How is pediatric rhabdomyosarcoma (RMS) treated?

Updated: Mar 28, 2019
  • Author: Roshni Dasgupta, MD, MPH; Chief Editor: Eugene S Kim, MD, FACS, FAAP  more...
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Any child with a suspected rhabdomyosarcoma (RMS) requires a tissue diagnosis confirmation and surgical staging. Thus, early surgical consultation is mandatory to allow accurate diagnosis and treatment planning.

In general, a small resectable tumor of nongenitourinary origin may be treated initially with complete resection. Contraindications for initial surgical excision include unresectable disease outside of the pelvis or retroperitoneum and disease that would necessitate disfiguring or disabling resection. A tumor that is unresectable or is resectable only through mutilating surgery may be treated with incisional biopsy or core needle biopsy (CNB), followed by chemotherapy, radiation therapy (RT), or both, as well as possible definitive surgical resection.

Wide local excision is avoided in head and neck tumors, particularly if excision will result in a significant cosmetic or functional defect. Orbital exenteration for orbital RMS should be performed only for local recurrence. Vaginal or uterine RMS in the pediatric patient is treated with biopsy, followed by chemotherapy with or without radiation and then second-look surgery. Residual disease may necessitate partial vaginectomy.

Primary bladder tumors are no longer treated with anterior pelvic exenteration. Instead, chemotherapy and, occasionally, RT for persistent disease are used. [28] This therapy has allowed a functional bladder to be retained in 60% of patients 4 years after diagnosis, with a survival rate of 89%.

Whereas earlier studies suggested that prognosis was not improved by a resection that did not remove all gross disease, subsequent studies suggested that pretreatment debulking of 50% or more of the tumor volume in patients with retroperitoneal and pelvic RMS may result in superior failure-free survival. [23]

The surgeon should evaluate the draining lymph node basin for selected sites (eg, extremity, trunk, or paratesticular location). The Intergroup Rhabdomyosarcoma Study (IRS) Group (IRSG) recommends aggressive nodal sampling; however, the surgeon may also consider lymph node mapping and sentinel lymph node biopsy (SLNB) with preoperative lymphoscintigraphy. [29, 30]

Certain sites (eg, extremity, trunk, and paratesticular) have a predilection for lymphatic metastases. Accordingly, SLNB with preoperative lymphoscintigraphy is recommended for these sites and has been found to be superior to positron emission tomography (PET) alone. However, these recommended protocols often are not followed, and this failure of procedure has an impact on patient survival. [24]

Investigations are under way through the Soft Tissue Sarcoma Committee of the Children’s Oncology Group. These studies seek to verify the stratification-based treatment algorithm. Future studies will aim to continue to improve survival while minimizing the morbidity and mortality resulting not only from the malignancy but also from its treatment.

In addition, it should be remembered that local control is achieved by a combination of resection and RT. Outcomes are best when both modalities are employed. If RT is to be held or diminished out of concern for long-term morbidity, it is important for the surgeon to utilize resection as the primary form of local control.

Novel therapies, including oncolytic viruses, dendritic-cell vaccines, and monoclonal antibodies, are under current clinical investigation.

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