Objective responses at the involved sites and patient status at last follow-up
| . | Response to treatment at the involved sites . | . | . | . | . | . | |||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Patient # . | Overall response . | Bone . | Cardiovascular system . | Retroperitoneum . | Endocrine system . | CNS . | Lung . | Other . | PET response . | Follow-up duration, (months)* . | Status at last follow-up . | Time on SRL + PDN, (months) . | Treatment at last follow-up . |
| 1 | PR | SD | SD | PR (reduction in peri-renal and mesenteric fibrosis) | — | — | — | Resolution of systemic symptoms and leg edema, no recurrence of bowel obstruction | Not performed | 108 | Alive | 108 | SRL + PDN |
| 2 | PR | SD | Complete resolution of pericarditis; and SD of atrial mass and coated aorta | PR (reduction in peri-renal and peri-aortic fibrosis) | Reduction in serum prolactin (42→17 ng/mL) | — | — | No reappearance of skin nodules | Not performed | 78 | Alive | 78 | SRL + PDN |
| 3 | PR | SD | — | CR (resolution of peri-aortic and mesenteric fibrosis) | — | SD | — | No reappearance of skin nodules | Negative at baseline | 76 | Alive | 76 | SRL + PDN |
| 4 | PR | SD | — | PR (reduction of periaortic and peri-renal fibrosis, and resolution of HDN) | Stable | — | SD | — | Negative at baseline | 70 | Alive | 69 | SRL + PDN |
| 5 | PR | SD | — | PR (reduction of peri-aortic fibrosis and resolution of HDN) | Stable | — | — | — | PMR | 25 | Deceased | 15 | None (withdrew SRL + PDN at month 15 for small-cell lung cancer) |
| 6 | PD | SD | — | — | — | PD | N/A | — | Not performed | 11 | Deceased | 11 | SRL + PDN |
| 7 | PR | SD | — | — | — | PR | — | Stable | Positive at baseline; follow-up PET not performed (lost to follow -up) | 11 | Alive (lost to follow-up) | 11 | SRL + PDN |
| 8 | PD | SD | PD (increase in myocardial infiltration) | PD (worsened HDN and bowel occlusions) | Stable | SD | Stable | Positive at baseline; not repeated for overt clinical progression | 32 | Alive | 32 | Vemurafenib (stopped SRL at month 25 for PD) | |
| 9 | SD | — | — | SD | — | — | — | Stable | SMD | 24 | Alive | 21 | SRL + PDN |
| 10 | SD | SD | SD (with complete resolution of pericardial effusion after drainage) | SD | Stable | — | — | Stable | CMR | 14 | Alive | 14 | SRL + PDN |
| . | Response to treatment at the involved sites . | . | . | . | . | . | |||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Patient # . | Overall response . | Bone . | Cardiovascular system . | Retroperitoneum . | Endocrine system . | CNS . | Lung . | Other . | PET response . | Follow-up duration, (months)* . | Status at last follow-up . | Time on SRL + PDN, (months) . | Treatment at last follow-up . |
| 1 | PR | SD | SD | PR (reduction in peri-renal and mesenteric fibrosis) | — | — | — | Resolution of systemic symptoms and leg edema, no recurrence of bowel obstruction | Not performed | 108 | Alive | 108 | SRL + PDN |
| 2 | PR | SD | Complete resolution of pericarditis; and SD of atrial mass and coated aorta | PR (reduction in peri-renal and peri-aortic fibrosis) | Reduction in serum prolactin (42→17 ng/mL) | — | — | No reappearance of skin nodules | Not performed | 78 | Alive | 78 | SRL + PDN |
| 3 | PR | SD | — | CR (resolution of peri-aortic and mesenteric fibrosis) | — | SD | — | No reappearance of skin nodules | Negative at baseline | 76 | Alive | 76 | SRL + PDN |
| 4 | PR | SD | — | PR (reduction of periaortic and peri-renal fibrosis, and resolution of HDN) | Stable | — | SD | — | Negative at baseline | 70 | Alive | 69 | SRL + PDN |
| 5 | PR | SD | — | PR (reduction of peri-aortic fibrosis and resolution of HDN) | Stable | — | — | — | PMR | 25 | Deceased | 15 | None (withdrew SRL + PDN at month 15 for small-cell lung cancer) |
| 6 | PD | SD | — | — | — | PD | N/A | — | Not performed | 11 | Deceased | 11 | SRL + PDN |
| 7 | PR | SD | — | — | — | PR | — | Stable | Positive at baseline; follow-up PET not performed (lost to follow -up) | 11 | Alive (lost to follow-up) | 11 | SRL + PDN |
| 8 | PD | SD | PD (increase in myocardial infiltration) | PD (worsened HDN and bowel occlusions) | Stable | SD | Stable | Positive at baseline; not repeated for overt clinical progression | 32 | Alive | 32 | Vemurafenib (stopped SRL at month 25 for PD) | |
| 9 | SD | — | — | SD | — | — | — | Stable | SMD | 24 | Alive | 21 | SRL + PDN |
| 10 | SD | SD | SD (with complete resolution of pericardial effusion after drainage) | SD | Stable | — | — | Stable | CMR | 14 | Alive | 14 | SRL + PDN |
Objective responses for measurable lesions were reported according to RECIST 1.0 as CR, PR, SD, or PD when assessed on measurable lesions by means of CT or MRI; for bone lesions, considered to be nonmeasurable, we classified responses according to RECIST 1.0 as CR, incomplete response/SD, or PD (see Methods). The response assessed by PET was reported as CMR, PMR, SMD, or progressive metabolic disease (see Methods). For other manifestations (eg, endocrine abnormalities and skin lesions), we provided descriptive responses. PET was performed at baseline and repeated only in patients with pathologic findings.
CMR, complete metabolic response; SMD, stable metabolic disease.
Follow-up duration has been calculated from the start of SRL + PDN treatment to last follow-up.