Myelodysplastic Syndromes (MDS)
Conditions
Keywords
MDS (all IPSS subtypes), Newly diagnosed
Brief 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.
Detailed 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.
Interventions
OTHERNo interventions
Only registration of clinical practice
Sponsors
Radboud University Medical Center
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Demographics | 14.5 years of follow-up (FU) | 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 |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Correlations | 14.5 years of FU | 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 |
| New prognostic scoring systems | 14.5 years of FU | To derive and validate new prognostic scoring systems |
| Scientific research in MDS | 14.5 years of FU | 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. |
Other
| Measure | Time frame | Description |
|---|---|---|
| Dissemination | 14.5 years of FU | To disseminate the results of the studies to all stakeholders involved |
Countries
Austria, Croatia, Czechia, Denmark, France, Germany, Greece, Israel, Italy, Netherlands, Poland, Portugal, Romania, Serbia, Spain, Sweden, Switzerland, United Kingdom
Contacts
Primary ContactCorine J van Marrewijk, PhD
Backup ContactRosalie Lubbers, MSc
Outcome results
None listed