.ORCID https://orcid.org/0000-0002-7113-4028
Jayashree MotwaniDepartment of Paediatric Haematology, Birmingham Children's Hospital, Birmingham, UK.
James S O'DonnellIrish Centre for Vascular Biology, School of Pharmacy and Biomolecular Sciences, Royal College of Surgeons in Ireland, Dublin, Ireland.
Christoph KönigsGoethe University, University Hospital Frankfurt, Department of Paediatrics and Adolescent Medicine, Frankfurt, Germany.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Over recent decades, management of people with hemophilia (PwH) has been greatly improved by scientific advances that have resulted in a rich and varied therapeutic landscape. Nevertheless, treatment limitations continue to drive innovation, and emerging options have the potential to realize further improvement. We advocate four general principles to optimize benefits from innovation: individualizing the treatment approach, targeting 'normal,' making the most of available resources, and considering treatment affordability. Ultimately, all PwH-men and women, of all ages and severities, and worldwide-should have access to treatment that fully prevents bleeding, while allowing personal, social, family, and professional lives of choice. Clearly, we are not there yet, but developing goals/milestones based on the principles we describe may help to achieve this.

Indexed as

hemophiliainnovationmanagementoutcomesquality of life

Identifiers

PMID39381602
PMCPMC11459663

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC
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.