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Objectives:
Allogeneic Hematopoietic Stem Cell Transplantation (HSCT) is a successful treatment option for multiple malignant diseases (i.e. leukemia) and non-malignant disorders (i.e. metabolic disorders, genetic disorders, immunodeficiencies). Unfortunately, transplantation from an HLA-related family member is only available in 30-40% of stem cell transplant recipients. The other patients requiring HSCT must then receive their stem cells from either a matched-unrelated donor (MUD) or from cord blood. One major limitation upon receiving these unrelated stem cells are acute and chronic graft-versus-host disease. Specifically looking at acute graft-versus-host disease (aGVHD), up to 30% of the recipients of stem cells from an HLA-identical related donor will develop greater or equal to grade 2 of aGVHD despite immunosuppressive prophylaxis. The percentages of patients who develop aGVHD from unrelated donors are even higher.
The current standard treatment for aGVHD is corticosteroids. Unfortunately, only 40% of matched-siblings HSCT cases and 25% of MUD SCT cases show a complete response to these steroids. Those patients who do not respond to corticosteroids can show a dismal outcome. Given the poor outcome with refractory GVHD, there has been a lot of interest in trying to predict who will get GVHD. These findings could lead to augmentation of GVHD prophylaxis.
The purpose of this study is to look at a series of identified biomarkers to predict aGVHD. Once blood is drawn from the SCT recipient, a multiplex ligation-dependent probe amplification (MLPA) will test different biomarkers in the blood to result in about 30-45 target sequences being examined simultaneously.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 1 | For Objective #1:
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| 2 | For Objectives #2 & #3:
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| Measure | Description | Time Frame |
|---|---|---|
| To show feasibility and reproducibility of performing a multiplex ligation-dependent amplification procedure (RT-MLPA) | Until September 2008 |
| Measure | Description | Time Frame |
|---|---|---|
| To describe the profile of changes in inflammatory gene products, using RT-MLPA, in pediatric patients receiving stem cell transplant | Until September 2008 | |
| To determine if change in a specific inflammatory product, or a combination of inflammatory products, can predict grade 2-4 acute graft-versus-host disease |
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Inclusion Criteria:
Exclusion Criteria:
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Patients undergoing a hematopoietic stem cell transplant at Children's Memorial Hospital
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| Name | Affiliation | Role |
|---|---|---|
| Reggie Duerst, MD | Ann & Robert H Lurie Children's Hospital of Chicago | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Lurie Children's Hospital | Chicago | Illinois | 60611 | United States |
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2.5 mL's of whole blood are obtained one time a week for the first 100 days of transplant
| Until September 2008 |