Clinical trial NCT00579397: Markers of Inflammation in Hematopoietic Stem Cell Transplant
Official title: Markers of Inflammation in Hematopoietic Stem Cell Transplant
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Acute Graft Versus Host Disease
Keywords: Markers of Inflammation
acute graft versus host disease
predicting aGVHD
Lead sponsor: Ann & Robert H Lurie Children's Hospital of Chicago
Sponsor class: OTHER
First posted: 2007-12-24
Enrollment: 30
Sex: ALL
Maximum age (years): 21
Standard ages: CHILD
ADULT
Accepts healthy volunteers: yes
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patients undergoing a hematopoietic stem cell transplant at Children's Memorial Hospital

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.

Eligibility criteria: 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

Primary outcomes:
measure: To show feasibility and reproducibility of performing a multiplex ligation-dependent amplification procedure (RT-MLPA)
timeFrame: Until September 2008
Secondary outcomes:
measure: To describe the profile of changes in inflammatory gene products, using RT-MLPA, in pediatric patients receiving stem cell transplant
timeFrame: Until September 2008
measure: 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
timeFrame: Until September 2008
Other outcomes:
Locations:
city: Chicago
country: United States
facility: Lurie Children's Hospital
state: Illinois
Study officials:
affiliation: Ann & Robert H Lurie Children's Hospital of Chicago
name: Reggie Duerst, MD
role: PRINCIPAL_INVESTIGATOR
References: