A Prospective, Multicentre European Registry for Newly Diagnosed Patients With Myelodysplastic Syndromes

Study Overview
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Brief Summary
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Study Objectives: To collect and describe demographics, disease-management, and treatment outcomes of Myelodysplastic Syndromes (MDS) patients who are newly diagnosed and classified according to the World Health Organization (WHO) criteria. To perform observational studies concerning relevant scientific research questions in MDS using clinical data and biological samples, and to present relevant research outcomes in the fields of diagnosis and prognostication, health related quality of life issues, health economics, and risk stratification for newly developed classes of drugs. To disseminate results of the studies to all stakeholders involved.

Detailed Description
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Methodology: Data on patients with MDS will be collected prospectively at diagnosis and at 6-months intervals after diagnosis. The data will be gathered by seventeen (or more) countries that are represented within the LeukemiaNet MDS Working Party and will be combined in one central European Database. Data analyses will be conducted by the Data Management Centre at the University of York in various sub studies, at specific time points as decided or requested by the steering committee, but at least once a year included in the European Registry and at the end of the follow-up period. Number of Patients & Centres Over 149 hematology centres in eighteen (or more) different countries (Austria, Croatia, Czech Republic, Denmark, France, Germany, Greece, Israel, Italy, The Netherlands, Romania, Spain, Sweden, Switzerland and United Kingdom) will participate as referral centres in this Registry. The recruitment target is a minimum of 3000 cases lower-risk MDS and 1000 higher-risk cases. Population: The study population will consist of newly diagnosed patients with all subtypes of MDS classified according to the WHO criteria, including therapy-related MDS and MDS-F, AML with 20-<30 percent marrow blasts (former RAEB-t), and CMML and other forms of mixed MDS/MPD. Study Duration: The enrolment time is scheduled to continue until at least December 2022 but extension of the recruitment period is possible. Patients will be followed until withdrawal (for any reason) or termination of the EUMDS Registry.

Official Title
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A Prospective, Multicentre European Registry for Newly Diagnosed Patients With Myelodysplastic Syndromes (MDS), Including Acute Myeloid Leukaemia With 20-<30 Percent Marrow Blasts (Former RAEB-t), and Chronic Myelomonocytic Leukaemia (CMML)

Conditions
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Myelodysplastic Syndromes (MDS)

Intervention / Treatment
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* Other: No interventions


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Patients must meet all of the following criteria Age > 18 years Newly diagnosed patient (within 100 days from the date of the diagnostic bone marrow (BM) aspirate) MDS classified according to current WHO criteria All sub groups of MDS Therapy-related MDS MDS with Fibrosis (MDS-F) AML with 20-<30 percent marrow blasts (former RAEB-t) CMML and other forms of mixed MDS/MPD IPSS and IPSS-R Risk group classification (mandatory) Able and willing to provide the written informed consent Exclusion Criteria: Age <18 years Patient unwilling or unable to give consent AML with ≥30 percent marrow blasts according to WHO Patients with inv(16), t(5;17) and t(8;21) are considered AML and therefore not eligible Patients with higher risk MDS progressed from a previously diagnosed lower risk MDS that was not registered within 100 days after first diagnosis of (lower risk) MDS

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| MDS patients<br>Patients with MDS according to current WHO criteria and International Prognostic Scoring System (IPSS) classification | Other: No interventions<br>* Only registration of clinical practice<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Demographics | The primary objective of this study is collect and describe demographics, clinical and lab manifestations, epidemiological data, genetic characteristics, HRQoL, disease-management, and treatment outcomes of MDS patients who are newly diagnosed and classified according to the WHO-2008 and WHO-2016 criteria | 14.5 years of follow-up (FU) | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Correlations | To investigate the relationship between: Clinical characteristics at inclusion and during follow-up Treatments received, including transfusions, and Responses to treatment Overall survival Disease progression General and disease specific HRQoL, and Karnofsky Performance Status Health Economics | 14.5 years of FU | 
| New prognostic scoring systems | To derive and validate new prognostic scoring systems | 14.5 years of FU | 
| Scientific research in MDS | To perform observational studies concerning relevant scientific research questions in MDS using clinical data and biological samples and to present relevant research outcomes in the fields of diagnosis & prognostication, HRQoL issues, health economics, risk stratification for newly developed classes of drugs. | 14.5 years of FU | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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MDS (all IPSS subtypes), Newly diagnosed