King’s research case study shows how personalised medicine can change lives

30 January 2024 - A research case published in the New England Journal of Medicine shows the benefits of tailoring treatment to an individual patient

Earlier this month, the Paediatric Liver centre at King’s College Hospital published a detailed case study in the New England Journal of Medicine, showcasing how personalised care, tailored to a patient’s individual needs, led to more effective treatment of a young child with auto-immune hepatitis.

Autoimmune hepatitis is a rare, life-long liver disease. It occurs when the body’s immune system causes damage to its own healthy liver cells, leading to inflammation in the liver.

Professor Dino Hadzic, one of the senior Paediatric Hepatologist at the Trust, explained: “At King’s, we see around 350 children with auto-immune hepatitis every year. This can be an extremely challenging chronic condition, and this girl was among the 10-20% of patients who unfortunately do not respond to standard treatment methods, such as steroids, and needed a different approach.

“After looking in more detail at her immune response, using one of the recently developed national genetic panels (next generation sequencing), we discovered that she was born with an extremely rare mistake in her body’s defence system (known as ‘inborn error of immunity’), which had likely triggered autoimmune hepatitis.”

The team at King’s looked at the underlying change in the girl’s DNA which was causing the condition, and identified a specific immune treatment that could help.

Professor Dino Hadzic added “Instead of using steroids, we began a novel treatment with an anti-inflammatory drug called baricitinib (JAK inhibitor), which targeted a particular part of the girl’s immune response pathway. This was very effective in treating her condition, and five years on, she is doing extremely well.

“In publishing this observation, we hope to demonstrate how moving away from the traditional ‘one-size-fits-all’ approach, and using precision medicine, where a treatment is tailored to an individual patient, can be genuinely life-changing.”