血友病领域持续创新的未来需求:改善各种严重程度的患者(包括女性和资源有限地区的患者)的治疗效果。

IF 3.4 3区 医学 Q2 HEMATOLOGY Therapeutic Advances in Hematology Pub Date : 2024-09-30 eCollection Date: 2024-01-01 DOI:10.1177/20406207241285143
Jan Blatný, Jan Astermark, Cristina Catarino, Gerry Dolan, Karin Fijnvandraat, Cédric Hermans, Katharina Holstein, Víctor Jiménez-Yuste, Robert Klamroth, Michelle Lavin, Peter J Lenting, Sébastien Lobet, Maria Elisa Mancuso, Jayashree Motwani, James S O'Donnell, Christoph Königs
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引用次数: 0

摘要

近几十年来,血友病(PwH)患者的治疗因科学进步而得到了极大的改善,治疗手段丰富多样。尽管如此,治疗上的局限性仍在不断推动创新,而新出现的治疗方案也有可能实现进一步的改善。我们主张采用四项一般原则来优化创新带来的益处:个性化治疗方法、以 "正常 "为目标、充分利用现有资源以及考虑治疗的可负担性。归根结底,全世界所有的男性和女性、所有年龄段和严重程度的男性和女性都应该能够获得既能充分预防出血,又能让个人、社会、家庭和职业生活有所选择的治疗。显然,我们还没有做到这一点,但根据我们所描述的原则制定目标/里程碑可能有助于实现这一目标。
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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.

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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来源期刊
CiteScore
4.30
自引率
0.00%
发文量
54
审稿时长
7 weeks
期刊介绍: Therapeutic Advances in Hematology delivers the highest quality peer-reviewed articles, reviews, and scholarly comment on pioneering efforts and innovative studies across all areas of hematology. The journal has a strong clinical and pharmacological focus and is aimed at clinicians and researchers in hematology, providing a forum in print and online for publishing the highest quality articles in this area.
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