PIKACHU (Stem cell transplantation)
Recruiting
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
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
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.
In order to participate in a study please refer to your/your child’s doctor.
Last reviewed
Last reviewed
March 23, 2026
Study details
- Study details
Official title
PK/PD of corticosteroids in graft-versus-host disease after hematopoietic cell transplantation in childrenDisease
Malignant leukemia (ALL, AML, etc.)
Metabolic disorder (Hurler, Fanconi, etc.)
Maximum number of patients
130Start date
October 1, 2020Status
Open for inclusionLocal principal investigator
Dr. C. A. LindemansSponsor
Prinses Máxima Center for pediatric oncologyApproval
The study of this new treatment has been reviewed by an accredited medical research ethics committee. This committee has decided that it is justified to ask patients to participate in this study. More information can be found at: CCMO.Trial registry number
Dutch Trial Registry (NTR) study NL8703
The above information is intended as a brief summary only and may not reflect the most up-to-date information. For full details and the current status of a protocol, physicians can contact the Princess Máxima Center directly.