RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Combining chemotherapy with stem cell transplantation may allow the doctor to give higher doses of chemotherapy drugs and kill more tumor cells. It is not yet known if donor stem cell transplantation is more effective with or without chemotherapy in treating myelodysplastic syndrome.

PURPOSE: Phase III trial to determine the effectiveness of donor stem cell transplantation with or without chemotherapy in treating children who have primary myelodysplastic syndrome.

Condition	Treatment or Intervention	Phase
atypical chronic myeloid leukemia Chronic Myelomonocytic Leukemia juvenile myelomonocytic leukemia myelodysplastic and myeloproliferative disease Myelodysplastic Syndromes	Drug: cytarabine  Drug: mercaptopurine  Procedure: allogeneic bone marrow transplantation  Procedure: biological markers  Procedure: biological response modifier therapy  Procedure: biopsies  Procedure: bone marrow ablation with stem cell support  Procedure: bone marrow transplantation  Procedure: chemotherapy  Procedure: diagnostic test  Procedure: peripheral blood stem cell transplantation	Phase III

Further Study Details: 

OBJECTIVES:

• 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.

PROJECTED ACCRUAL: Not specified

Ages Eligible for Study:  up to  18 Years,  Genders Eligible for Study:  Both

Criteria

DISEASE CHARACTERISTICS:

• 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

PATIENT CHARACTERISTICS: Age

• Under 19

Performance status

• Not specified

Life expectancy

• Not specified

Hematopoietic

• See Disease Characteristics

Hepatic

• Not specified

Renal

• Not specified

Other

• No other concurrent illness that would preclude study

PRIOR CONCURRENT THERAPY: Biologic therapy

• Not specified

Chemotherapy

• No prior chemotherapy for MDS

Endocrine therapy

• Not specified

Radiotherapy

• No prior radiotherapy for MDS

Surgery

• Not specified

Germany
      Universitaetskinderklinik - Universitaetsklinikum Freiburg, Freiburg,  79106,  Germany; Recruiting

Study chairs or principal investigators

Charlotte Niemeyer, MD,  Study Chair,  Universitaetskinderklinik - Universitaetsklinikum Freiburg   

Study ID Numbers:  CDR0000257581; EWOG-MDS-98; EU-20218; NCT00047268
Record last reviewed:  October 2002
Last Updated:  April 4, 2005
Record first received:  October 3, 2002
ClinicalTrials.gov Identifier:  NCT00047268
Health Authority: United States: Federal Government
ClinicalTrials.gov processed this record on 2005-04-08