Histology-specific therapy for advanced soft tissue sarcoma and benign connective tissue tumors

Current Treatment Options in Oncology

Volumn 13, Issue 3, 2012, Pages 285-298

  Silk, Ann W ^a   Schuetze, Scott M ^a  

^a UNIVERSITY OF MICHIGAN COMPREHENSIVE CANCER CENTER   (United States)

Author keywords

Connective tissue tumor; Giant cell tumor; Inflammatory myofibroblastic tumor; mTOR inhibitor; Pazopanib; PEComa; Sarcoma; Tyrosine kinase inhibitor

Indexed keywords

ANAPLASTIC LYMPHOMA KINASE; ANTHRACYCLINE; BEVACIZUMAB; CEDIRANIB; COLONY STIMULATING FACTOR 1; CRIZOTINIB; CYCLOPHOSPHAMIDE; DENOSUMAB; DOXORUBICIN; EVEROLIMUS; GEMCITABINE; IFOSFAMIDE; IMATINIB; OSTEOCLAST DIFFERENTIATION FACTOR; PACLITAXEL; PAZOPANIB; PLACEBO; PLATELET DERIVED GROWTH FACTOR RECEPTOR; PREDNISOLONE; RAPAMYCIN; RIDAFOROLIMUS; SORAFENIB; STEM CELL FACTOR; SUNITINIB; TAXANE DERIVATIVE; TEMSIROLIMUS; UNINDEXED DRUG; VASCULOTROPIN RECEPTOR 1; VASCULOTROPIN RECEPTOR 2; VASCULOTROPIN RECEPTOR 3;

ABDOMINAL PAIN; ADVANCED CANCER; ALVEOLAR SOFT PART SARCOMA; ANGIOMYOLIPOMA; ANGIOSARCOMA; ANOREXIA; ANTINEOPLASTIC ACTIVITY; ARTICLE; BENIGN TUMOR; BLOOD TOXICITY; BONE MARROW SUPPRESSION; BONE NECROSIS; CHONDROSARCOMA; CHORDOMA; CONNECTIVE TISSUE TUMOR; DERMATOFIBROSARCOMA PROTUBERANS; DESMOID TUMOR; DIARRHEA; DOSAGE SCHEDULE COMPARISON; DRUG DOSE REDUCTION; DRUG ERUPTION; DRUG RECEPTOR BINDING; DRUG WITHDRAWAL; DYSPNEA; EDEMA; EWING SARCOMA; FATIGUE; FEBRILE NEUTROPENIA; FIBROMATOSIS; GASTROINTESTINAL STROMAL TUMOR; HAND FOOT SYNDROME; HEPATITIS; HISTOPATHOLOGY; HUMAN; HYPERBILIRUBINEMIA; HYPERCALCEMIA; HYPERTENSION; HYPOPHOSPHATEMIA; HYPOPIGMENTATION; HYPOTHYROIDISM; INTERSTITIAL PNEUMONIA; LEIOMYOSARCOMA; LIPOSARCOMA; LYMPHANGIOLEIOMYOMATOSIS; LYMPHOCYTOPENIA; MALIGNANT PERIPHERAL NERVE SHEATH TUMOR; MOUTH DISEASE; MUCOSA INFLAMMATION; MULTICENTER STUDY (TOPIC); MULTIPLE CYCLE TREATMENT; NAUSEA; NEUTROPENIA; OSTEOCLASTOMA; OSTEOSARCOMA; PAIN; PERIVASCULAR EPITHELIOID CELL TUMOR; PHASE 1 CLINICAL TRIAL (TOPIC); PHASE 2 CLINICAL TRIAL (TOPIC); PHASE 3 CLINICAL TRIAL (TOPIC); PIGMENTED VILLONODULAR SYNOVITIS; PLASMA CELL GRANULOMA; PROGRESSION FREE SURVIVAL; RANDOMIZED CONTROLLED TRIAL (TOPIC); SIDE EFFECT; SOFT TISSUE SARCOMA; SOLITARY FIBROUS TUMOR; SPINDLE CELL SARCOMA; STOMATITIS; SWELLING; SYNOVIAL SARCOMA; TUMOR GROWTH; UTERUS SARCOMA;

