EWOG-MDS 2006

Author: Dipl.-Inform. MPH Peter Nöllke, Prof. Dr. med. Charlotte Niemeyer, erstellt 2003/07/24, Last modification: 2019/09/17

Title Prospective non–randomized multi-center study for epidemiology and characterization of Myelodysplastic Syndromes (MDS) and Juvenile Myelomonocytic Leukemia (JMML) in childhood
Disease Myelodysplastic Syndrome (MDS) and Juvenile Myelomonocytic Leukemia (JMML) in childhood
Type Trial aiming at the standardization of diagosis and therapy
Problem / Objectives

The aim of the study is to improve the accuracy of diagnosis for children and adolescents with MDS by a standardized review of morphology and standardized cytogenetic and molecular analyses.

Primary objectives

  • To evaluate the frequency of the different subtypes of MDS in childhood and adolescence by a standardized diagnostic approach
  • To evaluate the frequency of cytogenetic and molecular abnormalities: -Specifically using array-CGH to evaluate the frequency of subtle chromosomal imbalances, i.e.gains and losses of defined chromosomal regions, and amplifications.-Specifically using mFISH to identify unknown chromosomal aberrations, particularly subtle translocations involving new candidate genes, and to better define chromosomal breakpoints.

Secondary objectives

  • To assess survival for children and adolescents with MDS and JMML
  • To evaluate relapse rate, morbidity and mortality in children with MDS and JMML treated by HSCT
Inclusion Criteria
  • Confirmed diagnosis of MDS or JMML (morphology, cytogenetics)
  • Myeloid leukemia of Down syndrome (patients aged > 6 years).
  • Age: age less than 18 years
Exclusion Criteria
  • Denied informed consent and/or assent by caretakers/patient.
  • Fanconi anemia (diagnosed by chromosomal breakage, G2 cell cycle arrest, Western blot or mutational analysis) or other congenital bone marrow failure disorders (diagnosed clinically or by disease specific germ line mutations) without secondary MDS. Secondary MDS in congenital bone marrow failure is defined by a consistent acquired bone marrow abnormality as a) increase in blasts, b) acquired consistent, hromosomal abnormality, c) increasing bone marrow cellularity in the presence of blood pancytopenia
  • Shwachman syndrome or Fanconi anemia with a single aberration not typical of MDS.
  • Translocation characteristic for de novo AML like t(8;21)(q22;q22) [AML1/ETO fusion gene], t(15,17)(q22;q12) [PML/RARα rearrangement], inv(16)(p13q22) [CBFβ/MYH11rearrangement]
  • Myeloid leukemia of Down syndrome (patients aged < 6 years).
  • Participation in another interventional study within the last 4 weeks (except for therapy optimizing
  • studies in cancer or bone marrow failure, diagnostic protocols).
Recruitment 260
Status Start 01/01/2007; end of recruitment: 01/04/2013
Principal Investigator Prof. Dr. med. Charlotte Niemeyer
E-Mail mailto:ewog-mds@uniklinik-freiburg.de
URL http://www.ewog-mds.org/
Contact

Investigator

Prof. Dr. Charlotte Niemeyer
Universitätsklinikum Freiburg, ZKJ
Klinik IV: Pädiatrische Hämatologie und Onkologie
Mathildenstraße 1
79106 Freiburg i. Brsg.
Telefon +49 (761) 270 45060
Fax +49 (761) 270 45180
charlotte.niemeyer@uniklinik-freiburg.de

Data management

Alexandra Fischer
Universitätsklinikum Freiburg, ZKJ
Klinik IV, Sekretariat Onkologie
Mathildenstraße 1
79106 Freiburg i. Brsg.
Telefon +49 (761) 270 46170
Fax +49 (761) 270 46 230
alexandra.fischer@uniklinik-freiburg.de

Statistics

Peter Noellke
Universitätsklinik Freiburg
ZKJ, Klinik für Päd. Onkologie und Hämatologie
Mathildenstr. 1
79106 Freiburg
Telefon +49 (761) 270 46190
Fax +49 (761) 270 46230
peter.noellke@uniklinik-freiburg.de

Link(s) Geschützte Dokumente
Sponsoring Carreras-Stiftung, Förderverein für krebskranke Kinder e.V., Freiburg i. Br.