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Published in: Current Oncology Reports 8/2014

01-08-2014 | Sarcomas (SR Patel, Section Editor)

Advances in Therapy for Pediatric Sarcomas

Authors: Aaron Weiss, Jonathan Gill, John Goldberg, Joanne Lagmay, Holly Spraker-Perlman, Rajkumar Venkatramani, Damon Reed

Published in: Current Oncology Reports | Issue 8/2014

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Abstract

Pediatric sarcomas are relatively rare malignancies individually. As a group they are typically approached with combination chemotherapies in addition to local control. Fortunately, these malignancies have been approached through careful clinical trial collaboration to define risk groups and appropriately deliver local control measures and systemic therapies. Although local disease is typically approached with curative intent, therapy typically lasts over 6 months and has significant associated morbidities. It is more difficult to cure metastatic disease or induce sustained remissions. In this article, we discuss recent advances in the understanding of the disease process and highlight recent and future cooperative group trials in osteosarcoma, Ewing sarcoma, rhabdomyosarcoma, nonrhabdomyosarcoma soft tissue sarcomas, and desmoid tumor as well as discuss promising therapeutic approaches such as epigenetics and immunotherapy.
Literature
1.
go back to reference Ries LAG et al., editors. Cancer incidence and survival among children and adolescents: United States SEER Program 1975-1995. NIH publication no. 99-4649. Bethesda: National Cancer Institute; 1999. Ries LAG et al., editors. Cancer incidence and survival among children and adolescents: United States SEER Program 1975-1995. NIH publication no. 99-4649. Bethesda: National Cancer Institute; 1999.
2.
go back to reference Bleyer A et al., editors. Cancer epidemiology in older adolescents and young adults 15 to 29 years of age, including SEER incidence and survival: 1975-2000. NIH publication no. 06-5767. Bethesda: National Cancer Institute; 2006. Bleyer A et al., editors. Cancer epidemiology in older adolescents and young adults 15 to 29 years of age, including SEER incidence and survival: 1975-2000. NIH publication no. 06-5767. Bethesda: National Cancer Institute; 2006.
3.
go back to reference Bielack SS et al. Bone tumors in adolescents and young adults. Curr Treat Options in Oncol. 2008;9(1):67–80. Bielack SS et al. Bone tumors in adolescents and young adults. Curr Treat Options in Oncol. 2008;9(1):67–80.
4.
go back to reference Federman N et al. The multidisciplinary management of osteosarcoma. Curr Treat Options in Oncol. 2009;10(1–2):82–93. Federman N et al. The multidisciplinary management of osteosarcoma. Curr Treat Options in Oncol. 2009;10(1–2):82–93.
5.
go back to reference Kempf-Bielack B et al. Osteosarcoma relapse after combined modality therapy: an analysis of unselected patients in the Cooperative Osteosarcoma Study Group (COSS). J Clin Oncol. 2005;23(3):559–68.PubMed Kempf-Bielack B et al. Osteosarcoma relapse after combined modality therapy: an analysis of unselected patients in the Cooperative Osteosarcoma Study Group (COSS). J Clin Oncol. 2005;23(3):559–68.PubMed
6.
go back to reference Kager L et al. Primary metastatic osteosarcoma: presentation and outcome of patients treated on neoadjuvant Cooperative Osteosarcoma Study Group protocols. J Clin Oncol. 2003;21(10):2011–8.PubMed Kager L et al. Primary metastatic osteosarcoma: presentation and outcome of patients treated on neoadjuvant Cooperative Osteosarcoma Study Group protocols. J Clin Oncol. 2003;21(10):2011–8.PubMed
7.
go back to reference Bielack SS et al. Prognostic factors in high-grade osteosarcoma of the extremities or trunk: an analysis of 1,702 patients treated on neoadjuvant cooperative osteosarcoma study group protocols. J Clin Oncol. 2002;20(3):776–90.PubMed Bielack SS et al. Prognostic factors in high-grade osteosarcoma of the extremities or trunk: an analysis of 1,702 patients treated on neoadjuvant cooperative osteosarcoma study group protocols. J Clin Oncol. 2002;20(3):776–90.PubMed
8.
go back to reference Ferrari S et al. Neoadjuvant chemotherapy with high-dose ifosfamide, high-dose methotrexate, cisplatin, and doxorubicin for patients with localized osteosarcoma of the extremity: a joint study by the Italian and Scandinavian sarcoma groups. J Clin Oncol. 2005;23(34):8845–52.PubMed Ferrari S et al. Neoadjuvant chemotherapy with high-dose ifosfamide, high-dose methotrexate, cisplatin, and doxorubicin for patients with localized osteosarcoma of the extremity: a joint study by the Italian and Scandinavian sarcoma groups. J Clin Oncol. 2005;23(34):8845–52.PubMed
9.
go back to reference Meyers PA et al. Osteosarcoma: a randomized, prospective trial of the addition of ifosfamide and/or muramyl tripeptide to cisplatin, doxorubicin, and high-dose methotrexate. J Clin Oncol. 2005;23(9):2004–11.PubMed Meyers PA et al. Osteosarcoma: a randomized, prospective trial of the addition of ifosfamide and/or muramyl tripeptide to cisplatin, doxorubicin, and high-dose methotrexate. J Clin Oncol. 2005;23(9):2004–11.PubMed
10.
go back to reference Mirabello L, Troisi RJ, Savage SA. Osteosarcoma incidence and survival rates from 1973 to 2004: data from the Surveillance, Epidemiology, and End Results Program. Cancer. 2009;115(7):1531–43.PubMedCentralPubMed Mirabello L, Troisi RJ, Savage SA. Osteosarcoma incidence and survival rates from 1973 to 2004: data from the Surveillance, Epidemiology, and End Results Program. Cancer. 2009;115(7):1531–43.PubMedCentralPubMed
11.
go back to reference Chou AJ, Gorlick R. Chemotherapy resistance in osteosarcoma: current challenges and future directions. Expert Rev Anticancer Ther. 2006;6(7):1075–85.PubMed Chou AJ, Gorlick R. Chemotherapy resistance in osteosarcoma: current challenges and future directions. Expert Rev Anticancer Ther. 2006;6(7):1075–85.PubMed
12.
go back to reference Chen X et al. Recurrent somatic structural variations contribute to tumorigenesis in pediatric osteosarcoma. Cell Rep. 2014;7(1):104–12.PubMedCentralPubMed Chen X et al. Recurrent somatic structural variations contribute to tumorigenesis in pediatric osteosarcoma. Cell Rep. 2014;7(1):104–12.PubMedCentralPubMed
13.
go back to reference Clark JC, Dass CR, Choong PF. A review of clinical and molecular prognostic factors in osteosarcoma. J Cancer Res Clin Oncol. 2008;134(3):281–97.PubMed Clark JC, Dass CR, Choong PF. A review of clinical and molecular prognostic factors in osteosarcoma. J Cancer Res Clin Oncol. 2008;134(3):281–97.PubMed
14.
go back to reference Savage SA, Mirabello L. Using epidemiology and genomics to understand osteosarcoma etiology. Sarcoma. 2011;2011:548151.PubMedCentralPubMed Savage SA, Mirabello L. Using epidemiology and genomics to understand osteosarcoma etiology. Sarcoma. 2011;2011:548151.PubMedCentralPubMed
15.
go back to reference Sampson VB et al. A review of targeted therapies evaluated by the pediatric preclinical testing program for osteosarcoma. Front Oncol. 2013;3:132.PubMedCentralPubMed Sampson VB et al. A review of targeted therapies evaluated by the pediatric preclinical testing program for osteosarcoma. Front Oncol. 2013;3:132.PubMedCentralPubMed
16.
go back to reference Marina N et al. International collaboration is feasible in trials for rare conditions: the EURAMOS experience. Cancer Treat Res. 2009;152:339–53.PubMed Marina N et al. International collaboration is feasible in trials for rare conditions: the EURAMOS experience. Cancer Treat Res. 2009;152:339–53.PubMed
17.
go back to reference Bielack S et al. MAP plus maintenance pegylated interferon α-2b (MAPIfn) versus MAP alone in patients with resectable high-grade osteosarcoma and good histologic response to preoperative MAP: first results of the EURAMOS-1 "good response" randomization. J Clin Oncol. 2013;31(18 Suppl), LBA10504. Bielack S et al. MAP plus maintenance pegylated interferon α-2b (MAPIfn) versus MAP alone in patients with resectable high-grade osteosarcoma and good histologic response to preoperative MAP: first results of the EURAMOS-1 "good response" randomization. J Clin Oncol. 2013;31(18 Suppl), LBA10504.
