Table 2.

Toxicity and clinical response to treatment with the HD-CAR-1 product

UPN	Age	Gender	Disease	CART dose [CARTs/m2]	CRS grade	ICANS	Infection	Best response	Response at EOS	alloSCT after CARTs	Progression of disease/death [day after CARTs]
1	32	F	B-ALL	1 × 106	I	–	RSV	PD	PD	–	28 (CSF: 13 blasts /µl)/254
2	21	M	Pre-B-ALL	1 × 106	–	–	–	CR MRD +	CR MRD +	Yes	119 (BM: 8% blasts)/268
3†	67	F	B-ALL	1 × 106	–	–	–	CR MRD + (day 28)	n.r	–	56 (BM: 52% blasts)/76
4	32	F	B-ALL	5 × 106	–	–	–	CR MRD −	CR MRD −	–	–
5	63	M	Pre-B-ALL	5 × 106	–	–	Staph. epi	CR MRD −	CR MRD +	–	146 (extramedullary: malignant ascites)
6	28	F	B-ALL	5 × 106	I	–	–	CR MRD −	CR MRD-(metabolic CR)	–	120 (extramedullary: right mamma)
7	67	F	MPAL	20 × 106	–	–	–	CR MRD −	CR MRD +	–	–
8†	36	F	Pre-B-ALL	20 × 106	II	–	–	PD	n.r	Yes	23 (BM: 100% blast infiltration)/58
9	45	M	B-ALL	20 × 106	–	–	–	CR MRD −	CR MRD −	–	189 (CSF: 50–60% blasts)
10	47	F	B-ALL	20 × 106	–	–	–	CR MRD +	PD	Yes	93 (BM: 9% blasts)
11	37	M	B-ALL	50 × 106	–	–	Respiratory*	CR MRD −	CR MRD −
12†	36	M	B-ALL	50 × 106	II	–	E. coli sepsis	SD (day 26)	n.r	–	–/39 (TRM: septic MOV)
13	32	M	B-ALL	50 × 106	–	–	Respiratory*	CR MRD −	CR MRD-(metabolic CR)	–	–

ALL acute lymphoblastic leukemia, BM bone marrow, c-ALL common B cell leukemia, CART chimeric antigen receptor T cells, CR complete remission, CRS cytokine release syndrome, CSF cerebrospinal fluid, EOS end of study on day 90 after HD-CAR-1 treatment, F female, M male, MPAL mixed-phenotype acute leukemia, MOV multi-organ failure, MRD minimal residual disease, n.r. not reached, pre-ALL precursor B cell acute lymphoblastic leukemia, PD progressive disease, RSV respiratory syncytial virus, SD stable disease, TRM treatment-related mortality, UPN unique patient number

^†Patient died before reaching EOS

*No pathogen identified