How clinical trials and advanced therapies are transforming precision care for kids

17 September 2025 | Expected time to read: 2 minutes

Image of Dr Steven Keogh sitting outside leaning against table and smiling.

Paediatric medicine is constantly evolving, as researchers and clinicians all work hard to make treatments safer, more effective, less invasive, and more personalised to the specific needs of each child.  

Leading the way in children’s cancer care and advanced therapies 

Clinician-researchers like Dr Steven Keogh, an oncologist providing Blood Transplant and Cellular Therapies at The Children’s Hospital at Westmead (CHW), are not only delivering world-class, evidence-based care to children today, they are sharing their knowledge internationally to support better care for kids everywhere.  

For children with blood cancers and genetic conditions like sickle cell disease, this research has been transformative. 

Breakthroughs in treating sickle cell disease 

“Sickle cell anaemia is a genetic disease. When children with sickle cell anaemia progress through life, they have what’s called sickling crises, they can get severe pain and blocked blood vessels that impair organ function.  

“A bone marrow transplant is the only curative therapy for sickle cell disease,” said Dr Keogh. 

Image of young child smiling standing in front of blurred background.

Using precision care to improve treatment outcomes 

The clinicians and researchers at CHW have collaborated with world experts over many years to understand how to get the best outcomes for children receiving bone marrow transplants.  

Drawing on this research, Dr Keogh and his colleagues analyse a child’s blood results to calculate the exact amount of chemotherapy needed to stop blood cell production processes and prepare for a transplantation. Too small a dose can lead to a failed treatment, but too much would be highly toxic and cause sickness.  

“The pharmacokinetic lab is using 25 years of research and publications so that kids today can benefit from it and get the maximum chance of cure,” said Dr Keogh. 

Turning research breakthroughs into better futures 

For some kids, conditions that used to be life limiting can now be cured. Dr Keogh and his team recently published two of the world’s largest studies to help kids needing bone marrow transplants. 

Together, we can advance precision care for kids. By donating today, you’ll help deliver life-changing treatments faster and give sick kids the best chance at a healthier future. 

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