18.
go back to reference Ebb D et al. Phase II trial of trastuzumab in combination with cytotoxic chemotherapy for treatment of metastatic osteosarcoma with human epidermal growth factor receptor 2 overexpression: a report from the children's oncology group. J Clin Oncol. 2012;30(20):2545–51.PubMedCentralPubMed Ebb D et al. Phase II trial of trastuzumab in combination with cytotoxic chemotherapy for treatment of metastatic osteosarcoma with human epidermal growth factor receptor 2 overexpression: a report from the children's oncology group. J Clin Oncol. 2012;30(20):2545–51.PubMedCentralPubMed
19.
go back to reference Chou AJ et al. Addition of muramyl tripeptide to chemotherapy for patients with newly diagnosed metastatic osteosarcoma: a report from the Children's Oncology Group. Cancer. 2009;115(22):5339–48.PubMedCentralPubMed Chou AJ et al. Addition of muramyl tripeptide to chemotherapy for patients with newly diagnosed metastatic osteosarcoma: a report from the Children's Oncology Group. Cancer. 2009;115(22):5339–48.PubMedCentralPubMed
20.
go back to reference Meyers PA et al. Osteosarcoma: the addition of muramyl tripeptide to chemotherapy improves overall survival—a report from the Children's Oncology Group. J Clin Oncol. 2008;26(4):633–8.PubMed Meyers PA et al. Osteosarcoma: the addition of muramyl tripeptide to chemotherapy improves overall survival—a report from the Children's Oncology Group. J Clin Oncol. 2008;26(4):633–8.PubMed
21.
go back to reference Goldsby RE et al. Feasibility and dose discovery analysis of zoledronic acid with concurrent chemotherapy in the treatment of newly diagnosed metastatic osteosarcoma: a report from the Children's Oncology Group. Eur J Cancer. 2013;49(10):2384–91.PubMedCentralPubMed Goldsby RE et al. Feasibility and dose discovery analysis of zoledronic acid with concurrent chemotherapy in the treatment of newly diagnosed metastatic osteosarcoma: a report from the Children's Oncology Group. Eur J Cancer. 2013;49(10):2384–91.PubMedCentralPubMed
22.
go back to reference Arndt CA et al. Inhaled granulocyte-macrophage colony stimulating factor for first pulmonary recurrence of osteosarcoma: effects on disease-free survival and immunomodulation. a report from the Children's Oncology Group. Clin Cancer Res. 2010;16(15):4024–30.PubMedCentralPubMed Arndt CA et al. Inhaled granulocyte-macrophage colony stimulating factor for first pulmonary recurrence of osteosarcoma: effects on disease-free survival and immunomodulation. a report from the Children's Oncology Group. Clin Cancer Res. 2010;16(15):4024–30.PubMedCentralPubMed
23.
go back to reference Beaty 3rd O et al. A phase II trial and pharmacokinetic study of oxaliplatin in children with refractory solid tumors: a Children's Oncology Group study. Pediatr Blood Cancer. 2010;55(3):440–5.PubMed Beaty 3rd O et al. A phase II trial and pharmacokinetic study of oxaliplatin in children with refractory solid tumors: a Children's Oncology Group study. Pediatr Blood Cancer. 2010;55(3):440–5.PubMed
24.
go back to reference Bond M et al. A phase II study of imatinib mesylate in children with refractory or relapsed solid tumors: a Children's Oncology Group study. Pediatr Blood Cancer. 2008;50(2):254–8.PubMed Bond M et al. A phase II study of imatinib mesylate in children with refractory or relapsed solid tumors: a Children's Oncology Group study. Pediatr Blood Cancer. 2008;50(2):254–8.PubMed
25.
go back to reference Langevin AM et al. A phase II trial of rebeccamycin analogue (NSC #655649) in children with solid tumors: a Children's Oncology Group study. Pediatr Blood Cancer. 2008;50(3):577–80.PubMed Langevin AM et al. A phase II trial of rebeccamycin analogue (NSC #655649) in children with solid tumors: a Children's Oncology Group study. Pediatr Blood Cancer. 2008;50(3):577–80.PubMed
26.
go back to reference Saylors 3rd RL et al. Cyclophosphamide plus topotecan in children with recurrent or refractory solid tumors: a Pediatric Oncology Group phase II study. J Clin Oncol. 2001;19(15):3463–9.PubMed Saylors 3rd RL et al. Cyclophosphamide plus topotecan in children with recurrent or refractory solid tumors: a Pediatric Oncology Group phase II study. J Clin Oncol. 2001;19(15):3463–9.PubMed
27.
go back to reference Zwerdling T et al. Phase II investigation of docetaxel in pediatric patients with recurrent solid tumors: a report from the Children's Oncology Group. Cancer. 2006;106(8):1821–8.PubMed Zwerdling T et al. Phase II investigation of docetaxel in pediatric patients with recurrent solid tumors: a report from the Children's Oncology Group. Cancer. 2006;106(8):1821–8.PubMed
28.
go back to reference Jacobs S et al. Phase II trial of ixabepilone administered daily for five days in children and young adults with refractory solid tumors: a report from the children's oncology group. Clin Cancer Res. 2010;16(2):750–4.PubMedCentralPubMed Jacobs S et al. Phase II trial of ixabepilone administered daily for five days in children and young adults with refractory solid tumors: a report from the children's oncology group. Clin Cancer Res. 2010;16(2):750–4.PubMedCentralPubMed
29.
go back to reference Gorlick R et al. Children's Oncology Group's 2013 blueprint for research: bone tumors. Pediatr Blood Cancer. 2013;60(6):1009–15.PubMed Gorlick R et al. Children's Oncology Group's 2013 blueprint for research: bone tumors. Pediatr Blood Cancer. 2013;60(6):1009–15.PubMed
30.
go back to reference Chou AJ et al. Inhaled lipid cisplatin (ILC) in the treatment of patients with relapsed/progressive osteosarcoma metastatic to the lung. Pediatr Blood Cancer. 2012;60(4):580–6.PubMed Chou AJ et al. Inhaled lipid cisplatin (ILC) in the treatment of patients with relapsed/progressive osteosarcoma metastatic to the lung. Pediatr Blood Cancer. 2012;60(4):580–6.PubMed
31.
go back to reference Jordan MA, Wilson L. Microtubules as a target for anticancer drugs. Nat Rev Cancer. 2004;4(4):253–65.PubMed Jordan MA, Wilson L. Microtubules as a target for anticancer drugs. Nat Rev Cancer. 2004;4(4):253–65.PubMed
32.
