Title: Understanding treatment decision-making processes in families where a child or young person has relapsed/refractory rhabdomyosarcoma
Funder: Children's Cancer and Leukaemia Group

Abstract: In children whose rhabdomyosarcoma has not responded to treatment (refractory), or has come back after treatment (relapsed), there are difficult decisions to be made about what treatment to give next. Only around one in five children with relapsed or refractory rhabdomyosarcoma can be cured, and therefore there are choices to be made about how to prioritise their care. The options might include: # aggressive treatment aiming to cure # treatment to reduce the amount of disease, and therefore help symptoms # experimental trials of new treatments (also called early phase studies) # symptom control Choices made at these times may be extensively re-examined and become a cause of distress months and years after the decision. Within this project we will do two studies that are linked into understanding how decisions are made about treatments: # A “systematic review”, where we will look for all previous early phase studies in relapsed or refractory rhabdomyosarcoma and see how effective the different treatments are for different children. This will help to give families and professionals more accurate information about what to expect from the options available. # An interview study, where we speak to patients and families about how they have made, or are making, decisions about treatment in relapsed or refractory rhabdomyosarcoma. This will help us understand the decision-making process and how best to support families making these choices. The results of both of these studies will be combined in a best practice statement which will provide advice and support to clinicians and families about important things to consider when discussing treatment options. The project will include patients and parents in designing and performing the research to get the best results for families in the future. We will share all of our findings with families and professionals.

Lay abstract: Understanding treatment decision-making processes in families where a child or young person has relapsed/refractory rhabdomyosarcoma. In children whose rhabdomyosarcoma has not responded to treatment (refractory), or has come back after treatment (relapsed), there are difficult decisions to be made about what treatment to give next. Only around one in five children with relapsed or refractory rhabdomyosarcoma can be cured, and therefore there are choices to be made about how to prioritise their care. The options might include: # aggressive treatment aiming to cure # treatment to reduce the amount of disease, and therefore help symptoms # experimental trials of new treatments (also called early phase studies) # symptom control. Choices made at these times may be extensively re-examined and become a cause of distress months and years after the decision. Within this project we will do two studies that are linked into understanding how decisions are made about treatments: # A “systematic review”, where we will look for all previous early phase studies in relapsed or refractory rhabdomyosarcoma and see how effective the different treatments are for different children. This will help to give families and professionals more accurate information about what to expect from the options available. # An interview study, where we speak to patients and families about how they have made, or are making, decisions about treatment in relapsed or refractory rhabdomyosarcoma. This will help us understand the decision-making process and how best to support families making these choices. The results of both of these studies will be combined in a best practice statement which will provide advice and support to clinicians and families about important things to consider when discussing treatment options. The project will include patients and parents in designing and performing the research to get the best results for families in the future. We will share all of our findings with families and professionals.

Research activity codes: 7.1 INDIVIDUAL CARE NEEDS
Health categories: CANCER AND NEOPLASMS