Rhabdomyosarcoma - Childhood: Latest Research

Approved by the Cancer.Net Editorial Board, 07/2022

ON THIS PAGE: You will read about the scientific research being done now to learn more about rhabdomyosarcoma and how to treat it. Use the menu to see other pages.

Doctors are working to learn more about rhabdomyosarcoma, ways to prevent it, how to best treat it, and how to provide the best care to people diagnosed with this disease. The following areas of research may include new options for patients through clinical trials. Always talk with your child’s doctor about the best diagnostic and treatment options for your child.

  • Low-risk rhabdomyosarcoma. For children with rhabdomyosarcoma at a low risk of recurrence, research is focused on decreasing the side effects of treatment by decreasing the total dose of chemotherapy, limiting radiation therapy, and decreasing the total length of time in treatment.

  • Intermediate-risk rhabdomyosarcoma. For children with intermediate-risk rhabdomyosarcoma, clinical trials are aimed at testing the addition of newer targeted therapies, such as drugs that inhibit pathways that may be overexpressed in the tumor. The current Children's Oncology Group (COG) study for intermediate-risk rhabdomyosarcoma is also investigating the duration of therapy by adding a maintenance period of low-dose chemotherapy following the initial course of treatment.

  • Higher-risk rhabdomyosarcoma. For children with higher-risk rhabdomyosarcoma, research is aimed at finding new treatment options and better ways to deliver therapy. A recent COG study tested whether adding irinotecan/vincristine to standard VAC chemotherapy (see Types of Treatment) might improve outcomes. While survival was the same between the 2 treatment regimens, the addition of irinotecan/vincristine did reduce some side effects at an overall lower dose of cyclophosphamide. Another COG study for high-risk rhabdomyosarcoma tested whether irinotecan, vincristine, and chemotherapy given every 2 weeks might improve outcomes. While there was some improvement for children younger than 10 years old with embryonal rhabdomyosarcoma, no significant improvement was seen from this study. The current COG study is investigating intensification of therapy and length of therapy for children with high-risk disease.

  • Palliative care/supportive care. Clinical trials are also underway to find better ways of reducing symptoms and side effects of current rhabdomyosarcoma treatments to improve patients’ comfort and quality of life.

  • Kinase inhibitors. Kinase inhibitors are a type of cancer medication called targeted therapy. Targeted therapies are medications that target specific cancer genes, proteins, or the tissue environment that contributes to cancer growth and survival. This type of treatment blocks the growth and spread of cancer cells while limiting damage to healthy cells, usually leading to fewer side effects than other cancer medications. For patients with high-risk disease, current research is evaluating a type of targeted therapy that blocks the nutrients needed for a tumor to grow and spread. Learn more about targeted treatments.

  • New approaches to treatment. Research is ongoing to improve the understanding of rhabdomyosarcoma and how different drugs affect the disease. Investigations into the importance of gene translocations are very important and can help stratify patients between the various risk groups as well as by translocations that have improved diagnostic methods to better distinguish between the 2 major types of rhabdomyosarcoma. Additionally, ongoing studies are collecting and transplanting human rhabdomyosarcoma cells into animal models (called tumor xenografts) which safely permit testing of new investigational drugs.

  • Tissue collection. Families who have children with rhabdomyosarcoma are encouraged to consider enrolling their child in the COG study APEC14B1, "The Project: EveryChild Protocol: A Registry, Eligibility Screening, Biology and Outcome Study." This study collects tumor and tissue samples that are not needed for their own diagnosis, as well as demographic, treatment, and outcome information so that scientists can learn more about the disease's biology, investigate new treatments, and reduce short-term and long-term side effects

Looking for More about Latest Research?

If you would like more information about the latest areas of research in rhabdomyosarcoma, explore these related items that take you outside of this guide:

The next section in this guide is Coping with Treatment. It offers some guidance on how to cope with the physical, emotional, social, and financial changes that cancer and its treatment can bring. Use the menu to choose a different section to read in this guide.