Patient characteristics and procedures
| Patients . | S1 . | S2 . | P1 . | P2 . | P4 . | P5 . | P6 . | P8 . | TT2 . | TT3 . | TT5 . | TT6 . | TT7 . | TT8 . | TT9 . |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Disease | Infant ALL | Infant ALL | Pre-B ALL | Pre-B ALL | Pre-B ALL | Infant ALL | Pre-B ALL | Infant ALL | Infant ALL | Infant ALL | Infant ALL | Infant ALL | Infant ALL | Pre-B ALL | Infant ALL |
| Genetics | MLL | MLL | ETV6-RUNX1 | MLL | Tril t(2;17) | MLL | 47 XX, gain c18/X | MLL | MLL | MLL | MLL | MLL | MLL | ETV6-RUNX1 | MLL |
| Previous SCT | Y | Y | N | N | Y | N | Y | N | Y | Y | Y | Y | Y | N | Y |
| Previous CAR19 | N | N | N | N | N | N | N | N | N | Y | N | Y | N | Y | N |
| Previous BiteY | Y | Y | N | N | N | N | N | N | N | N | Y | N | N | Y | Y |
| Disease burden ∗PCR | 12% | 10% | 80% | 7%∗ | 3.4% | 11% | 1.3% | 1%∗ | 3.2% | 21% | 66% | 0.6% | 3.2% | 58% | 0.9% |
| Lymphodepletion | F90 C120 A1 | F150 C120 A1 | F150 C120 A1 | F150 C120 A1 | F150 C120 A1 | F120 C120 A1 | F150 C120 A1 | F150 C120 — | F150 C120 A1 | F150 C120 A1 | F150 C120 A1 | F150 C120 A1 | F150 C120 A1 | F150 C120 A1 | F150 C120 A1 |
| CRS | N | N | G ≥3 | G2 | G2 | G1 | G1 | G ≥3 | G1 | G1 | G1 | G1 | G2 | G2 | G2 |
| ICANS | N | G ≥3 | N | N | N | N | N | G1 | N | N | N | N | G ≥ 3 | G1 | N |
| Viral comps. | N | N | N | CMV ADV | N | N | BK | N | CMV | ADV | N | ADV | N | N | N |
| D28 Flow | ND | ND | NEG | NEG | ND | ND | ND | POS | ND | POS | NEG | POS | ND | NEG | ND |
| D28 PCR | NEG | NEG | POS | POS | NEG | NEG | NEG | POS | NEG | POS | NEG | POS | NEG | POS | NEG |
| Allo-SCT | Second MMUD RIC | Second MUD RIC | MUD MAC | MUD MAC | Alt MSD MAC | MUD RIC | Second MMUD RIC | N | Second MSD RIC | N | Second MUD RIC | N | Second MUD RIC | MUD MAC | N |
| Chemotherapy | F,C, Thio | F,C | C | C | C | F,T, Thio | F | N/A | F,C | N/A | F,C | N/A | F,C | E | N/A |
| ATG | Y | Y | Y | Y | N | Y | Y | N/A | N | N/A | Y | N/A | Y | N | N/A |
| Radiotherapy (Gy) | 4 | 2 | 14.4 | 14.4 | 14.4 | 0 | 2 | N/A | 2 | N/A | 2 | N/A | 2 | 12 | N/A |
| FU and outcome | AW | AW | DRM | DRM | TRM | AW | AW | DRM | AW | DRM | AW | DRM | DRM | DRM | DRM |
| Patients . | S1 . | S2 . | P1 . | P2 . | P4 . | P5 . | P6 . | P8 . | TT2 . | TT3 . | TT5 . | TT6 . | TT7 . | TT8 . | TT9 . |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Disease | Infant ALL | Infant ALL | Pre-B ALL | Pre-B ALL | Pre-B ALL | Infant ALL | Pre-B ALL | Infant ALL | Infant ALL | Infant ALL | Infant ALL | Infant ALL | Infant ALL | Pre-B ALL | Infant ALL |
