Veeranki Sreenivas P, Pednekar Priti, Graf Marlon, Tuly Rifat, Recht Michael, Batt Katharine
PRECISIONheor, Los Angeles, CA, USA.
American Thrombosis and Hemostasis Network, Rochester, NY, USA.
J Blood Med. 2021 Oct 21;12:913-928. doi: 10.2147/JBM.S334852. eCollection 2021.
Over the past decade, there has been an increase in novel therapeutic options to treat hemophilia A. It is still unclear how these novel treatments are used in the management of patients with hemophilia A, particularly those with challenging clinical scenarios who are typically excluded in clinical trials.
This study aimed to understand the areas of consensus and disagreement among hematologists regarding the preferences toward therapeutic approaches for difficult-to-treat patients with severe hemophilia A without inhibitors.
During February-June 2020, a three-round modified Delphi study was conducted to generate consensus among 13 US experts in the field of hemophilia. Experts were asked about their preferences toward therapeutic options for patients with challenging clinical situations, including age-related morbidities (eg, myocardial infarction, joint arthropathy), increasing demand for high-impact physical activities, early onset osteoporosis, and newborns with hemophilia A. Consensus was defined as ≥75% agreement between the panelists.
Consensus was reached on many, but not all cases, leaving uncertainty about appropriateness of therapeutic approaches for some patients where clinical evidence is not available or driven by physicians' or patients' preferences toward therapeutic options. A majority of panelists preferred FVIII replacement therapy rather than emicizumab prophylaxis for the challenging cases presented due to established evidence on safety, efficacy, and level of bleed protection for FVIII treatment.
Recommendations emerging from this study may help guide practicing hematologists in the management of challenging hemophilia A cases. Future studies are needed to address treatment options in the clinical cases where no consensus was reached.
在过去十年中，治疗甲型血友病的新型治疗选择有所增加。目前仍不清楚这些新型治疗方法在甲型血友病患者管理中是如何使用的，特别是那些具有挑战性临床情况的患者，他们通常被排除在临床试验之外。
本研究旨在了解血液科医生在治疗无抑制剂的重度甲型血友病难治性患者的治疗方法偏好方面的共识和分歧领域。
在2020年2月至6月期间，进行了三轮改良德尔菲研究，以在13名美国血友病领域专家中达成共识。专家们被问及他们对具有挑战性临床情况患者的治疗选择偏好，包括与年龄相关的疾病（如心肌梗死、关节病）、对高强度体育活动需求增加、早发性骨质疏松症以及甲型血友病新生儿。共识定义为小组成员之间≥75%的一致意见。
在许多但并非所有情况下都达成了共识，对于一些缺乏临床证据或由医生或患者对治疗选择的偏好驱动的患者，治疗方法的适用性仍存在不确定性。由于FVIII治疗在安全性、有效性和出血保护水平方面有既定证据，大多数小组成员在出现的具有挑战性病例中更喜欢FVIII替代疗法而不是emicizumab预防。
本研究得出的建议可能有助于指导执业血液科医生管理具有挑战性的甲型血友病病例。未来需要开展研究以解决未达成共识的临床病例中的治疗选择问题。