go back to reference Kolb EA et al. Initial testing (stage 1) of eribulin, a novel tubulin binding agent, by the pediatric preclinical testing program. Pediatr Blood Cancer. 2013;60(8):1325–32.PubMed Kolb EA et al. Initial testing (stage 1) of eribulin, a novel tubulin binding agent, by the pediatric preclinical testing program. Pediatr Blood Cancer. 2013;60(8):1325–32.PubMed
33.
go back to reference Lamoureux F et al. Therapeutic relevance of osteoprotegerin gene therapy in osteosarcoma: blockade of the vicious cycle between tumor cell proliferation and bone resorption. Cancer Res. 2007;67(15):7308–18.PubMed Lamoureux F et al. Therapeutic relevance of osteoprotegerin gene therapy in osteosarcoma: blockade of the vicious cycle between tumor cell proliferation and bone resorption. Cancer Res. 2007;67(15):7308–18.PubMed
34.
go back to reference Mori K et al. Receptor activator of nuclear factor-κB ligand (RANKL) directly modulates the gene expression profile of RANK-positive Saos-2 human osteosarcoma cells. Oncol Rep. 2007;18(6):1365–71.PubMed Mori K et al. Receptor activator of nuclear factor-κB ligand (RANKL) directly modulates the gene expression profile of RANK-positive Saos-2 human osteosarcoma cells. Oncol Rep. 2007;18(6):1365–71.PubMed
35.
go back to reference Heiner JP et al. Localization of GD2-specific monoclonal antibody 3F8 in human osteosarcoma. Cancer Res. 1987;47(20):5377–81.PubMed Heiner JP et al. Localization of GD2-specific monoclonal antibody 3F8 in human osteosarcoma. Cancer Res. 1987;47(20):5377–81.PubMed
36.
go back to reference Roth M et al. Ganglioside GD2 as a therapeutic target for antibody-mediated therapy in patients with osteosarcoma. Cancer. 2014;120(4):548–54.PubMed Roth M et al. Ganglioside GD2 as a therapeutic target for antibody-mediated therapy in patients with osteosarcoma. Cancer. 2014;120(4):548–54.PubMed
37.
go back to reference Maric G et al. Glycoprotein non-metastatic b (GPNMB): a metastatic mediator and emerging therapeutic target in cancer. Oncotargets Ther. 2013;6:839–52. Maric G et al. Glycoprotein non-metastatic b (GPNMB): a metastatic mediator and emerging therapeutic target in cancer. Oncotargets Ther. 2013;6:839–52.
38.
go back to reference Esiashvili N, Goodman M, Marcus Jr RB. Changes in incidence and survival of Ewing sarcoma patients over the past 3 decades: surveillance epidemiology and end results data. J Pediatr Hematol Oncol. 2008;30(6):425–30.PubMed Esiashvili N, Goodman M, Marcus Jr RB. Changes in incidence and survival of Ewing sarcoma patients over the past 3 decades: surveillance epidemiology and end results data. J Pediatr Hematol Oncol. 2008;30(6):425–30.PubMed
39.
go back to reference Grier HE et al. Addition of ifosfamide and etoposide to standard chemotherapy for Ewing's sarcoma and primitive neuroectodermal tumor of bone. N Engl J Med. 2003;348(8):694–701.PubMed Grier HE et al. Addition of ifosfamide and etoposide to standard chemotherapy for Ewing's sarcoma and primitive neuroectodermal tumor of bone. N Engl J Med. 2003;348(8):694–701.PubMed
40.
go back to reference Womer RB et al. Randomized controlled trial of interval-compressed chemotherapy for the treatment of localized Ewing sarcoma: a report from the Children's Oncology Group. J Clin Oncol. 2012;30(33):4148–54.PubMedCentralPubMed Womer RB et al. Randomized controlled trial of interval-compressed chemotherapy for the treatment of localized Ewing sarcoma: a report from the Children's Oncology Group. J Clin Oncol. 2012;30(33):4148–54.PubMedCentralPubMed
41.
go back to reference Cotterill SJ et al. Prognostic factors in Ewing's tumor of bone: analysis of 975 patients from the European Intergroup Cooperative Ewing's Sarcoma Study Group. J Clin Oncol. 2000;18(17):3108–14.PubMed Cotterill SJ et al. Prognostic factors in Ewing's tumor of bone: analysis of 975 patients from the European Intergroup Cooperative Ewing's Sarcoma Study Group. J Clin Oncol. 2000;18(17):3108–14.PubMed
42.
43.
go back to reference Erkizan HV et al. A small molecule blocking oncogenic protein EWS-FLI1 interaction with RNA helicase A inhibits growth of Ewing's sarcoma. Nat Med. 2009;15(7):750–6.PubMedCentralPubMed Erkizan HV et al. A small molecule blocking oncogenic protein EWS-FLI1 interaction with RNA helicase A inhibits growth of Ewing's sarcoma. Nat Med. 2009;15(7):750–6.PubMedCentralPubMed
44.
go back to reference Sankar S et al. Mechanism and relevance of EWS/FLI-mediated transcriptional repression in Ewing sarcoma. Oncogene. 2013;32(42):5089–100.PubMedCentralPubMed Sankar S et al. Mechanism and relevance of EWS/FLI-mediated transcriptional repression in Ewing sarcoma. Oncogene. 2013;32(42):5089–100.PubMedCentralPubMed
45.
go back to reference Bennani-Baiti IM et al. Lysine-specific demethylase 1 (LSD1/KDM1A/AOF2/BHC110) is expressed and is an epigenetic drug target in chondrosarcoma, Ewing's sarcoma, osteosarcoma, and rhabdomyosarcoma. Hum Pathol. 2012;43(8):1300–7.PubMed Bennani-Baiti IM et al. Lysine-specific demethylase 1 (LSD1/KDM1A/AOF2/BHC110) is expressed and is an epigenetic drug target in chondrosarcoma, Ewing's sarcoma, osteosarcoma, and rhabdomyosarcoma. Hum Pathol. 2012;43(8):1300–7.PubMed
46.
go back to reference Grohar PJ et al. Identification of an inhibitor of the EWS-FLI1 oncogenic transcription factor by high-throughput screening. J Natl Cancer Inst. 2011;103(12):962–78.PubMedCentralPubMed Grohar PJ et al. Identification of an inhibitor of the EWS-FLI1 oncogenic transcription factor by high-throughput screening. J Natl Cancer Inst. 2011;103(12):962–78.PubMedCentralPubMed
47.
go back to reference Fong PC et al. Inhibition of poly(ADP-ribose) polymerase in tumors from BRCA mutation carriers. N Engl J Med. 2009;361(2):123–34.PubMed Fong PC et al. Inhibition of poly(ADP-ribose) polymerase in tumors from BRCA mutation carriers. N Engl J Med. 2009;361(2):123–34.PubMed
48.
go back to reference Foulkes WD, Smith IE, Reis-Filho JS. Triple-negative breast cancer. N Engl J Med. 2010;363(20):1938–48.PubMed Foulkes WD, Smith IE, Reis-Filho JS. Triple-negative breast cancer. N Engl J Med. 2010;363(20):1938–48.PubMed
49.
go back to reference Carrle D, Bielack S. Osteosarcoma lung metastases detection and principles of multimodal therapy. Cancer Treat Res. 2009;152:165–84.PubMed Carrle D, Bielack S. Osteosarcoma lung metastases detection and principles of multimodal therapy. Cancer Treat Res. 2009;152:165–84.PubMed
50.
go back to reference Garnett MJ et al. Systematic identification of genomic markers of drug sensitivity in cancer cells. Nature. 2012;483(7391):570–5.PubMedCentralPubMed Garnett MJ et al. Systematic identification of genomic markers of drug sensitivity in cancer cells. Nature. 2012;483(7391):570–5.PubMedCentralPubMed
51.
