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

Myelodysplastic Syndromes (MDS) Clinical Trial

— EUMDS  

Official title:

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)

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT00600860
• Other study ID #: 883
• Status: Recruiting
• Start date: April 2008
• Est. completion date: June 30, 2023

Study information

• Verified date: December 2020
• Source: Radboud University
• Contact: Corine J van Marrewijk, PhD
• Phone: +31-24-3614794
• Email: corine.vanmarrewijk[@]radboudumc.nl
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

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.

Description:

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 4000
• Est. completion date: June 30, 2023
• Est. primary completion date: December 31, 2022
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Myelodysplastic Syndromes
• Myelodysplastic Syndromes (MDS)
• Preleukemia
• Syndrome

Intervention

Other:
• No interventions
Only registration of clinical practice

Locations

Country	Name	City	State
Austria	University of Innsbruck Fachartz fur Innere Medizin, Dept of Hematology	Innsbruck
Croatia	Clinical Hospital Merkur	Zagreb
Czechia	Institute of Haematology and Blood Transfusion U nemocnice	Prague
Denmark	Aarhus University Hospital	Aarhus
France	Hopital Avicenne Universite Paris, Dept of Hematology	Paris
Germany	Medizinische Klinik und Poliklinik fur Haematologie, Onkologie und Klinische Immunologie	Dusseldorf
Greece	University of Patras Medical School, Haematology Division, Dept of Internal Medicine	Patras
Israel	Tel-Aviv Sourasky Medical Center, Dept. of Medicine A	Tel-Aviv
Italy	University of Pavia Medical SChool, Dept of Hematology	Pavia
Netherlands	Radboud University Nijmegen Medical Centre, dept of Hematology	Nijmegen
Poland	Medical University, Dept of Hematology, Oncology and Internal Medicine	Warszawa
Portugal	Instituto Português de Oncologia de Lisboa,	Lisbon
Romania	Fundeni CLinical Institute, Clinic of Hematology	Bucharest
Serbia	Clinical Center of Vojvodina	Novi Sad
Spain	Hospital La Fe, Dept of Hematology	Valencia
Sweden	Karolinsky Institute Huddinge University Hospital, Dept of Medicine Division HAematology	Stockholm
Switzerland	Inselspital	Bern
United Kingdom	Leeds General Infirmary Dept of Hematology	Leeds

Sponsors (1)

Lead Sponsor	Collaborator
Radboud University

Countries where clinical trial is conducted

Austria,  Croatia,  Czechia,  Denmark,  France,  Germany,  Greece,  Israel,  Italy,  Netherlands,  Poland,  Portugal,  Romania,  Serbia,  Spain,  Sweden,  Switzerland,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	Dissemination	To disseminate the results of the studies to all stakeholders involved	14.5 years of FU
Primary	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	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
Secondary	New prognostic scoring systems	To derive and validate new prognostic scoring systems	14.5 years of FU
Secondary	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

External Links

•   European MDS network platform
•   European MDS Registry
•   Publication (PMID: 25907546): Validation of the revised international prognostic scoring system (IPSS-R) in patients with lower-risk myelodysplastic syndromes: a report from the prospective European LeukaemiaNet MDS (EUMDS) registry
•   Publication (PMID: 27926979): Erythropoiesis-stimulating agents significantly delay the onset of a regular transfusion need in nontransfused patients with lower-risk myelodysplastic syndrome
•   Publication (PMID: 28526957): Cytomorphology review of 100 newly diagnosed lower-risk MDS patients in the European LeukemiaNet MDS (EUMDS) registry reveals a high inter-observer concordance
•   Publication (PMID: 29122992): Labile plasma iron levels predict survival in patients with lower-risk myelodysplastic syndromes
•   Publication (PMID: 29407183): Prognostic impact of a suboptimal number of analyzed metaphases in normal karyotype lower-risk MDS
•   Publication (PMID: 29572506): Health-related quality of life in lower-risk MDS patients compared with age- and sex-matched reference populations: a European LeukemiaNet study
•   Publication (PMID: 30037803): The use of immunosuppressive therapy in MDS: clinical outcomes and their predictors in a large international patient cohort
•   Publication (PMID: 30126931): Early platelet count kinetics has prognostic value in lower-risk MDS
•   Publication (PMID: 31171638): Impact of red blood cell transfusion dose density on progression-free survival in lower-risk myelodysplastic syndromes patients
•   Publication (PMID: 31278207): Impact of treatment with iron chelation therapy in patients with lower-risk myelodysplastic syndromes participating in the European MDS registry