PB0698 - Determinants of desmopressin response in bleeding disorders: a systematic review and meta-analysis

Monday, June 26, 2023

18:30 – 19:30 ET

Room: Exhibition

Presenting Author(s)

Sebastiaan N.J Laan, MSc

PhD candidate
Department of Internal Medicine, Division of Thrombosis and Hemostasis, Einthoven Laboratory for Vascular and Regenerative Medicine, Leiden University Medical Center, Leiden, The Netherlands
Leiden, Zuid-Holland, Netherlands

Co-Author(s)

Jessica Del Castillo Alferez, Msc (she/her/hers)

PhD student
Department of Molecular Hematology, Sanquin Research, Amsterdam, The Netherlands.
Amsterdam, Noord-Holland, Netherlands
SC

Suzanne C. Cannegieter, MD PhD

Professor
Department of Clinical Epidemiology, Leiden University Medical Center
Leiden, Zuid-Holland, Netherlands
KF

Karin Fijnvandraat

Professor
Amsterdam UMC, University of Amsterdam, Emma Children’s Hospital, Pediatric Hematology Amsterdam, Netherlands
Amsterdam, Noord-Holland, Netherlands
MK

Marieke J.H.A. Kruip

Associate professor, hematologist, MD, PhD
Department of Hematology, Erasmus MC, Erasmus University Medical Center Rotterdam, Rotterdam, The Netherlands
Rotterdam, Zuid-Holland, Netherlands
Sl

Saskia le Cessie

professor
Department of Clinical Epidemiology – LUMC – Netherlands and Department of biomedical data sciences – LUMC - Netherlands
Leiden, Zuid-Holland, Netherlands
Ruben Bierings, PhD

Principal investigator
Department of Hematology, Erasmus MC, University Medical Center Rotterdam, the Netherlands
Rotterdam, Zuid-Holland, Netherlands
Jeroen C.J Eikenboom, MD. PhD

Professor, Head of Division of Thrombosis and Hemostasis
Leiden University Medical Center
Leiden, Zuid-Holland, Netherlands
Iris van Moort, PhD

Post Doc.
Department of Hematology – Erasmus MC, Erasmus University Medical Center - Netherlands
Rotterdam, Zuid-Holland, Netherlands

Background: Several bleeding disorders can be treated with desmopressin to prevent or stop bleeding. However, large inter-individual variability is observed in desmopressin response, and the determinants of response are largely unknown.

Aims: In this systematic review we aim to identify the factors that determine desmopressin response in patients.

Methods: A comprehensive search was performed in Pubmed, Embase, Web of Science, COCHRANE Library and Emcare to include papers until December 2022 of patients with various bleeding disorders receiving desmopressin. Desmopressin response rate and possible determinants for desmopressin response were collected. Desmopressin response was defined according to our definition (table 1). Response rate was also calculated based on the response definition previously reported which was either with a cut-off level or without. In addition, we determined whether disease type, weight, age, sex, blood group, mutations and base coagulation factor levels were possible determinants.

Results: Based on the in- and exclusion criteria, 105 articles were included from the 591 identified by our search strategy. These articles comprised 1826 patients; n=921 von Willebrand disease (VWD), 844 hemophilia A (HA), 27 platelet function defects (PFD) and 32 “other” that received desmopressin. Meta-analysis on the impact of the predicted determinants on the response is currently ongoing. Preliminary analysis of response rate (figure 1) according to our pre-defined definition, which was applied to 70 articles, demonstrated a complete response in 88.9% of patients with VWD and 48.0% in HA (patients and carriers). When no cut-off was used, response rate (from 48 articles) was 96.3%, 96.9% and 33.3% for VWD, HA and PFD, respectively. Additionally, we observed no indication for sex-specific response rates in patients with VWD or hemophilia.

Conclusion(s): Preliminary analysis showed that the response rate varied greatly per disease type. The response rate in each disease may be largely determined by the patients’ baseline characteristics.

Funding: Netherlands Organization Scientific Research (NWA.1160.18.038).