Sarcoma

Doxil for soft tissue sarcoma – pro

Doxorubicin has been used for soft tissue sarcoma(STS) since the first report in 1972, and more recently, its pegylated form has also been studied. A study by Judson et al, found that it had has equivalent activity to doxorubicin in STS with an improved toxicity profile. In this randomized phase II trial, Doxil (50 mg/m2 every four weeks) was compared to unencapsulated doxorubicin (75 mg/m2 every three weeks). Although Doxil was well tolerated and

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Sinitinib for Extraskeletal Myxoid Chondrosarcoma – pro

Extraskeletal myxoid chondrosarcoma (EMC) is a genetically distinct and rare sarcoma that tends to be indolent but to eventually recurr locally and metastastzise.  The 2009 review by Drilon highlighted ineffectiveness of chemotherapy and emphasized an agressive approach to local control. There are , however, two case reports showing excellent responses to chemotherapy.  Radiotherapy is sometimes helpful. Sutent was effective in two cases reported

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Sorafenib for desmoid tumors – pro

  Desmoid tumors, also called aggressive fibromatosis, deep musculoaponeurotic fibromatosis, and fibrosarcoma grade I of the desmoid type, do not generally metastasize but can cause pain and compromise organs. Few treatments for this condition are well established, although many novel agents are being studied. Among them is Nexavar (sorafenib). The basis for the interest in this drug is that it appears to inhibit MPNST cell growth in vitro.

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Votrient for GIST – pro

Pazopanib(Votrient) is an oral angiogenesis inhibitor targeting vascular endothelial growth factor receptor (VEGFR), platelet derived growth factor receptor (PDGFR), and KIT. These are important pathways for GIST tumors and several trials looked into using pazopanib for GIST. Although initial studies suggested that the drug is not effective for GIST, more recently a phase II study of 25 patients and a randomized study of 86 patients suggest that

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SCT for SVI – pro

Severe combined immunodeficiency (SCID) is a serious, life-threatening condition with high morbidity and mortality. SCID, a group of rare genetic disorders characterized by profound abnormalities in the development and function of the T and B lymphocytes and natural killer cells, was first reported more than 50 years ago. In the past two decades, great advances have been made in the understanding and treatment of SCID. A variety of molecular defects

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Docetaxel/ gemcitabine for sarcoma – pro

Docetaxel and gemcitabine is a well described regimen in soft tissue sarcoma. Hensley examined gemcitabine and docetaxel in a phase II study of patients with metastatic leiomyosarcoma. That study also examined the pharmacokinetics of gemcitabine in a 30- and 90-minute infusion schedule on separate cycles of therapy, described above. The study enrolled 34 patients, all but five with uterine leiomyosarcoma, and demonstrated a surprising response rate

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Topotecan and cytoxan for relapsed Ewing’s sarcoma – pro

Ewing sarcoma geenrally responds to chemotherapy. The prognosis in relapse is poor. Promising response rates have been reported for the combination of topotecan (TOPO) and cyclophosphamide (CYC). IN such cases, one can usually obtain a response and prolongation of life but not a cure. ESMO guidelines recommend palliative treatment in relapse and this would include well-based regimens such as topotecan and cytoxan. Hunold A, Weddeling N, Paulussen

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Irinotecan for rhabdomyosarcoma – pro

Rhabdomyosarcoma arising from skeletal muscle is the most common soft tissue sarcoma of childhood. Approximately 20% of patients present with disseminated disease and have a poor prognosis with combined modality therapy. The remaining 80% of patients present with localized disease and cure rates with combined modality therapy range from 50% to 90% depending on histology, site of disease and the ability of surgery to completely remove all gross and

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Adjuvant chemotherapy for oseosarcoma – pro

Despite an optimal loco-regional treatment, 35%–50% of patients with sarcoma will develop metastasis. Systemic chemotherapy is then the standard treatment; the active drugs are doxorubicin and ifosfamide and, to a lesser extent, dacarbazine. In the adjuvant setting, NCCN recommends "a combination of two of the following agents: doxorubicin, ifosfamide, high dose methotrexate and growth factors"(p.10). If there had been neoadjuvant chemo, as in

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Histiocytic sarcoma – pro

Histiocytic sarcoma (HS) is a rare neoplasm of uncertain etiology. Most recently, the diagnostic criteria for this entity have been revised with inclusion of diagnostic modalities such as immunohistochemical and cytogentic techniques. HS tends to have an aggressive clinical course and presents with systemic symptoms of fever, weight loss, adenopathy, hepatosplenomegly, rash, and pancytopenia. There is no standard treatment and no comparative studies

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