Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
. 2021 Sep 30:11:701400.
doi: 10.3389/fonc.2021.701400. eCollection 2021.

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

Juri Fuchs  1 , Anastasia Murtha-Lemekhova  1 , Markus Kessler  2 , Patrick Günther  2 , Alexander Fichtner  3 , Jan Pfeiffenberger  4 , Pascal Probst  1   5 , Katrin Hoffmann  1
Affiliations

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

Juri Fuchs et al. Front Oncol. .

Abstract

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 and progression-free survival of 51% and 50% for the total study population. For patients treated after 2000, 5-year OS and PFS was 65% and 59%, respectively. 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 and disease-related death.

Conclusion: This analysis provides comprehensive information on the largest number of patients 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 this orphan disease.

Protocol registration: PROSPERO (CRD42021228911) https://www.crd.york.ac.uk/prospero/display_record.php?ID=CRD42021228911.

Keywords: RELIVE initiative; biliary rhabdomyosarcoma; pediatric hepatobiliary surgery; pediatric liver surgery; pediatric liver tumors; pediatric oncology; pediatric rhabdomyosarcoma.

PubMed Disclaimer

Conflict of interest statement

The authors declare that the research was conducted in the absence of any commercial or financial relationships that could be construed as a potential conflict of interest.

Figures

Figure 1
Figure 1
PRISMA flow chart of the study selection and inclusion process.
Figure 2
Figure 2
(A) OS of the complete study population; (B) PFS of the complete study population; (C) comparing OS depending on the type of surgery showing significant higher OS in patients with limited or extended surgery compared to no surgery (95%-CI); (D) comparing OS of patients with surgery plus EBRT with those who received surgery without EBRT – no significant difference in OS between the two groups (95%-CI).
Figure 3
Figure 3
Cox proportional hazards model for risk of death, adjusted for 11 covariates. Numbers in paratheses represent 95%-CI. * marks significant results.
See this image and copyright information in PMC

References

    1. Aye JM, Xue W, Palmer JD, Walterhouse DO, Arnold MA, Heaton TE, et al. . Suboptimal Outcome for Patients With Biliary Rhabdomyosarcoma Treated on Low-Risk Clinical Trials: A Report From the Children's Oncology Group. Pediatr Blood Cancer (2021) 68(4):e28914. doi: 10.1002/pbc.28914 - DOI - PMC - PubMed
    1. Urla C, Warmann SW, Sparber-Sauer M, Schuck A, Leuschner I, Klingebiel T, et al. . Treatment and Outcome of the Patients With Rhabdomyosarcoma of the Biliary Tree: Experience of the Cooperative Weichteilsarkom Studiengruppe (CWS). BMC Cancer (2019) 19(1):945. doi: 10.1186/s12885-019-6172-5 - DOI - PMC - PubMed
    1. Guérin F, Rogers T, Minard-Colin V, Gaze MN, Terwisscha S, Van Noesel M, et al. . Outcome of Localized Liver-Bile Duct Rhabdomyosarcoma According to Local Therapy: A Report From the European Paediatric Soft-Tissue Sarcoma Study Group (EpSSG)-RMS 2005 Study. Pediatr Blood Cancer (2019) 66(7):e27725. doi: 10.1002/pbc.27725 - DOI - PubMed
    1. Perruccio K, Cecinati V, Scagnellato A, Provenzi M, Milano GM, Basso E, et al. . Biliary Tract Rhabdomyosarcoma: A Report From the Soft Tissue Sarcoma Committee of the Associazione Italiana Ematologia Oncologia Pediatrica. Tumori (2018) 104(3):232–7. doi: 10.5301/tj.5000692 - DOI - PubMed
    1. Spunt SL, Lobe TE, Pappo AS, Parham DM, Wharam MD, Jr, Arndt C, et al. . Aggressive Surgery is Unwarranted for Biliary Tract Rhabdomyosarcoma. J Pediatr Surg (2000) 35(2):309–16. doi: 10.1016/s0022-3468(00)90030-7 - DOI - PubMed

Publication types