The factor VIII treatment history of non-severe hemophilia A

Amal Abdi, Fabienne R. Kloosterman, Corien L. Eckhardt, Christoph Male, Giancarlo Castaman, Kathelijn Fischer, Erik A. M. Beckers, Marieke J. H. A. Kruip, Kathelijne Peerlinck, Maria Elisa Mancuso, Cristina Santoro, Charles R. Hay, Helen Platokouki, Johanna G. van Der Bom, Samantha C. Gouw, Karin Fijnvandraat*, Dan P. Hart, INSIGHT Study Group

*Corresponding author for this work

Research output: Contribution to journalArticleAcademicpeer-review


Background In patients with non-severe hemophilia A, we lack detailed knowledge on the timing of treatment with factor VIII (FVIII) concentrates. This knowledge could provide information about the expected treatment timing in patients with severe hemophilia A treated with non-replacement therapies. Objective To assess the FVIII treatment history in patients with non-severe hemophilia A. Methods Patients with non-severe hemophilia (baseline FVIII activity [FVIII:C] 2-40 IU/dL) were included from the INSIGHT study. The primary outcome was median age at first FVIII exposure (ED1). In a subgroup of patients for whom more detailed information was available, we analyzed the secondary outcomes: median age at first 20 EDs, annualized bleeding rate for all bleeds (ABR), joint bleeds (AJBR), and major spontaneous bleeds (ASmBR). Results In the total cohort (n = 1013), median baseline FVIII activity was 8 IU/dL (interquartile range [IQR] 4-15) and the median age at ED1 was 3.7 years (IQR 1.4-7.7). Median age at ED1 rose from 2.5 years (IQR 1.2-5.7) in patients with FVIII:C 2-5 IU/dL to 9.7 years (IQR 4.8-16.0) in patients with FVIII:C 25-40 IU/dL. In the subgroup (n = 104), median age at ED1, ED5, ED10, and ED20 was 4.0 years (IQR 1.4-7.6), 5.6 years (IQR 2.9-9.3), 7.5 years (IQR 4.4-11.3), and 10.2 years (IQR 6.5-14.2), respectively. Median ABR, AJBR, and ASmBR were 1.1 (IQR 0.5-2.6), 0.3 (IQR 0.1-0.7), and 0 (IQR 0-0), respectively. Conclusion This study demonstrates that in non-severe hemophilia A, the age at first FVIII exposure increases with baseline FVIII:C and that major spontaneous bleeds rarely occur.

Original languageEnglish
Pages (from-to)3203-3210
Number of pages8
JournalJournal of Thrombosis and Haemostasis
Issue number12
Early online date28 Sept 2020
Publication statusPublished - Dec 2020


  • factor VIII
  • hemophilia A
  • hemorrhage
  • joint bleed
  • treatment
  • RISK
  • IX


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