Table 1:

Patient and Disease Characteristics (n = 31)

Treating institution
JHH	18 (58.1)
St. Jude	13 (41.9)
Sex
Male	18 (58.1)
Female	13 (41.9)
Age at diagnosis	6.5 [0.3–21.0]
Age at time of CAR T-cell infusion	7.9 [0.8–23.6]
Indication for tisagenlecleucel
Primary refractory disease	11 (35.5)
Relapse 1	14 (45.2)
Relapse 2	5 (16.1)
Relapse 3+	1 (3.2)
High-risk genetics
BCR-ABL1	1 (3.2)
Ph-like	6 (19.4)
KMT2A rearranged	5 (19.4)
p53 alteration (without associated hypodiploidy)	2 (6.5)
TCF3-PBX1	1 (3.2)
Myc translocation	1 (3.2)
Low hypodiploidy	3 (9.7)
Monosomy 7	1 (3.2)
Prior allogeneic HCT	4 (12.9)
Prior CD19- or CD22-directed therapy
Any agent	8 (25.8)
Blinatumomab	6 (19.3)
Inotuzumab	5 (16.1)
CD19-CAR T-cell therapy	1 (3.2)
Pre-infusion disease evaluation *
Marrow burden (median disease % [range])
MRD-negative (n = 3)	—
MRD-positive >0 to <5% (n = 15)	0.6 [0.003–2.7]
≥5% (n = 13**)	52 [7.6–100]
CNS3	1 (3.2)
Non-CNS extramedullary disease^	3 (9.7)

JHH: Johns Hopkins Hospital; St. Jude: St. Jude Children’s Research Hospital; HCT: hematopoietic cell transplantation; MRD: minimal residual disease (by flow cytometry). Numerical data are presented as the n (%) or median [range]

^*After bridging chemotherapy and before CAR T-cell therapy

^**One patient without available disease % by flow cytometry was classified using morphologic blasts (52%), and one patient had disease confirmed by peripheral blood only and was empirically categorized as having 26% disease for this analysis

^{^}Increased metabolic uptake on PET-CT [evaluated in a subset of patients (n = 7)]