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Successful treatment of relapsed testicular embryonal rhabdomyosarcoma with Endostar and traditional chemotherapy: a case report.

ABSTRACT: Embryonal rhabdomyosarcoma (ERMS) has a low prevalence, poor prognosis, and limited treatment efficacy. We report a case of an 18-year-old male whose disease relapsed in the abdominal cavity after a testicular ERMS curative resection. The patient received eight sequential cycles of rescue therapy using cisplatin and isocyclophosphamide in combination with a vascular targeted drug, Endostar. The therapeutic effect of the combination regimen has been evaluated for complete response. This is the first case to report using Endostar and chemotherapy in relapsed ERMS, and the curative effect results in complete response. Endostar, a new vascular targeted drug, combined with chemotherapy may play a synergistic role and provide a reference for the treatment of ERMS.

SUBMITTER: Han T

PROVIDER: S-EPMC6124800 | biostudies-literature | 2018

REPOSITORIES: biostudies-literature

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Successful treatment of relapsed testicular embryonal rhabdomyosarcoma with Endostar and traditional chemotherapy: a case report.

Han Tao T Chen Jianjun J Luan Yuting Y Chen Xiaoxia X Yang Xiaodan X Zhang Yue Y Li Gao G Wang Di D Zheng Zhendong Z

OncoTargets and therapy 20180830

Embryonal rhabdomyosarcoma (ERMS) has a low prevalence, poor prognosis, and limited treatment efficacy. We report a case of an 18-year-old male whose disease relapsed in the abdominal cavity after a testicular ERMS curative resection. The patient received eight sequential cycles of rescue therapy using cisplatin and isocyclophosphamide in combination with a vascular targeted drug, Endostar. The therapeutic effect of the combination regimen has been evaluated for complete response. This is the fi ...[more]

PMID: 30214234

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Project description:BackgroundPatients with alveolar rhabdomyosarcoma (ARMS) have inferior outcomes compared to patients with embryonal rhabdomyosarcoma (ERMS) and more effective chemotherapy options are needed for these patients. Vinorelbine is a semisynthetic vinca alkaloid that has clinical activity in relapsed rhabdomyosarcoma (RMS) when used alone or in combination with cyclophosphamide.AimsThe goal of our study was to evaluate whether RMS histology subtype influences response rate to vinorelbine alone or in combination.Materials & methodsFive Phase 2 trials that enrolled RMS patients were included in the meta-analysis. Two studies evaluated vinorelbine alone, two studies evaluated vinorelbine in combination with low dose oral cyclophosphamide, and one study evaluated vinorelbine and intravenous cyclophosphamide in combination with temsirolimus or bevacizumab. All RMS patients had relapsed or refractory disease and had received at least one prior therapy. Response was reported according to RECIST1.1 and was defined as a complete or partial response. Response data was obtained from published results or from trial principal investigator. RMS NOS patients were grouped with ERMS patients for this analysis. Summary estimates comparing differences between ARMS and ERMS response rates were generated using a random-effects model to account for heterogeneity among the studies.ResultsOne hundred fifty-six enrolled patients evaluable for response were included in the meta-analysis, 85 ARMS, 64 ERMS and 7 RMS-NOS. The combined effect generated from the random-effects model demonstrated a 41% increase (p = 0.001, 95% CI; 0.21-0.60) in response to vinorelbine as a single agent or in combination in patients with ARMS compared to patients with ERMS. There was no significant difference in the rate of progressive disease between patients with ARMS compared to ERMS (p = 0.1, 95%CI; -0.26-0.02).DiscussionVinorelbine is an active agent for the treatment of relapsed or refractory RMS and a meta-analysis of Phase 2 studies shows that radiographic responses in patients with ARMS were significantly higher than ERMS or RMS-NOS.ConclusionThese data support further investigation of vinorelbine in newly diagnosed patients with RMS particularly those with alveolar histology.

Project description:BACKGROUND:Rhabdomyosarcoma (RMS) is the most common soft tissue sarcoma in the pediatric cancer population. Survival among metastatic RMS patients has remained dismal yet unimproved for years. We previously identified the class I-specific histone deacetylase inhibitor, entinostat (ENT), as a pharmacological agent that transcriptionally suppresses the PAX3:FOXO1 tumor-initiating fusion gene found in alveolar rhabdomyosarcoma (aRMS), and we further investigated the mechanism by which ENT suppresses PAX3:FOXO1 oncogene and demonstrated the preclinical efficacy of ENT in RMS orthotopic allograft and patient-derived xenograft (PDX) models. In this study, we investigated whether ENT also has antitumor activity in fusion-negative eRMS orthotopic allografts and PDX models either as a single agent or in combination with vincristine (VCR). METHODS:We tested the efficacy of ENT and VCR as single agents and in combination in orthotopic allograft and PDX mouse models of eRMS. We then performed CRISPR screening to identify which HDAC among the class I HDACs is responsible for tumor growth inhibition in eRMS. To analyze whether ENT treatment as a single agent or in combination with VCR induces myogenic differentiation, we performed hematoxylin and eosin (H&E) staining in tumors. RESULTS:ENT in combination with the chemotherapy VCR has synergistic antitumor activity in a subset of fusion-negative eRMS in orthotopic "allografts," although PDX mouse models were too hypersensitive to the VCR dose used to detect synergy. Mechanistic studies involving CRISPR suggest that HDAC3 inhibition is the primary mechanism of cell-autonomous cytoreduction in eRMS. Following cytoreduction in vivo, residual tumor cells in the allograft models treated with chemotherapy undergo a dramatic, entinostat-induced (70-100%) conversion to non-proliferative rhabdomyoblasts. CONCLUSION:Our results suggest that the targeting class I HDACs may provide a therapeutic benefit for selected patients with eRMS. ENT's preclinical in vivo efficacy makes ENT a rational drug candidate in a phase II clinical trial for eRMS.

Dataset Information

Successful treatment of relapsed testicular embryonal rhabdomyosarcoma with Endostar and traditional chemotherapy: a case report.

Publications

Successful treatment of relapsed testicular embryonal rhabdomyosarcoma with Endostar and traditional chemotherapy: a case report.

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