Clinical Sarcoma Research最新文献

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Dedifferentiated chondrosarcoma of the pelvis: clinical outcomes and current treatment. 骨盆去分化软骨肉瘤:临床结果和目前的治疗。
Clinical Sarcoma Research Pub Date : 2018-12-14 eCollection Date: 2018-01-01 DOI: 10.1186/s13569-018-0110-1
Johnathan R Lex, Scott Evans, Jonathan D Stevenson, Michael Parry, Lee M Jeys, Robert J Grimer
{"title":"Dedifferentiated chondrosarcoma of the pelvis: clinical outcomes and current treatment.","authors":"Johnathan R Lex,&nbsp;Scott Evans,&nbsp;Jonathan D Stevenson,&nbsp;Michael Parry,&nbsp;Lee M Jeys,&nbsp;Robert J Grimer","doi":"10.1186/s13569-018-0110-1","DOIUrl":"https://doi.org/10.1186/s13569-018-0110-1","url":null,"abstract":"<p><strong>Background: </strong>Dedifferentiated chondrosarcomas (CS) are a high-grade variant of CS that confers a 5-year survival of around 10-24%. Dedifferentiated CS arising from the pelvis confers an even worse prognosis.</p><p><strong>Questions: </strong>(1) What is the prognosis of patients with dedifferentiated CS of the pelvis? (2) Do wide margins or type of surgical intervention influence outcome? (3) Does the use of adjuvant therapy affect outcome?</p><p><strong>Methods: </strong>Patients were retrospectively reviewed from a prospectively collated musculoskeletal oncology database from 1995 to 2016. Thirty-one cases of dedifferentiated CS arising from the pelvis were included. Wide margins were defined as greater than 4 mm. The mean age was 55.6 years (range 33 to 76 years) and there were 19 males (61.3%) and 12 females (38.7%).</p><p><strong>Results: </strong>The disease presented at a locally or systemically advanced stage in 13 patients (41.9%). Eighteen patients (58.1%) underwent surgery with curative intent. Overall survival at 12 months was 15.4% for patients treated with palliative intent and 50% for those treated with surgery. In the surgical group, there were higher rates of disease-free survival in patients who underwent hindquarter amputation and those who received wide surgical margins (p = 0.047 and p = 0.019, respectively). Those who underwent hindquarter amputation were more likely to achieve wide margins (p = 0.05). Time to recurrent disease (local or systemic) was always less than 24 months. No hindquarter amputation for recurrent disease resulted in disease-free survival. No patient who received adjuvant therapy for palliative or recurrent disease had disease control.</p><p><strong>Conclusions: </strong>Pelvic dedifferentiated CS often presents at an advanced local or systemic stage and confers a poor prognosis. Achieving wide surgical margins (> 4 mm) provided the highest rate of long-term disease-free survival. Failing to achieve wide margins results in rapid disease recurrence, conferring deleterious consequences.</p>","PeriodicalId":10684,"journal":{"name":"Clinical Sarcoma Research","volume":"8 ","pages":"23"},"PeriodicalIF":0.0,"publicationDate":"2018-12-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1186/s13569-018-0110-1","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"36790952","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 22
Extraskeletal myxoid chondrosarcoma with massive pulmonary metastases. 骨外黏液样软骨肉瘤伴大量肺转移。
Clinical Sarcoma Research Pub Date : 2018-12-05 eCollection Date: 2018-01-01 DOI: 10.1186/s13569-018-0108-8
Luca Paoluzzi, Munir Ghesani
{"title":"Extraskeletal myxoid chondrosarcoma with massive pulmonary metastases.","authors":"Luca Paoluzzi,&nbsp;Munir Ghesani","doi":"10.1186/s13569-018-0108-8","DOIUrl":"https://doi.org/10.1186/s13569-018-0108-8","url":null,"abstract":"<p><strong>Background: </strong>Extraskeletal myxoid chondrosarcoma (EMC) is a rare malignant mesenchymal neoplasm of uncertain differentiation characterized by rearrangements of the NR4A3 gene. EMC often affects adults around the age of 50 and arise in the deep tissues of the proximal extremities and limb girdles. EMC is characterized by indolent growth rate but strong tendency to local recurrence and metastatic spread. No systemic treatment is specifically approved by the FDA for this disease and surgery has been traditionally the only potentially curative strategy.