Serial Response and Biomarker-Guided Steroid Taper for Children with GVHD

Pediatric Blood Cancer
John Levine, MD, MS
Icahn School of Medicine at Mount Sinai, Tisch Cancer Institute


Allogeneic hematopoietic stem cell transplants (stem cell transplant from a donor) [HSCT] are an important treatment for high-risk hematologic (blood) malignancies. However, these transplants can result in graft versus host disease (GVHD) which targets the skin, liver, and GI tract. This occurs in about 35% of pediatric allogeneic transplant receipts and GVHD is the leading cause of non-relapse mortality after allogeneic HSCTs. Current treatment of GVHD is with corticosteroids which can result in infections after high doses, and long-term exposure to steroids results in slowed growth, poor quality of life, mood disturbances, and learning impairments. While the majority of GVHD in pediatric patients is steroid responsive, clinical severity and early clinical responses to steroid treatment are poor predictors of outcomes and not helpful to guide therapy. The investigators conducting this study have developed a blood test (GVHD biomarkers) that predicts whether a patient will respond well to steroids. The primary objective of this phase II study is to identify patients with GVHD at very low risk for treatment failure so their treatment can be rapidly tapered (reduced) to avoid treatment complications utilizing the GVHD biomarkers. This trial is the first ever aimed at decreasing the toxicity of GVHD treatment by personalizing steroid dosing and rapidly tapering based upon reproducible biological measures.

Trial Registration: Identifier: NCT05090384