Pulmonary arterial hypertension (PAH), is a disease in which blood pressure in the pulmonary artery is elevated. As a result, the heart has to work very hard to pump blood around. PAH is a progressive disease, meaning the disease gets worse and worse. Eventually, patients die of heart failure. Every year, about 12 children in the Netherlands get this disease. After five years, only 60% of them are still alive.
PAH can occur due to a congenital heart defect or because the disease runs in the family (heredity). But you can also get the disease spontaneously or due to liver problems; how that works is not yet entirely clear. Adults can also get PAH, but Menno is investigating the disease in children. PAH is incurable and currently only treatments that slow down the disease are available. Despite those treatments, a large number of patients die from the disease or have to undergo a lung or heart transplant.
One of the existing treatments for children is a backpack with a pump containing medication. This medication goes into the body through an IV line. Menno explains how this disease limits children: 'They live with the knowledge that they can die from this disease. It can go wrong within 15 minutes if the pump fails, and there is also a risk of infection of the infusion line. That is why it is so important that we find new treatments for this disease. So that children can live a happier and longer life.'
To improve children's quality of life, Menno searches for new treatments. In particular, he focuses on making the large pulmonary arteries more flexible. 'We know that in this disease, the pulmonary arteries become stiffer and more rigid,' Menno explains. 'You can compare it to a rubber band. Normally, the vein is very flexible, which is why blood pressure is low. Due to the disease, blood vessels become stiff, just like a rubber band that you cannot stretch easily. When this happens, the pressure in the vessels becomes high, which changes the blood flow further down the smaller vessels and the disease progresses faster there. We also know that stiff pulmonary arteries keep increasing the workload of the heart, resulting in heart failure.'
Douwes wants to find out which disease processes cause pulmonary arteries to become stiffer. To do so, he will, among other things, examine the blood of children* with this disease for signalling substances. 'If I know exactly which disease processes cause the stiffness, we can eventually develop a treatment that counteracts these processes. Then we can slow down or maybe even prevent pulmonary arterial hypertension and right heart failure. Patients can then live longer with a better quality of life.'
The research is being conducted at the Beatrix Children's Hospital of the UMCG, which has great amount of expertise in congenital heart defects. In addition, Beatrix Children's Hospital UMCG is also the national centre of expertise for children with pulmonary arterial hypertension. In his research, Menno collaborates with several laboratories at UMCG, the Faculty of Science and Engineering at the University of Groningen, and the Sick Kids hospital in Toronto, Canada.
*In the care for children with this disease, data is collected in hospital examinations, such as an MRI scan or a blood draw. With the patient's consent, Menno uses that data for his research. So the children who take part in Menno's research do not have to undergo any additional examinations.
Menno receives the 'Senior Clinical Scientist' grant of 475,000 euros from the Dutch Heart Foundation. This is a person-specific grant, intended for development as a researcher and for establishing or maintaining an independent line of research. A condition for the grant is that it contributes to improving the heart health of Dutch society.