go back to reference Soldatenkov VA et al. Regulation of the human poly(ADP-ribose) polymerase promoter by the ETS transcription factor. Oncogene. 1999;18(27):3954–62.PubMed Soldatenkov VA et al. Regulation of the human poly(ADP-ribose) polymerase promoter by the ETS transcription factor. Oncogene. 1999;18(27):3954–62.PubMed
52.
go back to reference Olmos D et al. Safety, pharmacokinetics, and preliminary activity of the anti-IGF-1R antibody figitumumab (CP-751,871) in patients with sarcoma and Ewing's sarcoma: a phase 1 expansion cohort study. Lancet Oncol. 2010;11(2):129–35.PubMedCentralPubMed Olmos D et al. Safety, pharmacokinetics, and preliminary activity of the anti-IGF-1R antibody figitumumab (CP-751,871) in patients with sarcoma and Ewing's sarcoma: a phase 1 expansion cohort study. Lancet Oncol. 2010;11(2):129–35.PubMedCentralPubMed
53.
go back to reference Malempati S, Hawkins DS. Rhabdomyosarcoma: review of the Children's Oncology Group (COG) Soft-Tissue Sarcoma Committee experience and rationale for current COG studies. Pediatr Blood Cancer. 2012;59(1):5–10.PubMedCentralPubMed Malempati S, Hawkins DS. Rhabdomyosarcoma: review of the Children's Oncology Group (COG) Soft-Tissue Sarcoma Committee experience and rationale for current COG studies. Pediatr Blood Cancer. 2012;59(1):5–10.PubMedCentralPubMed
54.
go back to reference Juergens H et al. Preliminary efficacy of the anti-insulin-like growth factor type 1 receptor antibody figitumumab in patients with refractory Ewing sarcoma. J Clin Oncol. 2011;29(34):4534–40.PubMedCentralPubMed Juergens H et al. Preliminary efficacy of the anti-insulin-like growth factor type 1 receptor antibody figitumumab in patients with refractory Ewing sarcoma. J Clin Oncol. 2011;29(34):4534–40.PubMedCentralPubMed
55.
go back to reference Pappo AS et al. R1507, a monoclonal antibody to the insulin-like growth factor 1 receptor, in patients with recurrent or refractory Ewing sarcoma family of tumors: results of a phase II Sarcoma Alliance for Research Through Collaboration study. J Clin Oncol. 2011;29(34):4541–7.PubMedCentralPubMed Pappo AS et al. R1507, a monoclonal antibody to the insulin-like growth factor 1 receptor, in patients with recurrent or refractory Ewing sarcoma family of tumors: results of a phase II Sarcoma Alliance for Research Through Collaboration study. J Clin Oncol. 2011;29(34):4541–7.PubMedCentralPubMed
56.
go back to reference Anderson JL et al. Pediatric sarcomas: translating molecular pathogenesis of disease to novel therapeutic possibilities. Pediatr Res. 2012;72(2):112–21.PubMed Anderson JL et al. Pediatric sarcomas: translating molecular pathogenesis of disease to novel therapeutic possibilities. Pediatr Res. 2012;72(2):112–21.PubMed
57.
go back to reference O'Reilly KE et al. mTOR inhibition induces upstream receptor tyrosine kinase signaling and activates Akt. Cancer Res. 2006;66(3):1500–8.PubMedCentralPubMed O'Reilly KE et al. mTOR inhibition induces upstream receptor tyrosine kinase signaling and activates Akt. Cancer Res. 2006;66(3):1500–8.PubMedCentralPubMed
58.
go back to reference Schwartz GK et al. Cixutumumab and temsirolimus for patients with bone and soft-tissue sarcoma: a multicentre, open-label, phase 2 trial. Lancet Oncol. 2013;14(4):371–82.PubMedCentralPubMed Schwartz GK et al. Cixutumumab and temsirolimus for patients with bone and soft-tissue sarcoma: a multicentre, open-label, phase 2 trial. Lancet Oncol. 2013;14(4):371–82.PubMedCentralPubMed
59.
go back to reference Naing A et al. Insulin growth factor-receptor (IGF-1R) antibody cixutumumab combined with the mTOR inhibitor temsirolimus in patients with refractory Ewing's sarcoma family tumors. Clin Cancer Res. 2012;18(9):2625–31.PubMed Naing A et al. Insulin growth factor-receptor (IGF-1R) antibody cixutumumab combined with the mTOR inhibitor temsirolimus in patients with refractory Ewing's sarcoma family tumors. Clin Cancer Res. 2012;18(9):2625–31.PubMed
60.
go back to reference Ferrari A et al. Soft tissue sarcoma across the age spectrum: a population-based study from the surveillance epidemiology and end results database. Pediatr Blood Cancer. 2011;57(6):943–9.PubMed Ferrari A et al. Soft tissue sarcoma across the age spectrum: a population-based study from the surveillance epidemiology and end results database. Pediatr Blood Cancer. 2011;57(6):943–9.PubMed
61.
go back to reference Perez EA et al. Rhabdomyosarcoma in children: a SEER population based study. J Surg Res. 2011;170(2):e243–51.PubMed Perez EA et al. Rhabdomyosarcoma in children: a SEER population based study. J Surg Res. 2011;170(2):e243–51.PubMed
62.
go back to reference Barr FG. Gene fusions involving PAX and FOX family members in alveolar rhabdomyosarcoma. Oncogene. 2001;20(40):5736–46.PubMed Barr FG. Gene fusions involving PAX and FOX family members in alveolar rhabdomyosarcoma. Oncogene. 2001;20(40):5736–46.PubMed
63.
go back to reference Williamson D et al. Fusion gene-negative alveolar rhabdomyosarcoma is clinically and molecularly indistinguishable from embryonal rhabdomyosarcoma. J Clin Oncol. 2010;28(13):2151–8.PubMed Williamson D et al. Fusion gene-negative alveolar rhabdomyosarcoma is clinically and molecularly indistinguishable from embryonal rhabdomyosarcoma. J Clin Oncol. 2010;28(13):2151–8.PubMed
64.
go back to reference Skapek SX et al. PAX-FOXO1 fusion status drives unfavorable outcome for children with rhabdomyosarcoma: a Children's Oncology Group report. Pediatr Blood Cancer. 2013;60(9):1411–7.PubMed Skapek SX et al. PAX-FOXO1 fusion status drives unfavorable outcome for children with rhabdomyosarcoma: a Children's Oncology Group report. Pediatr Blood Cancer. 2013;60(9):1411–7.PubMed
65.
go back to reference Pappo AS et al. Two consecutive phase II window trials of irinotecan alone or in combination with vincristine for the treatment of metastatic rhabdomyosarcoma: the Children's Oncology Group. J Clin Oncol. 2007;25(4):362–9.PubMed Pappo AS et al. Two consecutive phase II window trials of irinotecan alone or in combination with vincristine for the treatment of metastatic rhabdomyosarcoma: the Children's Oncology Group. J Clin Oncol. 2007;25(4):362–9.PubMed
66.
go back to reference Children's Oncology Group Fall Meeting. 2013. Dallas. Children's Oncology Group Fall Meeting. 2013. Dallas.
67.
go back to reference Weigel B et al. Early results from Children's Oncology Group (COG) ARST0431: intensive multidrug therapy for patients with metastatic rhabdomyosarcoma (RMS). J Clin Oncol. 2010;28(15 Suppl):9503. Weigel B et al. Early results from Children's Oncology Group (COG) ARST0431: intensive multidrug therapy for patients with metastatic rhabdomyosarcoma (RMS). J Clin Oncol. 2010;28(15 Suppl):9503.
