Demartis Francesco, Batorova Angelika, Chambost Hervé, Eshghi Peyman, Karimi Mehran, Kavakli Kaan, El Fegoun Soraya Benchikh, Cepo Katarina, Vestergaard Lene Sommer, Benson Gary
Agenzia per l'Emofilia, Centro Malattie Emorragiche, Azienda Ospedaliero Universitaria Careggi, Firenze, Italy.
National Hemophilia Centre, Department of Hematology and Transfusion Medicine, Medical School of Comenius University, University Hospital, Bratislava, Slovakia.
TH Open. 2017 Dec 8;1(2):e130-e138. doi: 10.1055/s-0037-1608943. eCollection 2017 Jul.
Treating hemophilia A or B patients with inhibitors is particularly challenging, as they do not respond to replacement therapy with factor VIII or factor IX concentrates. A room temperature-stable formulation of recombinant activated factor VII (rFVIIa; NovoSeven ), which provides improved convenience and treatment access to patients compared with the earlier formulation of rFVIIa, was shown to be safe and effective in a post-authorization, multinational, observational study (Study Monitoring Antibodies against Room Temperature-stable factor 7 [SMART-7™]). In post hoc, subgroup analyses of SMART-7™ data, the hemostatic response following rFVIIa monotherapy in patients with hemophilia A or B with inhibitors by time to first treatment and in different age cohorts was assessed. A total of 482/618 bleeding episodes treated with rFVIIa monotherapy and with (1) valid efficacy assessment, (2) no missing time for bleed start, (3) no missing time for any dose administration, and (4) valid time to first treatment were included in the analyses. Data on the type and location of bleeding episodes treated with rFVIIa monotherapy were also collected. The majority of bleeding episodes treated with rFVIIa monotherapy were treated within 1 hour after bleeding onset (318/482 [66%]) and, among them, 96.5% (307/318) were effectively treated (i.e., bleeding stopped). Hemostatic efficacy remained high for bleeding episodes treated >1 to ≤4 hours after the onset, with 94/101 (93.1%) treated effectively. Cause and location of bleeding varied across the different age groups assessed. Real-world evidence from post hoc, subgroup analyses of SMART-7™ data confirmed that patients were able to treat themselves quickly and that early treatment with rFVIIa was associated with high efficacy.
治疗患有抑制物的A型或B型血友病患者极具挑战性，因为他们对使用凝血因子VIII或凝血因子IX浓缩物的替代疗法无反应。与早期的重组活化凝血因子VII（rFVIIa；诺其 ）制剂相比，一种室温稳定的rFVIIa制剂为患者提供了更高的便利性和治疗可及性，在一项批准后开展的多国观察性研究（抗室温稳定因子7抗体研究监测[SMART-7™]）中显示安全有效。在对SMART-7™数据进行事后亚组分析时，评估了患有抑制物的A型或B型血友病患者接受rFVIIa单药治疗后首次治疗时间以及不同年龄队列的止血反应。共有482/618例接受rFVIIa单药治疗的出血事件纳入分析，这些事件满足以下条件：(1) 有效疗效评估；(2) 出血开始时间无缺失；(3) 任何剂量给药时间无缺失；(4) 首次治疗时间有效。还收集了接受rFVIIa单药治疗的出血事件的类型和部位数据。接受rFVIIa单药治疗的出血事件大多在出血发作后1小时内得到治疗（318/482 [66%]），其中96.5%（307/318）得到有效治疗（即出血停止）。对于出血发作后>1至≤4小时接受治疗的出血事件，止血疗效仍然很高，94/101（93.1%）得到有效治疗。在评估的不同年龄组中，出血的原因和部位各不相同。对SMART-™数据进行事后亚组分析得出的真实世界证据证实，患者能够快速自我治疗，且早期使用rFVIIa治疗疗效高。
