Background The biliary tree is a rare location of pediatric rhabdomyosarcoma. Due to the low
incidence, there is a lack of evidence concerning therapeutic guidelines for this
tumor location. In particular, the impact of surgery is discussed controversially.
Purpose Objective is to generate evidence-based treatment guidelines for pediatric biliary
rhabdomyosarcoma (BRMS). All available published data on therapeutic regimens and
important prognostic factors are investigated with a focus on the role of surgery.
Methods A systematic literature search of MEDLINE, Web of Science, and CENTRAL was performed.
Patient data were entered individually. Data was pooled and qualitative and quantitative
analyses of demographic data, therapy, postoperative/interventional outcomes, relapse,
and survival were conducted. In an individual patient data analysis, cox regression
was applied to identify key factors predicting the outcome of patients with BRMS.
Results 65 studies met the inclusion criteria, providing data on 176 patients with BRMS.
Individual patient data analysis showed a 5-year overall survival of 51% for the total
study population. For patients treated after 2000, 5-year OS was 65%. Absence of surgical
tumor resection was an independent risk factor for death (Hazard ratio 8.9, 95%-CI
1.8-43.6, p = 0.007) and significantly associated with recurrent disease.
Conclusion This analysis provides comprehensive information on the largest number of patients
with hitherto reported in the literature. BRMS is still associated with high morbidity
and mortality. Surgical tumor resection is essential for appropriate oncological treatment
of BRMS. International cooperation studies are needed to enhance evidence and improve
the outcome of his orphan disease.
