Multimodality Treatment Linked to High OS Rate in Pediatric RMS of Head and Neck

An adjusted combination of chemotherapy, radiation therapy, and surgery induced a 5-year overall survival (OS) rate of 91.3% in pediatric patients with rhabdomyosarcomas (RMS) of the head and neck.

RMS of the head and neck is a rare differential diagnosis in head and neck tumors and may present with painless swelling, bloody secretion, dysphagia, exophthalmos, and vision impairment. In most cases, treatment of RMS of the head and neck combines polychemotherapy, radiotherapy, and surgery.

In a small (N = 28), 20-year retrospective study of patients with a mean age of 6.8 ± 5.0 years at diagnosis, researchers found that treatment with a combination of polychemotherapy, radiotherapy and surgery resulted in “excellent” OS and a median progression-free survival (PFS) of 46.0 ± 67.4 months (range, 8-251 months). Data was collected from patients treated at the Charité, Universitätsmedizin Berlin from May 1996 to June 2016. Data from patients’ health records and the German Childhood Cancer Registry were also reviewed.

Nine patients (32.1%) presented with cranial nerve deficit at initial diagnosis, including vision impairment by optic neuropathy (n = 6) and dysphagia by glossopharyngeal and vagus nerve deficit (n = 2). Fourteen patients (50%) presented with painless swelling, 2 patients (7.1%) experienced bleeding, and 2 (7.1%) had recurrent infections at the tumor site.

Most patients had embryonal disease (85.7%), followed by alveolar subtype (10.7%). One patient presented with unknown histological subtype (3.6%), and researchers did not observe any anaplastic or mixed-type RMS in the cohort. Seventeen patients (60.7%) were male and 11 (39.3%) were female.

Five patients (17.9%) had IRS stage 1 disease, 14.3% were stage 2, 50% were stage 3; 10.7% had stage 4 tumors, and 2 patients (7.1%) could not be assigned an IRS stage. The most common tumor locations were orbital (32.1%), parapharyngeal (14.2%), and paranasal sinuses (10.7%).

All patients received polychemotherapy. Twenty-four patients (85.7%) underwent adjuvant radiotherapy, 10 (35.7%) of whom had undergone previous surgical resection.

Ten patients (35.7%) received conventional radiation therapy, 14.3% received 3D conformal radiation therapy, 10.7% received proton beam therapy, and 7.1% were treated with intensity modulated radiation therapy. Another 5 patients (17.9%) underwent radiation therapy at another hospital.

Twelve patients (42.9%) received initial surgery with no clear resection margins on histopathological examination.

Twenty-six patients were included in the survival analysis. Two patients were lost to follow-up.

Researchers found no significant difference in OS for patients aged 0 to 10 years compared with those 10 to 18 years (P = .079). There was also no significant difference in OS for female versus male patients (P = .242).

Researchers also found no significant OS difference between nonparameningeal and parameningeal RMS (P = .637).

Three patients (10.7%) died of tumor progression during a mean follow-up time of 56.2 months (4.7 years)—2 had a RMS with parameningeal growth, and 1 had a RMS with nonparameningeal localization.

Seven patients (25.0%) experienced a local recurrence after a median time of 14.5 months (range, 8-40). Local recurrence was treated with radiation therapy, radiation and polychemotherapy, and polychemotherapy in 2 patients each. In 1 patient, local recurrence was treated with surgery and adjuvant irradiation.

Common side effects associated with chemotherapy and radiation therapy included neutropenia, mucositis, nausea and vomiting, and radiodermatitis. One patient had a secondary malignancy, a radiation-induced sarcoma of the right pterygopalatine fossa treated with ifosfamide, topotecan, and cyclophosphamide.

Häußler SM, Stromberger C, Olze, et al. Head and neck rhabdomyosarcoma in children: a 20-year retrospective study at a tertiary referral center [published online November 16, 2017]. J Cancer Res Clin Oncol. doi: 10.1007/s00432-017-2544-x.