Table 2.
Use of PDGFR kinase inhibitors in clinical trials for different tumors
| 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. |
[117,127-130] |
|
| Gastrointestinal stromal tumor |
Imatinib and other tyrosine kinase inhibitors against PDGFRα and Kit are used routinely in the clinic with good results |
[132-138] |
| 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. |
[169-172] |
| 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] |
CR, complete response; PR, partial response; SD, stable disease.