Abstract
BackgroundBiliary rhabdomyosarcoma (RMS) is the most common biliary tumor in children. The management of affected patients contains unique challenges because of the rarity of this tumor entity and its critical location at the porta hepatis, which can make achievement of a radical resection very difficult.MethodsIn a retrospective chart analysis we analysed children suffering from biliary RMS who were registered in three different CWS trials (CWS-96, CWS-2002P, and SoTiSaR registry).ResultsSeventeen patients (12 female, 5 male) with a median age of 4.3 years were assessed. The median follow-up was 42.2 months (10.7–202.5). The 5-year overall (OS) and event free survival (EFS) rates were 58% (45–71) and 47% (34–50), respectively. Patients > 10 years of age and those with alveolar histology had the worst prognosis (OS 0%). Patients with botryoid histology had an excellent survival (OS 100%) compared to those with non-botryoid histology (OS 38%, 22–54, p = 0.047). Microscopic complete tumor resection was achieved in almost all patients who received initial tumor biopsy followed by chemotherapy and delayed surgery.ConclusionPositive predictive factors for survival of children with biliary RMS are age ≤ 10 years and botryoid tumor histology. Primary surgery with intention of tumor resection should be avoided.
Highlights
Biliary rhabdomyosarcoma (RMS) is the most common biliary tumor in children
The objective of this study was to analyze the data of patients suffering from biliary RMS who were treated within two different Cooperative Weichteilsarkom Studiengruppe (CWS) trials and Soft Tissue Sarcoma Registry (SoTiSaR) (Soft Tissue Sarcoma) registry with regard to treatment concepts and outcome
Since 1981, 17 (5 male, 12 female) out of > 3500 patients (0.5%) with soft tissue sarcoma registered in the prospective CWS trials and in the SoTiSaR registry suffered from RMS of the biliary tree and fulfilled the inclusion criteria for this analysis (Table 1)
Summary
Biliary rhabdomyosarcoma (RMS) is the most common biliary tumor in children. The management of affected patients contains unique challenges because of the rarity of this tumor entity and its critical location at the porta hepatis, which can make achievement of a radical resection very difficult. Rhabdomyosarcoma (RMS) is the most common soft tissue sarcoma of the childhood, accounting for about 5% of all pediatric malignancies [1]. Rhabdomyosarcoma of the biliary tree represents only about 0.5% of all pediatric RMS [1, 2]. Biliary RMS usually arises in the common bile duct, but it can originate from anywhere along the biliary tree [1,2,3,4]. Late recognition of the biliary tree malignancy, its critical location, and frequent extension into the liver are the main factors responsible for diminished survival. While some authors recommend aggressive surgical resection of the tumor [4, 5], others questioned the necessity of aggressive surgical excision for this type of tumors [1].
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