68.
go back to reference Shern JF et al. Comprehensive genomic analysis of rhabdomyosarcoma reveals a landscape of alterations affecting a common genetic axis in fusion-positive and fusion-negative tumors. Cancer Discov. 2014;4(2):216–31.PubMed Shern JF et al. Comprehensive genomic analysis of rhabdomyosarcoma reveals a landscape of alterations affecting a common genetic axis in fusion-positive and fusion-negative tumors. Cancer Discov. 2014;4(2):216–31.PubMed
69.
go back to reference Shukla N et al. Oncogene mutation profiling of pediatric solid tumors reveals significant subsets of embryonal rhabdomyosarcoma and neuroblastoma with mutated genes in growth signaling pathways. Clin Cancer Res. 2012;18(3):748–57.PubMedCentralPubMed Shukla N et al. Oncogene mutation profiling of pediatric solid tumors reveals significant subsets of embryonal rhabdomyosarcoma and neuroblastoma with mutated genes in growth signaling pathways. Clin Cancer Res. 2012;18(3):748–57.PubMedCentralPubMed
70.
go back to reference Li SQ et al. Targeting wild-type and mutationally activated FGFR4 in rhabdomyosarcoma with the inhibitor ponatinib (AP24534). PLoS One. 2013;8(10):e76551.PubMedCentralPubMed Li SQ et al. Targeting wild-type and mutationally activated FGFR4 in rhabdomyosarcoma with the inhibitor ponatinib (AP24534). PLoS One. 2013;8(10):e76551.PubMedCentralPubMed
71.
go back to reference Gerber HP et al. Complete inhibition of rhabdomyosarcoma xenograft growth and neovascularization requires blockade of both tumor and host vascular endothelial growth factor. Cancer Res. 2000;60(22):6253–8.PubMed Gerber HP et al. Complete inhibition of rhabdomyosarcoma xenograft growth and neovascularization requires blockade of both tumor and host vascular endothelial growth factor. Cancer Res. 2000;60(22):6253–8.PubMed
72.
go back to reference Dilling MB et al. Rapamycin selectively inhibits the growth of childhood rhabdomyosarcoma cells through inhibition of signaling via the type I insulin-like growth factor receptor. Cancer Res. 1994;54(4):903–7.PubMed Dilling MB et al. Rapamycin selectively inhibits the growth of childhood rhabdomyosarcoma cells through inhibition of signaling via the type I insulin-like growth factor receptor. Cancer Res. 1994;54(4):903–7.PubMed
73.
go back to reference Houghton PJ et al. Initial testing (stage 1) of the mTOR inhibitor rapamycin by the Pediatric Preclinical Testing Program. Pediatr Blood Cancer. 2008;50(4):799–805.PubMed Houghton PJ et al. Initial testing (stage 1) of the mTOR inhibitor rapamycin by the Pediatric Preclinical Testing Program. Pediatr Blood Cancer. 2008;50(4):799–805.PubMed
74.
go back to reference Kolb EA et al. Initial testing (stage 1) of a monoclonal antibody (SCH 717454) against the IGF-1 receptor by the Pediatric Preclinical Testing Program. Pediatr Blood Cancer. 2008;50(6):1190–7.PubMed Kolb EA et al. Initial testing (stage 1) of a monoclonal antibody (SCH 717454) against the IGF-1 receptor by the Pediatric Preclinical Testing Program. Pediatr Blood Cancer. 2008;50(6):1190–7.PubMed
75.
go back to reference Ferrari A et al. Adult-type soft tissue sarcomas in pediatric-age patients: experience at the Istituto Nazionale Tumori in Milan. J Clin Oncol. 2005;23(18):4021–30.PubMed Ferrari A et al. Adult-type soft tissue sarcomas in pediatric-age patients: experience at the Istituto Nazionale Tumori in Milan. J Clin Oncol. 2005;23(18):4021–30.PubMed
76.
go back to reference Spunt SL et al. Prognostic factors for children and adolescents with surgically resected nonrhabdomyosarcoma soft tissue sarcoma: an analysis of 121 patients treated at St Jude Children's Research Hospital. J Clin Oncol. 1999;17(12):3697–705.PubMed Spunt SL et al. Prognostic factors for children and adolescents with surgically resected nonrhabdomyosarcoma soft tissue sarcoma: an analysis of 121 patients treated at St Jude Children's Research Hospital. J Clin Oncol. 1999;17(12):3697–705.PubMed
77.
go back to reference Blaes AH et al. Pathologic femur fractures after limb-sparing treatment of soft-tissue sarcomas. J Cancer Surviv. 2010;4(4):399–404.PubMed Blaes AH et al. Pathologic femur fractures after limb-sparing treatment of soft-tissue sarcomas. J Cancer Surviv. 2010;4(4):399–404.PubMed
78.
go back to reference Tukenova M et al. Radiation therapy and late mortality from second sarcoma, carcinoma, and hematological malignancies after a solid cancer in childhood. Int J Radiat Oncol Biol Phys. 2011;80(2):339–46.PubMed Tukenova M et al. Radiation therapy and late mortality from second sarcoma, carcinoma, and hematological malignancies after a solid cancer in childhood. Int J Radiat Oncol Biol Phys. 2011;80(2):339–46.PubMed
79.
go back to reference Pervaiz N et al. A systematic meta-analysis of randomized controlled trials of adjuvant chemotherapy for localized resectable soft-tissue sarcoma. Cancer. 2008;113(3):573–81.PubMed Pervaiz N et al. A systematic meta-analysis of randomized controlled trials of adjuvant chemotherapy for localized resectable soft-tissue sarcoma. Cancer. 2008;113(3):573–81.PubMed
80.
go back to reference Pappo AS et al. Phase II trial of neoadjuvant vincristine, ifosfamide, and doxorubicin with granulocyte colony-stimulating factor support in children and adolescents with advanced-stage nonrhabdomyosarcomatous soft tissue sarcomas: a Pediatric Oncology Group study. J Clin Oncol. 2005;23(18):4031–8.PubMed Pappo AS et al. Phase II trial of neoadjuvant vincristine, ifosfamide, and doxorubicin with granulocyte colony-stimulating factor support in children and adolescents with advanced-stage nonrhabdomyosarcomatous soft tissue sarcomas: a Pediatric Oncology Group study. J Clin Oncol. 2005;23(18):4031–8.PubMed
81.
go back to reference Pratt CB et al. Treatment of unresectable or metastatic pediatric soft tissue sarcomas with surgery, irradiation, and chemotherapy: a Pediatric Oncology Group study. Med Pediatr Oncol. 1998;30(4):201–9.PubMed Pratt CB et al. Treatment of unresectable or metastatic pediatric soft tissue sarcomas with surgery, irradiation, and chemotherapy: a Pediatric Oncology Group study. Med Pediatr Oncol. 1998;30(4):201–9.PubMed
82.
go back to reference Demetri GD et al. Efficacy and safety of sunitinib in patients with advanced gastrointestinal stromal tumour after failure of imatinib: a randomised controlled trial. Lancet. 2006;368(9544):1329–38.PubMed Demetri GD et al. Efficacy and safety of sunitinib in patients with advanced gastrointestinal stromal tumour after failure of imatinib: a randomised controlled trial. Lancet. 2006;368(9544):1329–38.PubMed
83.
go back to reference Gooskens SL et al. Imatinib mesylate for children with dermatofibrosarcoma protuberans (DFSP). Pediatr Blood Cancer. 2010;55(2):369–73.PubMed Gooskens SL et al. Imatinib mesylate for children with dermatofibrosarcoma protuberans (DFSP). Pediatr Blood Cancer. 2010;55(2):369–73.PubMed
84.
