Table 1. Patient Characteristics for Cases with SDH Mutations vs Original Exon 11 Mutations.
| SDH-mutant (n=12) |
Original KIT Exon 11 (n=282) |
p-value | |||
|---|---|---|---|---|---|
| No. | % | No. | % | ||
| Treatment Arm | |||||
| Low-Dose | 7 | 58% | 140 | 50% | |
| High-Dose | 5 | 42% | 142 | 50% | |
| AGE AT STUDY ENTRY | <0.0001 | ||||
| Median (range) | |||||
| < 40 years | 7 | 58% | 24 | 8% | |
| 40 to <50 years | 2 | 17% | 36 | 13% | |
| 50 to <60 years | 3 | 25% | 67 | 24% | |
| 60 to <70 years | 0 | 0% | 74 | 26% | |
| ≥ 70 years | 0 | 0% | 81 | 29% | |
| GENDER | 0.03 | ||||
| Female | 9 | 75% | 120 | 43% | |
| Male | 3 | 25% | 162 | 57% | |
| RACE | 0.45 | ||||
| White | 9 | 75% | 231 | 82% | |
| Black | 1 | 8% | 30 | 11% | |
| Asian | 2 | 17% | 16 | 6% | |
| Pacific Islander | 0 | 0% | 1 | <1% | |
| Native American | 0 | 0% | 1 | <1% | |
| Unknown | |||||
| Ethnicity | 0.14 | ||||
| Non-Hispanic | 10 | 83% | 266 | 94% | |
| Hispanic | 2 | 17% | 8 | 3% | |
| Unknown | 0 | 0% | 8 | 3% | |
| PERFORMANCE STATUS | 0.23 | ||||
| 0-1 | 12 | 100% | 237 | 85% | |
| 2-3 | 0 | 0% | 43 | 15% | |
| TYPE OF DISEASE | 0.34 | ||||
| Measurable | 12 | 100% | 262 | 93%% | |
| Non-measurable | 0 | 0% | 20 | 7% | |
| PRIOR CHEMOTHERAPY | 0.75 | ||||
| No | 9 | 82% | 206 | 78% | |
| Yes | 2 | 18% | 59 | 22% | |
| PRIOR SURGERY | 0.89 | ||||
| No | 1 | 8% | 20 | 7% | |
| Yes | 11 | 92% | 256 | 98% | |
| FAMILY HISTORY OF GIST | 0.08 | ||||
| No/unknown | 11 | 92% | 281 | 100% | |
| Yes | 1 | 8% | 1 | <1% | |
| PRIOR RT | 0.21 | ||||
| No | 11 | 100% | 238 | 88% | |
| Yes | 0 | 0% | 33 | 12% | |
| SMALL BOWEL ORIGIN | 0.02 | ||||
| No | 11 | 100% | 172 | 66% | |
| Yes | 0 | 0% | 90 | 34% | |
| Abdomen | 2 | 18% | 56 | 21% | |
| Stomach | 9 | 82% | 108 | 41% | |
| Small Bowel | 0 | 0% | 90 | 34% | |
| Other | 0 | 0% | 8 | 3% | |
| MAXIMUM DIAMETER | 0.35 | ||||
| < 4.0 cm | 2 | 17% | 63 | 22% | |
| 4.0 - 8.0 cm | 6 | 50% | 72 | 26% | |
| 8.0 - 12.0 cm | 1 | 8% | 67 | 24% | |
| ≥ 12.0 cm | 3 | 25% | 70 | 25% | |
| unknown | 0 | 0% | 10 | 3% | |
| TIME SINCE INITIAL DIAGNOSIS | 0.58 | ||||
| < 12 months | 0 | 0% | 7 | 2% | |
| ≥ 12 months | 12 | 100% | 275 | 98% | |
| Hemoglobin | 12.4 (9.6 – 14.0) | 12.8 (8.4 – 19.5) | |||
| ANC | 3.7 (1.7 – 5.7) | 4.6 (1.3 – 18.5) | |||
| WBC | 5.5 (3.7 – 8.2) | 7.1 (3.5 – 21.5) | |||
| Platelets | 233 (131 – 1188) | 271 (103 – 890) | |||
| Albumin | 4.2 (3.7 – 4.7) | 3.8 (2.0 – 8.3) | |||
| Bilirubin | 0.35 (0.2 – 0.6) | 0.52 (0.10 – 8.00) | |||
| Creatinine | 0.78 (0.09 – 1.10) | 0.9 (0.12 – 2.00) | |||
| Response Rate | 8% (0% – 48%) | 66% (60% – 71%) | 0.0001 | ||
| CR (confirmed and unconfirmed) | 0% (0% – 26%) | 6% (3% – 10%) | |||
| PR (confirmed and unconfirmed) | 8% (0% – 48%) | 59% (53% – 65%) | |||
| Stable | 50% (21% – 79%) | 18% (14% – 23%) | |||
| Increasing Disease / Not Assessable | 33% (10% – 65%) | 16% (12% – 21%) | |||
| Median OS | 116 mo. (22 mo. – 116 mo.) | 66 mo. (57 mo. – 78 mo.) | p=0.38* | ||
| Median PFS | 9 mo. (3 mo. – 58 mo.) | 24 (21mo. – 28 mo.) | p=0.07* | ||
*
Adjusted for all prognostically significant factors found in the analysis of eligible patients.