Patient response details
| Patient no. . | Clinical status before sorafenib . | Dose, mg . | Treatment duration, days . | WBC, × 109/L (blasts %) . | Best response . | Side effects (grade*) . | Emergence of sorafenib resistance . | Outcome at last visit on sorafenib (grade*) . | |
|---|---|---|---|---|---|---|---|---|---|
| Before sorafenib . | After 7 days of sorafenib . | ||||||||
| Sorafenib after allo-SCT | |||||||||
| 1 | Relapse day 87 after allo-SCT | 200-800 | 211 | 39.6 (78) | 1.5 (14) | HR, BMR | Neutropenia (IV), thrombopenia (IV), pneumonia hemolysis | Yes | Exitus letalis on day 216 in relapse |
| 2 | Relapse day 168 after allo-SCT | 200-800 | 221 | 73.5 (84) | 2.45 (0) | HR, BMR | Neutropenia (IV), thrombopenia (IV) | No | Exitus letalis on day 221 with cerebral mass, neutropenia (IV), thrombopenia (IV) |
| 3 | Relapse day 322 after allo-SCT | 800 | 81 | 18.6 (80, in BM) | 5.94 (NA) | CMR, CR | Maculopapulous exanthema of the skin (GVHD) | No | CR and ongoing CMR with sorafenib |
| Sorafenib before allo-SCT | |||||||||
| 4 | Relapse day 30 after first consolidation cycle | 400-800 | 50 | 44 (91) | 1.54 (6) | HR, BMR | Neutropenia (IV), thrombopenia (III), sepsis | No | Exitus letalis on day 58 in sepsis, neutropenia (IV), thrombopenia (IV) |
| 5 | Primary refractory | 800 | 13† | 36.2 (94) | 0.6 (40) | HR, BMR,‡ CMR | No | No | Relapse on day 111 after allo-SCT, sorafenib monotherapy 400 mg twice a day → ongoing CMR |
| 6 | Primary refractory | 800 | 82 | 8 (26) | 8(1) | HR | Hand-foot syndrome, hyperkeratosis | Yes | Relapse on day 71 RIC-allo-SCT → CR |
| Patient no. . | Clinical status before sorafenib . | Dose, mg . | Treatment duration, days . | WBC, × 109/L (blasts %) . | Best response . | Side effects (grade*) . | Emergence of sorafenib resistance . | Outcome at last visit on sorafenib (grade*) . | |
|---|---|---|---|---|---|---|---|---|---|
| Before sorafenib . | After 7 days of sorafenib . | ||||||||
| Sorafenib after allo-SCT | |||||||||
| 1 | Relapse day 87 after allo-SCT | 200-800 | 211 | 39.6 (78) | 1.5 (14) | HR, BMR | Neutropenia (IV), thrombopenia (IV), pneumonia hemolysis | Yes | Exitus letalis on day 216 in relapse |
| 2 | Relapse day 168 after allo-SCT | 200-800 | 221 | 73.5 (84) | 2.45 (0) | HR, BMR | Neutropenia (IV), thrombopenia (IV) | No | Exitus letalis on day 221 with cerebral mass, neutropenia (IV), thrombopenia (IV) |
| 3 | Relapse day 322 after allo-SCT | 800 | 81 | 18.6 (80, in BM) | 5.94 (NA) | CMR, CR | Maculopapulous exanthema of the skin (GVHD) | No | CR and ongoing CMR with sorafenib |
| Sorafenib before allo-SCT | |||||||||
| 4 | Relapse day 30 after first consolidation cycle | 400-800 | 50 | 44 (91) | 1.54 (6) | HR, BMR | Neutropenia (IV), thrombopenia (III), sepsis | No | Exitus letalis on day 58 in sepsis, neutropenia (IV), thrombopenia (IV) |
| 5 | Primary refractory | 800 | 13† | 36.2 (94) | 0.6 (40) | HR, BMR,‡ CMR | No | No | Relapse on day 111 after allo-SCT, sorafenib monotherapy 400 mg twice a day → ongoing CMR |
| 6 | Primary refractory | 800 | 82 | 8 (26) | 8(1) | HR | Hand-foot syndrome, hyperkeratosis | Yes | Relapse on day 71 RIC-allo-SCT → CR |
WBC indicates peripheral white blood cell count; allo-SCT, allogeneic stem cell transplantation; HR, hematologic remission; BMR, bone marrow remission; CMR, complete molecular remission; CR, complete remission; and RIC, reduced-intensity conditioning therapy.
According to the National Cancer Institute common toxicity criteria, version 2 (http://www.fda.gov/cder/cancer/toxicityframe.htm).
This patient was treated for only 14 days before SCT, when a donor became available and conditioning could be started. A hematologic relapse occurred 111 days after SCT, and the patient responded again to sorafenib (400 mg twice a day), achieving a CMR.
BMR is not according to Cheson et al.14 Bone marrow was hypocellular (Figure 1B); but after only 14 days, nucleated cells consisted of still more than 50% blasts.