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ELIANA was the global trial behind the first CAR-T approval: a single infusion of tisagenlecleucel put four in five children and young adults with relapsed B-cell acute lymphoblastic leukaemia and no standard treatment remaining into remission, and most responders were still in remission without further therapy at the five-year update. Severe cytokine release syndrome affected almost half.
ELIANA, trial NCT02435849 sponsored by Novartis and published in the New England Journal of Medicine in 2018, was the global trial behind the first CAR-T approval: four in five children and young adults with relapsed or refractory B-cell acute lymphoblastic leukaemia and no standard treatment remaining went into remission after a single tisagenlecleucel infusion, and most responders stayed there for years. 75 patients were infused for the primary analysis (79 by the three-year update), the remission rate within three months was 81 percent with all responders MRD-negative, and the five-year update in JCO in 2023 showed durable survival in most responders without further therapy, at the cost of severe cytokine release syndrome in almost half. Whether children who relapse can be rescued with a second CAR-T is the open question.
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.
79 treated.
| Endpoint | Arm | n | Value | HR (95% CI) | p | Source |
|---|---|---|---|---|---|---|
| Overall remission rate within 3 monthsprimary | Tisagenlecleucel | 75 | 81% | - | - | link |
| Overall survival at 12 months | Tisagenlecleucel | - | 76% | - | - | link |
| Overall survival at 5 years | Tisagenlecleucel | - | 55% | - | - | link |
Shares Stephan A. Grupp, ELIANA: the global trial that made tisagenlecleucel the first approved CAR-T therapy for children and young adults with relapsed ALL, Tisagenlecleucel, Cytokine release syndrome (CRS).
Shares Emily Whitehead, Tisagenlecleucel, CD19, CAR-T cell therapy.
Shares Emily Whitehead, ELIANA: the global trial that made tisagenlecleucel the first approved CAR-T therapy for children and young adults with relapsed ALL, Tisagenlecleucel, CD19.
Shares ELIANA: the global trial that made tisagenlecleucel the first approved CAR-T therapy for children and young adults with relapsed ALL, Relapsed and refractory acute lymphoblastic leukaemia in children, Cytokine release syndrome (CRS), CD19.
Shares Tisagenlecleucel, Novartis, CAR-T cell therapy, Acute lymphoblastic leukaemia.
Shares Emily Whitehead, ELIANA: the global trial that made tisagenlecleucel the first approved CAR-T therapy for children and young adults with relapsed ALL, CAR-T cell therapy.
Shares ELIANA: the global trial that made tisagenlecleucel the first approved CAR-T therapy for children and young adults with relapsed ALL, Cytokine release syndrome (CRS), Manufacturing cost and time for living and radioactive medicines, CD19.
Shares Manufacturing cost and time for living and radioactive medicines, CD19, CAR-T cell therapy, Acute lymphoblastic leukaemia.