NCCN Publishes New Guidelines Focusing on Pediatric Rhabdomyosarcoma

The NCCN has recently published new practical guidelines for pediatric soft tissue sarcoma care, according to a press release from the NCCN.^1,2 These guidelines focus specifically on rhabdomyosarcoma (RMS), the most common soft tissue sarcoma in patients younger than 20 years.

Treatment for RMS generally consists of chemotherapy, surgery, and radiation therapy, which all must be adapted to fit the patient’s risk group, age, and developmental stage. The guidelines were curated with the intent to balance the goal of cure with minimizing the risk of acute adverse effects (AEs).

“Most cancers that occur in children are fundamentally different from cancers occurring in adults,” said Stephen Skapek, MD, chief of the Division of Pediatric Hematology-Oncology at Duke Cancer Institute and chair of the NCCN Guidelines Panel for Pediatric Soft Tissue Sarcoma, in the press release.¹ “RMS can be divided into specific subtypes that are driven by different genetic changes that can influence outcomes. Those changes and other clinical and pathologic features are all incorporated into the multifaceted treatments. That’s why it was essential to form a group of leading, multidisciplinary experts from across the country to put together a road map for diagnosing, risk-stratifying, and treating these patients.”

The guidelines offer site-specific considerations, including head or neck mass; soft tissue mass of the extremity; soft tissue mass of the trunk; mass of the vulva, vagina, or uterus fundus/cervix; paratesticular mass; bladder or prostate mass; and biliary/liver mass. For each category, the guidelines include a map of treatments based on various patient characteristics.

Criteria for diagnosis of very low risk, low risk, intermediate risk, and high risk are outlined, with each outlining potential treatment options for most patient statuses.

One section outlines surveillance requirements for patients with no active disease. It includes history and physical examination every 3 to 6 months for 2 to 3 years, then every 6 to 12 months for years 4 and 5, then annually; imaging consisting of primary site and chest CT or chest x-ray every 3 to 6 months for 2 to 3 years, every 6 to 12 months for years 4 and 5, then annually; and whole-body fluorodeoxyglucose-PET imaging as clinically indicated.

Furthermore, the principles of imaging, pathologic assessment, ancillary techniques useful in the diagnosis of RMS, surgery, lymph node disease assessment, radiation therapy, and systemic therapy are among those included in the guidelines.

For systemic therapy, vincristine and dactinomycin (Cosmegen) are prevalent in treatment regimens across very low–, low-, intermediate-, and high-risk RMS, sometimes joined with cyclophosphamide and paired with maintenance therapy.

In a section about the principle of survivorship, where consequential risks of treatment are outlined, some notable factors include vasospastic attacks with chemotherapy. Additionally, the use of radiotherapy may confer a risk of subsequent benign or malignant neoplasm, pulmonary toxicity, cardiac toxicity, functional asplenia, and renal toxicity, among others. Risks associated with surgery include amputation-related complications, cystectomy-related complications, bowel obstruction, urinary incontinence, asplenia, pulmonary dysfunction, and hypothyroidism.

Douglas Hawkins, MD, vice chair of the NCCN panel, group chair of Children’s Oncology Group, professor of hematology-oncology at the University of Washington School of Medicine, and clinician at Seattle Children’s Hospital, added, “Children are most likely to be diagnosed with RMS at a very young age: toddlers or early elementary school. When treating someone so young, you are not looking to just prolong survival; the goal is a full cure with minimal [adverse] effects and zero recurrence.”¹

References

1. New guidelines from NCCN detail fundamental differences in cancer in children compared to adults. News release. NCCN. February 17, 2026. Accessed February 20, 2026. https://tinyurl.com/4hmycssb
2. NCCN. Clinical Practice Guidelines in Oncology. Pediatric soft tissue sarcoma, version 1.2026. Accessed February 20, 2026. https://tinyurl.com/32p4t886