go back to reference Heinrich MC et al. Kinase mutations and imatinib response in patients with metastatic gastrointestinal stromal tumor. J Clin Oncol. 2003;21(23):4342–9.PubMed Heinrich MC et al. Kinase mutations and imatinib response in patients with metastatic gastrointestinal stromal tumor. J Clin Oncol. 2003;21(23):4342–9.PubMed
85.
go back to reference McArthur GA et al. Molecular and clinical analysis of locally advanced dermatofibrosarcoma protuberans treated with imatinib: Imatinib Target Exploration Consortium study B2225. J Clin Oncol. 2005;23(4):866–73.PubMed McArthur GA et al. Molecular and clinical analysis of locally advanced dermatofibrosarcoma protuberans treated with imatinib: Imatinib Target Exploration Consortium study B2225. J Clin Oncol. 2005;23(4):866–73.PubMed
86.
go back to reference Kadoch C, Crabtree GR. Reversible disruption of mSWI/SNF (BAF) complexes by the SS18-SSX oncogenic fusion in synovial sarcoma. Cell. 2013;153(1):71–85.PubMedCentralPubMed Kadoch C, Crabtree GR. Reversible disruption of mSWI/SNF (BAF) complexes by the SS18-SSX oncogenic fusion in synovial sarcoma. Cell. 2013;153(1):71–85.PubMedCentralPubMed
87.
go back to reference Barretina J et al. Subtype-specific genomic alterations define new targets for soft-tissue sarcoma therapy. Nat Genet. 2010;42(8):715–21.PubMedCentralPubMed Barretina J et al. Subtype-specific genomic alterations define new targets for soft-tissue sarcoma therapy. Nat Genet. 2010;42(8):715–21.PubMedCentralPubMed
88.
go back to reference Thomas RK et al. High-throughput oncogene mutation profiling in human cancer. Nat Genet. 2007;39(3):347–51.PubMed Thomas RK et al. High-throughput oncogene mutation profiling in human cancer. Nat Genet. 2007;39(3):347–51.PubMed
89.
go back to reference Wardelmann E et al. Soft tissue sarcoma: from molecular diagnosis to selection of treatment. Pathological diagnosis of soft tissue sarcoma amid molecular biology and targeted therapies. Ann Oncol. 2010;21 Suppl 7:265–9. Wardelmann E et al. Soft tissue sarcoma: from molecular diagnosis to selection of treatment. Pathological diagnosis of soft tissue sarcoma amid molecular biology and targeted therapies. Ann Oncol. 2010;21 Suppl 7:265–9.
90.
go back to reference Holtkamp N et al. Mutation and expression of PDGFRA and KIT in malignant peripheral nerve sheath tumors, and its implications for imatinib sensitivity. Carcinogenesis. 2006;27(3):664–71.PubMed Holtkamp N et al. Mutation and expression of PDGFRA and KIT in malignant peripheral nerve sheath tumors, and its implications for imatinib sensitivity. Carcinogenesis. 2006;27(3):664–71.PubMed
91.
go back to reference Park MS, Ravi V, Araujo DM. Inhibiting the VEGF-VEGFR pathway in angiosarcoma, epithelioid hemangioendothelioma, and hemangiopericytoma/solitary fibrous tumor. Curr Opin Oncol. 2010;22(4):351–5.PubMed Park MS, Ravi V, Araujo DM. Inhibiting the VEGF-VEGFR pathway in angiosarcoma, epithelioid hemangioendothelioma, and hemangiopericytoma/solitary fibrous tumor. Curr Opin Oncol. 2010;22(4):351–5.PubMed
92.
go back to reference Potti A et al. Determination of vascular endothelial growth factor (VEGF) overexpression in soft tissue sarcomas and the role of overexpression in leiomyosarcoma. J Cancer Res Clin Oncol. 2004;130(1):52–6.PubMed Potti A et al. Determination of vascular endothelial growth factor (VEGF) overexpression in soft tissue sarcomas and the role of overexpression in leiomyosarcoma. J Cancer Res Clin Oncol. 2004;130(1):52–6.PubMed
93.
go back to reference Tamborini E et al. Expression of ligand-activated KIT and platelet-derived growth factor receptor beta tyrosine kinase receptors in synovial sarcoma. Clin Cancer Res. 2004;10(3):938–43.PubMed Tamborini E et al. Expression of ligand-activated KIT and platelet-derived growth factor receptor beta tyrosine kinase receptors in synovial sarcoma. Clin Cancer Res. 2004;10(3):938–43.PubMed
94.
go back to reference Hurwitz HI et al. Phase I trial of pazopanib in patients with advanced cancer. Clin Cancer Res. 2009;15(12):4220–7.PubMed Hurwitz HI et al. Phase I trial of pazopanib in patients with advanced cancer. Clin Cancer Res. 2009;15(12):4220–7.PubMed
95.
go back to reference Sleijfer S 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(19):3126–32.PubMed Sleijfer S 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(19):3126–32.PubMed
96.
go back to reference van der Graaf WT et al. Pazopanib for metastatic soft-tissue sarcoma (PALETTE): a randomised, double-blind, placebo-controlled phase 3 trial. Lancet. 2012;379(9829):1879–86.PubMed van der Graaf WT et al. Pazopanib for metastatic soft-tissue sarcoma (PALETTE): a randomised, double-blind, placebo-controlled phase 3 trial. Lancet. 2012;379(9829):1879–86.PubMed
97.
go back to reference Glade Bender JL et al. Phase I pharmacokinetic and pharmacodynamic study of pazopanib in children with soft tissue sarcoma and other refractory solid tumors: a Children’s Oncology Group Phase I Consortium report. J Clin Oncol. 2013;31(24):3034–43.PubMedCentralPubMed Glade Bender JL et al. Phase I pharmacokinetic and pharmacodynamic study of pazopanib in children with soft tissue sarcoma and other refractory solid tumors: a Children’s Oncology Group Phase I Consortium report. J Clin Oncol. 2013;31(24):3034–43.PubMedCentralPubMed
98.
go back to reference Abou-Alfa GK, et al. Doxorubicin plus sorafenib vs doxorubicin alone in patients with advanced hepatocellular carcinoma: a randomized trial. JAMA. 304(19):2154-60. Abou-Alfa GK, et al. Doxorubicin plus sorafenib vs doxorubicin alone in patients with advanced hepatocellular carcinoma: a randomized trial. JAMA. 304(19):2154-60.
99.
go back to reference Wu YL et al. Intercalated combination of chemotherapy and erlotinib for patients with advanced stage non-small-cell lung cancer (FASTACT-2): a randomised, double-blind trial. Lancet Oncol. 2013;14(8):777–86.PubMed Wu YL et al. Intercalated combination of chemotherapy and erlotinib for patients with advanced stage non-small-cell lung cancer (FASTACT-2): a randomised, double-blind trial. Lancet Oncol. 2013;14(8):777–86.PubMed
100.
go back to reference Eilber FC et al. Treatment-induced pathologic necrosis: a predictor of local recurrence and survival in patients receiving neoadjuvant therapy for high-grade extremity soft tissue sarcomas. J Clin Oncol. 2001;19(13):3203–9.PubMed Eilber FC et al. Treatment-induced pathologic necrosis: a predictor of local recurrence and survival in patients receiving neoadjuvant therapy for high-grade extremity soft tissue sarcomas. J Clin Oncol. 2001;19(13):3203–9.PubMed
101.
go back to reference Alebouyeh M et al. Aggressive intra-abdominal fibromatosis in children and response to chemotherapy. Pediatr Hematol Oncol. 2005;22(6):447–51.PubMed Alebouyeh M et al. Aggressive intra-abdominal fibromatosis in children and response to chemotherapy. Pediatr Hematol Oncol. 2005;22(6):447–51.PubMed
102.
