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. 2020 Dec;18(12):3203-3210.
doi: 10.1111/jth.15076. Epub 2020 Sep 28.

The factor VIII treatment history of non-severe hemophilia A

Amal Abdi  1 , Fabienne R Kloosterman  1 , Corien L Eckhardt  1 , Christoph Male  2 , Giancarlo Castaman  3 , Kathelijn Fischer  4 , Erik A M Beckers  5 , Marieke J H A Kruip  6 , Kathelijne Peerlinck  7 , Maria Elisa Mancuso  8 , Cristina Santoro  9 , Charles R Hay  10 , Helen Platokouki  11 , Johanna G van der Bom  12   13 , Samantha C Gouw  1   12 , Karin Fijnvandraat  1   14 , Dan P Hart  15 ; INSIGHT Study Group
Affiliations

The factor VIII treatment history of non-severe hemophilia A

Amal Abdi et al. J Thromb Haemost. 2020 Dec.

Abstract

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.

Keywords: factor VIII; hemophilia A; hemorrhage; joint bleed; treatment.

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Conflict of interest statement

FK, AA, CE, EB, KP, CH, HP, JB, SG have nothing to disclose. CM has received research grant awards from Biotest and CSL Behring. He and/or his institution has received advisory of lecturing honoraria from Bayer, Biotest, CSL Behring, Novo Nordisk, Roche. GC received fees to act as a speaker at or to participate in Advisory Board meetings from Ablynx, Bayer, CSL Behring, Kedrion, Novo Nordisk, Shire/Takeda, Sobi, Roche, Uniqure, and Werfen. GC received unrestricted research grants from CSL Bhering, Pfizer, and Sobi. K. Fischer has received speaker's fees from Bayer, Baxter/Shire, Sobi/Biogen, CSL Behring, Octapharma, Pfizer, NovoNordisk, and has performed consultancy for Bayer, Baxter, Biogen, CSL‐Behring,Freeline, Novo Nordisk, Pfizer, Roche, and Sobi. She and/or her institution has received research support from Bayer, Pfizer, Baxter/Shire, and Novo Nordisk. MK received unrestricted research grants from ZonMW, Innovatiefonds, Bayer, Boehringer‐Ingelheim, Pfizer, Daiichi‐Sankyo for research outside of this work. MM received fees to act as an advisor/consultant/speaker for Bayer, CSL Behring, Novo Nordisk, Pfizer, Roche, Sobi, Bioverativ, Shire/Takeda, Octapharma, Kedrion, Grifols, Biotest, and Catalyst. CS has received consultancy or speaker fees by Bayer, CSL Behring, Shire/Takeda, Novo Nordisk, Sobi, Novartis, Pfizer, Amgen, and Roche. The institution of K. Fijnvandraat has received unrestricted research grants from CSL Behring, Novo Nordisk, and received consultancy fees from Grifols, Takeda, and Novo Nordisk. DPH has received research grant awards from Bayer, Octapharma, and Takeda. He and/or his institution have received advisory or lecturing honoraria from Bayer, Biomarin, Biotest, Grifols, Octapharma, Pfizer, Roche, Sanofi, Sobi, Takeda, UniQure.

Figures

Figure 1
Figure 1
Flowchart of patients included in the study
Figure 2
Figure 2
Age at first exposure to factor VIII (FVIII) treatment stratified for FVIII baseline activity in categories
See this image and copyright information in PMC

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