New insights into treatment of rare fetal blood disease

The LUMC is a Dutch Expertise Center for Fetal Medicine and one of the largest fetal treatment centers in the world

Led by the Leiden University Medical Center (LUMC), the first international multicenter study into treatment strategies and outcomes for babies with fetal and neonatal allo-immune thrombocytopenia (FNAIT) has been published in The Lancet Haematology. This is a rare blood disorder during pregnancy that can lead to brain haemorrhages in the baby.

FNAIT arises because the platelets, small cell fragments in our blood that prevent bleeding, of the unborn baby are broken down by antibodies from the mother. This puts the baby at risk of severe brain haemorrhage. “Due to the rarity of FNAIT, we don’t really know yet how this disease is treated in different hospitals, and therefore we don’t know what the optimal treatment strategy for newborns is,” explains Thijs de Vos, researcher at both the Neonatology Department of the LUMC and Sanquin Research, “In this study, therefore, we compared the treatment and outcomes of approximately 400 children across Australia, the United States and Europe.”

New insights
De Vos and colleagues write in The Lancet Haematology that there are major differences between centers in treatment strategies after childbirth. Particularly in the use of platelet transfusions. “While some centers do not administer transfusions, others use the method as standard treatment at FNAIT,” says De Vos. “Our study thus shows that there is no consensus on the treatment of FNAIT, while also exposing important questions.”

For example, the researchers’ data suggests that the use of platelets in transfusions leads to an increase in the baby’s platelet count. “But we’re not sure if this actually reduces the risk of brain haemorrhage. We would like to answer that question,” says the doctor-researcher.

Basis for clinical studies
“Our research provides new insights into the treatment of this rare disease. This will allow treatment guidelines to be developed and the data can also be used to set up clinical studies,” said Enrico Lopriore, Professor of Neonatology and Fetal Medicine. “Think of a randomized study in which two different blood transfusion strategies are compared. In this way we will hopefully arrive at the optimal treatment of FNAIT in the future.”

The LUMC is a Dutch Center of Expertise for Fetal Medicine and one of the largest fetal treatment centers in the world. The LUMC and blood bank Sanquin are together pioneers in the field of FNAIT research by combining their respective clinical and diagnostic expertise. This study was therefore a collaboration between the Neonatology and Obstetrics departments of the LUMC and Sanquin.

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International study offers new insights into treatment of rare fetal blood disease
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