Rhabdomyosarcoma - Childhood: Latest Research

Approved by the Cancer.Net Editorial Board, 10/2023

ON THIS PAGE: You will read about the scientific research being done 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. 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. The recently completed Children's Oncology Group (COG) study for intermediate-risk rhabdomyosarcoma also investigated the duration of therapy by adding a period of maintenance therapy of low-dose chemotherapy following the initial course of treatment. Results from this study are still being evaluated.

  • High-risk rhabdomyosarcoma. For children with high-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 chemotherapy to standard vincristine, dactinomycin, and cyclophosphamide chemotherapy (VAC therapy; 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 with an overall lower dose of cyclophosphamide. Another COG study for high-risk rhabdomyosarcoma tested whether giving irinotecan, vincristine, and other chemotherapy every 2 weeks might improve outcomes. There was some improvement for children younger than 10 years old with embryonal rhabdomyosarcoma, but no significant improvement was seen from this study. The current COG study is studying a different formulation of vincristine, called vinorelbine (available as a generic drug), combined with dactinomycin and cyclophosphamide. This study is also looking at the length of therapy by including a maintenance phase of low-dose chemotherapy after the first round of treatment.

  • Palliative and 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. For patients with high-risk disease, current research is evaluating this type of targeted therapy, which 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. Studies examining gene translocations are very important. This research can help better classify a disease's risk group as well as improve ways to distinguish the type of rhabdomyosarcoma. Additionally, ongoing studies are collecting and transplanting human rhabdomyosarcoma cells into animal models (called tumor xenografts), which safely permit testing of new experimental 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 the child's own diagnosis, as well as demographic, treatment, and outcome information. Through this study, 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:

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