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Donor Stem Cell Transplant With or Without Chemotherapy in Treating Children With Primary Myelodysplastic Syndrome

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00047268
Recruitment Status : Unknown
Verified July 2007 by National Cancer Institute (NCI).
Recruitment status was:  Active, not recruiting
First Posted : January 27, 2003
Last Update Posted : September 17, 2013
Information provided by:
National Cancer Institute (NCI)

Brief Summary:

RATIONALE: Giving chemotherapy before a donor stem cell transplant helps stop the patient's immune system from rejecting the donor's stem cells. When the healthy stem cells from a donor are infused into the patient they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets. It is not yet known whether donor stem cell transplant is more effective with or without chemotherapy in treating primary myelodysplastic syndrome.

PURPOSE: This phase III trial is studying how well donor stem cell transplant given with chemotherapy works and compares it with donor stem cell transplant without chemotherapy in treating children with primary myelodysplastic syndrome.

Condition or disease Intervention/treatment Phase
Leukemia Myelodysplastic/Myeloproliferative Neoplasms Drug: cytarabine Drug: mercaptopurine Other: laboratory biomarker analysis Procedure: allogeneic bone marrow transplantation Procedure: biopsy Procedure: peripheral blood stem cell transplantation Phase 3

Detailed Description:


  • Determine, by a standard approach, the frequency of different FAB subtypes in children with primary myelodysplastic syndromes.
  • Determine the frequency of cytogenetic and molecular abnormalities in these patients.
  • Determine the survival of patients treated with allogeneic stem cell transplantation with or without induction chemotherapy.
  • Determine the rate of complete remission in patients treated with these regimens.
  • Determine the event-free survival of patients treated with these regimens.
  • Determine the relapse rate, morbidity, and mortality of patients treated with these regimens.
  • Determine different subsets of patients who benefit from these regimens.

OUTLINE: This is a multicenter study. Patients are stratified according to FAB subtype (refractory anemia (RA) or RA with ringed sideroblasts (RARS) vs RA with excess blasts (RAEB) vs RAEB in transformation (RAEB-t) vs juvenile myelomonocytic leukemia (JMML)).

Patients undergo complete medical and physical examination. Patients are screened for the following aberrations: -7, +8, +21, t(8;21), t(15;17), and inv(16). Smears of peripheral blood and bone marrow, as well as bone marrow biopsies and all cytogenetic and molecular studies performed on blood or bone marrow, are evaluated by a panel of international experts.

Patients with progressive RA or RARS undergo allogeneic stem cell transplantation (ASCT) according to EWOG-MDS SCT studies. Patients with stable RA or RARS wait for an optimal donor before undergoing ASCT. Patients with RAEB with fewer than 15% bone marrow blasts undergo ASCT. Patients with RAEB with at least 15% bone marrow blasts and patients with RAEB-t with fewer than 30% bone marrow blasts receive standard acute myeloid leukemia (AML) induction therapy and then undergo ASCT. Patients with RAEB-t with at least 30% bone marrow blasts are considered for standard AML induction therapy.

Patients with advanced JMML undergo evaluation for splenectomy and receive chemotherapy with mercaptopurine and cytarabine every 3-4 weeks (for 1-4 doses). Patients then undergo ASCT.

Patients are followed every 6 months.


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Study Type : Interventional  (Clinical Trial)
Masking: None (Open Label)
Primary Purpose: Diagnostic
Official Title: Prospective Study of the Diagnosis and Treatment of Myelodysplastic Syndromes (MDS) in Childhood
Study Start Date : July 1998

Primary Outcome Measures :
  1. Patient numbers in the different FAB subtypes

Secondary Outcome Measures :
  1. Survival
  2. Event-free survival

Information from the National Library of Medicine

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Ages Eligible for Study:   up to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No


  • Morphologically confirmed primary myelodysplastic syndromes (MDS)

    • Diagnosed between July 1, 1998 and June 30, 2002
  • No prior aplastic anemia
  • No prior congenital bone marrow failure syndrome, such as:

    • Fanconi's anemia
    • Kostmann syndrome
    • Shwachman syndrome
    • Dyskeratosis congenital
    • Amegakaryocytic thrombocytopenia
    • Diamond-Blackfan anemia
  • No Down syndrome
  • None of the following cytogenetic or molecular abnormalities:

    • t(8;21)(q22;q22)
    • t(15;17)(q22;q12)
    • inv(16)(p13;q22)
  • No typical clinical and cytogenetic features of acute myeloid leukemia FAB M7 (i.e., acute megakaryocytic leukemia) with fewer than 30% blasts in bone marrow or peripheral blood



  • Under 19

Performance status

  • Not specified

Life expectancy

  • Not specified


  • See Disease Characteristics


  • Not specified


  • Not specified


  • No other concurrent illness that would preclude study


Biologic therapy

  • Not specified


  • No prior chemotherapy for MDS

Endocrine therapy

  • Not specified


  • No prior radiotherapy for MDS


  • Not specified

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00047268

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Universitaetskinderklinik - Universitaetsklinikum Freiburg
Freiburg, Germany, D-79106
Sponsors and Collaborators
European Working Group of MDS in Childhood
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Study Chair: Charlotte Niemeyer, MD Universitaetskinderklinik - Universitaetsklinikum Freiburg
Publications automatically indexed to this study by Identifier (NCT Number):
Layout table for additonal information Identifier: NCT00047268    
Other Study ID Numbers: CDR0000257581
First Posted: January 27, 2003    Key Record Dates
Last Update Posted: September 17, 2013
Last Verified: July 2007
Keywords provided by National Cancer Institute (NCI):
juvenile myelomonocytic leukemia
childhood myelodysplastic syndromes
atypical chronic myeloid leukemia, BCR-ABL1 negative
myelodysplastic/myeloproliferative neoplasm, unclassifiable
chronic myelomonocytic leukemia
Additional relevant MeSH terms:
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Myelodysplastic Syndromes
Myeloproliferative Disorders
Myelodysplastic-Myeloproliferative Diseases
Neoplasms by Histologic Type
Bone Marrow Diseases
Hematologic Diseases
Precancerous Conditions
Antimetabolites, Antineoplastic
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents
Antiviral Agents
Anti-Infective Agents
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Nucleic Acid Synthesis Inhibitors
Enzyme Inhibitors