</p><p><strong>Case presentation: </strong>A 41-year-old Caucasian woman originally presented with a 14.8 cm left thigh mass. She was managed with wide local resection but after 2 years she developed recurrent disease in the pelvis and in the lungs; the lung involvement was characterized by innumerable nodules without any significant respiratory symptoms. After failing three clinical trials, she experienced prolonged disease control while on treatment with the tyrosine kinase inhibitor (TKI) pazopanib and radiation therapy delivered to the pelvic lesion. Dose reduction of pazopanib due to severe diarrhea was followed by rapid disease progression in the pelvis requiring vascular stenting; increase in tumor growth after discontinuation of a TKI has been described in other malignancies and is a possibility in this specific patient.</p><p><strong>Conclusion: </strong>While surgical management of EMC with or without radiation therapy is still the preferable approach when feasible, small series support the use of tyrosine kinase inhibitors and possible new immunotherapies in selected patients. Basket trials focusing on diseases with unique genomic features such as EMC will hopefully provide a better understanding of new options for care.</p>","PeriodicalId":10684,"journal":{"name":"Clinical Sarcoma Research","volume":"8 ","pages":"20"},"PeriodicalIF":0.0,"publicationDate":"2018-12-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1186/s13569-018-0108-8","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"36758164","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 7
A phase II study of temsirolimus and liposomal doxorubicin for patients with recurrent and refractory bone and soft tissue sarcomas. 替西莫司和阿霉素脂质体治疗复发性和难治性骨和软组织肉瘤的II期研究。
Clinical Sarcoma Research Pub Date : 2018-11-05 eCollection Date: 2018-01-01 DOI: 10.1186/s13569-018-0107-9
Matteo M Trucco, Christian F Meyer, Katherine A Thornton, Preeti Shah, Allen R Chen, Breelyn A Wilky, Maria A Carrera-Haro, Lillian C Boyer, Margaret F Ferreira, Umber Shafique, Jonathan D Powell, David M Loeb
{"title":"A phase II study of temsirolimus and liposomal doxorubicin for patients with recurrent and refractory bone and soft tissue sarcomas.","authors":"Matteo M Trucco,&nbsp;Christian F Meyer,&nbsp;Katherine A Thornton,&nbsp;Preeti Shah,&nbsp;Allen R Chen,&nbsp;Breelyn A Wilky,&nbsp;Maria A Carrera-Haro,&nbsp;Lillian C Boyer,&nbsp;Margaret F Ferreira,&nbsp;Umber Shafique,&nbsp;Jonathan D Powell,&nbsp;David M Loeb","doi":"10.1186/s13569-018-0107-9","DOIUrl":"https://doi.org/10.1186/s13569-018-0107-9","url":null,"abstract":"<p><strong>Background: </strong>Relapsed and refractory sarcomas continue to have poor survival rates. The cancer stem cell (CSC) theory provides a tractable explanation for the observation that recurrences occur despite dramatic responses to upfront chemotherapy. Preclinical studies demonstrated that inhibition of the mechanistic target of rapamycin (mTOR) sensitizes the CSC population to chemotherapy.</p><p><strong>Methods: </strong>Here we present the results of the Phase II portion of a Phase I/II clinical trial that aimed to overcome the chemoresistance of sarcoma CSC by combining the mTOR inhibitor temsirolimus (20 mg/m<sup>2</sup> weekly) with the chemotherapeutic agent liposomal doxorubicin (30 mg/m<sup>2</sup> monthly).</p><p><strong>Results: </strong>Fifteen patients with relapsed/refractory sarcoma were evaluable at this recommended Phase 2 dose level. The median progression free survival was 315 days (range 27-799). Response rate, defined as stable disease or better for 60 days, was 53%. Nine of the patients had been previously treated with doxorubicin. Therapy was well tolerated. In a small number of patients, pre- and post- treatment tumor biopsies were available for assessment of ALDH expression as a marker of CSCs and showed a correlation between response and decreased ALDH expression. We also found a correlation between biopsy-proven inhibition of mTOR and response.