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ELIANA

ELIANA was the global trial behind the first CAR-T approval: four in five children with no options left went into remission, and many stayed there for years.

75 infused (97 enrolled): ORR 81% at 3 months, all MRD-negative; 12-month EFS 50%, OS 76%; 5-year update: EFS 44% and OS 55% in the infused population with no further therapy in most responders. NEJM 2018; JCO 2023 update. Grade 3-4 CRS 46%.

Setting
Relapsed or refractory B-ALL, patients aged 3-25: single infusion of tisagenlecleucel
Phase
Phase 2
Sponsor
Novartis
Registry
Headline result
ORR 81%; 12-month OS 76%; 5-year OS 55%.
Reported
2018
Enrolled
97
Replication
ENSIGN (US) and real-world CIBMTR registry data (>500 children) reproduce ~85% CR and comparable durability.

Outcomes

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In plain words
What these results mean for people, not percentages
97 people took part
Overall remission rate within 3 monthsprimaryother endpoint
  • 81 out of 100 people reached this endpoint with Tisagenlecleucel.
  • This endpoint is not one of the standard survival or response measures; read it alongside the trial's primary result.
Overall survival at 12 monthssurvival endpoint
  • 76 out of 100 people alive at 12 months with Tisagenlecleucel.
  • Overall survival counts deaths from any cause, so it is the most direct measure of whether a treatment helps people live longer.
Overall survival at 5 yearssurvival endpoint
  • 55 out of 100 people alive at 5 years with Tisagenlecleucel.
  • Overall survival counts deaths from any cause, so it is the most direct measure of whether a treatment helps people live longer.
Be careful
  • There was no comparison group, so this number cannot tell you how much of the effect is due to the treatment.
  • These results apply to the people the trial enrolled: Relapsed or refractory B-ALL, patients aged 3-25: single infusion of tisagenlecleucel. People in a different situation may not see the same effect.
  • Only 97 people took part, so the numbers are less certain than in a large trial.

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.

97 participants enrolled.

Overall remission rate within 3 monthsprimary
Tisagenlecleucel81 of 100
n = 75
Source
Overall survival at 12 months
Tisagenlecleucel76 of 100
Overall survival at 5 years
Tisagenlecleucel55 of 100
Source
EndpointArmnValueHR (95% CI)pSource
Overall remission rate within 3 monthsprimaryTisagenlecleucel7581%link
Overall survival at 12 monthsTisagenlecleucel76%
Overall survival at 5 yearsTisagenlecleucel55%link
Replication
ENSIGN (US) and real-world CIBMTR registry data (>500 children) reproduce ~85% CR and comparable durability.

Key papers

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Connected

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