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MDS-RIGHT Report Summary

Project ID: 634789
Funded under: H2020-EU.3.1.

Periodic Reporting for period 1 - MDS-RIGHT (Providing the right care to the right patient with MyeloDysplastic Syndrome at the right time)

Reporting period: 2015-05-01 to 2016-10-31

Summary of the context and overall objectives of the project

Healthy aging is one of the major challenges for European society, as ageing is associated with more health problems and healthcare (HC) utilisation. Both myelodysplastic syndromes (MDS) and anemia are conditions occurring more often in the elderly, limiting quality of life (QoL) and increasing the pressure on HC use with the expected shift to older age.
MDS is a chronic bone marrow malignancy most often seen in elderly aged 75 or older, which is often complicated by severe anaemia (decrease in amount of red blood cells), infections or bleeding complications. The majority (75%) of patients with MDS are classified as lower-risk, which is associated with an 8-years loss of life expectancy compared to healthy elderly population. A considerable number of lower-risk MDS-patients (in total an estimated 2 million European citizens) remain ‘under diagnosed’ as anemia of the elderly (AoE), due to insufficient awareness of the relevance of a right diagnosis and the reluctance to use bone marrow puncture (required for MDS diagnosis) in frail elderly, as this is often regarded too invasive and demanding. Consequently, these people do not have access to proper care. The MDS-RIGHT project aims to address the complex challenges associated with correct diagnosis and treatment of patients with MDS or AoE, using data from a comprehensive well-established European myelodysplastic syndrome Registry (EUMDS) as well as a community based Dutch population (LifeLines). The main goal is to compare long-term outcomes and cost, as well as approaches to diagnosis and treatment of MDS and AoE; to assess (epi)genetic abnormalities; and to develop more effective and safer evidence-based, tailored interventions for the elderly patients with lower-risk MDS or AoE, resulting in better treatment compliance and more cost-effective and safer use of HCI for our elderly European citizens with lower-risk MDS and/or anaemia.
Existing HC for MDS is challenging and complex, as MDS is a heterogeneous disorder and the available HCI do not cure MDS, but generally aim to ameliorate symptoms of BM failure (in particular anaemia) and to improve QoL. The goal of MDS treatment is to help patients to remain active, mobile and independent. The EUMDS registry ideally suited for comparing the effectiveness of currently available HCI for this predominantly elderly patient group and perfectly fits the topic PHC17-2014: ‘Comparing the effectiveness of existing HCI in the elderly’, as it encompasses longitudinal observational data from more than 2,100 lower-risk MDS patients, with >8 years follow-up, in 16 EU countries and Israel. Comparisons of this ‘real life’ data will help identify which treatment strategies are most cost-effective. In addition, data from EUMDS and LifeLines will facilitate development and validation of new, more effective diagnostic and prognostic procedures, enabling identification of specific patient groups more likely to benefit from HCI, contributing to more personalized HC. Outcomes of MDS-RIGHT will contribute to improved evidence-based treatment guidelines, allowing the HC community to use resources more cost-effectively. These new guidelines and other outcomes of the MDS-RIGHT will be distributed to all stakeholders (e.g. doctors, patient groups, HC regulators, pharmaceutical companies) by means of a new to establish ‘European MDS competence network’.
In order to achieve the above, MDS-RIGHT encompasses 6 work packages, each dedicated to address one of the six objectives:
1) Compare outcome and costs of existing HCIs by using data from the comprehensive EUMDS Registry. This will provide robust evidence to underpin sustainable use of HC resources
2) Enhancing compliance with diagnostic procedures in MDS, by introducing new diagnostic methods. This will increase the number of correctly and timely diagnosed MDS patients, and prevent underdiagnosis
3) Maximising QoL of patients by restricting MDS-specific interventions to patients with the right diagnosis (that is: MDS) who are likely to benefit from these HCIs. This will improve QoL of patients through better tailoring of these interventions
4) Providing models that can better predict the likely response of a patient to a certain treatment, so called ‘treatment-outcome prediction models’, to support personalised decision making and robust economic analyses. Improved predictive and response criteria will reduce overtreatment with the expensive therapies, thereby contributing to more cost-effective HC
5) Develop improved, evidence-based diagnostic and therapeutic guidelines, based on the outcomes of objectives 1-4
6) Establish a European MDS competence network encompassing all stakeholders for dissemination and utilization of up-to-date, evidence-based and regulatory guidance

Work performed from the beginning of the project to the end of the period covered by the report and main results achieved so far

So far, MDS-RIGHT is well on track. Detailed statistical analysis plans are made, impact of HCI on the established core outcome sets (COS): overall survival (OS) and progression-free survival (PFS) are evaluated for iron chelation on OS (ASH 2016 abstract), and ESAs on OS and PFS (JIM 2016). Also, QoL in lower-risk MDS patients is compared to a reference population (manuscript in preparation), outcomes in MDS are evaluated to define new COS, and time to the first transfusion in subjects responding to ESA therapy is evaluated as new COS. For the validation of novel molecular and flow cytometric diagnostic and prognostic markers, DNA from 1000 lower-risk MDS patients has been sequenced. Collection and transfer of DNA will continue. Samples from >400 AoE individuals were identified in LifeLines, and first samples are sequenced to provide insight into the frequency and type of molecular defects in (unexplained) AoE in a general population, and to elucidate the relation between unexplained AoE, age-related clonal hematopoiesis and MDS.
A detailed plan for dissemination of results, and a comprehensive catalogue of MDS collaborative groups, existing documents, and national contact points has been developed to create a multi-stakeholder European MDS competence network for sharing MDS information and knowledge. A network website ( is established as central hub of the stakeholder network, including a project specific section (; resources, including general MDS information and the catalogue, and relevant approved guidelines. Several specific recommendations, including those for use of allogeneic stem cell transplantation in MDS (Witte T de, et al, accepted Blood 2017), and those for CMML (joint EHA-ELN) will be added. The website will be gradually expanded with dynamic web-based recommendation facilities (currently being programmed and formatted by a dedicated website Task Force), including an interactive algorithm for personalized therapeutic recommendation; a news & events and a community section, including a moderated blog to encourage and facilitate MDS stakeholder interaction and exchange of views on aspects of MDS management and research. The progress of activities of the MDS-RIGHT project is continuously monitored by project management and the project partners.

Progress beyond the state of the art and expected potential impact (including the socio-economic impact and the wider societal implications of the project so far)

During this first reporting period, a solid basis has been founded within the MDS-RIGHT project for achieving the expected impacts.

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