CC BY 4.0 · TH Open 2017; 01(02): e130-e138
DOI: 10.1055/s-0037-1608943
Original Article
Georg Thieme Verlag KG Stuttgart · New York

Real-World Early Treatment with Room Temperature–Stable Recombinant Factor VIIa in Hemophilia A/B and Inhibitors: SMART-7™ Post Hoc Analyses

Francesco Demartis
1  Agenzia per l'Emofilia, Centro Malattie Emorragiche, Azienda Ospedaliero Universitaria Careggi, Firenze, Italy
Angelika Batorova
2  National Hemophilia Centre, Department of Hematology and Transfusion Medicine, Medical School of Comenius University, University Hospital, Bratislava, Slovakia
Hervé Chambost
3  Service d'Hématologie Oncologie Pédiatrique, La Timone, APHM, and Inserm, UMR 1062, Faculté de Médecine, Aix-Marseille Université, Marseille, France
Peyman Eshghi
4  Pediatric Congenital Hematologic Disorders Research Center, Shahid Beheshti University of Medical Sciences, Tehran, Iran
Mehran Karimi
5  Hematology Research Center, Shiraz University of Medical Sciences, Shiraz, Iran
Kaan Kavakli
6  Department of Hematology, Ege University Children's Hospital, Izmir, Turkey
Soraya Benchikh El Fegoun
7  Biopharm Operations, Novo Nordisk Healthcare AG, Zurich, Switzerland
Katarina Cepo
8  Medical & Science Biopharm, Novo Nordisk A/S, Søborg, Denmark
Lene Sommer Vestergaard
9  Biostatistics LCM Haem, GH & Concizumab, Novo Nordisk A/S, Søborg, Denmark
Gary Benson
10  Northern Ireland Haemophilia Comprehensive Care Centre, Belfast City Hospital, Belfast, United Kingdom
› Institutsangaben
Weitere Informationen


31. August 2017

02. November 2017

08. Dezember 2017 (online)


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.

Authors' Contribution

F.D., A.B., H.C., P.E., M.K., K.K., and G.B. performed the research. S.B.E.F., K.C., and L.S.V. analyzed the data. All authors had full access to the data, contributed to writing the manuscript, and approved the final version for submission.


SMART-7™ was a post-authorization safety study funded by Novo Nordisk A/S, Denmark.