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Clinical Trial
. 2021 Apr;68(4):e28914.
doi: 10.1002/pbc.28914. Epub 2021 Jan 26.

Suboptimal outcome for patients with biliary rhabdomyosarcoma treated on low-risk clinical trials: A report from the Children's Oncology Group

Jamie M Aye  1 , Wei Xue  2 , Joshua D Palmer  3 , David O Walterhouse  4 , Michael A Arnold  5   6 , Todd E Heaton  7 , Rajkumar Venkatramani  8
Affiliations
Clinical Trial

Suboptimal outcome for patients with biliary rhabdomyosarcoma treated on low-risk clinical trials: A report from the Children's Oncology Group

Jamie M Aye et al. Pediatr Blood Cancer. 2021 Apr.

Abstract

Background: Biliary rhabdomyosarcoma (RMS) is the most common biliary tumor in children. The biliary tract is classified as a favorable primary site. Therefore, patients with localized biliary RMS were included in two consecutive low-risk studies, D9602 and ARST0331, by the Children's Oncology Group (COG). The outcome for these patients treated with low-risk therapy has not been reported.

Procedure: Patients with biliary RMS enrolled on COG low-risk trials D9602 or ARST0331 were analyzed. All patients received systemic chemotherapy and those with Group II (microscopic residual) or Group III (macroscopic residual) disease received 36-50.4 Gy adjuvant radiotherapy (RT). Delayed primary excision (DPE) was allowed on both studies.

Results: Seventeen patients with biliary RMS were treated on D9602 (n = 7) or ARST0331 (n = 10). Median age was 3.5 years (range 1.7-10.3). Ten (59%) patients had tumors >5 cm and 14 (82%) had Group III disease. Fifteen (88%) patients received RT. The 5-year event-free survival (EFS) and overall survival (OS) were 70.6% (95% confidence interval [CI]: 46.9-94.3%) and 76.5% (95% CI: 54.6-98.4%), respectively. The majority of patients (80%) who received RT did not have disease recurrence while both patients who did not receive RT had local relapse. Five (36%) of 14 patients with Group III disease underwent DPE; two experienced a local relapse. In the nine patients without DPE, two developed local relapse.

Conclusions: Patients with localized biliary RMS treated on low-risk studies had suboptimal outcomes. These patients may benefit from therapy on intermediate-risk studies.

Keywords: biliary rhabdomyosarcoma; chemotherapy; radiotherapy; surgery.

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Conflict of interest statement

Conflict of Interest

The authors declare no conflicts of interest. The content is solely the responsibility of the authors and does not necessarily represent the official views of the National Institutes of Health.

Figures

FIGURE 1
FIGURE 1
Event-free and overall survival.
FIGURE 2
FIGURE 2
Event-free and overall survival based on tumor size.
See this image and copyright information in PMC

Comment in

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