Markers of Inflammation in Hematopoietic Stem Cell Transplant

Ann & Robert H Lurie Children's Hospital of Chicago

Status: Completed

Conditions: Acute Graft Versus Host Disease

Study type: Observational

Funder types: Other

Identifiers

NCT00579397

SCT 0407

Details and patient eligibility

About

Objectives:

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

Full description

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.

Enrollment: 30 patients

Sex: All

Ages: Under 21 years old

Volunteers: Accepts Healthy Volunteers

Inclusion criteria

• 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

Exclusion criteria

• Inability for subject/parent to understand study and therefore unable to consent
• Children under 7.0 kgs

Trial design

30 participants in 2 patient groups

1

Description:

For Objective #1: * Healthy adult volunteers

2

Description:

For Objectives #2 \& #3: * Recipients undergoing an allogeneic stem cell transplant