PB0525 - Real-world Bleeding Rates on Emicizumab using Digital Treatment Diary data

Monday, June 24, 2024

13:45 – 14:45 ICT

Room: Exhibition Hall

Presenting Author(s)

Martijn R. Brands, MD

Physician-researcher
Emma Children’s Hospital, Amsterdam UMC location AMC, University of Amsterdam, Amsterdam, the Netherlands
Amsterdam, Noord-Holland, Netherlands

Co-Author(s)

LT

Liesbeth Taal

Data manager
Department of Clinical Epidemiology, Leiden University Medical Center, Leiden, the Netherlands
Leiden, Zuid-Holland, Netherlands
MD

Mariëtte H.E. Driessens

Patient representative
Netherlands Hemophilia Patient Society (NVHP), Nijkerk, the Netherlands
Nijkerk, Gelderland, Netherlands
Cv

Caroline M.E. van Veen

Project manager
HemoNED Foundation, Leiden, the Netherlands
Leiden, Zuid-Holland, Netherlands
MK

Marieke J.H.A. Kruip

Hematologist
Department of Hematology, Erasmus MC, Erasmus University Medical Centre Rotterdam, Rotterdam, the Netherlands
Rotterdam, Zuid-Holland, Netherlands
Pd

Paul L. den Exter

Hematologist
Department of Thrombosis and Hemostasis, Leiden University Medical Center, Leiden, the Netherlands
Leiden, Zuid-Holland, Netherlands
BL

Britta A.P. Laros-van Gorkom

Hematologist
Department of Hematology, Radboud University Medical Center, Nijmegen, the Netherlands
Nijmegen, Gelderland, Netherlands
MS

Marjet A. Stein-Wit

Pediatric hematologist
Department of Pediatrics, University Medical Center Groningen, Groningen, the Netherlands
Groningen, Groningen, Netherlands
Kathelijn Fischer

Pediatric Hematologist
Centre for Benign Haematology, Thrombosis and Haemostasis, Van Creveldkliniek, University Medical Centre Utrecht, Utrecht University, Utrecht, The Netherlands
Utrecht, Utrecht, Netherlands
SM

Stephan Meijer

Patient representative
Netherlands Hemophilia Patient Society (NVHP), Nijkerk, the Netherlands
Nijkerk, Gelderland, Netherlands
Karina Meijer

MD, PhD, professor
Department of Hematology, University Medical Center Groningen, University of Groningen, Groningen, The Netherlands
Groningen, Groningen, Netherlands
Marlene Beijlevelt, MSc

Advanced Nurse Practitioner
Amsterdam UMC
Amsterdam, Noord-Holland, Netherlands
KF

Karin Fijnvandraat

Professor
Emma Kinderziekenhuis/AMC, University of Amsterdam, Netherlands
Amsterdam, Noord-Holland, Netherlands

Co-Author(s)

SG

Samantha C. Gouw, MD PhD

Pediatric hematologist
Department of Pediatric Hematology, Emma Children’s Hospital, Amsterdam UMC location University of Amsterdam, Amsterdam, the Netherlands
Amsterdam, Noord-Holland, Netherlands

Background: People with congenital bleeding disorders in the Netherlands use a digital treatment diary to log bleeds and prophylactic and therapeutic infusions. Although many people with severe hemophilia A currently use emicizumab, its efficacy has not yet been evaluated in a real-world setting in the Netherlands using nationwide treatment diary data.

Aims: Assess real-world bleeding rates on emicizumab in an unselected cohort and assess the reliability of treatment diary data for clinical research.

Methods: Dutch people with severe hemophilia A of all ages using emicizumab who have used the treatment diary after the start of emicizumab were eligible. Data obtained between 2019 and October 2023 were analyzed. Bleeds treated with FVIII concentrate (documented in treatment diaries and/or electronic health records (EHRs)) were included. Annual bleeding rates (ABR), annual joint bleeding rates (AJBR) and proportions of people with zero treated bleeds were assessed. Treatment diary data were verified with EHR data.

Results: In total, 232 persons were included. Their median age was 29 years (interquartile range (IQR) 15-53). The median duration of emicizumab use was 27 months (IQR 15-31). Median (IQR) real-world treated ABR and AJBR were 0.6 (0.0-1.4) and 0.0 (0.0-0.7) respectively. At 24 weeks follow-up, 64% (149/232) had zero treated bleeds and 81% (187/232) had zero treated joint bleeds. At 1 year, this was 49% (113/232) and 72% (167/232) respectively.
Of 456 bleeds, 42% (192/456) were documented in both treatment diaries and EHRs, 23% (104/456) only in treatment diaries, and 35% (160/456) only in EHRs. Of 352 EHR-documented bleeds, 55% (192/352) were reported in treatment diaries.

Conclusion(s): Real-world bleeding rates in Dutch people with severe hemophilia A using emicizumab, determined using routinely collected digital treatment diary and EHR data, were comparable to other real-world studies. Here we aim to showcase the value and limitations of the treatment diary to evaluate treatment efficacy.