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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

Blatný, Jan ; Astermark, Jan LU ; Catarino, Cristina ; Dolan, Gerry ; Fijnvandraat, Karin ; Hermans, Cédric ; Holstein, Katharina ; Jiménez-Yuste, Víctor ; Klamroth, Robert and Lavin, Michelle , et al. (2024) In Therapeutic advances in hematology 15.
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.... (More)

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.

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organization
publishing date
type
Contribution to journal
publication status
published
subject
keywords
hemophilia, innovation, management, outcomes, quality of life
in
Therapeutic advances in hematology
volume
15
publisher
SAGE Publications
external identifiers
  • scopus:85206105390
  • pmid:39381602
ISSN
2040-6207
DOI
10.1177/20406207241285143
language
English
LU publication?
yes
id
10ed7981-1984-4440-bba1-f7bc48b8034a
date added to LUP
2024-12-18 13:19:24
date last changed
2025-07-17 06:26:25
@article{10ed7981-1984-4440-bba1-f7bc48b8034a,
  abstract     = {{<p>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.</p>}},
  author       = {{Blatný, Jan and Astermark, Jan and Catarino, Cristina and Dolan, Gerry and Fijnvandraat, Karin and Hermans, Cédric and Holstein, Katharina and Jiménez-Yuste, Víctor and Klamroth, Robert and Lavin, Michelle and Lenting, Peter J. and Lobet, Sébastien and Mancuso, Maria Elisa and Motwani, Jayashree and O’Donnell, James S. and Königs, Christoph}},
  issn         = {{2040-6207}},
  keywords     = {{hemophilia; innovation; management; outcomes; quality of life}},
  language     = {{eng}},
  publisher    = {{SAGE Publications}},
  series       = {{Therapeutic advances in hematology}},
  title        = {{Future needs for continuing innovation in hemophilia : improving outcomes for individuals of all severities, including women and those in resource-constrained regions}},
  url          = {{http://dx.doi.org/10.1177/20406207241285143}},
  doi          = {{10.1177/20406207241285143}},
  volume       = {{15}},
  year         = {{2024}},
}