Donor Stem Cell Transplant With or Without Chemotherapy in Treating Children With Primary Myelodysplastic Syndrome
NCT ID: NCT00047268
Last Updated: 2013-09-17
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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UNKNOWN
PHASE3
INTERVENTIONAL
1998-07-31
Brief Summary
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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.
Detailed Description
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* 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
Conditions
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Keywords
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Study Design
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DIAGNOSTIC
NONE
Interventions
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cytarabine
mercaptopurine
laboratory biomarker analysis
allogeneic bone marrow transplantation
biopsy
peripheral blood stem cell transplantation
Eligibility Criteria
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Inclusion Criteria
* 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
18 Years
ALL
No
Sponsors
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European Working Group of MDS in Childhood
OTHER
Principal Investigators
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Charlotte Niemeyer, MD
Role: STUDY_CHAIR
Universitaetskinderklinik - Universitaetsklinikum Freiburg
Locations
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Universitaetskinderklinik - Universitaetsklinikum Freiburg
Freiburg im Breisgau, , Germany
Countries
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References
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Drexler B, Schwarz-Furlan S, Baumann I, Rudelius M, Nollke P, Lebrecht D, Ramamoorthy S, Rotari N, Karow A, Hirabayashi S, Beier F, Behrens YL, Gohring G, Kalb R, Wlodarski MW, Strahm B, Erlacher M, Niemeyer CM, Yoshimi A. Long-term outcomes of patients with refractory cytopenia of childhood under observation only. Blood Adv. 2025 Aug 26;9(16):4279-4285. doi: 10.1182/bloodadvances.2025016136.
Pastor VB, Sahoo SS, Boklan J, Schwabe GC, Saribeyoglu E, Strahm B, Lebrecht D, Voss M, Bryceson YT, Erlacher M, Ehninger G, Niewisch M, Schlegelberger B, Baumann I, Achermann JC, Shimamura A, Hochrein J, Tedgard U, Nilsson L, Hasle H, Boerries M, Busch H, Niemeyer CM, Wlodarski MW. Constitutional SAMD9L mutations cause familial myelodysplastic syndrome and transient monosomy 7. Haematologica. 2018 Mar;103(3):427-437. doi: 10.3324/haematol.2017.180778. Epub 2017 Dec 7.
Gohring G, Michalova K, Beverloo HB, Betts D, Harbott J, Haas OA, Kerndrup G, Sainati L, Bergstraesser E, Hasle H, Stary J, Trebo M, van den Heuvel-Eibrink MM, Zecca M, van Wering ER, Fischer A, Noellke P, Strahm B, Locatelli F, Niemeyer CM, Schlegelberger B. Complex karyotype newly defined: the strongest prognostic factor in advanced childhood myelodysplastic syndrome. Blood. 2010 Nov 11;116(19):3766-9. doi: 10.1182/blood-2010-04-280313. Epub 2010 Aug 27.
Other Identifiers
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EWOG-MDS-98
Identifier Type: -
Identifier Source: secondary_id
EU-20218
Identifier Type: -
Identifier Source: secondary_id
CDR0000257581
Identifier Type: -
Identifier Source: org_study_id