ABCDEFGH
1
EvidenceTherapeutic ClassDrug NameBrand NameTargetPatientsResultsReference
2
Phase 2 TrialImmune Checkpoint InhibitorDurvalumab + tremelimumabImfinzi + (no brand name)PD-L1 + CTLA451 PRSomaiah et al. Lancet Oncol 2022
3
Phase 2 TrialImmune Checkpoint InhibitorPembrolizumabKeytrudaPD-1343 PR, median PFS 6.6 monthsBlay et al. J Clin Oncol 2021
4
Phase 2 TrialTargeted TherapyApatinibAitanVEGF278 PR, median PFS 18 monthsLiu et al. Lancet Oncol 2020
5
Phase 2 TrialTherapeutic VaccineGI-6301TarmogenBrachyury24No difference in ORR for vaccine vs. placeboDeMaria et al. J Clin Oncol 2020
6
Phase 2 TrialImmune Checkpoint InhibitorPembrolizumabKeytrudaPD-1121 PR, median PFS 5.7 monthsBlay et al. Ann Oncol. 2019
7
Phase 2 TrialTargeted TherapyDasatinibSprycelPDGRF, c-KIT32median PFS 6.3 monthsSchuetze et al. Cancer 2016
8
Phase 2 TrialTargeted TherapySorafenibNexavarmultiple RTK271 PR, 73% SD (at 9 months)Bompas et al. Ann Oncol. 2015
9
Phase 2 TrialTargeted TherapyLapatinibTykerbEGFR1813 SDStacchiotti et al. Ann Oncol. 2013
10
Phase 2 TrialTargeted TherapyImatinibGleevecPDGFR501 PR, 70% SD, median PFS 9.2 monthsStacchiotti et al. Journal of Clinical Oncology, 2012
11
Phase 2 TrialTargeted TherapyImatinib + everolimusGleevec + AfinitorPDGFR + mTOR431 PR, 86% SDStacchiotti et al. Cancer, 2018
12
Phase 2 TrialTargeted TherapySunitinibSutentmultiple RTK94 SDGeorge et al. J Clin Oncol. 2009
13
Phase 2 TrialCytotoxic TherapyRubitecanOrathecinN/A151 PRChugh et al. J Clin Oncol. 2005
14
15
Phase 1 TrialTherapeutic VaccineMVA-BN-BrachyuryBrachyury101 PR, 2 SDDeMaria et al. J Clin Oncol 2021
16
Phase 1 TrialTargeted Therapy + RadiationNilotinibTasignaPDGFR23median PFS 58.15 monthsCote et al. Int J Radiat Oncol Biol Phys
2018
17
Phase 1 TrialTargeted TherapyTazemetostatTazverikEZH221 CR, 1 PRChi et al. 2017 (Abstract)
18
Phase 1 TrialTherapeutic VaccineGI-6301TarmogenBrachyury10median PFS 8.3 monthsHeery et al. Cancer Immunol Res. 2015
19
Phase 1 TrialTargeted Therapy + Cytotoxic TherapyImatinib mesyate + metronomic cyclophosphamideGleevec + CytoxanPDGFR7median PFS 10.2 monthsAdenis et al. British Journal of Cancer, 2013
20
21
Case SeriesCytotoxic Therapy +/- Targeted TherapyCisplatin +/- imatinibPlatinol +/- GleevecPDGFR (imatinib)3227 SD, median PFS 8 monthsBaldi et al. Cancer 2022
22
Case SeriesImmune Checkpoint InhibitorNivolumabOpdivoPD-121 PRWilliamson et al. NPJ Precis Oncol 2021
23
Case SeriesTargeted TherapyErlotinib Tarceva EGFR 314 PR, 14 SD, median PFS 6.2 monthsMir et al. J Clin Oncol 2021
24
Case SeriesTargeted TherapyImatinibGleevecPDGFR623 PR, 43 SDLebellec et al. Eur J Cancer 2017
25
Case SeriesTargeted TherapyErlotinibTarcevaEGFR51 PR, 4 SDLebellec et al. Eur J Cancer 2017
26
Case SeriesTargeted TherapySorafenibNexavarmultiple RTK111PR, 9 SDLebellec et al. Eur J Cancer 2017
27
Case SeriesTargeted TherapyPazopanib + SunitinibVotrient + Sutentmultiple RTK5median PFS 8.5 monthsLipplaa et al. Clin Sarcoma Res 2016
