CML Pediatric ITK Response According to Molecular Identification at Diagnosis
This study focuses on children under 18 diagnosed with Chronic Myeloid Leukaemia (CML). We know that modern medicines called tyrosine kinase inhibitors (TKIs) have greatly improved CML treatment, but sometimes they don't work as well as expected, even in children. Children's CML can be different from adult CML, suggesting there might be different underlying reasons for the disease. This research aims to find specific genetic patterns (a 'molecular signature') in children with CML. By looking at existing DNA and RNA samples from when they were first diagnosed, we want to see if these patterns can help predict how well they will respond to TKI treatment. This could help doctors make more personalised treatment choices for children in the future.
At a glance
What is this study about?
Chronic Myeloid Leukaemia (CML) is a type of cancer that affects the blood and bone marrow. While new medicines called tyrosine kinase inhibitors (TKIs) have been very successful in treating CML, we still sometimes see cases where the treatment doesn't work as well as we'd hope, even in children. CML in children can be a bit different from CML in adults, and scientists believe there might be unique biological reasons for these differences and how children respond to treatment.
This study is trying to understand why some children respond better to TKIs than others. We know that certain changes in our genes can affect how cancer behaves and how it responds to medicines. While some of these genetic changes are known, there's still a lot to learn, especially in children with CML. Researchers will be looking closely at DNA and RNA samples taken from children when they were first diagnosed with CML.
The main goal is to find a 'molecular signature' – a specific pattern of genetic changes – that can help predict how a child will respond to TKI treatment. By analysing these samples using advanced techniques like Next Generation Sequencing, the team hopes to identify markers that show whether a child is likely to respond well, or if they might need a different approach. This kind of research is really important because it could lead to more personalised and effective treatments for children with CML in the future.
Key takeaways
- This study examines CML in children aged 18 and under.
- It uses existing blood samples (DNA and RNA) from diagnosis.
- The goal is to find genetic patterns that predict TKI treatment response.
- No new tests or clinic visits are required for participants.
- The research aims to improve future CML treatments for children.
Who may be eligible?
This study is looking back at information from children who have already been diagnosed and treated for CML. To be included, the child must have been 18 years old or younger when they were first diagnosed. They also must have had a specific genetic change called the Philadelphia chromosome and BCR::ABL1 transcript, which confirms the CML diagnosis.
Their CML needed to be in the 'chronic phase' (the earliest stage) when diagnosed, and their first treatment should have been one of the TKI medicines. It's okay if they had some initial medicine called hydroxyurea before starting TKIs. Crucially, there needs to be a stored DNA sample available from their diagnosis, and for a smaller group, an RNA sample too.
Children are not included if they were over 18 at diagnosis, or if their CML was in a more advanced accelerated or blastic phase at diagnosis. Also, if their first treatment for CML was anything other than a TKI medicine, they wouldn't be included in this particular study.
Could this study suit you?
Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.
- Was your child under 18 when diagnosed with CML?
- Did they have the 'Philadelphia chromosome' genetic change?
- Was their CML in the 'chronic phase' at diagnosis?
- Did they start treatment with a TKI medicine?
- Are there stored DNA samples from their diagnosis available?
What does participation involve?
This study is a 'retrospective' study, meaning it looks at information and samples that have already been collected from children who have been treated for CML in the past. This means you do not need to do anything or attend any appointments for this study. The researchers will be using existing stored DNA and RNA samples that were taken as part of your child's routine care at the time of their diagnosis. They will also look at your child's treatment records to see how they responded to the TKI medicines. There are no extra tests, visits, or medications involved for your child or family as part of this research.
Potential risks and benefits
Locations (1)
- CHU de Bordeaux, Service Hématologie BiologiqueVerified postcodeBordeaux, France· Recruiting
Common questions
What is CML?
CML stands for Chronic Myeloid Leukaemia, which is a type of cancer of the blood and bone marrow.
What are TKIs?
TKIs (Tyrosine Kinase Inhibitors) are types of medicines that specifically target and block the growth signals in CML cells.
Why is this study only for children?
CML in children can behave differently from CML in adults, so this study focuses specifically on understanding the disease in younger patients.
Will my child have to do anything for this study?
No, this study uses existing, stored medical information and samples, so your child won't need to do anything or attend extra appointments.
How could this study help in the future?
By understanding genetic patterns, this study hopes to help doctors predict which CML treatments might work best for children, leading to more effective and personalised care.
How to find out more
Stéphanie DULUCQ
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
Discussion
Community discussion
Powered by our forum at community.patient.info. Please be respectful — this is not medical advice.