ENESTop
A Phase II, Single Arm, Open Label Study of Treatment-free Remission in Chronic Myeloid Leukemia (CML) Chronic Phase (CP) Patients After Achieving Sustained MR4.5 on Nilotinib
Phase 2NCT01698905
- Population
- Adults with chronic-phase CML, at least 3 years of TKI therapy, sustained deep molecular response achieved after switching from imatinib to nilotinib
- Design
- Single-arm phase 2 trial. Patients in chronic-phase CML who had received at least 3 years of TKI therapy and reached sustained deep molecular response only after switching from imatinib to nilotinib completed a 1-year nilotinib consolidation phase; 126 then attempted TFR. Nilotinib was restarted on loss of major molecular response (MMR) or confirmed loss of MR4.
- Arms
- Entered TFR phase after nilotinib consolidation (experimental, n = 126): Second-line nilotinib, 1-year consolidation, then discontinuation; restart on loss of MMR or confirmed loss of MR4
- People
- Hughes TP, senior author Mahon FX
Show the numbers
| Endpoint | Analysis | Entered TFR phase after nilotinib consolidation | Control | Effect |
|---|---|---|---|---|
| TFR126 patients who attempted TFRProportion in treatment-free remission at 48 weeks after stopping nilotinib (the abstract labels the 48-week analysis as the primary analysis). Patients leave TFR on loss of MMR or confirmed loss of MR4. | primaryHughes 2021 | 57.9% | — | 73 of 126 patients. Reported in the 5-year update paper; the original 48-week publication was not fetched. |
| TFR126 patients who attempted TFRProportion in treatment-free remission at 5 years after stopping nilotinib. Patients leave TFR on loss of MMR or confirmed loss of MR4. | long term-updateHughes 2021 | 42.9% at 5 y | — | 54 of 126 patients. Since the 48-week analysis, 58% (11 of 19) of patients who left the TFR phase had not lost molecular response and stopped for other reasons. 59 patients restarted nilotinib after loss of MMR or confirmed loss of MR4: 98.3% regained MMR, 94.9% MR4 and 93.2% MR4.5. No disease progression or CML-related deaths. Cardiovascular events accumulated with longer nilotinib exposure in patients who restarted. |
| Endpoint | Analysis | Entered TFR phase after nilotinib consolidation | Control | Effect |
|---|---|---|---|---|
| TFR126 patients who attempted TFRProportion in treatment-free remission at 48 weeks after stopping nilotinib (the abstract labels the 48-week analysis as the primary analysis). Patients leave TFR on loss of MMR or confirmed loss of MR4. | primaryHughes 2021 | 57.9% | — | 73 of 126 patients. Reported in the 5-year update paper; the original 48-week publication was not fetched. |
| TFR126 patients who attempted TFRProportion in treatment-free remission at 5 years after stopping nilotinib. Patients leave TFR on loss of MMR or confirmed loss of MR4. | long term-updateHughes 2021 | 42.9% at 5 y | — | 54 of 126 patients. Since the 48-week analysis, 58% (11 of 19) of patients who left the TFR phase had not lost molecular response and stopped for other reasons. 59 patients restarted nilotinib after loss of MMR or confirmed loss of MR4: 98.3% regained MMR, 94.9% MR4 and 93.2% MR4.5. No disease progression or CML-related deaths. Cardiovascular events accumulated with longer nilotinib exposure in patients who restarted. |