Who can enter
- Children with prednisolone prophylaxis (prevention) or treatment of graft-versus-host disease after hematopoetic stem cell transplantation.
- Age: 0-20 years.
Goal
The goal of this study is to find the right dose of prednisolone for every child to either prevent or treat graft-versus-host disease after hematopoetic stem cell transplantation.
Background
Our immune system has the task to respond to “foreign intruders” in our body. Along with a hematopoietic stem cell transplantation, the patient receives a new immune system from the donor which can cause an immune response to the patient’s own body. This is called graft-versus-host disease (GVHD), or reversed rejection disease. GVHD occurs in approximately 40% of stem cell recipients and is therefore one of the biggest obstacles in stem cell transplantation.
Cornerstone of treatment for GVHD is high dose of systemic corticosteroids. The most common used medicine in this class is prednisolone and is used to either prevent or treat GVHD. Every child receives the same dose of corticosteroids (according to their bodyweight), but the results between patients are highly variable. In 3 out of 10 children, the treatment with prednisolone fails, meaning GVHD will still develop or will not be cured. Unfortunately, there is even a chance a patient will not survive the immune response.
We think that the prednisolone dose, and thereby the effect in children after stem cell transplantation can be improved. This relationship between dose and effect is not yet investigated in patients with GVHD and will be the first step to improve the current treatment.