</p><p><strong>Conclusions: </strong>Our study adds to the literature supporting the addition of mTOR inhibition to chemotherapy agents for the treatment of sarcomas, and proposes that a mechanism by which mTOR inhibition enhances the efficacy of chemotherapy may be through sensitizing the chemoresistant CSC population. Further study, ideally with pre- and post-therapy assessment of ALDH expression in tumor cells, is warranted.<i>Trial registration</i> The trial was registered on clinicaltrials.gov (NCT00949325) on 30 July 2009. http://www.editorialmanager.com/csrj/default.aspx.</p>","PeriodicalId":10684,"journal":{"name":"Clinical Sarcoma Research","volume":"8 ","pages":"21"},"PeriodicalIF":0.0,"publicationDate":"2018-11-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1186/s13569-018-0107-9","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"36707779","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 21
Descending thoracic aortic aneurysm revealing metastasis of a soft tissue fibrosarcoma: a case report and review of the literature. 显示软组织纤维肉瘤转移的胸降主动脉瘤一例报告及文献复习。
Clinical Sarcoma Research Pub Date : 2018-10-26 eCollection Date: 2018-01-01 DOI: 10.1186/s13569-018-0109-7
Clotilde Delerce, Olivia Bailly, Amine Bouhamama, Sophie Couchon, Frank Pilleul, Arnaud Thivolet, Charles Mastier
{"title":"Descending thoracic aortic aneurysm revealing metastasis of a soft tissue fibrosarcoma: a case report and review of the literature.","authors":"Clotilde Delerce,&nbsp;Olivia Bailly,&nbsp;Amine Bouhamama,&nbsp;Sophie Couchon,&nbsp;Frank Pilleul,&nbsp;Arnaud Thivolet,&nbsp;Charles Mastier","doi":"10.1186/s13569-018-0109-7","DOIUrl":"https://doi.org/10.1186/s13569-018-0109-7","url":null,"abstract":"<p><strong>Background: </strong>Review of the first documented case of aortic wall metastasis from a limb sarcoma.</p><p><strong>Case presentation: </strong>In a 56-year-old woman with a diagnosis of a high-grade limb fibrosarcoma, an aortic metastasis was revealed by a fast growing aneurysm of the descending thoracic aorta. This was managed with an endoprosthesis.</p><p><strong>Conclusion: </strong>The presence of an aneurysm in a patient with a sarcoma with a high potential for metastasis and poor cardiovascular risk factors should alert physicians.</p>","PeriodicalId":10684,"journal":{"name":"Clinical Sarcoma Research","volume":"8 ","pages":"22"},"PeriodicalIF":0.0,"publicationDate":"2018-10-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1186/s13569-018-0109-7","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"36704102","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 2
Preoperative radiotherapy of soft-tissue sarcomas: surgical and radiologic parameters associated with local control and survival. 软组织肉瘤的术前放疗:与局部控制和生存相关的外科和放射学参数。
Clinical Sarcoma Research Pub Date : 2018-10-05 eCollection Date: 2018-01-01 DOI: 10.1186/s13569-018-0106-x
Panagiotis Tsagozis, Otte Brosjö, Mikael Skorpil
{"title":"Preoperative radiotherapy of soft-tissue sarcomas: surgical and radiologic parameters associated with local control and survival.","authors":"Panagiotis Tsagozis,&nbsp;Otte Brosjö,&nbsp;Mikael Skorpil","doi":"10.1186/s13569-018-0106-x","DOIUrl":"https://doi.org/10.1186/s13569-018-0106-x","url":null,"abstract":"<p><strong>Background: </strong>Preoperative radiotherapy is often used to facilitate excision of soft-tissue sarcomas. We aimed define factors that affect local tumour control and patient survival.</p><p><strong>Methods: </strong>A single institution registry study of 89 patients with non-metastatic soft-tissue sarcomas having preoperative radiotherapy between 1994 and 2014. Radiologic (presence of peritumoural oedema and volume change following radiotherapy) and histopathologic (tumour volume, grade and surgical margin) parameters were recorded. Outcomes were the events of local recurrence, amputation, metastasis and death.