Table 2.
Primary, Secondary, and Key Subgroup Efficacy Outcomes(intention-to-treat population; N = 65)
| Outcome | No. of Patients | % |
|---|---|---|
| Overall complete response | 13 | 20 |
| 95% CI | 11.1 to 31.8 | |
| CR | 7 | 11 |
| 95% CI | 4.4 to 20.9 | |
| CRi | 6 | 9 |
| 95% CI | 3.5 to 19.0 | |
| PR | 6 | 9 |
| 95% CI | 3.5 to 19.0 | |
| BMB | 4 | 6 |
| 95% CI | 1.7 to 15.0 | |
| Overall response (CR, CRi, PR, and BMB) | 23 | 35 |
| 95% CI | 23.8 to 48.3 | |
| CR and CRi, based on baseline disease status | ||
| Relapsed ALL (n = 36) | 9 | 25 |
| Relapsed and refractory ALL (n = 29) | 4 | 14 |
| CR and CRi based on ALL lineage | ||
| B-cell lineage (n = 55) | 11 | 20 |
| T-cell lineage (n = 10) | 2 | 20 |
| CR and CRi based on VSLI line of therapy | ||
| 3rd line (n = 32) | 6 | 19 |
| 4th line (n = 24) | 5 | 21 |
| 5th or greater line (n = 9) | 2 | 22 |
| CR and CRi based on prior HCT | ||
| Prior HCT (n = 31) | 8 | 26 |
| No prior HCT (n = 34) | 5 | 15 |
| CR and CRi based on prior clofarabine treatment | ||
| Prior clofarabine in a multidrug regimen (n = 3) | 0 | 0 |
| Prior clofarabine as a single agent (n = 4) | 2 | 50 |
| No prior clofarabine treatment (n = 58) | 11 | 19 |
| CR and CRi based on baseline bone marrow cytogenetics | ||
| Unfavorable (n = 33) | 5 | 15 |
| Intermediate (n = 23) | 6 | 26 |
| CR and CRi based on baseline age group, years | ||
| 18 to 29 (n = 29) | 10 | 34 |
| 30 to 59 (n = 28) | 2 | 7 |
| ≥ 60 (n = 8) | 1 | 13 |
| CR and CRi based on dose reduction | ||
| Required dose reduction (n = 15) | 7 | 47 |
| No required dose reduction (n = 50) | 6 | 12 |
| Time to CR and CRi, days | ||
| Median | 54 | |
| Range | 25 to 81 | |
| CR and CRi duration (uncensored), weeks | ||
| Median | 23 | |
| Range | 5 to 66 | |
| Overall survival, months | ||
| Median | 4.6 | |
| Range | < 1 to > 25 | |
| Post-VSLI HCT | 12 | 18.5 |
Abbreviations: ALL, acute lymphoblastic leukemia; BMB, bone marrow blast response; CR, complete response; CRi, CR with incomplete hematologic recovery; HCT, hematopoietic cell transplantation; PR, partial remission; VSLI, vincristine sulfate liposome injection.