28
Case SeriesTargeted TherapyImatinibGleevecPDGFR4634 SDHindi et al. Euro J Cancer 2015
29
Case SeriesTargeted TherapyImatinibGleevecPDGFR22 SDRohatgi et al. Eurasian J Med 2015
30
Case SeriesTargeted TherapyErlotinib + BevacizumabTarceva + AvastinEGFR, VEGF33 SDAsklund et. al. Acta Oncol. 2014 Jan
31
Case SeriesTargeted TherapyImatinibGleevecPDGFR171 SDFerraresi et al. BMC Cancer. 2010
32
Case SeriesTargeted TherapyImatinib + SirolimusGleevec + RapamunePDGFR + mTOR98 SDStacchiotti et al. Ann Oncol. 2009
33
Case SeriesTargeted TherapyImatinibGleevecPDGFR31 PR, 2 SDGeoerger et al. Eur J Cancer 2009
34
Case SeriesTargeted TherapyImatinibGleevecPDGFR64 symptomatic improvementCasali et al. Cancer 2004
35
36
Case ReportCytotoxic TherapyIfosfomide + etoposide; vincristine + doxorubicin + cyclophosphamide; ifosfamide + carboplatin + etoposide; pazopanib1 (pediatric)1 PR (>90%, for 5 years)Miyahara et al. J Pediatr Hematol Oncol 2020
37
Case ReportTargeted TherapyPazopanib VotrientVEGFR/PDGFR11 SDRibeiro et al. Case Rep Oncol Med. 2018
38
Case ReportTargeted TherapyTemsirolimusToriselmTOR11 SDLebellec et al. Eur J Cancer 2017
39
Case ReportImmune Checkpoint InhibitorNivolumabOpdivoPD-111 PRMigliorini et al. OncoImmunology 2017
40
Case ReportImmune Checkpoint InhibitorPembrolizumabKeytrudaPD-111 PRMigliorini et al. OncoImmunology 2017
41
Case ReportTargeted TherapyPazopanib VotrientVEGFR/PDGFR11PRJagersberg et al. Acta Neurochir. 2017
42
Case ReportTherapeutic VaccineMVX-ONCO-1N/A11 PRMigliorini et al. OncoImmunology 2017
43
Case ReportTargeted TherapyErlotinib + LinsitinibTarceva + LinsitinibEGFR + IGF1R11 PRAleksic et al. Frontiers in Oncology 2017
44
Case ReportTargeted TherapyErlotinibTarcevaEGFR11 PRHouessinon et al. Case Reports Oncol. 2015
45
Case ReportTargeted TherapySorafenibNexavarmultiple RTK11 SDSvoboda et al. J Am Acad Dermatol. 2012
46
Case ReportCytoxoic TherapyVincristine + cyclophosphamide +doxorubicin + etoposide + ifosfamide1 (pediatric)1 CR (durable for 3+ years)Al-Rahawan MM et al. Pediatr Blood Cancer 2012
47
Case ReportTargeted TherapyThalidomideThalomide11 PRChay et al. J Clin Oncol. 2011
48
Case ReportTargeted TherapyCetuximab + GefitinibErbitux + IressaEGFR11 PRLinden et al. Acta Oncol. 2009
49
Case ReportTargeted TherapyErlotinibTarcevaEGFR11 PRSinghal et al. Anticancer Drugs. 2009
50
Case ReportTargeted TherapyCetuximab + GefitinibErbitux + IressaEGFR11 PRHof et al. Onkologie. 2006
51
Case ReportTargeted Therapy + Cytotoxic TherapyThalidomide + liposomal doxorubicinThalomid + Doxil11 SDSchönegger et al. Anticancer Drugs. 2005
52
Case ReportCytotoxic TherapyIfosfamide + doxorubicin1 (pediatric)1 PRScimeca eta al. J Pediatr Hematol Oncol. 1996
53
54
55
56
57
58
59
60
61
62
63
64
65
66
67
68
69
70
71
72
73
74
75
76
77
78
79
80
81
82
83
84
85
86
87
88
89
90
91
92
93
94
95
96
97
98
99
100