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

NCT00047268 | Unknown Phase 3

• 3 other identifiers: CDR0000257581EWOG-MDS-98EU-20218
• Study Type: interventional
• Target: N/A
• Locations: 1 country
• Sites: 1

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.

Conditions

Leukemia; Myelodysplastic/Myeloproliferative Neoplasms

Keywords

juvenile myelomonocytic leukemia; childhood myelodysplastic syndromes; atypical chronic myeloid leukemia, BCR-ABL1 negative; myelodysplastic/myeloproliferative neoplasm, unclassifiable; chronic myelomonocytic leukemia

Outcome Measures

Primary Outcomes (1)

• Patient numbers in the different FAB subtypes

Secondary Outcomes (2)

• Survival

• Event-free survival

Interventions

cytarabine DRUG

mercaptopurine DRUG

laboratory biomarker analysis OTHER

allogeneic bone marrow transplantation PROCEDURE

biopsy PROCEDURE

peripheral blood stem cell transplantation PROCEDURE

Eligibility Criteria

• Age: Up to 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

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

Contact the study team to discuss eligibility requirements. They can help determine if this study is right for you.

Sponsors & Collaborators

• European Working Group of MDS in Childhood: lead

Study Sites (1)

Universitaetskinderklinik - Universitaetsklinikum Freiburg

Freiburg im Breisgau, D-79106, Germany

Location

Related Publications (3)

• 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.

  PMID: 40554414 DERIVED

• 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.

  PMID: 29217778 DERIVED

• 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.

  PMID: 20802024 DERIVED

MeSH Terms

Conditions

Leukemia; Myelodysplastic-Myeloproliferative Diseases; Leukemia, Myelomonocytic, Juvenile; Leukemia, Myeloid, Chronic, Atypical, BCR-ABL Negative; Myeloproliferative Disorders; Leukemia, Myelomonocytic, Chronic

Interventions

Cytarabine; Mercaptopurine; Biopsy; Peripheral Blood Stem Cell Transplantation

Condition Hierarchy (Ancestors)

Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases; Bone Marrow Diseases; Leukemia, Myeloid; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Cytidine; Pyrimidine Nucleosides; Pyrimidines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Arabinonucleosides; Nucleosides; Nucleic Acids, Nucleotides, and Nucleosides; Sulfhydryl Compounds; Sulfur Compounds; Organic Chemicals; Purines; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring; Cytodiagnosis; Cytological Techniques; Clinical Laboratory Techniques; Diagnostic Techniques and Procedures; Diagnosis; Specimen Handling; Diagnostic Techniques, Surgical; Surgical Procedures, Operative; Investigative Techniques; Hematopoietic Stem Cell Transplantation; Stem Cell Transplantation; Cell Transplantation; Cell- and Tissue-Based Therapy; Biological Therapy; Therapeutics; Transplantation

Study Officials

• Charlotte Niemeyer, MD

  Universitaetskinderklinik - Universitaetsklinikum Freiburg

  STUDY CHAIR

Study Design

• Study Type: interventional
• Phase: phase 3
• Masking: NONE
• Purpose: DIAGNOSTIC
• Sponsor Type: OTHER

Study Record Dates

• First Submitted: October 3, 2002
• First Posted: January 27, 2003
• Study Start: July 1, 1998
• Last Updated: September 17, 2013

Record last verified: 2007-07

Locations

DE (1)