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

Primary Purpose

Leukemia, Myelodysplastic/Myeloproliferative Neoplasms

Status
Unknown status
Phase
Phase 3
Locations
Germany
Study Type
Interventional
Intervention
cytarabine
mercaptopurine
laboratory biomarker analysis
allogeneic bone marrow transplantation
biopsy
peripheral blood stem cell transplantation
Sponsored by
European Working Group of MDS in Childhood
About
Eligibility
Locations
Outcomes
Full info

About this trial

This is an interventional diagnostic trial for Leukemia focused on measuring juvenile myelomonocytic leukemia, childhood myelodysplastic syndromes, atypical chronic myeloid leukemia, BCR-ABL1 negative, myelodysplastic/myeloproliferative neoplasm, unclassifiable, chronic myelomonocytic leukemia

Eligibility Criteria

undefined - 18 Years (Child, Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Universitaetskinderklinik - Universitaetsklinikum Freiburg

Outcomes

Primary Outcome Measures

Patient numbers in the different FAB subtypes

Secondary Outcome Measures

Survival
Event-free survival

Full Information

First Posted
October 3, 2002
Last Updated
September 16, 2013
Sponsor
European Working Group of MDS in Childhood
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1. Study Identification

Unique Protocol Identification Number
NCT00047268
Brief Title
Donor Stem Cell Transplant With or Without Chemotherapy in Treating Children With Primary Myelodysplastic Syndrome
Official Title
Prospective Study of the Diagnosis and Treatment of Myelodysplastic Syndromes (MDS) in Childhood
Study Type
Interventional

2. Study Status

Record Verification Date
July 2007
Overall Recruitment Status
Unknown status
Study Start Date
July 1998 (undefined)
Primary Completion Date
undefined (undefined)
Study Completion Date
undefined (undefined)

3. Sponsor/Collaborators

Name of the Sponsor
European Working Group of MDS in Childhood

4. Oversight

5. Study Description

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.
Detailed 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

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
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

7. Study Design

Primary Purpose
Diagnostic
Study Phase
Phase 3
Masking
None (Open Label)

8. Arms, Groups, and Interventions

Intervention Type
Drug
Intervention Name(s)
cytarabine
Intervention Type
Drug
Intervention Name(s)
mercaptopurine
Intervention Type
Other
Intervention Name(s)
laboratory biomarker analysis
Intervention Type
Procedure
Intervention Name(s)
allogeneic bone marrow transplantation
Intervention Type
Procedure
Intervention Name(s)
biopsy
Intervention Type
Procedure
Intervention Name(s)
peripheral blood stem cell transplantation
Primary Outcome Measure Information:
Title
Patient numbers in the different FAB subtypes
Secondary Outcome Measure Information:
Title
Survival
Title
Event-free survival

10. Eligibility

Sex
All
Maximum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Charlotte Niemeyer, MD
Organizational Affiliation
Universitaetskinderklinik - Universitaetsklinikum Freiburg
Official's Role
Study Chair
Facility Information:
Facility Name
Universitaetskinderklinik - Universitaetsklinikum Freiburg
City
Freiburg
ZIP/Postal Code
D-79106
Country
Germany

12. IPD Sharing Statement

Citations:
PubMed Identifier
29217778
Citation
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.
Results Reference
derived
PubMed Identifier
20802024
Citation
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.
Results Reference
derived

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

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