The standard treatment for acute graft-vs-host disease (GVHD) is to suppress the activity of
the donor immune cells using steroid medications such as prednisone. Although most GVHD,
especially in children, responds well to treatment, sometimes (around 1/3 of the time) there
is either no response to steroids or the response does not last. In those cases, the GVHD can
become dangerous and even life-threatening. Unfortunately, doctors cannot predict who will
have a good response to treatment based on symptom severity or initial response to steroids.
As a result, nearly all children who develop GVHD are treated with long courses of high dose
steroids even though that means many patients receive more treatment than they probably need.
Steroid treatment can cause short-term complications like infections, high blood sugar, high
blood pressure, muscle weakness, depression, anxiety, and problems sleeping and long-term
complications like bone damage, cataracts in the eyes, and decreased growth. 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.
The doctors conducting this research have developed a blood test (GVHD biomarkers) that
predicts whether a patient will respond well to steroids. The study team found that children
who have low GVHD biomarkers at the start of treatment and for the first two weeks of
treatment have a very high response rate to steroids. In this study, the study team will
monitor GVHD symptoms and biomarkers during treatment and taper steroids quickly in patients
who have GVHD that is expected to respond very well to treatment. The study team will assess
how many patients respond well to lower steroid dosing and what steroid complications
develop. The study team will also use surveys to obtain the patient's own assessment of their
quality of life (down to age 5 years).