Summary of various responses in 31 patients
| Patient no. . | ONLS score . | Organomegaly response . | Extravascular volume overload response . | sPAP response . | Hematologic response . | Serum VEGF, pg/mL . | ||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 0 mo . | 3 mo . | 6 mo . | 12 mo . | 15 mo . | Last visit* . | 0 mo . | Last visit† . | |||||
| 1 | 1 | 1 | 0 | 0 | 0 | 0 (21 mo) | NR | R | R | PR | 728 | 223 |
| 2 | 1 | 0 | 0 | 0 | 0 | 0 (27 mo) | R | R | NS | CR | 1298 | 412 |
| 3 | 1 | 0 | 0 | 0 | 0 | 0 (21 mo) | R | R | NS | SD | 309 | 231 |
| 4 | 3 | 2 | 1 | 1 | 1 | 1 (27 mo) | R | R | NS | CR | 2197 | 127 |
| 5 | 3 | 2 | 1 | 1 | 0 | 0 (21 mo) | R | R | R | PR | 1010 | 478 |
| 6 | 8 | 6 | 3 | 1 | 1 | 1 (36 mo) | R | NS | NS | PR | 380 | 236 |
| 7 | 8 | 6 | 5 | 4 | 2 | 2 (36 mo) | R | R | R | CR | 1537 | 459 |
| 8 | 7 | 5 | 2 | 2 | 2 | 2 (27 mo) | R | R | NS | CR | 475 | 120 |
| 9 | 2 | 2 | 1 | 1 | 1 | 1 (30 mo) | R | R | R | PR | 877 | 400 |
| 10 | 5 | 2 | 2 | 2 | 2 | 2 (30 mo) | R | R | NS | PR | 1147 | 134 |
| 11 | 3 | 2 | 1 | 1 | 1 | 1 (27 mo) | R | R | NS | CR | 1131 | 205 |
| 12 | 9 | 6 | 5 | 4 | 3 | 3 (21 mo) | NR | NR | NS | PR | 666 | 364 |
| 13 | 9 | 4 | 4 | 3 | 3 | 3 (21 mo) | R | R | R | SD | 697 | 533 |
| 14 | 8 | 6 | 5 | 4 | 3 | 3 (21 mo) | R | R | NR | SD | 974 | 588 |
| 15 | 7 | 3 | 2 | 2 | 2 | 2 (21 mo) | NR | R | R | CR | 1249 | 359 |
| 16 | 8 | 5 | 4 | 4 | 3 | 3 (21 mo) | NR | R | NS | PR | 1290 | 168 |
| 17 | 2 | 1 | 0 | 0 | 0 | 0 (24 mo) | R | R | R | CR | 1081 | 204 |
| 18 | 4 | 2 | 0 | 0 | 0 | 0 (21 mo) | R | R | R | PR | 1196 | 377 |
| 19 | 1 | 0 | 0 | 0 | 0 | 0 (24 mo) | NS | NS | NS | CR | 344 | 196 |
| 20 | 2 | 2 | 2 | 0 | 0 | 0 (21 mo) | R | R | R | PR | 1369 | 275 |
| 21 | 2 | 1 | 1 | 1 | 1 | 1 (24 mo) | NR | R | R | SD | 1047 | 310 |
| 22 | 10 | 10 | 8 | 5 | 4 | 4 (18 mo) | NR | NR | R | PR | 1600 | 373 |
| 23 | 4 | 3 | 3 | 3 | 3 | 3 (18 mo) | NR | NR | R | SD | 1590 | 248 |
| 24 | 5 | 4 | 3 | 2 | 1 | 1 (21 mo) | R | R | R | PR | 473 | 190 |
| 25 | 6 | 3 | 3 | 3 | 2 | 2 (27 mo) | R | R | R | CR | 1048 | 461 |
| 26 | 4 | 3 | 2 | 0 | 0 | 0 (30 mo) | NS | R | NS | CR | 249 | 183 |
| 27 | 4 | 3 | 2 | 1 | 1 | 1 (18 mo) | R | R | NS | CR | 927 | 312 |
| 28 | 5 | 5 | 3 | 2 | 2 | 2 (15 mo) | NR | R | NS | PR | 789 | 454 |
| 29 | 6 | 5 | 5 | 3 | 2 | 2 (15 mo) | NS | R | NS | CR | 452 | 320 |
| 30 | 4 | 4 | 3 | 3 | 2 | 2 (15 mo) | NR | R | NS | SD | 1590 | 368 |
| 31 | 5 | 5 | 5 | 3 | 3 | 3 (15 mo) | NR | NR | NS | PR | 2334 | 782 |
| Response rate | 100% | 64.3% | 71.4% | 93.3% | 80.6% | 95.8% | ||||||
| Patient no. . | ONLS score . | Organomegaly response . | Extravascular volume overload response . | sPAP response . | Hematologic response . | Serum VEGF, pg/mL . | ||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 0 mo . | 3 mo . | 6 mo . | 12 mo . | 15 mo . | Last visit* . | 0 mo . | Last visit† . | |||||
