OC 43.4 - Effectiveness of emicizumab under real-world conditions in patients of all ages with hemophilia A with and without FVIII inhibitors: interim analysis of the non-interventional study EMIIL

Monday, June 26, 2023

15:30 – 15:45 ET

Room: 710B

Session Available As: On-Demand

Presenting Author(s)

Johannes Oldenburg, MD (he/him/his)

Professor, Chairman and Director
University Clinic Bonn AöR, Institute of Experimental Haematology and Transfusion Medicine, Bonn, Germany
Bonn, Nordrhein-Westfalen, Germany

Co-Author(s)

Carmen Escuriola-Ettingshausen, MD

Physician
Haemophilia Centre Rhine-Main, Mörfelden-Walldorf, Germany
Mörfelden-Walldorf, Hessen, Germany
Pierre Fontana, MD PhD (he/him/his)

Head of Geneva Platelet Group
Division of Angiology and Haemostasis, University Hospitals of Geneva, Switzerland
Geneva, Geneve, Switzerland
SW

Swee Wenning

Physician
SRH Kurpfalzkrankenhaus, Heidelberg, Germany
Heidelberg, Baden-Wurttemberg, Germany
CP

Christian Pfrepper, MD

Physician
Division of Hemostaseology, University Hospital Leipzig, Germany
Leipzig, Sachsen, Germany
BH

Bianca Heidersdorf

Medical Manager Hemophilia
Roche Pharma AG, Grenzach-Wyhlen, Germany
Grenzach-Wyhlen, Baden-Wurttemberg, Germany
PU

Patrick Udvardi

Medical Science Liaison
Roche Pharma Schweiz AG, Basel, Switzerland
Basel, Basel-Stadt, Switzerland
MA

Manuela Albisetti

Director
University Children’s Hospital Zurich, Zurich, Switzerland
Zürich, Zurich, Switzerland

Background: Emicizumab is a monoclonal, humanized bispecific antibody bridging coagulation factors IXa and X and thereby replacing the coagulation function of activated factor VIII (FVIII) even in the presence of FVIII inhibitors. Emicizumab is approved in the EU for routine prophylaxis in adult and pediatric patients with hemophilia A (PwHA) with FVIII inhibitors and severe hemophilia A without FVIII inhibitors. In clinical trial settings, subcutaneous administration of emicizumab has demonstrated a positive benefit/risk profile. However, real-world data on long-term effectiveness is limited.

Aims: To better understand the long-term effectiveness of emicizumab prophylaxis in PwHA with and without FVIII inhibitors under real-world conditions.

Methods: EMIIL (ISRCTN58752772) is an ongoing 5-year single-arm, two cohorts, prospective, multicenter non-interventional study in Germany and Switzerland collecting primary observational data in PwHA newly treated with emicizumab. Based on the presence of FVIII inhibitors, patients are enrolled in one of two cohorts (Figure 1). The primary endpoint is the annualized bleeding rate (ABR) of treated bleeds, estimated using a generalized linear model (GLM). Here we report results from the second interim analysis.

Results: At data cut-off (09 May 2022), a total of 85 patients (mean [SD] age 26.7 [21.3] years) were available for evaluation. Of those, 35.3% were children ( < 12 years), 4.7% adolescents, 56.5% adults < 65 years, and 3.5% ≥65 years. After a median treatment duration of 419 days (range 15 - 873), 71.8% of patients had zero treated bleeds and 87.1% had zero treated spontaneous bleeds. Additional analyses of bleeding incidences in weekly intervals and by age group will be presented. The GLM-based ABR was 0.636 (95% CI 0.088-4.621). No patient developed FVIII inhibitors.

Conclusion(s): The initial results on the effectiveness of emicizumab in a real-world setting, particularly the results on ABR and the proportion of patients with zero bleeds support findings from previous clinical trials.

Learning Objectives:

Describe the indication and use of subcutaneous treatment with emicizumab
Interpret the beneficial effect of emicizumab on bleeding endpoints in a real-world setting
Recognize the consistent efficacy across all bleeding endpoints and the favorable safety profile of emicizumab treatment in patients with severe hemophilia A