Clinical trial NCT00898118: Studying Blood Samples in Young Patients With Cytopenia After a Donor Stem Cell Transplant
Official title: Serial Analysis of Chimerism in Patients With Refractory Cytopenia (RC) Transplanted With Reduced Intensity Conditioning (RIC)
Study type: OBSERVATIONAL
Overall status: UNKNOWN
Conditions: Leukemia
Myelodysplastic Syndromes
Keywords: refractory cytopenia with multilineage dysplasia
childhood myelodysplastic syndromes
previously treated myelodysplastic syndromes
secondary myelodysplastic syndromes
Lead sponsor: National Cancer Institute (NCI)
Sponsor class: NIH
First posted: 2009-05-12
Enrollment: 125
Sex: ALL
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no

Brief summary: RATIONALE: Studying samples of blood from patients with cancer in the laboratory may help doctors learn more about changes that occur in DNA and identify biomarkers related to cancer.

PURPOSE: This research study is looking at blood samples in young patients with cytopenia after undergoing a donor stem cell transplant.

Detailed description: OBJECTIVES:

Primary

* To study hematopoietic chimerism in whole blood and different cell populations (i.e., CD14, CD15, CD 56, CD3, and CD19) as well as in dendritic cells and regulatory T cells after allogeneic hematopoietic stem cell transplantation with reduced intensity conditioning in patients with refractory cytopenia.
* To compare the results of chimerism obtained with standard short tandem nucleotide polymorphism PCR (sensitivity 1%) with those obtained with single nucleotide polymorphisms PCR (sensitivity 0.1- 0.01%).

Secondary

* To evaluate the relationship between mixed chimerism and hematological engraftment, overall survival, and event-free survival.
* To study the impact of mixed chimerism in plasmacytoid dendritic and regulatory T cells on the incidence of acute and chronic graft-versus-host-disease.

OUTLINE: This is a multicenter study.

Peripheral blood is collected from patients and donors prior to hematopoietic stem cell transplantation (HSCT). Patients also undergo blood sample collection on days 30, 60, 100, and 180 after transplantation. Peripheral blood cells are enriched and separated into lineage-specific subpopulations (i.e., CD3, CD14, CD15, CD19, and CD56) which are then divided equally for either DNA isolation via PCR or for flow cytometry. DNA concentrations in pre-HSCT donor and patient samples and in post-HSCT subpopulation samples are determined using quantitative real-time PCR. Samples are also analyzed for quantification of chimerism and detection of genetic markers via short tandem repeats- and sequence nucleotide polymorphism-based chimerism analyses.

Eligibility criteria: DISEASE CHARACTERISTICS:

* Diagnosed with refractory cytopenia

  * Hypocellular bone marrow and normal karyotype
* Underwent stem cell transplantation (SCT) from an HLA identical (8/8) sibling, an HLA identical (10/10) relative, or an HLA identical or single allelic disparate unrelated donor

  * Received a preparative regimen including either thiotepa or fludarabine phosphate
* Concurrently enrolled on EWOG-MDS-2006

PATIENT CHARACTERISTICS:

* Not specified

PRIOR CONCURRENT THERAPY:

* See Disease Characteristics

Primary outcomes:
measure: Number of patients with complete chimerism as measured by standard short tandem nucleotide polymorphism PCR in whole blood and the different cell populations
measure: Number of patients with complete chimerism as measured by single nucleotide polymorphisms PCR in the different cell populations
Secondary outcomes:
measure: Number of patients with mixed chimerism and full hematological recovery at day 100
measure: Number of patients with mixed chimerism and acute or chronic graft-versus-host disease
Other outcomes:
Locations:
city: Vienna
country: Austria
facility: St. Anna Children's Hospital
city: Ghent
country: Belgium
facility: Ghent University
city: Prague
country: Czech Republic
facility: University Hospital Motol
city: Aarhus
country: Denmark
facility: Aarhus Universitetshospital - Aarhus Sygehus
city: Freiburg
country: Germany
facility: European Working Group of MDS in Childhood
city: Freiburg
country: Germany
facility: Universitaetskinderklinik - Universitaetsklinikum Freiburg
city: South Giovanni Rotondo
country: Italy
facility: IRCCS "Casa Sollievo della Sofferenza"
city: Rotterdam
country: Netherlands
facility: Erasmus MC - Sophia Children's Hospital
city: Wroclaw
country: Poland
facility: Akademia Medyczna im. Piastow Slaskich
city: Zurich
country: Switzerland
facility: University Children's Hospital
Study officials:
affiliation: European Working Group of MDS in Childhood
name: Peter Bader, MD
role: PRINCIPAL_INVESTIGATOR
References: