Trial document
DRKS00000001
Trial Description
Title
EWOG - MDS 98 Myelodysplastic Syndromes in Childhood
Prospective Study of the Diagnosis and Treatment
of Myelodysplastic Syndromes (MDS) in Childhood - EWOG-MDS 98
Trial Acronym
EWOG-MDS 98
URL of the Trial
Brief Summary in Lay Language
Myelodysplastic Syndrome of childhood is a rare disorder that is charcetrized by a reduced production of blood cells and the risk of leukemia development. The prognosis for children diagnosed with MDS is unfavorable. The aim of the study is to introduce standard diagnostic tests and subsequently collect data about the frequency of the differnt subtypes of MDS. Furthermore, the survival of children with MDS should be improved by standardized treatment with chemotherapy and/or bone marrow transplantation.
Brief Summary in Scientific Language
Myelodysplastic Syndromes (MDS) in childhood are a heterogenous group of clonal stem cell disorders accounting for less than 10% of all hematological malignancies in childhood. MDS is characterized by peripheral blood cytopenia, ineffective hematopoiesis, dysplasia of all 3 cell lineages and a high risk of transformation in acute leukemia. Classification of MDS in Childhood has been confusing and inconsistent, while the FAB classification has generally been accepted for adults with MDS. Prognosis of most children with MDS is poor and there is no consensus in the literature concerning the optimal treatment strategy. In general, allogeneic stem cell transplantation (SCT) is the therapy of choice.
The primary objective of this multi-center non-randomized study is to facilitate the diagnostic procedures for all children and adolescents with MDS (study patients)by a standardized review of morphology and standardized cytogenetic and molecular analyses. All cases of MDS will be classified according to a modified FAB classification. Using this approach the frequency of the differnt FAB subtypes and cytogenetic and molecular abnormalities are to be assessed.
The secondary objective of the study is to improve the survival for children and adolescents with primary MDS(protocol patients) over that reported in the literature. In the presence of an HLA-matched family donor, early SCT is the treatment of choice for MDS patients of all FAB subtypes. SCT will be performed according to the ongoing EWOG-MDS study. In the presence of a bone marrow blast count > 15%, AML induction therapy is recommended prior to SCT. AML therapy for these protocol patients will be administered according to the National AML studies. For patients lacking an HLA-identical family donor, SCT from an unrelated volunteer is recommended, though AML therapy without SCT can be offered as an alternative treatment option.
Do you plan to share individual participant data with other researchers?
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Description IPD sharing plan:
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Organizational Data
- DRKS00000001
- 2008/08/07
- 2002/10/03
- yes
- Approved
- 099/98, Ethik-Kommission der Albert-Ludwigs-Universität Freiburg
Secondary IDs
- NCT00047268 (ClinicalTrials.gov)
- UKF000876 (Register Klinischer Studien des Universitätsklinikums Freiburg)
Health Condition or Problem studied
- D46.9 - Myelodysplastic syndrome, unspecified
- Myelodysplastic syndrome, unspecified
Interventions/Observational Groups
-
1.Diagnosis for all children and adolescents with MDS (study patients)by a standardized review of morphology and standardized cytogenetic and molecular analyses
2.Therapy: monitoring without treatment -
1.Diagnosis for all children and adolescents with MDS (study patients)by a standardized review of morphology and standardized cytogenetic and molecular analyses
2.Therapy: treatment according to the national AML therapy -
1.Diagnosis for all children and adolescents with MDS (study patients)by a standardized review of morphology and standardized cytogenetic and molecular analyses
2.Therapy: SCT will be performed according to the ongoing EWOG-MDS study
Characteristics
- Interventional
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- Non-randomized controlled trial
- Open (masking not used)
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- Active control (effective treament of control group)
- Treatment
- Parallel
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Primary Outcome
- To evaluate the frequency of the different FAB subtypes by a standardized approach
- To evaluate the frequency of cytogenetic and molecular abnormalities
Secondary Outcome
- To improve survival for children and adolescents with primary MDS over that reported in the
literature
- To assess the rate of complete remission (CR) and the event-free survival (EFS) in children
with primary MDS treated according to AML therapy
- To evaluate the relapse rate, morbidity and mortality in children with primary MDS treated by
SCT
- To identify different subsets of patients with primary MDS benefiting from SCT or AML-type
therapy
Countries of Recruitment
- Germany
- Denmark
- Sweden
- Norway
- Finland
- Iceland
- Netherlands
- Switzerland
- Austria
- Italy
- Czech Republic
Locations of Recruitment
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Recruitment
- Actual
- 1998/07/03
- 850
- Multicenter trial
- International
Inclusion Criteria
- Both, male and female
- no minimum age
- 18 Years
Additional Inclusion Criteria
All children and adolescents with MDS under the age of 19 diagnosed between July 1998 and June 2002 are registered as study patients.
