1. To show feasibility and reproducibility of performing a multiplex ligation-dependent
amplification procedure (RT-MLPA)
2. To describe the profile of changes in inflammatory gene products, using RT-MLPA, in
pediatric patients receiving stem cell transplant
3. To determine if changes in a specific inflammatory product, or a combination of
inflammatory products, can predict grade 2-4 acute graft-versus-host disease
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.
- Objective #1:
- Healthy adult volunteers, affiliated to Children's Memorial Hospital
- Male or female
- Objective #2 & #3:
- Recipient undergoing an allogeneic stem cell transplant
- Receiving related or unrelated cord blood, related or unrelated bone marrow or
peripheral blood stem cells
- Any pre-transplant regimen
- Ages of 0-21 years old
- Male or female
- Inability for subject/parent to understand study and therefore unable to consent
- Children under 7.0 kgs