PB1130 - Real-world evidence on emicizumab in 119 Hemophilia A patients with inhibitors from the French FranceCoag cohort

Tuesday, July 12, 2022

6:30 PM – 7:30 PM

Location: Exhibition

Presentation Format: In-Person

Co-Author(s)

AH

Annie Harroche

Pediatric Hematologist
CRC-MHC Hôpital Necker-Enfants malades, Paris, France
Paris, Ile-de-France, France
YR

Yohann Repesse

Hematologist
Department of Hemostasis, University Hospital of Caen
Caen, Basse-Normandie, France
AR

Antoine Rauch, MD, PhD

University of Lille, Inserm, CHU Lille, Institut Pasteur de Lille, U1011-EGID, France
Lille, Nord-Pas-de-Calais, France
FH

FATIHA HADIM

MEDICAL LEAD ROCHE , MD
ROCHE FRANCE
Boulogne Billancourt, Ile-de-France, France
AG

Aurélie Gaude

Medical Manager Hemophilia, PhD
Chugai France
La défense, Ile-de-France, France
DP

David Pau

BioStatistics and Data Science
Roche S.A.S
Boulgone Billancourt, Ile-de-France, France
SP

Sophie Pibre

Medical Evidence Strategy Lead
Roche S.A.S
Boulgone Billancourt, Ile-de-France, France

Presenting Author(s)

RD

Roseline D'Oiron

Hematologist
Haemophilia Treatment Centre, Bicêtre Hospital, University Hospital of Paris (APHP), Kremlin-Bicêtre, France.
paris, Centre, France

Background: Emicizumab has demonstrated significant reductions in bleeding episodes in the HAVEN program for Hemophilia A with inhibitors patients (HAwI). However, limited data are available in a real-life setting.

Aims: This study aimed to describe treated bleeds during emicizumab treatment in HAwI, patient and disease characteristics, and emicizumab tolerability.

Methods: In this non-interventional secondary data use study, data of HAwI having initiated emicizumab in France from June 2017 to December 2020 were extracted from the national FranceCoag database in October 2021. The primary endpoint was the treated bleeds' rate under emicizumab. Descriptive analysis was performed. Treated Bleeding-Free Survival (TBFS) was described using Kaplan-Meier method.

Results: Among a total of 119 eligible patients analyzed, 112 (94.1%) were severe, 5 (4.2%) moderate, and 2 (1.7%) minor HAwI. Patient age distribution at emicizumab initiation was < 2 yo(10.9%)/ [2-18[(33.6%)/ [18-65[(50.4%)/ ≥65 yo(5.0%); their median historical peak of inhibitor titer was 301 UB/mL (IQR 48-1,780) and inhibitor peak was 2.6 (0.5-10.0) over the 3 months before emicizumab initiation.
102 patients with ≥6 months treatment duration were retained for effectiveness analysis. Over follow-up (median: 96.5 weeks, IQR 67.9-131.7), patients received emicizumab during 89.1 weeks (IQR 54.2-127.1). Under emicizumab, 78 patients (76.5%) experienced no bleeds; 24 (23.5%, 95%CI:[15.7%-32.9%]) had 37 bleeds (3 patients presented bleed(s) during the loading dose), including 17 patients with 1 event and 7 patients with 2 or more events.
16 joint bleeds occurred in 12 patients (11.8%, 95%CI:[6.2%-19.7%]), 2 cerebral hemorrhages in 1 (1.0%, 95%CI:[0.02%-5.34%]) and/or 1 life-threatening retroperitoneal hemorrhage in 1 (1.0%, 95%CI:[0.02%-5.34%]).
The overall mean annual treated bleeds’ rate under emicizumab was 0.23± 0.79 (joint bleeds 0.16±0.46). TBFS at 26, 52 and 104 weeks was 95%, 84% and 73% respectively.
No safety signal was identified.

Conclusion(s): Using FranceCoag prospective cohort, this real-world study allows updating emicizumab knowledge in a large and non-selected HAwI population.