| 1 | 1 | 1 | 0 | 0 | 0 | 0 (21 mo) | NR | R | R | PR | 728 | 223 |
| 2 | 1 | 0 | 0 | 0 | 0 | 0 (27 mo) | R | R | NS | CR | 1298 | 412 |
| 3 | 1 | 0 | 0 | 0 | 0 | 0 (21 mo) | R | R | NS | SD | 309 | 231 |
| 4 | 3 | 2 | 1 | 1 | 1 | 1 (27 mo) | R | R | NS | CR | 2197 | 127 |
| 5 | 3 | 2 | 1 | 1 | 0 | 0 (21 mo) | R | R | R | PR | 1010 | 478 |
| 6 | 8 | 6 | 3 | 1 | 1 | 1 (36 mo) | R | NS | NS | PR | 380 | 236 |
| 7 | 8 | 6 | 5 | 4 | 2 | 2 (36 mo) | R | R | R | CR | 1537 | 459 |
| 8 | 7 | 5 | 2 | 2 | 2 | 2 (27 mo) | R | R | NS | CR | 475 | 120 |
| 9 | 2 | 2 | 1 | 1 | 1 | 1 (30 mo) | R | R | R | PR | 877 | 400 |
| 10 | 5 | 2 | 2 | 2 | 2 | 2 (30 mo) | R | R | NS | PR | 1147 | 134 |
| 11 | 3 | 2 | 1 | 1 | 1 | 1 (27 mo) | R | R | NS | CR | 1131 | 205 |
| 12 | 9 | 6 | 5 | 4 | 3 | 3 (21 mo) | NR | NR | NS | PR | 666 | 364 |
| 13 | 9 | 4 | 4 | 3 | 3 | 3 (21 mo) | R | R | R | SD | 697 | 533 |
| 14 | 8 | 6 | 5 | 4 | 3 | 3 (21 mo) | R | R | NR | SD | 974 | 588 |
| 15 | 7 | 3 | 2 | 2 | 2 | 2 (21 mo) | NR | R | R | CR | 1249 | 359 |
| 16 | 8 | 5 | 4 | 4 | 3 | 3 (21 mo) | NR | R | NS | PR | 1290 | 168 |
| 17 | 2 | 1 | 0 | 0 | 0 | 0 (24 mo) | R | R | R | CR | 1081 | 204 |
| 18 | 4 | 2 | 0 | 0 | 0 | 0 (21 mo) | R | R | R | PR | 1196 | 377 |
| 19 | 1 | 0 | 0 | 0 | 0 | 0 (24 mo) | NS | NS | NS | CR | 344 | 196 |
| 20 | 2 | 2 | 2 | 0 | 0 | 0 (21 mo) | R | R | R | PR | 1369 | 275 |
| 21 | 2 | 1 | 1 | 1 | 1 | 1 (24 mo) | NR | R | R | SD | 1047 | 310 |
| 22 | 10 | 10 | 8 | 5 | 4 | 4 (18 mo) | NR | NR | R | PR | 1600 | 373 |
| 23 | 4 | 3 | 3 | 3 | 3 | 3 (18 mo) | NR | NR | R | SD | 1590 | 248 |
| 24 | 5 | 4 | 3 | 2 | 1 | 1 (21 mo) | R | R | R | PR | 473 | 190 |
| 25 | 6 | 3 | 3 | 3 | 2 | 2 (27 mo) | R | R | R | CR | 1048 | 461 |
| 26 | 4 | 3 | 2 | 0 | 0 | 0 (30 mo) | NS | R | NS | CR | 249 | 183 |
| 27 | 4 | 3 | 2 | 1 | 1 | 1 (18 mo) | R | R | NS | CR | 927 | 312 |
| 28 | 5 | 5 | 3 | 2 | 2 | 2 (15 mo) | NR | R | NS | PR | 789 | 454 |
| 29 | 6 | 5 | 5 | 3 | 2 | 2 (15 mo) | NS | R | NS | CR | 452 | 320 |
| 30 | 4 | 4 | 3 | 3 | 2 | 2 (15 mo) | NR | R | NS | SD | 1590 | 368 |
| 31 | 5 | 5 | 5 | 3 | 3 | 3 (15 mo) | NR | NR | NS | PR | 2334 | 782 |
| Response rate | 100% | 64.3% | 71.4% | 93.3% | 80.6% | 95.8% | ||||||
ONLS indicates overall neuropathy limitation scale; sPAP, systolic pulmonary arterial pressure; VEGF, vascular endothelial growth factor; NR, nonresponse; R, response; PR, partial remission; NS, without related symptoms; CR, complete remission; and SD, stable disease.
Time in parentheses is the actual time after treatment at the last visit.
Same visit as when the last ONLS score was performed.