ANTINEOPLASTIC AGENTS; CLINICAL TRIALS AS TOPIC; HUMANS; NEOPLASM GRADING; NEOPLASM STAGING; NEOPLASMS, CONNECTIVE TISSUE; PROTEIN KINASE INHIBITORS; SARCOMA; TOR SERINE-THREONINE KINASES; TREATMENT OUTCOME;

EID: 84865692760     PISSN: 15272729     EISSN: 15346277     Source Type: Journal    
DOI: 10.1007/s11864-012-0194-4     Document Type: Article

References (43)

1. RG Maki JK Wathen SR Patel, et al. 2007 Randomized phase II study of gemcitabine and docetaxel compared with gemcitabine alone in patients with metastatic soft tissue sarcomas: results of sarcoma alliance for research through collaboration study 002 J Clin Oncol 25 2755 2763 17602081 10.1200/JCO.2006.10.4117 1:CAS:528:DC%2BD2sXosValtLY%3D
2. N Penel BN Bui J-O Bay, et al. 2008 Phase II trial of weekly paclitaxel for unresectable angiosarcoma: the ANGIOTAX Study J Clin Oncol 26 5269 5274 18809609 10.1200/JCO.2008.17.3146 1:CAS:528:DC%2BD1cXhsVyqsrbJ
3. R Chugh JK Wathen RG Maki, et al. 2009 Phase II multicenter trial of imatinib in 10 histologic subtypes of sarcoma using a Bayesian hierarchical statistical model J Clin Oncol 27 3148 3153 19451433 10.1200/JCO.2008.20.5054 1:CAS:528:DC%2BD1MXhtFWitb3L
4. Rutkowski P, Van Glabbeke M, Rankin CJ, et al. Imatinib mesylate in advanced dermatofibrosarcoma protuberans: pooled analysis of two phase II clinical trials. J Clin Oncol. 2010;28:1772-1779. Analysis of patients with advanced dermatofibrosarcoma protuberans treated with imatinib suggesting that a dose of 400 mg daily is as effective and better tolerated than 400 mg twice daily.
5. Cassier PA, Gelderblom H, Stacchiotti S, et al. Efficacy of imatinib mesylate for the treatment of locally advanced and/or metastatic tenosynovial giant cell tumor/pigmented villonodular synovitis. Cancer 2012;118:1649-1655. Series documenting activity of imatinib in managing patients with recurrent or unresectable diffuse-type tenosynovial giant cell tumor.
6. RG Maki DR D'Adamo ML Keohan, et al. 2009 Phase II study of sorafenib in patients with metastatic or recurrent sarcomas J Clin Oncol 27 3133 3140 19451436 10.1200/JCO.2008.20.4495 1:CAS:528:DC%2BD1MXhtFWitb3J
7. S Pacey M Ratain K Flaherty, et al. 2011 Efficacy and safety of sorafenib in a subset of patients with advanced soft tissue sarcoma from a Phase II randomized discontinuation trial Investigational New Drugs 29 481 488 20016927 10.1007/s10637-009-9367-9 1:CAS:528:DC%2BC3MXkvVSqtbc%3D
8. Ray-Coquard I, Italiano A, Bompas E, et al. Sorafenib for patients with advanced angiosarcoma: a phase II trial from the French Sarcoma Group (GSF/GETO). Oncologist 2012;17(2):260-6.
9. M von Mehren C Rankin JR Goldblum, et al. 2012 Phase 2 Southwest Oncology Group-directed intergroup trial (S0505) of sorafenib in advanced soft tissue sarcomas Cancer 118 770 776 10.1002/cncr.26334
10. G Grignani E Palmerini P Dileo, et al. 2012 A phase II trial of sorafenib in relapsed and unresectable high-grade osteosarcoma after failure of standard multimodal therapy: an Italian Sarcoma Group study Ann Oncol 23 508 516 21527590 10.1093/annonc/mdr151 1:STN:280:DC%2BC387nt1ynsQ%3D%3D
