Table 5. Rank-ordered PFS by DFMO dose, ODC genotype and urinary polyamines.
| Patient # | PFS (days) | Response after Cycle 1 (DFMO) | Best Response (CT/MIBG) | Status or Reason off study* | DFMO Dose (mg/m2) | ODC SNP rs2302615/ rs2302616 | UPA **Cycle 1 Day 1 (μmol/g Creatinine) | UPA **Cycle 1 Day 8 (μmol/g Creatinine) | DAS increase from Cycle 1 Day 1 |
|---|---|---|---|---|---|---|---|---|---|
| 1 | 1573 | SD | SD/PR | Alive (PF) | 500 | GA/TG | NA*** | 15.7 | NA |
| 2 | 1559 | SD | SD | Alive (PF) | 500 | GG/TG | 19.72 | 11.69 | No |
| 3 | 663 | SD | SD/PR | Alive (PF) | 1500 | GG/TT | 9 | 5.61 | Yes |
| 4 | 418 | SD | SD | PD | 750 | GA/GG | 2.25 | 7.8 | No |
| 5 | 239 | SD | SD | PD | 1000 | GG/TG | 40.12 | 8.71 | No |
| 6 | 209 | PD | (CT Neg)/PR | PD | 1500 | GA/TG | 12.23 | 3.98 | No |
| 7 | 136 | PD | SD | 2nd Leukemia | 1500 | GG/GG | 4.58 | 6.99 | No |
| 8 | 103 | SD | SD | PD | 750 | GG/GG | 5.04 | NA*** | No |
| 9 | 94 | SD | SD | PD | 500 | GG/TG | 10.18 | 4.2 | Yes |
| 10 | 67 | SD | PD | PD | 750 | GG/GG | 26.75 | 22.08 | Yes |
| 11 | 64 | PD | PD | PD | 1000 | AA/GG | 4.97 | 3.89 | Yes |
| 12 | 62 | SD | SD | PD | 1500 | AA/GG | 2.85 | 3.77 | No |
| 13 | 62 | SD | SD | PD | 1500 | GA/TG | 11.53 | 8.81 | Yes |
| 14 | 62 | SD | SD | PD | 1500 | GA/TG | 15.35 | 7.38 | Yes |
| 15 | 59 | PD | PD | PD | 1000 | GA/GG | 6.8 | 3.28 | Yes |
| 16 | 57 | SD | SD | PD | 750 | GG/GG | 2.16 | 1.94 | Yes |
| 17 | 31 | PD | PD | PD | 750 | GA/GG | 15.34 | 13.46 | Yes |
| 18 | 21 | PD | PD | PD | 1500 | GG/TG | 7.49 | 5.93 | Yes |
*PF = progression free, PD = progressive disease
2nd Leukemia = secondary leukemia
**Substrates for the tissue polyamine exporter SLC3A2 include the sum of putrescine, N1AcSpd, N8AcSpd and DAS; D1C1 = day 1, cycle 1, D8C1 = day 8 cycle 1
***NA = samples not available