Long-term prophylaxis: what are our options and how to define success?

IF 2.9 3区 教育学 Q1 EDUCATION, SCIENTIFIC DISCIPLINES Hematology. American Society of Hematology. Education Program Pub Date : 2022-12-09 DOI:10.1182/hematology.2022000397
Marilyn Jean Manco-Johnson, Beth Boulden Warren
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引用次数: 2

Abstract

Currently, we are at an enviable place in hemophilia treatment. Although full prophylaxis with standard half-life recombinant or plasma-derived factor concentrates has been definitively shown to be inadequate for full protection against bleeding and arthropathy, a number of novel therapies with improved hemostatic enhancement are clinically available or in promising clinical trials. In order to compare outcomes among a number of very efficacious therapies, it is necessary to have sensitive tools employed in long-term follow-up for several years for participants with no or minimal joint disease. The tool kit must be comprehensive, with outcomes of bleeding, factor level restoration or hemostatic capacity, joint structure, joint function, pain, quality of life, and patient satisfaction. This article reviews the history of prophylaxis, the promise of emerging therapies, and the sensitive tools used to assess long-term efficacy for joint structure and function.

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长期预防:我们的选择是什么?如何定义成功?
目前,我们在血友病治疗方面处于令人羡慕的地位。虽然标准半衰期重组或血浆衍生因子浓缩物的全面预防已被明确证明不足以完全保护出血和关节病,但许多改善止血增强的新疗法已在临床上可用或在有希望的临床试验中。为了比较一些非常有效的治疗方法的结果,有必要对没有或只有轻微关节疾病的参与者进行数年的长期随访,使用敏感的工具。整套工具必须是全面的,包括出血、因子水平恢复或止血能力、关节结构、关节功能、疼痛、生活质量和患者满意度。本文回顾了预防的历史,新兴疗法的前景,以及用于评估关节结构和功能长期疗效的敏感工具。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
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来源期刊
Hematology. American Society of Hematology. Education Program
Hematology. American Society of Hematology. Education Program EDUCATION, SCIENTIFIC DISCIPLINES-HEMATOLOGY
CiteScore
4.70
自引率
3.30%
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0
期刊介绍: Hematology, the ASH Education Program, is published annually by the American Society of Hematology (ASH) in one volume per year.
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