Exclusion Criteria
- Children with Down syndrome
- Children with the following cytogenetic or molecular abnormalities:
t(8;21)(q22;q22) [AML1/ETO fusion gene]
t(15;17)(q22;q12) [PML/RAR´´ rearrangement]
inv(16)(p13q22) [CBF$/MYH11rearrangement]
- Children with the typical clinical and cytogenetic features of AML FAB M7 who present with < 30% blasts in the BM and PB
- Concomitant illness precluding therapy according to protocol
- No informed consent by the patient or legal guardian
- Morphological diagnosis could not be confirmed
Addresses
-
start of 1:1-Block address primary-sponsor
- Universitätsklinikum Freiburg
- Hugstetter Str. 49
- 79095 Freiburg
- Germany
end of 1:1-Block address primary-sponsorstart of 1:1-Block address contact primary-sponsor- [---]*
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end of 1:1-Block address contact primary-sponsor -
start of 1:1-Block address scientific-contact
- Zentrum für Kinder- und Jugendmedizin des Universitätsklinikums Freiburg, Klinik IV, Pädiatrische Hämatologie/Onkologie
- Ms. Prof. Dr. med Charlotte Niemeyer
- Mathildenstr.1
- 79106 Freiburg
- Germany
end of 1:1-Block address scientific-contactstart of 1:1-Block address contact scientific-contact- 0761-270-4506
- 0761-270-4618
- charlotte.niemeyer at uniklinik-freiburg.de
- [---]*
end of 1:1-Block address contact scientific-contact -
start of 1:1-Block address public-contact
- Zentrum für Kinder- und Jugendmedizin des Universitätsklinikums Freiburg, Klinik IV, Pädiatrische Hämatologie/Onkologie
- Ms. Prof. Dr. med Charlotte Niemeyer
- Mathildenstr.1
- 79106 Freiburg
- Germany
end of 1:1-Block address public-contactstart of 1:1-Block address contact public-contact- 0761-270-4506
- 0761-270-4618
- charlotte.niemeyer at uniklinik-freiburg.de
- [---]*
end of 1:1-Block address contact public-contact
Sources of Monetary or Material Support
-
start of 1:1-Block address materialSupport
- Zentrum für Kinder- und Jugendmedizin des Universitätsklinikums Freiburg, Klinik IV, Pädiatrische Hämatologie/Onkologie
- Ms. Prof. Dr. med Charlotte Niemeyer
- Mathildenstr.1
- 79106 Freiburg
- Germany
end of 1:1-Block address materialSupportstart of 1:1-Block address contact materialSupport- 0761-270-4506
- 0761-270-4518
- charlotte.niemeyer at uniklinik-freiburg.de
- http://www.uniklinik-freiburg.de/kinderklinik/live/fachabteilungen/klinik4.html
end of 1:1-Block address contact materialSupport
Status
- Recruiting complete, follow-up complete
- 2006/12/31