Table 1.
Baseline characteristics and outcomes in the three patients.
| Variables | Patient 1 | Patient 2 | Patient 3 | Reference ranges | Units |
|---|---|---|---|---|---|
| Age | 56 | 56 | 72 | Years | |
| Sex | M | F | F | ||
| Stage at initial diagnosis | CP | CP | APa | ||
| Variables prior to enrollment on trial | |||||
| Hemoglobin | 131 | 98 | 105 | 140–180 for men, 95–133 for women | g/L |
| Absolute neutrophil count | 2.4 | 1.76 | 0.82 | 1.7–7.3 | ×109/L |
| Platelet count | 175 | 97 | 110 | 140–440 | ×109/L |
| ABL kinase domain mutation analysis | No mutations identified | No mutations identified | Testing canceled due to MRD of 0.448 IS | ||
| Bone marrow morphology | Trilineage hematopoiesis, no evidence of CML | Cellular marrow (50%) with trilineage hematopoiesis, no evidence of CML | Cellular (30%) marrow with dyspoietic trilineage hematopoiesis; 1% blasts | ||
| Conventional cytogenetics (% of normal metaphases) | 100 | 100 | 90% (2 metaphases with trisomy 8) | ||
| Treatment related variables | |||||
| Duration on frontline TKI | 36.5 | 6.4 | 30.4 | Months | |
| Duration on second line TKI | 52.7 | 23 | 31.5 | Months | |
| Duration on single agent TKI | 89.2 | 29.4 | 61.9 | Months | |
| Duration on TKI and AZA | 37.1 | 27.0 | 25.0 | Months | |
| No. of cycles of AZA | 38 | 27 | 19 | ||
| Duration of cycles | 28 (26–43) | 28 (27–41) | 35 (27–63) | Days (range) | |
| BCR-ABL transcript level at enrollment | 1.7255 | 1.2495 | 0.1085 | International Scale | |
| Outcome related variables | |||||
| BCR-ABL transcript levels at last follow up | 0.0032 | 0.0032 | 0.0032 | International Scale | |
| Time to achieve sustained MMR | 13.5 | 10.1 | 0.9 | Months | |
| Duration of MMR | 23.6 | 17.0 | 24.0 | Months | |
| Time to achieve MR4.5 | 19.8 | 27.0 | 4.4 | Months | |
| Total duration of MR4.5 | 15.2 | NA | 13.6 | Months | |
| Variables while on AZA-TKI combinationb | |||||
| Hemoglobin | 125 (116–132) | 104 (91–109) | 94 (86–102) | 140–180 for men, 95–133 for women | g/L |
| Absolute neutrophil count | 2.13 (1.57–3.74) | 1.7 (1.31–2.7) | 0.64 (0.34–1.31) | 1.7–7.3 | ×109/L |
| Platelet count | 206 (123–289) | 147 (86–172) | 140 (107–176) | 140–440 | ×109/L |
| Bone marrow morphology at six months | Cellular marrow with adequate trilineage maturation | Cellular marrow (30–40%) with mild erythroid hyperplasia. No morphologic evidence of CML | Hypocellular marrow (15–20%) with mild dysgranulopoiesis and mild dyserythropoiesisc | ||
| Conventional cytogenetics at 6 months (% of normal metaphases) | 100 | 100 | 100c | ||
a
By blasts and clonal evolution criteria.
b
Where applicable, values measured at the end of the cycle; expressed as median and range.
c
At nine months.
NA: not applicable.