Because stem cell transplantation (SCT) is still evolving, it is important to do research on this. The Stem Cell Transplantation Center at Wilhelmina Children's Hospital – part of University Medical Center (UMC) Utrecht – is conducting a number of studies in the field of SCT.

Doctors at the center ask parents and children for permission before including them in the studies. Although participation takes place on a voluntary basis, almost all patients take part. The studies will result in: 

• improvements in SCT, which also means the procedure will become safer 
• improvements in quality of life and quality of care

The role of viruses

Viruses increase the risk of death following an SCT. But viruses can also cause other problems. For example, they seem to be involved in the development of graft-versus-host disease (GVHD). In GVHD, the donor cells reject the recipient. Doctors at the center are conducting a number of different studies to try and find out how the body’s ability to defend itself against viruses either develops or doesn’t develop following such a transplant. They also want to find out what role viruses play in the onset of GVHD. Once we understand how it works, it will become easier to target treatment for the viruses. Because the center often uses umbilical cord blood for donor cells, doctors are also looking for ways to boost the body’s immune system after an SCT using cord blood.

Long-term outcomes of stem cell transplants for metabolic disorders

Within Europe, the Stem Cell Transplantation Center and the Department of Metabolic Disorders at Wilhelmina Children's Hospital play a major role in important international studies on stem cell transplantation for metabolic disorders. Over the next two years, Mieke Aldenhoven will be doing research on the long-term outcomes of SCTs in patients with Hurler syndrome. In this syndrome (which is a hereditary metabolic disorder), none of the body’s organs function properly. During the first year, Aldenhoven will visit 80 percent of all Hurler patients in Europe who had an SCT in the past twenty years. During the second year, she will visit such patients in the United States. A total of around two hundred Hurler patients who have had such transplants will participate in the study.

With her study, Aldenhoven wants to: 

• Learn which sources of stem cells provide the best outcomes: bone marrow, umbilical cord blood, or 'ordinary' blood 
• Provide clarity with regard to the selection criteria: when should transplantation be done and when not? 
• Identify the longer-term outcomes of successful SCTs in children 
• Improve post-SCT care for children with congenital metabolic disorders

Optimal dosage of medicine

What is the best dosage for chemotherapy and other medicines for children? Doctors at the Stem Cell Transplantation Center are now studying this. To do so, they are working together with: 

• Pharmacists from Wilhelmina Children's Hospital 
• The Leiden/Amsterdam Center for Drug Research (LACDR) 
• The stem cell transplantation center in Leiden

“We always used to give children a standard amount of chemotherapy,” says Jaap Jan Boelens. He is a pediatric immunologist at Wilhelmina Children's Hospital. “But because every child breaks this down in a different way, the effects are also very different. The same dose will be too much for one child and not enough for another. For example, too much chemotherapy results in more side effects. Too little chemotherapy increases the risk of rejection or recurrence of the disease. This is why we’re looking for the optimal dosage. We’re the first in the world to do this kind of research.”