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

J 2024

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

BLATNÝ, Jan; Jan ASTERMARK; Cristina CATARINO; Gerry DOLAN; Karin FIJNVANDRAAT et al.

Základní údaje

Originální název

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

Autoři

Vydání

THERAPEUTIC ADVANCES IN HEMATOLOGY, London, SAGE PUBLICATIONS LTD, 2024, 2040-6207

Další údaje

Jazyk

angličtina

Typ výsledku

Článek v odborném periodiku

Obor

30205 Hematology

Stát vydavatele

Velká Británie a Severní Irsko

Utajení

není předmětem státního či obchodního tajemství

Odkazy

URL

Impakt faktor

Impact factor: 3.100

Označené pro přenos do RIV

Ano

Kód RIV

RIV/00216224:14110/24:00138888

Organizační jednotka

Lékařská fakulta

Klíčová slova anglicky

hemophilia; innovation; management; outcomes; quality of life

Štítky

14110321, rivok

Příznaky

Mezinárodní význam, Recenzováno

Změněno: 20. 2. 2025 09:06, Mgr. Tereza Miškechová

Anotace

V originále

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.

Citovat

BLATNÝ, Jan; Jan ASTERMARK; Cristina CATARINO; Gerry DOLAN; Karin FIJNVANDRAAT; Cedric HERMANS; Katharina HOLSTEIN; Victor JIMENEZ-YUSTE; Robert KLAMROTH; Michelle LAVIN; Peter J LENTING; Sebastien LOBET; Maria Elisa MANCUSO; Jayashree MOTWANI; James S O'DONNELL a Christoph KOENIGS. Future needs for continuing innovation in hemophilia: improving outcomes for individuals of all severities, including women and those in resource-constrained regions. THERAPEUTIC ADVANCES IN HEMATOLOGY. London: SAGE PUBLICATIONS LTD, 2024, roč. 15, č. 2024, s. 1-8. ISSN 2040-6207. Dostupné z: https://doi.org/10.1177/20406207241285143.

@article{2478480,
   author = {Blatný, Jan and Astermark, Jan and Catarino, Cristina and Dolan, Gerry and Fijnvandraat, Karin and Hermans, Cedric and Holstein, Katharina and JimenezandYuste, Victor and Klamroth, Robert and Lavin, Michelle and Lenting, Peter J and Lobet, Sebastien and Mancuso, Maria Elisa and Motwani, Jayashree and O'Donnell, James S and Koenigs, Christoph},
   article_location = {London},
   article_number = {2024},
   doi = {https://doi.org/10.1177/20406207241285143},
   keywords = {hemophilia; innovation; management; outcomes; quality of life},
   language = {eng},
   issn = {2040-6207},
   journal = {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 = {https://journals.sagepub.com/doi/epdf/10.1177/20406207241285143},
   volume = {15},
   year = {2024}
}

TY  - JOUR
ID  - 2478480
AU  - Blatný, Jan - Astermark, Jan - Catarino, Cristina - Dolan, Gerry - Fijnvandraat, Karin - Hermans, Cedric - Holstein, Katharina - Jimenez-Yuste, Victor - Klamroth, Robert - Lavin, Michelle - Lenting, Peter J - Lobet, Sebastien - Mancuso, Maria Elisa - Motwani, Jayashree - O'Donnell, James S - Koenigs, Christoph
PY  - 2024
TI  - Future needs for continuing innovation in hemophilia: improving outcomes for individuals of all severities, including women and those in resource-constrained regions
JF  - THERAPEUTIC ADVANCES IN HEMATOLOGY
VL  - 15
IS  - 2024
SP  - 1-8
EP  - 1-8
PB  - SAGE PUBLICATIONS LTD
SN  - 20406207
KW  - hemophilia
KW  - innovation
KW  - management
KW  - outcomes
KW  - quality of life
UR  - https://journals.sagepub.com/doi/epdf/10.1177/20406207241285143
N2  - 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.
ER  -

BLATNÝ, Jan; Jan ASTERMARK; Cristina CATARINO; Gerry DOLAN; Karin FIJNVANDRAAT; Cedric HERMANS; Katharina HOLSTEIN; Victor JIMENEZ-YUSTE; Robert KLAMROTH; Michelle LAVIN; Peter J LENTING; Sebastien LOBET; Maria Elisa MANCUSO; Jayashree MOTWANI; James S O'DONNELL a Christoph KOENIGS. Future needs for continuing innovation in hemophilia: improving outcomes for individuals of all severities, including women and those in resource-constrained regions. \textit{THERAPEUTIC ADVANCES IN HEMATOLOGY}. London: SAGE PUBLICATIONS LTD, 2024, roč.~15, č.~2024, s.~1-8. ISSN~2040-6207. Dostupné z: https://doi.org/10.1177/20406207241285143.

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