Study for Epidemiology and Characterization of Myelodysplastic Syndromes (MDS) and Juvenile Myelomonocytic Leucemia (JMML) in Childhood

N/AUnknownNCT00662090

University Hospital Freiburg260 enrolled

Overview

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 analysis. The primary objectives of the study are: * 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. The secondary objectives of the study are: * 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

Study Type

OBSERVATIONAL

Enrollment

260

Conditions

Myelodysplastic Syndromes Juvenile Myelomonocytic Leukemia

Eligibility

Sex: ALLMax age: 215 Months

Medical Language ↔ Plain English

Inclusion Criteria: * Written informed consent by the caretakers and whenever possible the patient's assent. * Confirmed diagnosis of MDS or JMML (morphology, cytogenetics) * Myeloid leukemia of Down syndrome (patients aged \> 6 years). * Age less than 18 years Exclusion Criteria: * Denied informed consent and/or assent by caretakers/patient. * Myeloid leukemia of Down syndrome (patients \< 6 years). * Participation in another study within the last 4 weeks (except for therapy optimizing studies in cancer or bone marrow failure disorders and studies in diagnostics).

Outcomes

Primary Outcomes

To evaluate the frequency of the different subtypes of MDS in childhood and adolescence by a standardized diagnostic approach

Time frame: 5 years

To evaluate the frequency of cytogenetic and molecular abnormalities

Time frame: 5 years

Secondary Outcomes

To assess survival for children and adolescents with MDS and JMML

Time frame: 5 years

To evaluate relapse rate, morbidity and mortality in children with MDS and JMML treated by HSCT

Time frame: 5 years

Study for Epidemiology and Characterization of Myelodysplastic Syndromes (MDS) and Juvenile Myelomonocytic Leucemia (JMML) in Childhood

Overview

Conditions

Eligibility

Locations (1)

Outcomes

Primary Outcomes

Secondary Outcomes