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

Leukemia Clinical Trial

Official title:

Prospective Study of the Diagnosis and Treatment of Myelodysplastic Syndromes (MDS) in Childhood

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT00047268
• Other study ID #: CDR0000257581
• Secondary ID: EWOG-MDS-98EU-20
• Status: Active, not recruiting
• Phase: Phase 3
• First received: October 3, 2002
• Last updated: September 16, 2013
• Start date: July 1998

Study information

• Verified date: July 2007
• Source: National Cancer Institute (NCI)
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Federal Government
• Study type: Interventional

Clinical Trial 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.

Description:

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

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 0
• Accepts healthy volunteers: No
• Gender: Both
• Age group: N/A to 18 Years

Eligibility

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

Study Design

Masking: Open Label, Primary Purpose: Diagnostic

Related Conditions & MeSH terms

• Leukemia
• Myelodysplastic Syndromes
• Myelodysplastic-Myeloproliferative Diseases
• Myelodysplastic/Myeloproliferative Neoplasms
• Myeloproliferative Disorders
• Preleukemia

Intervention

Drug:
• cytarabine

• mercaptopurine

Other:
• laboratory biomarker analysis

Procedure:
• allogeneic bone marrow transplantation

• biopsy

• peripheral blood stem cell transplantation

Locations

Country	Name	City	State
Germany	Universitaetskinderklinik - Universitaetsklinikum Freiburg	Freiburg

Sponsors (1)

Lead Sponsor	Collaborator
European Working Group of MDS in Childhood

Country where clinical trial is conducted

Germany, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Patient numbers in the different FAB subtypes			No
Secondary	Survival			No
Secondary	Event-free survival			No