11. HI Hurwitz A Dowlati S Saini, et al. 2009 Phase I trial of pazopanib in patients with advanced cancer Clin Cancer Res 15 4220 4227 19509175 10.1158/1078-0432.CCR-08-2740 1:CAS:528:DC%2BD1MXntlOitr4%3D
12. Sleijfer S, Ray-Coquard I, Papai Z, et al. Pazopanib, a multikinase angiogenesis inhibitor, in patients with relapsed or refractory advanced soft tissue sarcoma: a phase II study from the European Organisation for Research and Treatment of Cancer-Soft Tissue and Bone Sarcoma Group (EORTC Study 62043). J Clin Oncol. 2009;27:3126-3132. Pazopanib was well-tolerated and had clinical activity in patients with advanced leiomyosarcomas, synovial sarcomas, and other STS types, but not adipocytic sarcomas.
13. Van Der Graaf WT, Blay J, Chawla SP, et al. PALETTE: A randomized, double-blind, phase III trial of pazopanib versus placebo in patients (pts) with soft-tissue sarcoma (STS) whose disease has progressed during or following prior chemotherapy-An EORTC STBSG Global Network Study (EORTC 62072). Abstract presented at the 47th Annual Meeting of the American Society of Clinical Oncology. Chicago, IL; June 3-7, 2011. Abstract LBA10002. The PALETTE study showed that pazopanib significantly improved PFS (median: 20 vs 7 weeks; HR∈=∈0.31, 95 % CI 0.24-0.40 ; P∈<∈0.0001).
14. S George P Merriam RG Maki, et al. 2009 Multicenter phase II trial of sunitinib in the treatment of nongastrointestinal stromal tumor sarcomas J Clin Oncol 27 3154 3160 19451429 10.1200/JCO.2008.20.9890 1:CAS:528: DC%2BD1MXhtFWitb3E
15. Stacchiotti S, Negri T, Zaffaroni N, et al. Sunitinib in advanced alveolar soft part sarcoma: evidence of a direct antitumor effect. Ann Oncol. 2011;22:1682-1690. One of the first trials to demonstrate a significant objective response rate in alveolar soft part sarcoma treated with a drug.
16. A Ghose Z Tariq S Veltri 2012 Treatment of multidrug resistant advanced alveolar soft part sarcoma with sunitinib Am J Ther 19 e56 e58 20634674 10.1097/MJT.0b013e3181e70d20
17. NCCN Guidelines, version 1. Soft Tissue Sarcoma. 2012. Available at: http://www.nccn.org/index.asp. Accessed March 18, 2012.
18. Gardner K, Judson I, Leahy M, et al. Activity of cediranib, a highly potent and selective VEGF signaling inhibitor, in alveolar soft part sarcoma. Abstract presented at the 45th Annual Meeting of the American Society of Clinical Oncology. Orlando, FL; May 29-June 2, 2009. Abstract 10523.
19. Kummar S, Strassberger A, Monks A, et al. An evaluation of cediranib as a new agent for alveolar soft part sarcoma (ASPS). Abstract presented at the 47th Annual Meeting of the American Society of Clinical Oncology. Chicago, IL; June 3-7, 2011. Abstract 10001.
20. US National Institutes of Health Clinical Trials: Sunitinib or cediranib for alveolar soft part sarcoma. 2012. Available at: http://clinicaltrials.gov/ ct2/show/NCT01391962. Accessed March 3, 2012.
