Markers of Inflammation in Hematopoietic Stem Cell Transplant

Completed

• Conditions: Acute Graft Versus Host Disease

• Registration Number: NCT00579397
• Lead Sponsor: Ann & Robert H Lurie Children's Hospital of Chicago

Brief Summary

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

Detailed 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.

Study Timeline

• Study Start: 2007-04
• Study First Submitted: 2007-12-21
• Study First Submitted QC: 2007-12-21
• Study First Posted: 2007-12-24
• Primary Completion: 2008-10
• Study Completion: 2012-12-12
• Status Verified: 2019-08
• Last Update Submitted: 2019-08-28
• Last Update Posted: 2019-08-30

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 30

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

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
To show feasibility and reproducibility of performing a multiplex ligation-dependent amplification procedure (RT-MLPA)	Until September 2008

Secondary Outcome Measures

Name	Time	Method
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	Until September 2008
To describe the profile of changes in inflammatory gene products, using RT-MLPA, in pediatric patients receiving stem cell transplant	Until September 2008

Trial Locations

Locations (1)

Lurie Children's Hospital; 🇺🇸 Chicago, Illinois, United States