Treatment Response and Biomarker-Guided Steroid Taper for Children with GVHD
Treatment Response and Biomarker-Guided Steroid Taper for Children with GVHD
This phase II trial studies the treatment response for patients with acute graft-versus-host disease (GVHD). GVHD occurs when donor immune cells attack the healthy tissue of a bone marrow or stem cell transplant patient. The standard treatment for GVHD is to lower the activity of the donor cells by using steroid medications such as prednisone. But steroid treatment may cause many complications and the risk of these complications increases with higher doses of steroids and longer treatment. It is important to find ways to decrease the steroid treatment in patients who do not need long courses. Researchers are doing this study to find out how many subjects respond well to lower steroid dosing based on a blood test (GVHD biomarker) and if they develop fewer complications.
Miscellaneous,
Pediatrics
Phase II
Both
Therapy (NOS)
Prednisone
Kitko, Carrie
International
Vanderbilt University
08-30-2022
Treatment
VICCPED2213
NCT05090384
Eligibility
0 Years
BOTH
NO
Inclusion Criteria:
Newly diagnosed GVHD that meets criteria for Minnesota standard risk except GVHD that is limited to skin rash
Ann Arbor 1 GVHD by biomarkers
GVHD not previously treated systemically (topical therapies and non-absorbed steroids are allowed)
Any donor type, human leukocyte antigen (HLA)-match, conditioning regimen is acceptable
Age 0-21 years at the time of screening
Performance score (Lansky/Karnofsky) >= 70%
Signed and dated written informed consent obtained from patient or legal representative and assent from pediatric patients capable of providing assent
Exclusion Criteria:
Patients treated for GVHD with > 0.5 mg/kg prednisone for any duration or any steroid treatment for GVHD for more than 3 days prior to confirmation of eligibility
Patients receiving corticosteroids > 0.1 mg/kg prednisone (or other steroid equivalent) for any indication within 7 days before the onset of acute GVHD except for adrenal insufficiency, premedication for transfusions/IV medications, or intermittent use for symptom control such as nausea/vomiting
Relapsed, progressing, or persistent malignancy or other condition (e.g., known declining donor chimerism) requiring withdrawal of systemic immune suppression
Patients with uncontrolled infection (i.e., progressive symptoms related to infection despite treatment, persistently positive microbiological cultures despite treatment, viral reactivations unresponsive to treatment, or any other evidence of severe infection)
Severe organ dysfunction including requirement for dialysis, mechanical ventilation, or oxygen supplementation exceeding 40% fraction of inspired oxygen (FiO2) within 7 days of enrollment
Significant liver disease evidenced by direct bilirubin > 2 mg/dl or alanine aminotransferase (ALT) or aspartate aminotransferase (AST) > 5 times the upper limit of normal
Creatinine clearance or estimated glomerular filtration rate
A clinical presentation resembling first occurrence chronic GVHD or overlap syndrome developing before or present at the time of enrollment
Patients who are pregnant
Newly diagnosed GVHD that meets criteria for Minnesota standard risk except GVHD that is limited to skin rash
Ann Arbor 1 GVHD by biomarkers
GVHD not previously treated systemically (topical therapies and non-absorbed steroids are allowed)
Any donor type, human leukocyte antigen (HLA)-match, conditioning regimen is acceptable
Age 0-21 years at the time of screening
Performance score (Lansky/Karnofsky) >= 70%
Signed and dated written informed consent obtained from patient or legal representative and assent from pediatric patients capable of providing assent
Exclusion Criteria:
Patients treated for GVHD with > 0.5 mg/kg prednisone for any duration or any steroid treatment for GVHD for more than 3 days prior to confirmation of eligibility
Patients receiving corticosteroids > 0.1 mg/kg prednisone (or other steroid equivalent) for any indication within 7 days before the onset of acute GVHD except for adrenal insufficiency, premedication for transfusions/IV medications, or intermittent use for symptom control such as nausea/vomiting
Relapsed, progressing, or persistent malignancy or other condition (e.g., known declining donor chimerism) requiring withdrawal of systemic immune suppression
Patients with uncontrolled infection (i.e., progressive symptoms related to infection despite treatment, persistently positive microbiological cultures despite treatment, viral reactivations unresponsive to treatment, or any other evidence of severe infection)
Severe organ dysfunction including requirement for dialysis, mechanical ventilation, or oxygen supplementation exceeding 40% fraction of inspired oxygen (FiO2) within 7 days of enrollment
Significant liver disease evidenced by direct bilirubin > 2 mg/dl or alanine aminotransferase (ALT) or aspartate aminotransferase (AST) > 5 times the upper limit of normal
Creatinine clearance or estimated glomerular filtration rate
A clinical presentation resembling first occurrence chronic GVHD or overlap syndrome developing before or present at the time of enrollment
Patients who are pregnant
