Table 1.

Disposition and dosing of infused patients

Disposition	Patients
Received tisagenlecleucel infusion, n*	137
In ELIANA, n	79
In ENSIGN, n	58
Median time from enrollment to infusion, days (range)	43 (24–133)
Median follow-up duration from infusion, months (range)†	24 (0.1–36.5)
Median dose, CAR-positive viable T cells (range)	1.0×108 (0.03×108–2.6×108)
In patients >50 kg (n=46), cells (range)	1.7×108 (0.1×108–2.5×108)
In patients ≤50 kg (n=91), cells/kg body weight (range)	3.3×106 (0.2×106–5.4×106)
Death following tisagenlecleucel infusion, n/N (%)	44/137 (32)
≤30 days postinfusion	4 (3)
Leukemia progression	2 (1)
AE‡	2 (1)
>30 days postinfusion	40 (29)
Leukemia progression	33 (24)
Other, prior to any further anticancer therapy§	2 (1)
Other, following treatment with additional anticancer therapy¶	5 (4)

*Screening for ELIANA began April 8, 2015 at 25 sites in 11 countries across four continents.4 Screening for ENSIGN began August 14, 2014 at 13 sites in the USA.28

†Data cut-off dates were April 13, 2018 for ELIANA and October 6, 2017 for ENSIGN.

‡Due to cerebral hemorrhage possibly related to tisagenlecleucel treatment in the setting of coagulopathy on day 15, and embolic infectious stroke (mucormycosis) on day 25.

§Both were due to infections possibly related to tisagenlecleucel treatment: systemic candidiasis associated with prolonged pancytopenia on day 62, and HHV-6-positive encephalitis associated with a history of prolonged neutropenia and lymphopenia on day 53.

¶Due to pneumonia on day 506, veno-occlusive disease following HSCT on day 359, other complications from HSCT on day 461, acute respiratory failure on day 125, and unknown reason on day 464.

AE, adverse event; CAR, chimeric antigen receptor; HHV-6, human herpesvirus 6; HSCT, hematopoietic stem cell transplant.