</p><p><strong>Results: </strong>Local recurrence rate was low (12%) and marginal excision gave equal local control to wide excision. Pelvic localization was associated with a higher risk for amputation. The absence of peritumoural oedema on MRI defined a subgroup of tumours with more favourable oncologic outcome. Reduction of tumour volume following radiotherapy was also associated with better patient survival. Both these radiologic parameters were associated with lower tumour grade. Tumour necrosis was not significant for patient survival. The local complication rate, mainly wound healing problems and infection, was high (40%), but did not lead to any amputation.</p><p><strong>Conclusion: </strong>Preoperative radiotherapy of high-risk soft-tissue sarcomas allows for good local control rate at the expense of local wound complications, which are however manageable. Marginal excision is sufficient for local control. Absence of peritumoural oedema on MRI, as well as tumour size reduction following radiotherapy are associated to superior patient survival and can be used ass early prognostic factors.</p>","PeriodicalId":10684,"journal":{"name":"Clinical Sarcoma Research","volume":"8 ","pages":"19"},"PeriodicalIF":0.0,"publicationDate":"2018-10-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1186/s13569-018-0106-x","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"36632456","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 14
Periostin expression in neoplastic and non-neoplastic diseases of bone and joint. 骨与关节肿瘤性和非肿瘤性疾病中骨膜增生蛋白的表达。
Clinical Sarcoma Research Pub Date : 2018-09-05 eCollection Date: 2018-01-01 DOI: 10.1186/s13569-018-0105-y
Jennifer M Brown, Akiro Mantoku, Afsie Sabokbar, Udo Oppermann, A Bass Hassan, Akiro Kudo, Nick Athanasou
{"title":"Periostin expression in neoplastic and non-neoplastic diseases of bone and joint.","authors":"Jennifer M Brown, Akiro Mantoku, Afsie Sabokbar, Udo Oppermann, A Bass Hassan, Akiro Kudo, Nick Athanasou","doi":"10.1186/s13569-018-0105-y","DOIUrl":"10.1186/s13569-018-0105-y","url":null,"abstract":"<p><strong>Background: </strong>Periostin is a matricellular protein that is expressed in bone and joint tissues. To determine the expression of periostin in primary bone tumours and to assess whether it plays a role in tumour progression, we carried out immunohistochemistry and ELISA for periostin in a range of neoplastic and non-neoplastic bone and joint lesions.</p><p><strong>Methods: </strong>140 formalin-fixed paraffin-embedded sections of bone tumours and tumour-like lesions were stained by an indirect immunoperoxidase technique with a polyclonal anti-periostin antibody. Periostin expression was also assessed in rheumatoid arthritis (RA) and non-inflammatory osteoarthritis (OA) synovium and synovial fluid immunohistochemistry and ELISA respectively.</p><p><strong>Results: </strong>Periostin was most strongly expressed in osteoid/woven bone of neoplastic and non-neoplastic bone-forming lesions, including osteoblastoma, osteosarcoma, fibrous dysplasia, osteofibrous dysplasia, fracture callus and myositis ossificans, and mineralised chondroid matrix/woven bone in chondroblastoma and clear cell chondrosarcoma. Reactive host bone at the edge of growing tumours, particularly in areas of increased vascularity and fibrosis, also stained strongly for periostin. Vascular elements in RA synovium strongly expressed periostin, and synovial fluid levels of periostin were higher in RA than OA.</p><p><strong>Conclusions: </strong>In keeping with its known role in modulating the synthesis of collagen and other extracellular matrix proteins in bone, strong periostin expression was noted in benign and malignant lesions forming an osteoid or osteoid-like matrix. Periostin was also noted in other bone tumours and was found in areas of reactive bone and increased vascularity at the edge of growing tumours, consistent with its involvement in tissue remodelling and angiogenesis associated with tumour progression.</p>","PeriodicalId":10684,"journal":{"name":"Clinical Sarcoma Research","volume":"8 ","pages":"18"},"PeriodicalIF":0.0,"publicationDate":"2018-09-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6123976/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"36481187","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Low-grade central fibroblastic osteosarcoma may be differentiated from its mimicker desmoplastic fibroma by genetic analysis. 通过基因分析可将低级别中枢成纤维性骨肉瘤与其相似的结缔组织增生纤维瘤区分开来。
