Division of Hematology and Hematological Malignancies, Department of Medicine, University of Utah, Salt Lake City, Utah.
Division of Hematology, Department of Medicine, University of Colorado, Aurora, Colorado, USA.
Curr Opin Hematol. 2020 Sep;27(5):295-301. doi: 10.1097/MOH.0000000000000600.
Nonsevere hemophilia A (NSHA) patients have received relatively little clinical and research attention as compared with their severe counterparts. There is increasing recognition that despite their milder bleeding phenotype, the management of NSHA can be a challenge, with most management decisions largely inferred from severe hemophilia A data. This review focuses on some of the more recent developments in the field of NSHA.
Epidemiologic studies suggest that NSHA remain under-recognized and under-diagnosed globally. As the NSHA population ages, they are susceptible to age-related comorbidities. Large cohort studies of NSHA report that the most common primary cause of death is malignancy. NSHA patients have a lifetime risk of inhibitor development with increasing exposure to factor VIII concentrate. Even so, not all patients with inhibitors will require eradication treatment, irrespective of bleeding phenotype at time of inhibitor development.
As there are currently no evidence-based strategies for inhibitor eradication in NSHA patients, preventive strategies are critical to mitigate inhibitor risk in NSHA. There is a need for active surveillance of NSHA patients by hemophilia treatment centers to address hemophilia-related issues and other age-related comorbidities, in collaboration with primary care physicians and other subspecialists.
与重度血友病 A 患者相比，非重度血友病 A（NSHA）患者得到的临床和研究关注相对较少。越来越多的人认识到，尽管他们的出血表型较轻，但 NSHA 的管理可能是一个挑战，大多数管理决策主要是从重度血友病 A 数据中推断出来的。本综述重点介绍了 NSHA 领域的一些最新进展。
流行病学研究表明，全球范围内 NSHA 仍然未被充分认识和诊断。随着 NSHA 人群年龄的增长，他们易患与年龄相关的合并症。对 NSHA 的大型队列研究报告称，最常见的主要死亡原因是恶性肿瘤。随着因子 VIII 浓缩物暴露量的增加，NSHA 患者终生存在产生抑制剂的风险。即便如此，并非所有有抑制剂的患者都需要进行清除治疗，无论在抑制剂出现时的出血表型如何。
由于目前在 NSHA 患者中没有针对抑制剂清除的循证策略，因此预防策略对于减轻 NSHA 中的抑制剂风险至关重要。有必要由血友病治疗中心对 NSHA 患者进行主动监测，以解决血友病相关问题和其他与年龄相关的合并症，并与初级保健医生和其他亚专科医生合作。
