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

Abstract

Objectives To evaluate the impact of local therapies on the outcome of patients with liver‐bile duct rhabdomyosarcoma (LBDRMS). Methods Data of 30 patients included in the EpSSG‐RMS 2005 study were analyzed. Results The median age at diagnosis was 3 years (11 months–8 years). All patients had non‐alveolar histology. Fifteen patients had a tumor > 5 cm and six had enlarged regional lymph nodes on imaging. Eight patients (27%) had primary surgery (1 R0). Six of them received external beam radiotherapy (EBRT). All are in first complete remission (CR1) except one (R1, EBRT+, local relapse, death). Six patients (20%) received EBRT without surgery: one had local relapse and died. Sixteen patients (53%) underwent delayed surgery, with 12 achieving R0 margins, which were higher than those in the primary surgery group (P = 0.003). Three patients with R0 margins received EBRT; one had a metastatic relapse and died. Nine patients with R0 resection did not receive EBRT, three relapsed locally (two deaths). Four R1 patients received additional EBRT without relapses. Local relapse occurred in two among 19 patients with EBRT and three among 11 without EBRT (P = 0.326). At a median follow‐up of 61 months (48–84 months), five patients died; all had a tumor size > 5 cm (P = 0.01). The five‐year overall survival was 85% (95% CI, 65–94), and event‐free survival was 76% (95% CI, 54–89). Conclusion This analysis did not show any significant difference in outcome between irradiated and nonirradiated patients. Local relapse in LBDRMS is related to initial tumor size and is often fatal.

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

“…This is the first systematic review and meta-analysis investigating biliary tract rhabdomyosarcoma in children. The number of included patients is more than five times higher than the largest reported cohort so far ( 3 ), thus providing comparatively strong evidence for this very rare condition.…”

“…Studies were included upon reception of sufficient information. Three studies that reported exclusively on patients with BRMS, but provided only limited access to IPD, were included for the pooled data analysis but were not suited for IPD analyses ( 1 – 3 ). In 11 cases, the same patient was reported by more than one case report or study.…”

“…A major focus of this study was to investigate the role of surgery in the therapy of BRMS, which has been discussed controversially in recent years ( 1 – 3 , 5 , 29 ). Our results suggest that surgical tumor resection should be a mainstay in the treatment of BRMS in children.…”

“…Tumors were staged according to previously investigated risk factors in pediatric BRMS, i.e., tumor size (≤ 5 cm vs. > 5 cm), nodal involvement, and presence of distant metastases ( 3 , 5 ). Patients were classified into clinical risk groups after initial surgery or biopsy using the Intergroup Rhabdomyosarcoma Study Group (IRS) guidelines (I: Complete surgical resection (R0); II: Microscopically incomplete surgical resection (R1); III: Gross tumor residual after surgery/biopsy (R2); IV: Metastatic disease) ( 22 ).…”

“…This scepsis may be based on the observation of complete remission in some patients with BRMS after sole chemotherapeutic treatment. In contrast, other authors emphasized the advantages of complete surgical tumor resection in pediatric RMS ( 12 , 13 ) and the need for local therapies for BRMS even in complete remission after chemotherapy ( 3 ). However, reliable evidence is missing.…”