Innovative new trial designs mean it will be possible to test new medicines in rare diseases such as childhood Lupus.
Systemic lupus erythematosus (often called Lupus or SLE) is an autoimmune disease. It typically affects women between the ages of 15 and 45, but can start in younger children.
There are many things that can trigger Lupus, and symptoms can vary from patient to patient. It often causes immune cells in the body to become hyperactive and produce autoantibodies. People with Lupus are often very tired, have joint pain, and their skin may be sensitive to sunlight. Lupus can also lead to damage in internal organs such as the kidneys. Severe forms affecting the kidneys or brain are called Lupus nephritis or Neuropsychiatric Lupus.
What did the authors hope to find?
The authors wanted to summarise the needs and challenges of testing medicines in children and young people with Lupus. They hoped that this would be useful to design future trials of new medicines in children and young people with Lupus.
How was the review performed?
This is a review article. This means that no special drug study was undertaken. The authors used published articles, medical guidelines and legal documents from the US and Europe to complete their review. They looked at all the evidence to see how studies of new medicines have been done in other rare diseases in children.
What were the main findings of the review?
The main finding was that no medicine has ever been approved specifically to treat children with Lupus, or kidney disease related to Lupus that starts in childhood. At the moment, doctors rely on the evidence collected in adults when deciding which drugs to use in children with Lupus, but there are differences in severity and
organ involvement in childhood Lupus compared to the disease in adults.
It is important to be able to test medicines that might be used in childhood really in children with the disease – this helps to ensure that there are no effects on safety, growth or development in children. Getting enough people with rare diseases to take part in trials can be difficult. The authors mention that a childhood-specific research network can be useful to quickly recruit children into clinical trials. For ethical and safety reasons, the most commonly used trial designs for testing medicines in adults are not recommended for use in children. In the future, innovative study designs will help doctors to complete trials in children with Lupus.
Are these findings new?
Yes, this is the first comprehensive review addressing the need for clinical trials in children with Lupus.
What are the limitations of this study?
This is not a prospective study, and there are no published clinical trials in children with Lupus to support the findings and proposals of the review.
What do the authors plan on doing with this information?
The authors plan to use the analysis to help design future clinical trials in children with Lupus.
What does this mean for me?
If you or a child you care for has Lupus, there may be new treatment options in the future – or you may be able to take part in a clinical trial. If you are interested in finding out more, you should talk to your doctor.
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Date prepared: April 2019
Summary based on research article published on: 19 September 2018
From: Brunner H, et al. Clinical trials in children and adolescents with systemic lupus erythematosus: methodological aspects, regulatory landscape and future opportunities.
Ann Rheum Dis 2019;78:162–170. doi:10.1136/annrheumdis-2018-213198
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