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Figure 1. Study Design
AML, acute myeloid leukemia; CR, complete response; CRi, complete response with incomplete blood count recovery; MLFS, morphologic
leukemia-free state.
Eligibility Criteria
Table 1. Patient Disposition
| N=41 | ||
| Number of Patients Active | 25 | |
| Number of Patients Discontinued | 16 | |
| Reasons for Discontinuation | ||
| Progressive disease | 6 | |
| Adverse event | 6 | |
| Other | 4 |
Other includes patient or physician decision.
Table 2. Baseline Characteristics
| N=41 | ||
| Age (years) | ||
| Median | 76 | |
| Range | 69-84 | |
| Gender, n (%) | ||
| Male | 24 (59) | |
| Female | 17 (41) | |
| AML Disease Status, n (%) | ||
| Newly diagnosed de novo | 29 (71) | |
| Secondary (AHD and treatment related) | 12 (29) | |
| ECOG Status, n (%) | ||
| 0-1 | 33 (80) | |
| 2 | 8 (20) | |
| Bone Marrow Blasts at Baseline | ||
| Median | 38 | |
| 20-29% Range, n (%) | 13 (32) | |
| 30-50% Range, n (%) | 15 (36) | |
| >50% Range, n (%) | 13 (32) | |
| Cytogenetic Risk Category, n (%) | ||
| Intermediate | 23 (56) | |
| High | 17 (41) | |
| Not Evaluable | 1 (3) |
AHD, antecedent hematologic disorder; AML, acute myeloid leukemia; ECOG, Eastern Cooperative Oncology Group.
Table 3. Treatment Emergent Adverse Events All Causality in 10% of Patients
| All Grades (%) N=41 | Grades 3-4 (%) N=41 | |||
| Hematologic | ||||
| Febrile Neutropenia | 12 (29) | 10 (24) | ||
| Thrombocytopenia | 11 (27) | 10 (24) | ||
| Anemia | 9 (22) | 4 (10) | ||
| Neutropenia | 4 (10) | 4 (10) | ||
| Leukopenia | 4 (10) | 1 (2) | ||
| Non-Hematologic | ||||
| Nausea | 18 (44) | 2 (5) | ||
| Constipation | 17 (41) | 0 | ||
| Fatigue | 17 (41) | 4 (10) | ||
| Peripheral Edema | 6 (15) | 0 | ||
| Vomiting | 6 (15) | 0 | ||
| Diarrhea | 5 (12) | 1 (2) | ||
| Dizziness | 5 (12) | 0 | ||
| Headache | 5 (12) | 1 (2) | ||
| Hypokalemia | 5 (12) | 0 | ||
| Pyrexia | 4 (10) | 0 | ||
| Cellulitis | 4 (10) | 4 (10) | ||
| Rash | 4 (10) | 0 | ||
| Hypotension | 4 (10) | 0 | ||
| Cough | 4 (10) | 0 | ||
| Dyspnea | 4 (10) | 0 | ||
| QTc Prolongation* | 2 (5) | 1 (2) |
*QTc events were seen in <10% of patients, however are noted here.
Table 4. Treatment-Emergent Adverse Events Leading to Drug Discontinuation
| Discontinuation | ||||||
| AE Term | Grade | (Cycle/Day) | Outcome | |||
| Peripheral Motor Neuropathy | 3 | 3/1 | Resolved | |||
| Parainfluenza | 3 | 3/22 | Resolved | |||
| Prolonged QTc/AF | 3 | 2/15 | Resolved | |||
| Subdural Hematoma | 5 | 3/22 | Fatal | |||
| Sepsis | 5 | 2/3 | Fatal | |||
| Sepsis | 5 | 2/14 | Fatal |
AE, adverse event; AF, atrial fibrillation.
| Interim Response Assessment | ||
| n=33* (%) | ||
| CR/CRi/MLFS (Primary endpoint) | 15 (45) | |
| CR | 9 (27) | |
| CRi | 4 (12) | |
| MLFS | 2 (6) | |
| PR/PRi | 3 (10) | |
| Stable Disease | 4 (12) | |
| Progressive Disease | 6 (18) | |
| Clinical Benefit** | 1 (3) | |
| No Clinical Benefit | 4 (12) |
CR, complete response; CRi, complete response with incomplete blood count recovery; MLFS, morphologic leukemia-free state;
PR, partial response; PRi, partial response with incomplete blood count recovery.
patients who have had at least 1 on-study disease assessment OR discontinued study therapy prior to an on-study disease assessment due to adverse event or other reasons.
**Patients did not meet strict International Working Group (IWG) response criteria, but were determined to have clinical benefit by Investigator.
Figure 2. Interim Efficacy and Duration on Study
AE, adverse event; AML, acute myeloid leukemia; CR, complete response; CRi, complete response with incomplete blood count recovery;
MLFS, morphologic leukemia-free state; PR, partial response; PRi, partial response with incomplete blood count recovery.
Figure 3. Event-Free Survival
Figure 4. Overall Survival
| DISCLOSURES | ||
| G. Garcia-Manero receives consultancy fees from MEI Pharma; E. Atallah reports no relevant conflicts of interest to disclose; O. Odenike receives honoraria and advisory fees from Sunesis Pharmaceuticals, Incyte, Sanofi-Aventis, Algeta Pharmaceuticals, and Spectrum Pharmaceuticals; B.C. Medeiros receives research funding from MEI Pharma; J. Cortes receives research funding from Celgene; V. Esquibel is an employee of MEI Pharma; S. Cha was employed by MEI Pharma at the time of abstract submission; S.K. Khaled receives research funding from Sequenom. |