Paroxysmal nocturnal hemoglobinuria: Review of the patient experience and treatment landscape

IF 6.9 2区 医学 Q1 HEMATOLOGY
Anem Waheed , Jamile Shammo , David Dingli
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引用次数: 0

Abstract

Paroxysmal nocturnal hemoglobinuria (PNH) is a rare disorder caused by complement-mediated hemolysis and thrombosis through the alternative pathway. The most common symptom of PNH is fatigue due to chronic anemia, which can negatively impact quality of life (QoL) and affect overall well-being. The currently approved therapies for PNH significantly limit intravascular hemolysis (IVH) and reduce the risk of thrombosis; however, they are associated with an infusion schedule that can become burdensome, and not all patients experience complete disease control. Several new complement inhibitors are in development that address the need for convenient routes of administration and aim to provide better disease control. With the variety of new treatment options on the horizon, hematologic markers as well as QoL concerns, patient opinion, and lifestyle factors should be considered to choose the optimal PNH treatment for each specific patient.

阵发性夜间血红蛋白尿:回顾患者的经验和治疗前景
阵发性夜间血红蛋白尿(PNH)是一种罕见的疾病,由补体介导的溶血和血栓通过替代途径引起。PNH最常见的症状是慢性贫血引起的疲劳,这会对生活质量(QoL)产生负面影响,影响整体健康。目前批准的PNH治疗可显著限制血管内溶血(IVH)并降低血栓形成风险,然而,它们与输注计划相关,可能变得负担沉重,并且并非所有患者都能完全控制疾病。几种新的补体抑制剂正在开发中,以满足方便给药途径的需要,并旨在提供更好的疾病控制。随着各种新的治疗方案的出现,血液学指标、生活质量、患者意见和生活方式因素应被考虑,以选择最佳的PNH治疗每个特定的患者。
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来源期刊
Blood Reviews
Blood Reviews 医学-血液学
CiteScore
13.80
自引率
1.40%
发文量
78
期刊介绍: Blood Reviews, a highly regarded international journal, serves as a vital information hub, offering comprehensive evaluations of clinical practices and research insights from esteemed experts. Specially commissioned, peer-reviewed articles authored by leading researchers and practitioners ensure extensive global coverage across all sub-specialties of hematology.
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