go back to reference Reitamo JJ, Scheinin TM, Hayry P. The desmoid syndrome. New aspects in the cause, pathogenesis and treatment of the desmoid tumor. Am J Surg. 1986;151(2):230–7.PubMed Reitamo JJ, Scheinin TM, Hayry P. The desmoid syndrome. New aspects in the cause, pathogenesis and treatment of the desmoid tumor. Am J Surg. 1986;151(2):230–7.PubMed
103.
go back to reference Bertario L et al. Genotype and phenotype factors as determinants of desmoid tumors in patients with familial adenomatous polyposis. Int J Cancer. 2001;95(2):102–7.PubMed Bertario L et al. Genotype and phenotype factors as determinants of desmoid tumors in patients with familial adenomatous polyposis. Int J Cancer. 2001;95(2):102–7.PubMed
104.
go back to reference Gomez Garcia EB, Knoers NV. Gardner's syndrome (familial adenomatous polyposis): a cilia-related disorder. Lancet Oncol. 2009;10(7):727–35.PubMed Gomez Garcia EB, Knoers NV. Gardner's syndrome (familial adenomatous polyposis): a cilia-related disorder. Lancet Oncol. 2009;10(7):727–35.PubMed
105.
go back to reference Pressey JG et al. Sirolimus therapy for fibromatosis and multifocal renal cell carcinoma in a child with tuberous sclerosis complex. Pediatr Blood Cancer. 2010;54(7):1035–7.PubMed Pressey JG et al. Sirolimus therapy for fibromatosis and multifocal renal cell carcinoma in a child with tuberous sclerosis complex. Pediatr Blood Cancer. 2010;54(7):1035–7.PubMed
106.
go back to reference Buitendijk S et al. Pediatric aggressive fibromatosis: a retrospective analysis of 13 patients and review of literature. Cancer. 2005;104(5):1090–9.PubMed Buitendijk S et al. Pediatric aggressive fibromatosis: a retrospective analysis of 13 patients and review of literature. Cancer. 2005;104(5):1090–9.PubMed
107.
go back to reference Faulkner LB et al. Pediatric desmoid tumor: retrospective analysis of 63 cases. J Clin Oncol. 1995;13(11):2813–8.PubMed Faulkner LB et al. Pediatric desmoid tumor: retrospective analysis of 63 cases. J Clin Oncol. 1995;13(11):2813–8.PubMed
108.
go back to reference Melis M, Zager JS, Sondak VK. Multimodality management of desmoid tumors: how important is a negative surgical margin? J Surg Oncol. 2008;98(8):594–602.PubMed Melis M, Zager JS, Sondak VK. Multimodality management of desmoid tumors: how important is a negative surgical margin? J Surg Oncol. 2008;98(8):594–602.PubMed
109.
go back to reference Jabbari S et al. Successful treatment of high risk and recurrent pediatric desmoids using radiation as a component of multimodality therapy. Int J Radiat Oncol Biol Phys. 2009;75(1):177–82.PubMed Jabbari S et al. Successful treatment of high risk and recurrent pediatric desmoids using radiation as a component of multimodality therapy. Int J Radiat Oncol Biol Phys. 2009;75(1):177–82.PubMed
110.
go back to reference Merchant TE et al. Long-term results with radiation therapy for pediatric desmoid tumors. Int J Radiat Oncol Biol Phys. 2000;47(5):1267–71.PubMed Merchant TE et al. Long-term results with radiation therapy for pediatric desmoid tumors. Int J Radiat Oncol Biol Phys. 2000;47(5):1267–71.PubMed
111.
go back to reference Ayala AG et al. Desmoid fibromatosis: a clinicopathologic study of 25 children. Semin Diagn Pathol. 1986;3(2):138–50.PubMed Ayala AG et al. Desmoid fibromatosis: a clinicopathologic study of 25 children. Semin Diagn Pathol. 1986;3(2):138–50.PubMed
112.
go back to reference Constantinidou A et al. Pegylated liposomal doxorubicin, an effective, well-tolerated treatment for refractory aggressive fibromatosis. Eur J Cancer. 2009;45(17):2930–4.PubMed Constantinidou A et al. Pegylated liposomal doxorubicin, an effective, well-tolerated treatment for refractory aggressive fibromatosis. Eur J Cancer. 2009;45(17):2930–4.PubMed
113.
go back to reference Heinrich MC et al. Clinical and molecular studies of the effect of imatinib on advanced aggressive fibromatosis (desmoid tumor). J Clin Oncol. 2006;24(7):1195–203.PubMed Heinrich MC et al. Clinical and molecular studies of the effect of imatinib on advanced aggressive fibromatosis (desmoid tumor). J Clin Oncol. 2006;24(7):1195–203.PubMed
114.
go back to reference Meazza C et al. Aggressive fibromatosis in children and adolescents: the Italian experience. Cancer. 2010;116(1):233–40.PubMed Meazza C et al. Aggressive fibromatosis in children and adolescents: the Italian experience. Cancer. 2010;116(1):233–40.PubMed
115.
go back to reference Raney B et al. Nonsurgical management of children with recurrent or unresectable fibromatosis. Pediatrics. 1987;79(3):394–8.PubMed Raney B et al. Nonsurgical management of children with recurrent or unresectable fibromatosis. Pediatrics. 1987;79(3):394–8.PubMed
116.
go back to reference Skapek SX et al. Vinblastine and methotrexate for desmoid fibromatosis in children: results of a Pediatric Oncology Group phase II trial. J Clin Oncol. 2007;25(5):501–6.PubMed Skapek SX et al. Vinblastine and methotrexate for desmoid fibromatosis in children: results of a Pediatric Oncology Group phase II trial. J Clin Oncol. 2007;25(5):501–6.PubMed
117.
go back to reference Skapek SX et al. Safety and efficacy of high-dose tamoxifen and sulindac for desmoid tumor in children: results of a Children's Oncology Group (COG) phase II study. Pediatr Blood Cancer. 2013;60(7):1108–12.PubMed Skapek SX et al. Safety and efficacy of high-dose tamoxifen and sulindac for desmoid tumor in children: results of a Children's Oncology Group (COG) phase II study. Pediatr Blood Cancer. 2013;60(7):1108–12.PubMed
118.
go back to reference Fiore M et al. Desmoid-type fibromatosis: a front-line conservative approach to select patients for surgical treatment. Ann Surg Oncol. 2009;16(9):2587–93.PubMed Fiore M et al. Desmoid-type fibromatosis: a front-line conservative approach to select patients for surgical treatment. Ann Surg Oncol. 2009;16(9):2587–93.PubMed
119.
go back to reference Gronchi A et al. Sporadic desmoid-type fibromatosis: a stepwise approach to a non-metastasising neoplasm—a position paper from the Italian and the French sarcoma group. Ann Oncol. 2014;25(3):578–83.PubMed Gronchi A et al. Sporadic desmoid-type fibromatosis: a stepwise approach to a non-metastasising neoplasm—a position paper from the Italian and the French sarcoma group. Ann Oncol. 2014;25(3):578–83.PubMed
120.
go back to reference Lazar AJ et al. Specific mutations in the β-catenin gene (CTNNB1) correlate with local recurrence in sporadic desmoid tumors. Am J Pathol. 2008;173(5):1518–27.PubMedCentralPubMed Lazar AJ et al. Specific mutations in the β-catenin gene (CTNNB1) correlate with local recurrence in sporadic desmoid tumors. Am J Pathol. 2008;173(5):1518–27.PubMedCentralPubMed
121.
