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Table 2 Use of PDGFR kinase inhibitors in clinical trials for different tumors

From: Targeting the PDGF signaling pathway in tumor treatment

Tumor type Results of patient studies Refs
Glioblastoma multifome Only limited effects of single agent treatment by imatinib in Phase II and Phase III studies. [103, 104]
No significant effect of imatinib treatment in combination with hydroxyurea. [105, 106, 108]
Chordoma 1 PR and 35 SD out of 50 patients treated, were observed in a Phase II study. [110]
Meningeoma No or only modest effect of imatinib as single agent or combined with hydroxyurea. [111, 112]
Among 9 patients preselected for PDGFR expression, 7 SD were noted. [113]
Dermatofibrosarcoma protuberance In a Phase II study, 4 CR and 4 PR out of 12 patients treated were recorded. [126]
In other Phase II trials, PR was noticed in about half of the patients. [117, 127130]
Gastrointestinal stromal tumor Imatinib and other tyrosine kinase inhibitors against PDGFRα and Kit are used routinely in the clinic with good results [132138]
Soft tissue sarcoma In a Phase III study with 369 patients a median progression-free survival of 4.6 months was noted in patients treated with pazopanib compared with 1.6 months for untreated controls [149]
Osteosarcoma No advantage of treatment with imatinib as single agent. [141]
Some effect of imatinib in combination with everolimus in treatment of synovial sarcoma. [142]
Chronic myeloproliferative diseases Patients with CMML with rearrangement of PDGFRβ responded to imatinib. [168]
Hypereosinophilic syndrome Patients with HES responded to imatinib. [169172]
Patients who developed resistance to imatinib responded to nilotinib or sorafenib. [174, 175]
Prostate cancer Out of 44 patients with hormone-refractory prostate cancer treated with sunitinib, 1 had PR, 3 a decline in prostate specific antigen of >50%, and 9 had a significant improvement in pain. [181]
No increased survival upon treatment with imatinib. [195, 196]
Non-small cell lung cancer Combination treatment with imatinib and docetaxel yielded 1 PR and 4 SD out of 20 treated patients. [213]
2 PR and 7 SD were observed after treatment of 18 patients with sunitinib. [214]
Neuroblastoma Little or no effect by imatinib as single agent treatment of children with relapsed or refractory neuroblastoma. [236]
  1. CR, complete response; PR, partial response; SD, stable disease.