Pencil Beam Scanning Proton Therapy for Rhabdomyosarcoma of the Biliary Tract

Pater, Luke; Turpin, Brian; Mascia, Anthony

doi:10.7759/cureus.1747

Cited by 4 publications

(2 citation statements)

References 9 publications

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“…In addition to VAC, patients with nonbiliary RMS treated with VA with or without RT on D9602 also developed SOS 27 . RT itself is also a risk factor for the development of SOS 28 . Both patients in our analysis who developed moderate to severe SOS had Group III disease and were less than 36 months of age.…”

Section: Discussionmentioning

confidence: 74%

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

Aye

Xue

Palmer

et al. 2021

Pediatric Blood & Cancer

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Background Biliary rhabdomyosarcoma (RMS) is the most common biliary tumor in children. The biliary tract is classified as a favorable primary site. Therefore, patients with localized biliary RMS were included in two consecutive low‐risk studies, D9602 and ARST0331, by the Children's Oncology Group (COG). The outcome for these patients treated with low‐risk therapy has not been reported. Procedure Patients with biliary RMS enrolled on COG low‐risk trials D9602 or ARST0331 were analyzed. All patients received systemic chemotherapy and those with Group II (microscopic residual) or Group III (macroscopic residual) disease received 36‐50.4 Gy adjuvant radiotherapy (RT). Delayed primary excision (DPE) was allowed on both studies. Results Seventeen patients with biliary RMS were treated on D9602 (n = 7) or ARST0331 (n = 10). Median age was 3.5 years (range 1.7‐10.3). Ten (59%) patients had tumors >5 cm and 14 (82%) had Group III disease. Fifteen (88%) patients received RT. The 5‐year event‐free survival (EFS) and overall survival (OS) were 70.6% (95% confidence interval [CI]: 46.9‐94.3%) and 76.5% (95% CI: 54.6‐98.4%), respectively. The majority of patients (80%) who received RT did not have disease recurrence while both patients who did not receive RT had local relapse. Five (36%) of 14 patients with Group III disease underwent DPE; two experienced a local relapse. In the nine patients without DPE, two developed local relapse. Conclusions Patients with localized biliary RMS treated on low‐risk studies had suboptimal outcomes. These patients may benefit from therapy on intermediate‐risk studies.

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Section: Discussionmentioning

confidence: 74%

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

Aye

Xue

Palmer

et al. 2021

Pediatric Blood & Cancer

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“…On the contrary, surgery with intent of tumor resection should be evaluated for all patients with BRMS. On the other hand, complete remission and long-term survival without any surgical tumor resection has been reported for BRMS ( 4 , 5 , 30 ). Predictors or risk factors that allow for an identification of those patients who do not need surgery have not been determined yet ( 28 , 31 ).…”

Section: Discussionmentioning

confidence: 99%

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

et al. 2021

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BackgroundThe 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.PurposeObjective 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.MethodsA 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.Results65 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.ConclusionThis 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 RegistrationPROSPERO (CRD42021228911) https://www.crd.york.ac.uk/prospero/display_record.php?ID=CRD42021228911.

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Rhabdomyosarcoma of the biliary tract in a child: a case report

Ran,

Gong,

Rui

et al. 2024

Front. Pediatr.

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Pediatric rhabdomyosarcoma of the biliary tract (BRMS) is extremely rare. Here, we present a case of a 2-year-old child who was initially misdiagnosed with choledocholithiasis upon admission. The diagnosis was later confirmed as BRMS through endoscopic retrograde cholangiopancreatography (ERCP). The patient was cured through surgery followed by chemotherapy. Due to the lack of specific early symptoms and the challenges in imaging differentiation, particularly in pediatric patients, clinical awareness of this condition needs to be heightened. Our findings indicate that ERCP is currently the optimal diagnostic tool for this disease, and a combination of surgery and chemotherapy can yield better therapeutic outcomes.

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Pencil Beam Scanning Proton Therapy for Rhabdomyosarcoma of the Biliary Tract

Cited by 4 publications

References 9 publications

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

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

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

Rhabdomyosarcoma of the biliary tract in a child: a case report

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