21. MM Mita AC Mita QS Chu, et al. 2008 Phase I trial of the novel mammalian target of rapamycin inhibitor deforolimus (AP23573; MK-8669) administered intravenously daily for 5 days every 2 weeks to patients with advanced malignancies J Clin Oncol 26 361 367 18202410 10.1200/JCO.2007.12.0345 1:CAS:528:DC%2BD1cXitVGntLk%3D
22. Chawla SP, Staddon AP, Baker LH, et al. Phase II study of the mammalian target of rapamycin inhibitor ridaforolimus in patients with advanced bone and soft tissue sarcomas. J Clin Oncol 2012, 30:78-84. In this relatively large study of patients with unresectable or metastatic sarcoma, 29 % had stable disease or partial response at 16 weeks, but the objective response rate was less than 5 %.
23. Chawla SP, Blay J, Ray-Coquard IL, et al. Results of the phase III, placebo-controlled trial (SUCCEED) evaluating the mTOR inhibitor ridaforolimus (R) as maintenance therapy in advanced sarcoma patients (pts) following clinical benefit from prior standard cytotoxic chemotherapy (CT). Abstract presented at the 47th Annual Meeting of the American Society of Clinical Oncology. Chicago, IL; June 3-7, 2011. Abstract 10005.
24. S Okuno H Bailey MR Mahoney, et al. 2011 A phase 2 study of temsirolimus (CCI-779) in patients with soft tissue sarcomas Cancer 117 3468 3475 21287536 10.1002/cncr.25928 1:CAS:528:DC%2BC3MXptVOntrw%3D
25. Italiano A, Kind Ml, Stoeckle E, Jones N, Coindre J-M, Bui B. Temsirolimus in advanced leiomyosarcomas: patterns of response and correlation with the activation of the mammalian target of rapamycin pathway. Anticancer Drugs. 2011;22:463-467.
26. R Quek Q Wang JA Morgan, et al. 2011 Combination mTOR and IGF-1R inhibition: phase I trial of everolimus and figitumumab in patients with advanced sarcomas and other solid tumors Clin Cancer Res 17 871 879 21177764 10.1158/1078-0432.CCR-10-2621 1:CAS:528:DC%2BC3MXitVWjs7c%3D
27. K Bullock W Petros I Younis, et al. 2011 A phase I study of bevacizumab (B) in combination with everolimus (E) and erlotinib (E) in advanced cancer (BEE) Cancer Chemother Pharmacol 67 465 474 21079958 10.1007/s00280-010-1507-6 1:CAS:528:DC%2BC3MXht1enu78%3D
28. Wagner AJ, Malinowska-Kolodziej I, Morgan JA, et al. Clinical activity of mTOR inhibition with sirolimus in malignant perivascular epithelioid cell tumors: targeting the pathogenic activation of mTORC1 in tumors. J Clin Oncol. 2010;28:835-840. Small series demonstrating that malignant PEComa may respond to treatment with mTOR inhibitor.
29. A Italiano C Delcambre I Hostein, et al. 2010 Treatment with the mTOR inhibitor temsirolimus in patients with malignant PEComa Ann Oncol 21 1135 1137 20215136 10.1093/annonc/mdq044 1:STN:280:DC%2BC3c3pt1ejsw%3D%3D
30. SL Dabora DN Franz S Ashwal, et al. 2011 Multicenter phase 2 trial of sirolimus for tuberous sclerosis: kidney angiomyolipomas and other tumors regress and VEGF- D levels decrease PLoS One 6 e23379 21915260 10.1371/journal.pone.0023379 1:CAS:528:DC%2BC3MXht1ent7bO
31. DM Davies PJ de Vries SR Johnson, et al. 2011 Sirolimus therapy for angiomyolipoma in tuberous sclerosis and sporadic lymphangioleiomyomatosis: a phase 2 trial Clin Cancer Res 17 4071 4081 21525172 10.1158/1078-0432.CCR-11- 0445 1:CAS:528:DC%2BC3MXnsFKgtL0%3D