Clinical Sarcoma Research Pub Date : 2018-08-23 eCollection Date: 2018-01-01 DOI: 10.1186/s13569-018-0104-z
Wangzhao Song, Eva van den Berg, Thomas C Kwee, Paul C Jutte, Anne-Marie Cleton-Jansen, Judith V M G Bovée, Albert J Suurmeijer
{"title":"Low-grade central fibroblastic osteosarcoma may be differentiated from its mimicker desmoplastic fibroma by genetic analysis.","authors":"Wangzhao Song,&nbsp;Eva van den Berg,&nbsp;Thomas C Kwee,&nbsp;Paul C Jutte,&nbsp;Anne-Marie Cleton-Jansen,&nbsp;Judith V M G Bovée,&nbsp;Albert J Suurmeijer","doi":"10.1186/s13569-018-0104-z","DOIUrl":"https://doi.org/10.1186/s13569-018-0104-z","url":null,"abstract":"<p><strong>Background: </strong>We studied two cases of rare fibrous bone tumors, namely desmoplastic fibroma (DF) and low-grade central osteosarcoma (LGCOS) resembling desmoplastic fibroma (DF-like LGCOS). As the clinical presentation, imaging features and histopathology of DF and DF-like LGOS show much overlap, the objective of this study was to investigate the value of cytogenetic analysis, molecular pathology and immunohistochemistry in discrimination of these two mimickers.</p><p><strong>Case presentation: </strong>A mutation in <i>CTNNB</i> (S45F) and nuclear beta-catenin immunostaining were observed in DF. DF-LGCOS had amplification of <i>CDK4</i> and showed strong nuclear expression of CDK4 by IHC. Moreover, the karyotype of DF-LGCOS showed an interstitial heterozygous deletion of the long arm of chromosome 13 (q12q32), associated with loss of the <i>RB1</i> tumor suppressor gene.</p><p><strong>Conclusions: </strong>Karyotyping and molecular genetic analysis may contribute to a conclusive diagnosis.</p>","PeriodicalId":10684,"journal":{"name":"Clinical Sarcoma Research","volume":"8 ","pages":"16"},"PeriodicalIF":0.0,"publicationDate":"2018-08-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1186/s13569-018-0104-z","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"36442818","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 8
Report from the 4th European Bone Sarcoma Networking meeting: focus on osteosarcoma 第四届欧洲骨肉瘤网络会议报告:聚焦骨肉瘤
Clinical Sarcoma Research Pub Date : 2018-08-10 DOI: 10.1186/s13569-018-0103-0
S. Strauss, J. Anninga, Rubina Baglio, D. Baumhoer, S. Behjati, S. Bielack, K. Boye, J. Broto, A. Cleton-Jansen, A. Degasperi, Abigail Evans, F. Fagioli, M. Fiocco, N. Gaspar, D. Heymann, N. Hindi, C. Lancia, O. Myklebost, M. Nathrath, F. Redini, K. Scotlandi, E. Tirtei, Michel Vanden Eynden, J. Whelan
{"title":"Report from the 4th European Bone Sarcoma Networking meeting: focus on osteosarcoma","authors":"S. Strauss, J. Anninga, Rubina Baglio, D. Baumhoer, S. Behjati, S. Bielack, K. Boye, J. Broto, A. Cleton-Jansen, A. Degasperi, Abigail Evans, F. Fagioli, M. Fiocco, N. Gaspar, D. Heymann, N. Hindi, C. Lancia, O. Myklebost, M. Nathrath, F. Redini, K. Scotlandi, E. Tirtei, Michel Vanden Eynden, J. Whelan","doi":"10.1186/s13569-018-0103-0","DOIUrl":"https://doi.org/10.1186/s13569-018-0103-0","url":null,"abstract":"","PeriodicalId":10684,"journal":{"name":"Clinical Sarcoma Research","volume":"8 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2018-08-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1186/s13569-018-0103-0","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"48659299","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 3
PDGF/PDGFR effects in osteosarcoma and the "add-on" strategy. PDGF/PDGFR在骨肉瘤中的作用和“附加”策略。
Clinical Sarcoma Research Pub Date : 2018-08-02 eCollection Date: 2018-01-01 DOI: 10.1186/s13569-018-0102-1
Jie Xu, Lu Xie, Wei Guo
