Clinical trial NCT00600860: A Prospective, Multicentre European Registry for Newly Diagnosed Patients With Myelodysplastic Syndromes
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)
Study type: OBSERVATIONAL
Overall status: UNKNOWN
Conditions: Myelodysplastic Syndromes (MDS)
Keywords: MDS (all IPSS subtypes)
Newly diagnosed
Lead sponsor: Radboud University Medical Center
Sponsor class: OTHER
First posted: 2008-01-25
Enrollment: 4000
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: Clinic

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.

Eligibility criteria: 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

Primary outcomes:
measure: Demographics
timeFrame: 14.5 years of follow-up (FU)
Secondary outcomes:
measure: Correlations
timeFrame: 14.5 years of FU
measure: New prognostic scoring systems
timeFrame: 14.5 years of FU
measure: Scientific research in MDS
timeFrame: 14.5 years of FU
Other outcomes:
measure: Dissemination
timeFrame: 14.5 years of FU
Locations:
city: Innsbruck
country: Austria
facility: University of Innsbruck Fachartz fur Innere Medizin, Dept of Hematology
city: Zagreb
country: Croatia
facility: Clinical Hospital Merkur
city: Prague
country: Czechia
facility: Institute of Haematology and Blood Transfusion U nemocnice
city: Aarhus
country: Denmark
facility: Aarhus University Hospital
city: Paris
country: France
facility: Hopital Avicenne Universite Paris, Dept of Hematology
city: Dusseldorf
country: Germany
facility: Medizinische Klinik und Poliklinik fur Haematologie, Onkologie und Klinische Immunologie
city: Patras
country: Greece
facility: University of Patras Medical School, Haematology Division, Dept of Internal Medicine
city: Tel-Aviv
country: Israel
facility: Tel-Aviv Sourasky Medical Center, Dept. of Medicine A
city: Pavia
country: Italy
facility: University of Pavia Medical SChool, Dept of Hematology
city: Nijmegen
country: Netherlands
facility: Radboud University Nijmegen Medical Centre, dept of Hematology
city: Warszawa
country: Poland
facility: Medical University, Dept of Hematology, Oncology and Internal Medicine
city: Lisbon
country: Portugal
facility: Instituto Português de Oncologia de Lisboa,
city: Bucharest
country: Romania
facility: Fundeni CLinical Institute, Clinic of Hematology
city: Novi Sad
country: Serbia
facility: Clinical Center of Vojvodina
city: Valencia
country: Spain
facility: Hospital La Fe, Dept of Hematology
city: Stockholm
country: Sweden
facility: Karolinsky Institute Huddinge University Hospital, Dept of Medicine Division HAematology
city: Bern
country: Switzerland
facility: Inselspital
city: Leeds
country: United Kingdom
facility: Leeds General Infirmary Dept of Hematology
Study officials:
affiliation: Leeds General Infirmary
name: David Bowen, PhD
role: STUDY_CHAIR
affiliation: Radboud University Medical Center
name: Theo de Witte, Prof Dr
role: STUDY_DIRECTOR
References:
citation: de Swart L, Crouch S, Hoeks M, Smith A, Langemeijer S, Fenaux P, Symeonidis A, Cermak J, Hellstrom-Lindberg E, Stauder R, Sanz G, Mittelman M, Holm MS, Malcovati L, Madry K, Germing U, Tatic A, Savic A, Almeida AM, Gredelj-Simec N, Guerci-Bresler A, Beyne-Rauzy O, Culligan D, Kotsianidis I, Itzykson R, van Marrewijk C, Blijlevens N, Bowen D, de Witte T; EUMDS Registry Participants. Impact of red blood cell transfusion dose density on progression-free survival in patients with lower-risk myelodysplastic syndromes. Haematologica. 2020 Mar;105(3):632-639. doi: 10.3324/haematol.2018.212217. Epub 2019 Jun 6.
citation: Itzykson R, Crouch S, Travaglino E, Smith A, Symeonidis A, Hellstrom-Lindberg E, Sanz G, Cermak J, Stauder R, Elena C, Germing U, Mittelman M, Langemeijer S, Madry K, Tatic A, Holm MS, Almeida AM, Savic A, Simec NG, Luno E, Culligan D, Guerci-Bresler A, Malcovati L, van Marrewijk C, Bowen D, de Witte T, Fenaux P; European MDS Registry members. Early platelet count kinetics has prognostic value in lower-risk myelodysplastic syndromes. Blood Adv. 2018 Aug 28;2(16):2079-2089. doi: 10.1182/bloodadvances.2018020495.
citation: de Swart L, Reiniers C, Bagguley T, van Marrewijk C, Bowen D, Hellstrom-Lindberg E, Tatic A, Symeonidis A, Huls G, Cermak J, van de Loosdrecht AA, Garelius H, Culligan D, Macheta M, Spanoudakis M, Panagiotidis P, Krejci M, Blijlevens N, Langemeijer S, Droste J, Swinkels DW, Smith A, de Witte T; EUMDS Steering Committee. Labile plasma iron levels predict survival in patients with lower-risk myelodysplastic syndromes. Haematologica. 2018 Jan;103(1):69-79. doi: 10.3324/haematol.2017.171884. Epub 2017 Nov 9.
citation: Garelius HK, Johnston WT, Smith AG, Park S, de Swart L, Fenaux P, Symeonidis A, Sanz G, Cermak J, Stauder R, Malcovati L, Mittelman M, van de Loosdrecht AA, van Marrewijk CJ, Bowen D, Crouch S, de Witte TJ, Hellstrom-Lindberg E. Erythropoiesis-stimulating agents significantly delay the onset of a regular transfusion need in nontransfused patients with lower-risk myelodysplastic syndrome. J Intern Med. 2017 Mar;281(3):284-299. doi: 10.1111/joim.12579. Epub 2016 Dec 7.