32. N Wolff W Kabbani T Bradley G Raj L Watumull J Brugarolas 2010 Sirolimus and temsirolimus for epithelioid angiomyolipoma J Clin Oncol 28 e65 e68 20048172 10.1200/JCO.2009.26.3061
33. K Shitara Y Yatabe A Mizota T Sano Y Nimura K Muro 2011 Dramatic tumor response to everolimus for malignant epithelioid angiomyolipoma Jpn J Clin Oncol 41 814 816 21415002 10.1093/jjco/hyr035
34. FX McCormack Y Inoue J Moss, et al. 2011 Efficacy and safety of sirolimus in lymphangioleiomyomatosis N Engl J Med 364 1595 1606 21410393 10.1056/NEJMoa1100391 1:CAS:528:DC%2BC3MXlsFegtr0%3D
35. Stacchiotti S, Palassini E, Sanfilippo R, et al. Gemcitabine in advanced angiosarcoma: a retrospective case series analysis from the Italian Rare Cancer Network. Ann Oncol. 2012;23(2):501-8.
36. O Mir J Domont A Cioffi, et al. 2011 Feasibility of metronomic oral cyclophosphamide plus prednisolone in elderly patients with inoperable or metastatic soft tissue sarcoma Eur J Cancer 47 515 519 21251814 10.1016/j.ejca.2010.11.025 1:CAS:528:DC%2BC3MXhvVyltLg%3D
37. RB West BP Rubin MA Miller, et al. 2006 A landscape effect in tenosynovial giant-cell tumor from activation of CSF1 expression by a translocation in a minority of tumor cells Proc Natl Acad Sci USA 103 690 695 16407111 10.1073/pnas.0507321103 1:CAS:528:DC%2BD28XhtVOiu7c%3D
38. J-Y Blay H El Sayadi P Thiesse J Garret I Ray-Coquard 2008 Complete response to imatinib in relapsing pigmented villonodular synovitis/tenosynovial giant cell tumor (PVNS/TGCT) Ann Oncol 19 821 822 18296418 10.1093/annonc/mdn033
39. N Penel A Le Cesne BN Bui, et al. 2011 Imatinib for progressive and recurrent aggressive fibromatosis (desmoid tumors): an FNCLCC/French Sarcoma Group phase II trial with a long-term follow-up Ann Oncol 22 452 457 20622000 10.1093/annonc/mdq341 1:STN:280:DC%2BC3M7ltFSlsQ%3D%3D
40. R Chugh JK Wathen SR Patel, et al. 2010 Efficacy of imatinib in aggressive fibromatosis: results of a phase II multicenter Sarcoma Alliance for Research through Collaboration (SARC) trial Clin Cancer Res 16 4884 4891 20724445 10.1158/1078-0432.CCR-10-1177 1:CAS:528:DC%2BC3cXht1aktbnL
41. MM Gounder RA Lefkowitz ML Keohan, et al. 2011 Activity of sorafenib against desmoid tumor/deep fibromatosis Clin Cancer Res 17 4082 4090 21447727 10.1158/1078-0432.CCR-10-3322 1:CAS:528:DC%2BC3MXnsFKgtLc%3D
42. Butrynski JE, D'Adamo DR, Hornick JL, et al. Crizotinib in ALK-rearranged inflammatory myofibroblastic tumor. N Engl J Med. 2010;363:1727-1733. This case report of two patients led to the recent update to the NCCN guidelines to include crizotinib as an option for management of patients with ALK∈+∈IMT.
43. Thomas D, Henshaw R, Skubitz K, et al. Denosumab in patients with giant-cell tumour of bone: an open-label, phase 2 study. The Lancet Oncology 2010;11:275-280. Phase II study demonstrating significant activity of denosumab, a monoclonal antibody to RANK ligand, in the management of patients with locally advanced or metastatic giant cell tumor of bone.