Lessons from the European and Israel National MDS Registry

Abstract

On behalf of the Israeli MDS working group and EUMDS Registry/ XIII Eurasian Hematology Oncology Congress (EHOC) 2022. The myelodysplastic syndromes (MDS) are a group of clonal stem cell diseases with cytopenias and a tendency to transform to leukemia. Despite the progress, there is still lack of real world data about the disease. In 2008, top European experts launched the EUMDS Registry. In this project, including 19 countries and 150 sites, epidemiological, clinical and lab data on newly diagnosed MDS patients, are collected and analyzed. As of today, 3300 patients have been recruited. In 2012, the Israel MDS group joined the project. We have contributed data on 360 patients, # 4 in the contributors. This project led to more than 100 abstracts in international meetings and 40 publications in first line journals. We will mention some of these studies. de Swart et al. summarized data on the first 1000 patients, validated the IPSS-R prognostic classification, showing its superiority on IPSS (de Swart L. BJH 2015). Another study, focusing on Quality of life (QoL) of LR-MDS patients revealed that these patients suffer from depression, anxiety, pain, discomfort and mobility difficulties, compared to controls (Stauder R, Leukemia 2018) The effects of Erythroid Stimulating Agents (ESAs,) was evaluated: Garelius et al. showed that ESA administration delays RBC transfusion dependency (Garelius HK. J Intern Med 2017). Since in most countries ESA is given to transfusion-dependent MDS patients, it might change the paradigm. Recently, we demonstrated that ESA treatment, is associated with improved outcomes and overall survival (Garelius HK. EHA 2022; submitted). Since prognostic factors are often determined at disease presentation, it was important to develop dynamic parameters. We showed that a rapid decline > 25% in the platelet count within 6 months is an adverse prognostic marker (Itzykson R. Bl Adv 2018) A study focusing on the mutational status was presented at ASH 2021 showing that LR-MDS patients can be grouped into 3 clusters, with a correlation to the clinical status (Malcovati L. ASH 2021) The Israeli group, independently and as a part of the EUMDS, was involved in several projects. Oster et al. suggested a non-invasive calculator to assess the probability of or excluding MDS diagnosis, based on patient characteristics, avoiding BM examination (Oster HS. Bl Adv 2021). We also investigated the correlation between Hb level and QoL. This correlation was found to be partial, and the decrease in QoL was not linear. This suggests that other factors other than Hb might play a role in determining QoL (Haring Y. ASH 2021). We recently presented data on lymphoid aggregates in BM biopsies, suggesting a possible association with poor prognosis (Book-Rabinowitz. Int MDS Symposium, Toronto 2021; submitted). In summary , the EUMDS registry is a platform of a scientific project, and an example of international collaboration. Such projects, especially in relatively uncommon diseases, allow collection of enough data to allow meaningful conclusions that might change paradigm and improve patient care. We call all participants of this meeting to join us and improve the quality of this wonderful important project.