| Genetics | MLL | MLL | ETV6-RUNX1 | MLL | Tril t(2;17) | MLL | 47 XX, gain c18/X | MLL | MLL | MLL | MLL | MLL | MLL | ETV6-RUNX1 | MLL |
| Previous SCT | Y | Y | N | N | Y | N | Y | N | Y | Y | Y | Y | Y | N | Y |
| Previous CAR19 | N | N | N | N | N | N | N | N | N | Y | N | Y | N | Y | N |
| Previous BiteY | Y | Y | N | N | N | N | N | N | N | N | Y | N | N | Y | Y |
| Disease burden ∗PCR | 12% | 10% | 80% | 7%∗ | 3.4% | 11% | 1.3% | 1%∗ | 3.2% | 21% | 66% | 0.6% | 3.2% | 58% | 0.9% |
| Lymphodepletion | F90 C120 A1 | F150 C120 A1 | F150 C120 A1 | F150 C120 A1 | F150 C120 A1 | F120 C120 A1 | F150 C120 A1 | F150 C120 — | F150 C120 A1 | F150 C120 A1 | F150 C120 A1 | F150 C120 A1 | F150 C120 A1 | F150 C120 A1 | F150 C120 A1 |
| CRS | N | N | G ≥3 | G2 | G2 | G1 | G1 | G ≥3 | G1 | G1 | G1 | G1 | G2 | G2 | G2 |
| ICANS | N | G ≥3 | N | N | N | N | N | G1 | N | N | N | N | G ≥ 3 | G1 | N |
| Viral comps. | N | N | N | CMV ADV | N | N | BK | N | CMV | ADV | N | ADV | N | N | N |
| D28 Flow | ND | ND | NEG | NEG | ND | ND | ND | POS | ND | POS | NEG | POS | ND | NEG | ND |
| D28 PCR | NEG | NEG | POS | POS | NEG | NEG | NEG | POS | NEG | POS | NEG | POS | NEG | POS | NEG |
| Allo-SCT | Second MMUD RIC | Second MUD RIC | MUD MAC | MUD MAC | Alt MSD MAC | MUD RIC | Second MMUD RIC | N | Second MSD RIC | N | Second MUD RIC | N | Second MUD RIC | MUD MAC | N |
| Chemotherapy | F,C, Thio | F,C | C | C | C | F,T, Thio | F | N/A | F,C | N/A | F,C | N/A | F,C | E | N/A |
| ATG | Y | Y | Y | Y | N | Y | Y | N/A | N | N/A | Y | N/A | Y | N | N/A |
| Radiotherapy (Gy) | 4 | 2 | 14.4 | 14.4 | 14.4 | 0 | 2 | N/A | 2 | N/A | 2 | N/A | 2 | 12 | N/A |
| FU and outcome | AW | AW | DRM | DRM | TRM | AW | AW | DRM | AW | DRM | AW | DRM | DRM | DRM | DRM |
A, alemtuzumab; ADV, adenovirus; Alt, alternative; ATG, antithymocyte globulin; AW, alive and well; BK, BK virus; C, cyclophosphamide; CMV, cytomegalovirus; CRS, cytokine release syndrome; DRM, disease-related mortality; E, etoposide; F, fludarabine; FU, follow up; G, grade; ICANS, immune effector cell–associated neurotoxicity syndrome; MAC, myeloablative conditioning; MMUD, mismatched unrelated donor; MSD, matched sibling donor; MUD, matched unrelated donor; N, no; N/A, not applicable; Neg, negative; ND, not determined; Pos, positive; T, treosulfan; TRM, treatment-related mortality; Thio, thiotepa; Viral comps., viral reactivation in the first 28 days after allo-CAR19; Y, yes.
Disease burden was quantified by flow cytometry unless by PCR where indicated.