Treatment and outcome of the patients with rhabdomyosarcoma of the biliary tree: Experience of the Cooperative Weichteilsarkom Studiengruppe (CWS)

Abstract

Background 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. Methods In 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). Results Seventeen 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. Conclusion Positive 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.

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

“…Consistent with prior studies, our analysis demonstrates patients with biliary RMS are typically less than 10 years of age with Group III, embryonal disease 4,8,20 . IRS IV was the first study to incorporate staging in addition to grouping as part of RMS risk stratification, and at the time, the biliary tract was considered an unfavorable site for staging 13,21 .…”

“…Meanwhile, in the five patients who were alive with a complete response at the end of therapy, surgery was not radical and was preceded or followed by chemotherapy and RT with no serious sequelae. Additionally, a German cooperative group reported on 12 patients with localized, biliary RMS recommended DPE alone for patients with resectable Group III embryonal disease and DPE followed by RT for patients with Group III alveolar disease or incompletely resectable Group III embryonal disease 8 . Their report demonstrated a 5‐year EFS of 37% (95% CI: 17‐57%); however, direct comparison to our series is difficult as their study included alveolar histology.…”

“…Biliary RMS occurs in young patients with a median age of 3.4 years, and 90% of patients present before 5 years of age with jaundice accompanied by abdominal distention, choluria, acholic stools, nausea, vomiting, or fever that may be mistaken for a choledochal cyst or infectious hepatitis 4–7 . The majority of cases are of embryonal histology 4,8 . Biliary RMS typically arises in the common bile duct but may originate anywhere along the biliary tree 5,6,9 .…”

Biliary Rhabdomyosarcoma in Pediatric Patients: A Systematic Review and Meta-Analysis of Individual Patient Data

“…The prognosis of patients older than 10 years is significantly worse, which has been suggested by other authors before ( 2 , 27 ). We report a higher prevalence of BRMS in male children, which has been indicated by most, but not all case series before ( 2 – 5 ). In contrast to previous assumptions ( 28 ), we found that alveolar histology can be present in BRMS and is associated with poor outcome.…”

Lactate in Sarcoma Microenvironment: Much More than just a Waste Product

“…Although there exist more than 70 subtypes, it is possible to divide them into two main categories: soft tissue sarcomas (STSs), which are derived from fat, muscle, nerve, sheath and blood vessels, representing less than 1% of all new cancer diagnoses, and bone sarcomas [1,2]. Among STSs, liposarcoma (LPS) and undifferentiated pleomorphic sarcoma are the most common subtypes in adults, while rhabdomyosarcoma is one of the highest incidences of tumors in children [3,4]. Concerning bone sarcomas, Ewing's sarcoma (EWS) is one of the most frequently diagnosed in children together with osteosarcoma (OS), which is also a common subtype in adults as well as chondrosarcoma [5].…”