go back to reference Tejpar S et al. Predominance of beta-catenin mutations and beta-catenin dysregulation in sporadic aggressive fibromatosis (desmoid tumor). Oncogene. 1999;18(47):6615–20.PubMed Tejpar S et al. Predominance of beta-catenin mutations and beta-catenin dysregulation in sporadic aggressive fibromatosis (desmoid tumor). Oncogene. 1999;18(47):6615–20.PubMed
122.
go back to reference Goldberg JM. Immunotherapy of sarcomas. Curr Opin Oncol. 2013;25(4):390–7.PubMed Goldberg JM. Immunotherapy of sarcomas. Curr Opin Oncol. 2013;25(4):390–7.PubMed
123.
go back to reference Strbo N, Podack ER. Secreted heat shock protein gp96-Ig: an innovative vaccine approach. Am J Reprod Immunol. 2008;59(5):407–16.PubMed Strbo N, Podack ER. Secreted heat shock protein gp96-Ig: an innovative vaccine approach. Am J Reprod Immunol. 2008;59(5):407–16.PubMed
124.
go back to reference Hodi FS et al. Improved survival with ipilimumab in patients with metastatic melanoma. N Engl J Med. 2010;363(8):711–23.PubMedCentralPubMed Hodi FS et al. Improved survival with ipilimumab in patients with metastatic melanoma. N Engl J Med. 2010;363(8):711–23.PubMedCentralPubMed
125.
go back to reference Brinkrolf P et al. A high proportion of bone marrow T cells with regulatory phenotype (CD4+CD25hiFoxP3+) in Ewing sarcoma patients is associated with metastatic disease. Int J Cancer. 2009;125(4):879–86.PubMed Brinkrolf P et al. A high proportion of bone marrow T cells with regulatory phenotype (CD4+CD25hiFoxP3+) in Ewing sarcoma patients is associated with metastatic disease. Int J Cancer. 2009;125(4):879–86.PubMed
126.
go back to reference Salama AK, Hodi FS. Cytotoxic T-lymphocyte-associated antigen-4. Clin Cancer Res. 2011;17(14):4622–8.PubMed Salama AK, Hodi FS. Cytotoxic T-lymphocyte-associated antigen-4. Clin Cancer Res. 2011;17(14):4622–8.PubMed
127.
go back to reference Topalian SL et al. Safety, activity, and immune correlates of anti-PD-1 antibody in cancer. N Engl J Med. 2012;366(26):2443–54.PubMedCentralPubMed Topalian SL et al. Safety, activity, and immune correlates of anti-PD-1 antibody in cancer. N Engl J Med. 2012;366(26):2443–54.PubMedCentralPubMed
128.
go back to reference Maki RG et al. A pilot study of anti-CTLA4 antibody ipilimumab in patients with synovial sarcoma. Sarcoma. 2013;2013:168145.PubMedCentralPubMed Maki RG et al. A pilot study of anti-CTLA4 antibody ipilimumab in patients with synovial sarcoma. Sarcoma. 2013;2013:168145.PubMedCentralPubMed
129.
go back to reference Robbins PF et al. Tumor regression in patients with metastatic synovial cell sarcoma and melanoma using genetically engineered lymphocytes reactive with NY-ESO-1. J Clin Oncol. 2011;29(7):917–24.PubMedCentralPubMed Robbins PF et al. Tumor regression in patients with metastatic synovial cell sarcoma and melanoma using genetically engineered lymphocytes reactive with NY-ESO-1. J Clin Oncol. 2011;29(7):917–24.PubMedCentralPubMed
130.
go back to reference Mackall CL et al. A pilot study of consolidative immunotherapy in patients with high-risk pediatric sarcomas. Clin Cancer Res. 2008;14(15):4850–8.PubMedCentralPubMed Mackall CL et al. A pilot study of consolidative immunotherapy in patients with high-risk pediatric sarcomas. Clin Cancer Res. 2008;14(15):4850–8.PubMedCentralPubMed
131.
go back to reference Burdach S et al. Allogeneic and autologous stem-cell transplantation in advanced Ewing tumors. An update after long-term follow-up from two centers of the European Intergroup study EICESS. Stem-cell transplant programs at Dusseldorf University Medical Center, Germany and St. Anna Kinderspital, Vienna, Austria. Ann Oncol. 2000;11(11):1451–62.PubMed Burdach S et al. Allogeneic and autologous stem-cell transplantation in advanced Ewing tumors. An update after long-term follow-up from two centers of the European Intergroup study EICESS. Stem-cell transplant programs at Dusseldorf University Medical Center, Germany and St. Anna Kinderspital, Vienna, Austria. Ann Oncol. 2000;11(11):1451–62.PubMed
132.
go back to reference Gore L et al. A phase I and pharmacokinetic study of the oral histone deacetylase inhibitor, MS-275, in patients with refractory solid tumors and lymphomas. Clin Cancer Res. 2008;14(14):4517–25.PubMedCentralPubMed Gore L et al. A phase I and pharmacokinetic study of the oral histone deacetylase inhibitor, MS-275, in patients with refractory solid tumors and lymphomas. Clin Cancer Res. 2008;14(14):4517–25.PubMedCentralPubMed
133.
go back to reference Berghuis D et al. Histone deacetylase inhibitors enhance expression of NKG2D ligands in Ewing sarcoma and sensitize for natural killer cell-mediated cytolysis. Clin Sarcoma Res. 2012;2(1):8.PubMedCentralPubMed Berghuis D et al. Histone deacetylase inhibitors enhance expression of NKG2D ligands in Ewing sarcoma and sensitize for natural killer cell-mediated cytolysis. Clin Sarcoma Res. 2012;2(1):8.PubMedCentralPubMed
134.
go back to reference Rao-Bindal K et al. The histone deacetylase inhibitor, MS-275 (entinostat), downregulates c-FLIP, sensitizes osteosarcoma cells to FasL, and induces the regression of osteosarcoma lung metastases. Curr Cancer Drug Targets. 2013;13(4):411–22.PubMedCentralPubMed Rao-Bindal K et al. The histone deacetylase inhibitor, MS-275 (entinostat), downregulates c-FLIP, sensitizes osteosarcoma cells to FasL, and induces the regression of osteosarcoma lung metastases. Curr Cancer Drug Targets. 2013;13(4):411–22.PubMedCentralPubMed
135.
go back to reference Keshelava N et al. Initial testing (stage 1) of vorinostat (SAHA) by the Pediatric Preclinical Testing Program. Pediatr Blood Cancer. 2009;53(3):505–8.PubMedCentralPubMed Keshelava N et al. Initial testing (stage 1) of vorinostat (SAHA) by the Pediatric Preclinical Testing Program. Pediatr Blood Cancer. 2009;53(3):505–8.PubMedCentralPubMed
136.
go back to reference Krishnadas DK et al. Decitabine facilitates immune recognition of sarcoma cells by upregulating CT antigens, MHC molecules, and ICAM-1. Tumor Biol. 2014. doi:10.1007/s13277-014-1764-9. Krishnadas DK et al. Decitabine facilitates immune recognition of sarcoma cells by upregulating CT antigens, MHC molecules, and ICAM-1. Tumor Biol. 2014. doi:10.​1007/​s13277-014-1764-9.
Metadata
Title
Advances in Therapy for Pediatric Sarcomas
Authors
Aaron Weiss
Jonathan Gill
John Goldberg
Joanne Lagmay
Holly Spraker-Perlman
Rajkumar Venkatramani
Damon Reed
Publication date
01-08-2014
Publisher
Springer US
Published in
Current Oncology Reports / Issue 8/2014
Print ISSN: 1523-3790
Electronic ISSN: 1534-6269
DOI
https://doi.org/10.1007/s11912-014-0395-z

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