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

. 2024 ; 15 () : 20406207241285143. [epub] 20240930

Status PubMed-not-MEDLINE Language English Country Great Britain, England Media electronic-ecollection

Document type Journal Article, Review

Links

PubMed 39381602
PubMed Central PMC11459663
DOI 10.1177/20406207241285143
PII: 10.1177_20406207241285143
Knihovny.cz E-resources

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.

Amsterdam UMC University of Amsterdam Emma Children's Hospital Pediatric Hematology Amsterdam Netherlands

Center for Thrombosis and Hemorrhagic Diseases IRCCS Humanitas Research Hospital Rozzano Milan Italy and Humanitas University Pieve Emanuele Milan Italy

Centre for Haemostasis and Thrombosis St Thomas' Comprehensive Care Centre London UK

Department of Haematology and Oncology University Medical Centre Hamburg Eppendorf Hamburg Germany

Department of Haematology La Paz University Hospital IdiPaz Autónoma University of Madrid Madrid Spain

Department 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

Department of Paediatric Haematology Birmingham Children's Hospital Birmingham UK

Department of Translational Medicine Lund University and Department of Hematology Oncology and Radiation Physics Skåne University Hospital Malmö Sweden

Division of Haematology Saint Luc University Hospital Catholic University of Louvain Brussels Belgium

Goethe University University Hospital Frankfurt Department of Paediatrics and Adolescent Medicine Frankfurt Germany

Hospital Bory Nemocnica Bory a s Ivana Kadlečíka 2 Bratislava 841 06 Slovakia

Immunochemotherapy Department Congenital Coagulopathies Comprehensive Care Centre Santa Maria University Hospital Lisbon University Lisbon Portugal

Irish Centre for Vascular Biology School of Pharmacy and Biomedical Sciences Royal College of Surgeons in Ireland Dublin Ireland

Irish Centre for Vascular Biology School of Pharmacy and Biomolecular Sciences Royal College of Surgeons in Ireland Dublin Ireland

National Coagulation Centre St James' Hospital Dublin Ireland

Neuromusculoskeletal Lab Brussels Belgium

Service d'ergothérapie et de kinésithérapie Cliniques universitaires Saint Luc Université catholique de Louvain Brussels Belgium

Université Paris Saclay INSERM Hémostase Inflammation Thrombose HITh U1176 94276 Le Kremlin Bicêtre France

University Hospital Brno Masaryk University Brno Czech Republic

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