{"title":"PDGF/PDGFR effects in osteosarcoma and the \"add-on\" strategy.","authors":"Jie Xu,&nbsp;Lu Xie,&nbsp;Wei Guo","doi":"10.1186/s13569-018-0102-1","DOIUrl":"https://doi.org/10.1186/s13569-018-0102-1","url":null,"abstract":"<p><p>New treatment options for advanced osteosarcoma have remained limited. The platelet-derived growth factor (PDGF)/platelet-derived growth factor receptor (PDGFR) pathway plays an important role in the development and metastasis of osteosarcoma, via either direct autocrine stimulation of tumor cells, or paracrine stimulation on tumor stromal cells. It promotes angiogenesis to overcome hypoxia in the tumor microenvironment, and modulates tumor interstitial fluid pressure to control the influx and efflux of other agents. Targeting the PDGF/PDGFR pathway is a promising therapeutic method to overcome drug resistance and improve patients' outcome in osteosarcoma. Further evidence is needed to define the detailed mechanism. Results from clinical trials using PDGF/PDGFR inhibitor as a single agent were disappointing, both in osteosarcoma and soft tissue sarcoma. However, when combined with other agents, named as \"add-on\" strategy, a synergistic antitumor effect has been confirmed in soft tissue sarcoma, and should be attempted in osteosarcoma.</p>","PeriodicalId":10684,"journal":{"name":"Clinical Sarcoma Research","volume":"8 ","pages":"15"},"PeriodicalIF":0.0,"publicationDate":"2018-08-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1186/s13569-018-0102-1","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"36376661","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 26
Tenosynovial giant cell tumor: case report of a patient effectively treated with pexidartinib (PLX3397) and review of the literature. 腱鞘巨细胞瘤:培西达替尼(PLX3397)有效治疗1例并文献复习
Clinical Sarcoma Research Pub Date : 2018-07-10 eCollection Date: 2018-01-01 DOI: 10.1186/s13569-018-0101-2
Nicholas Giustini, Nicholas M Bernthal, Susan V Bukata, Arun S Singh
{"title":"Tenosynovial giant cell tumor: case report of a patient effectively treated with pexidartinib (PLX3397) and review of the literature.","authors":"Nicholas Giustini,&nbsp;Nicholas M Bernthal,&nbsp;Susan V Bukata,&nbsp;Arun S Singh","doi":"10.1186/s13569-018-0101-2","DOIUrl":"https://doi.org/10.1186/s13569-018-0101-2","url":null,"abstract":"<p><strong>Background: </strong>Tenosynovial giant cell tumors (TGCTs) or giant cell tumors of tendon sheath are neoplasms that arise in the synovium. They can be categorized as nodular (localized) or diffuse type (D-TGCT). Historically, surgery has been the mainstay of therapy, but diffuse type disease recurs at a high rate and treatment often requires increasingly morbid procedures. Elucidation of the importance of the colony-stimulating factor (CSF1)/CSF1 receptor (CSF1R) pathway in the pathogenesis of this disease has created significant interest in targeting this pathway as a novel TGCT treatment approach. Pexidartinib, a selective tyrosine kinase inhibitor against CSF1R, showed an 83% disease control rate (52% with partial response and 31% with stable disease) in a recent phase 1 study of patients with TGCT.</p><p><strong>Case presentation: </strong>We present an illustrative example of a TGCT patient who would have required a morbid operation who derived considerable clinical benefit from pexidartinib treatment. Her tumor volume decreased by 48% after 4 months of treatment, and 55 months after starting treatment the patient exhibits continued disease stability with minimal clinical symptoms, and significant improvement in functional status.</p><p><strong>Conclusions: </strong>This case illustrates the effectiveness of systemic therapy in controlling a disease associated with high surgical morbidity. This approach may be especially useful in the treatment of extra-articular disease which often invades neurovascular bundles; as the effectiveness in metastatic disease is still unknown. In the future, systemic treatment for TGCT may be appropriate for the neoadjuvant setting to decrease disease burden prior to surgery with the aim of decreasing recurrence rates. However, properly designed prospective studies will need to be carried out to answer these questions.</p>","PeriodicalId":10684,"journal":{"name":"Clinical Sarcoma Research","volume":"8 ","pages":"14"},"PeriodicalIF":0.0,"publicationDate":"2018-07-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1186/s13569-018-0101-2","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"36308255","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 35
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