European Soft Tissue Sarcoma Group Research Articles

Rhabdomyosarcoma of the biliary tract: a case report

Rhabdomyosarcoma (RMS) of the biliary tract is a rare tumor that commonly arises from the common bile duct. The most common clinical symptoms are obstructive jaundice and abdominal pain. Although diagnosis is often difficult and is frequently made during surgery, diagnostic imaging techniques including ultrasound, computerized tomography scan, and magnetic resonance cholangiopancreatography remain useful in the diagnosis and evaluation of biliary tree anatomy. In order to improve prognosis, different rhabdomyosarcoma study groups have adopted multidisciplinary treatment approach. Herein we describe a case of three-year-old child with Embryonal rhabdomyosarcoma originating in the common bile duct who was treated with surgery, chemotherapy according to European soft tissue sarcoma group (EpSSG) protocol and adjuvant postoperative intensity modulated radiotherapy to surgical bed with 6 MV photons to a dose of 41, 4Gy in 23 fractions. One year and a half after the end of therapy, the patient is still disease free. Although Rhabdomyosarcoma of the biliary tract is a rare tumor, it should be considered in the differential diagnosis of patients who have obstructive jaundice and a cystic mass within the common bile duct. Once believed to be an incurable disease, the prognosis of patients with biliary rhabdomyosarcoma has improved with a multidisciplinary treatment approach.

A phase II study evaluating addition of bevacizumab to chemotherapy in childhood and adolescent patients presenting with metastatic rhabdomyosarcoma (RMS) and non-rhabdomyosarcoma soft tissue sarcoma (NRSTS).

TPS330 Background: Goals of this study, conduct of which was agreed with the European Medicines Evaluation Agency, are to evaluate the efficacy and safety profile of bevacizumab (Bv) administered with chemotherapy in metastatic RMS and NRSTS in childhood and adolescence, and to characterize the pharmacokinetic (PK) profile of Bv across all age subsets of the study population. Despite therapeutic advances, outcomes of metastatic RMS and NRSTS remain poor. Preclinical reports implicate angiogenesis in the pathogenesis of these tumors (Cancer Res 2006; 66:8770-78). Bevacizumab (Bv), a recombinant humanized monoclonal antibody to VEGF (Cancer Res 1997; 57:4593-99), is approved in several oncology indications in adults. A recent phase I study (J Clin Oncol 2008; 26:399-405) indicated that Bv is well tolerated in children with refractory solid tumors and yielded PK data that support further studies of Bv in childhood cancer. Methods: Approximately 150 patients, aged from > 6 months to < 18 years, who present with metastatic RMS or NRSTS, will be randomized to receive 18 months of standard combined modality therapy, as currently recommended by the European Soft Tissue Sarcoma Group, either alone or with Bv. Treatment will be in two phases: induction therapy [9 three-weekly cycles including 4 cycles of IVADo (ifosfamide, vincristine, actinomycin D and doxorubicin) followed by 5 cycles of IVA (i.e., without doxorubicin)] and maintenance therapy (twelve 4-weekly cycles of vinorelbine and cyclophosphamide). Local therapy will be considered after the 6th induction cycle. Primary endpoint will be event-free survival. PK sampling will be performed on all patients randomized to the experimental arm during the first 4 cycles of induction. After the primary efficacy and safety analysis, all patients who have not met the primary endpoint will be followed for at least 47 months for survival and long-term effects of treatment. The study enrolled 17 patients between July 2008 and December 2009. Author Disclosure Employment or Leadership Position Consultant or Advisory Role Stock Ownership Honoraria Research Funding Expert Testimony Other Remuneration Roche