NMTRC003/003B (DFMO maintenance)
NMTRC003 was the single-arm study, compared against historical patients, that got eflornithine approved as maintenance; the design remains debated.
EFS HR 0.48 and OS HR 0.32 versus a propensity-matched external control from ANBL0032. FDA approved December 2023 acknowledging the externally controlled design in a rare paediatric setting. A randomised trial has not been done.
- The treated group had about 52 percent lower chance of the event at any given time (hazard ratio 0.48).
- The absolute difference, how many more people out of 100 were helped, is not reported here.
- This is a surrogate endpoint: it measures the cancer being controlled or absent on scans and tests, which often, but not always, translates into living longer.
- These results apply to the people the trial enrolled: High-risk neuroblastoma in remission after standard therapy including anti-GD2: 2 years of oral eflornithine (single arm, externally controlled). People in a different situation may not see the same effect.
- The trial selected people by a biomarker (GD2); the result should not be assumed for people whose cancer does not have it.
Numbers are from the trial as recorded here; see the source links in the table below. This is orientation, not medical advice: ask your team how closely the trial population matches you.
105 participants enrolled.
| Endpoint | Arm | n | Value | HR (95% CI) | p | Source |
|---|---|---|---|---|---|---|
| Event-free survivalprimary | Eflornithine | 90 | — | 0.48 (0.27–0.85) | — | link |
| External control (ANBL0032) | 270 | — |
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