Research Team: Dr Bob Phillips, Dr Jess Morgan, Dr Gemma Bryan, Lucy Beresford and Connor Evans
Read the latest project updates
Rhabdomyosarcoma, a cancer that usually affects muscles, is the most common soft-tissue sarcoma in children. Overall, around 65% of children with rhabdomyosarcoma will survive, but the cure rates vary depending on different characteristics of the disease.
If children suffer from relapse (where the disease comes back after treatment), or do not respond to first-line therapy (refractory), treatment with curative intent may be available, but for many children, this is not successful.
This means that families have difficult choices to make about how to approach their child’s care. Choices made by families are often re-examined and can be a cause of distress after a decision has been made.
The original REFoRMS project was funded by CCLG to incorporate two studies:
- A systematic review to look at all the available evidence on the treatment options that have been evaluated for children with relapsed and refractory rhabdomyosarcoma. We are looking at how effective these treatments are, and whether the effectiveness of a treatment varies in different patient groups.
- An interview study to help to understand how patients and their families make decisions about the potential treatment options, and what factors influence these decisions. The study will also investigate how patients and families’ feel about the decisions they made in the past, and whether that has changed over time.
The findings from the two studies will be combined to create a best practice statement, which will provide advice and support for clinicians and families who are making difficult decisions about treatment options. This will help healthcare professionals to support families during the decision making process, and help to reduce the amount of decisional regret that they may experience in the future.
We are working alongside parents and clinical experts in this project to make sure that the research is going to be helpful for families in the future.
REFoRMS systematic review
The REFoRMS team have now completed the first stage of their project, the systematic review that looks at the different treatments for relapsed and refractory rhabdomyosarcoma and how effective these treatments are.
They reviewed data from 129 relevant published research studies, 99 clinical trials and over 1,100 children. You can find out more about their findings below:
Results booklet for parents and families
Supplementary materials for the systematic review report
Watch our webinar about the project
Interview study
The interview study has now finished recruirment. We are analysing the findings and hope to have these ready to share in early 2024.
Living-REFoRMS
This new project, funded by CCLG and Alice’s Arc, builds on the original REFoRMS work and plans to do two things:
- Develop a living systematic review of studies into new treatments (early phase studies) in relapsed and refractory rhabdomyosarcoma called Living-REFoRMS. Living systematic reviews are a relatively new method where, instead of only having one search of the literature, the evidence is regularly searched, assessed, and summarised so that the information is the most up-to-date that it can be. This living systematic review will look for completed research and currently open trials in this area, from all around the world.
- Develop a high-quality, regularly updated, online resource to share the findings from Living-REFoRMS that will be accessible to families and clinicians, and will help and support decision making in relapsed and refractory rhabdomyosarcoma. The online resource will be developed with and hosted by the CCLG, and we will use their experience in communicating complex childhood cancer information to ensure it best serves the community.
About Alice's Arc
Alice’s Arc was founded by Sara and David Wakeling to raise funds for research into rhabdomyosarcoma while their daughter, Alice, was on treatment for the disease.
Alice was first diagnosed with stage 4 alveolar rhabdomyosarcoma, aged three, in March 2015.
Following 20 months of chemotherapy at Great Ormond Street Hospital (GOSH) and proton therapy in the USA, Alice spent one year in remission before her cancer returned in February 2018. She passed away, aged seven, in October 2019, over four and a half years after her diagnosis.
Living-REFoRMS updates
The first Living-REFoRMS update searches were performed in October 2022 and the findings are now available.
The second update searches were performed in April 2023 and the findings are now available.
The third update searches were done in November 2023 and the findings are now available.
The fourth update searches were done in May 2024 and the findings are now available.
The fifth update searches were done in October 2024 and the findings are now available.
The work on developing the online resource will be starting in Autumn 2023 - watch this space, and our Twitter feed, for more information about how to get involved!
For more information:
REFoRMS team email address: reforms-project@york.ac.uk
Twitter account: @REFoRMS_Rhabdo
Phone:+44 793 804 4956