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EvidenceTherapeutic ClassDrug NameBrand NameTargetPatientsResultsReference
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Phase 2 Trial
Immune Checkpoint Inhibitor
PembrolizumabKeytrudaPD1343 PR, median PFS 6.6 monthsBlay et al. J Clin Oncol 2021
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Phase 2 TrialTherapeutic VaccineGI-6301TarmogenBrachyury24No difference in ORR for vaccine vs. placeboDeMaria et al. J Clin Oncol 2020
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Phase 2 Trial
Immune Checkpoint Inhibitor
Durvalumab + tremelimumab
Imfinzi + (no brand name)PD1 + CTLA451 PRSomaiah et al. J Clin Oncol 2020
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Phase 2 Trial
Immune Checkpoint Inhibitor
PembrolizumabKeytrudaPD1121 PR, median PFS 5.7 monthsBlay et al. Ann Oncol. 2019
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Phase 2 TrialTargeted TherapyDasatinibSprycelPDGRF, c-KIT32median PFS 6.3 monthsSchuetze et al. Cancer 2016
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Phase 2 TrialTargeted TherapySorafenibNexavarmultiple RTK271 PR, 73% SD (at 9 months)Bompas et al. Ann Oncol. 2015
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Phase 2 TrialTargeted TherapyLapatinibTykerbEGFR1813 SDStacchiotti et al. Ann Oncol. 2013
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Phase 2 TrialTargeted TherapyImatinibGleevecPDGFR501 PR, 70% SD, median PFS 9.2 monthsStacchiotti et al. Journal of Clinical Oncology, 2012
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Phase 2 TrialTargeted TherapyImatinib + everolimusGleevec + AfinitorPDGFR + mTOR431 PR, 86% SDStacchiotti et al. Cancer, 2018
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Phase 2 TrialTargeted TherapySunitinibSutentmultiple RTK94 SDGeorge et al. J Clin Oncol. 2009
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Phase 2 TrialCytotoxic TherapyRubitecanOrathecinN/A151 PRChugh et al. J Clin Oncol. 2005
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Phase 1 TrialTherapeutic VaccineMVA-BN-BrachyuryBrachyury101 PR, 2 SDDeMaria et al. J Clin Oncol 2021
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Phase 1 TrialTargeted Therapy + RadiationNilotinibTasignaPDGFR23median PFS 58.15 monthsCote et al. Int J Radiat Oncol Biol Phys
2018
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Phase 1 TrialTargeted TherapyTazemetostatTazverikEZH221 CR, 1 PRChi et al. 2017 (Abstract)
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Phase 1 TrialTherapeutic VaccineGI-6301TarmogenBrachyury10median PFS 8.3 monthsHeery et al. Cancer Immunol Res. 2015
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Phase 1 TrialTargeted Therapy + Cytotoxic TherapyImatinib mesyate + metronomic cyclophosphamideGleevec + CytoxanPDGFR7median PFS 10.2 monthsAdenis et al. British Journal of Cancer, 2013
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Case SeriesTargeted TherapyErlotinib Tarceva EGFR 314 PR, 14 SD, median PFS 6.2 monthsMir et al. J Clin Oncol 2021
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Case SeriesTargeted TherapyImatinibGleevecPDGFR623 PR, 43 SDLebellec et al. Eur J Cancer 2017
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Case SeriesTargeted TherapyErlotinibTarcevaEGFR51 PR, 4 SDLebellec et al. Eur J Cancer 2017
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Case SeriesTargeted TherapySorafenibNexavarmultiple RTK111PR, 9 SDLebellec et al. Eur J Cancer 2017
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Case SeriesTargeted TherapyPazopanib + SunitinibVotrient + Sutentmultiple RTK5median PFS 8.5 monthsLipplaa et al. Clin Sarcoma Res 2016
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Case SeriesTargeted TherapyImatinibGleevecPDGFR4634 SDHindi et al. Euro J Cancer 2015
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Case SeriesTargeted TherapyImatinibGleevecPDGFR22 SDRohatgi et al. Eurasian J Med 2015
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Case SeriesTargeted TherapyErlotinib + BevacizumabTarceva + AvastinEGFR, VEGF33 SDAsklund et. al. Acta Oncol. 2014 Jan
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Case SeriesTargeted TherapyImatinibGleevecPDGFR171 SDFerraresi et al. BMC Cancer. 2010
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Case SeriesTargeted TherapyImatinib + SirolimusGleevec + RapamunePDGFR + mTOR98 SDStacchiotti et al. Ann Oncol. 2009
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Case SeriesTargeted TherapyImatinibGleevecPDGFR31 PR, 2 SDGeoerger et al. Eur J Cancer 2009
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Case SeriesTargeted TherapyImatinibGleevecPDGFR64 symptomatic improvementCasali et al. Cancer 2004
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Case ReportTargeted TherapyPazopanib VotrientVEGFR/PDGFR11 SDRibeiro et al. Case Rep Oncol Med. 2018
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Case ReportTargeted TherapyTemsirolimusToriselmTOR11 SDLebellec et al. Eur J Cancer 2017
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Case Report
Immune Checkpoint Inhibitor
NivolumabOpdivoPD111 PRMigliorini et al. OncoImmunology 2017
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Case Report
Immune Checkpoint Inhibitor
PembrolizumabKeytrudaPD111 PRMigliorini et al. OncoImmunology 2017
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Case ReportTargeted TherapyPazopanib VotrientVEGFR/PDGFR11PRJagersberg et al. Acta Neurochir. 2017
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Case ReportTherapeutic VaccineMVX-ONCO-1N/A11 PRMigliorini et al. OncoImmunology 2017
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Case ReportTargeted TherapyErlotinib + LinsitinibTarceva + LinsitinibEGFR + IGF1R11 PRAleksic et al. Frontiers in Oncology 2017
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Case ReportTargeted TherapyErlotinibTarcevaEGFR11 PRHouessinon et al. Case Reports Oncol. 2015
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Case ReportTargeted TherapySorafenibNexavarmultiple RTK11 SDSvoboda et al. J Am Acad Dermatol. 2012
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Case ReportCytoxoic TherapyVincristine + clophosphamide +doxorubicin + etoposide + ifosfamide11 CR (durable for 3+ years)Al-Rahawan MM et al. Pediatr Blood Cancer 2012
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Case ReportTargeted TherapyThalidomideThalomide11 PRChay et al. J Clin Oncol. 2011
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Case ReportTargeted TherapyCetuximab + GefitinibErbitux + IressaEGFR11 PRLinden et al. Acta Oncol. 2009
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Case ReportTargeted TherapyErlotinibTarcevaEGFR11 PRSinghal et al. Anticancer Drugs. 2009
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Case ReportTargeted TherapyCetuximab + GefitinibErbitux + IressaEGFR11 PRHof et al. Onkologie. 2006
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Case ReportTargeted Therapy + Cytotoxic TherapyThalidomide + liposomal doxorubicinThalomid + Doxil11 SDSchönegger et al. Anticancer Drugs. 2005
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Case ReportCytotoxic TherapyIfosfamide + doxorubicin11 PRScimeca eta al. J Pediatr Hematol Oncol. 1996
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