Characteristics of patients with EMD
| UPN . | Preinfusion disease evaluation . | Postinfusion response . | Notable genetics . | Clinical outcome† (mo to event/last contact) . | |||
|---|---|---|---|---|---|---|---|
| Focal EMD . | Bone marrow∗ . | CSF . | Focal EMD . | Bone marrow∗ . | |||
| 1‡ | Soft tissue | 0.06% | CNS1 | Progressive | CR and MRD neg | IKZF1 truncation and hyperdiploid | EMD progression (1) |
| 2‡ | Orbit and focal bone | NGS pos | CNS1 | Mixed (1 mo), resolved by 3 mo | CR and MRD neg | KMT2A::RELA | EMD relapse at a new site (5) |
| 3 | Pleura | 32% | CNS1 | NE | CR and NGS pos | NF1 and RASGRP4 alterations | Consolidative HCT (1) |
| 4 | Testicular and lymph nodes | 33% | CNS1 | Resolved | CR and MRD neg | KMT2A::MLLT3 | Ongoing BCA and remission (15) |
| 5 | Focal bone | 34% | CNS1 | Resolved | CR and MRD neg | None detected | Consolidative HCT (3) |
| 6 | Pancreas, kidney, and bowel | 45% | CNS1 | NE | Progression | Hypodiploid | Marrow nonresponse (1) |
| 7 | Focal bone | NGS pos | CNS1 | Stable | CR and MRD neg | None detected | Consolidative HCT (2) |
| 8‡ | Focal bone | NGS pos | CNS1 | Resolved | CR and MRD neg | KMT2A::MLLT10 | Repeat CD19 CAR infusion for the loss of BCA (4) |
| 9 | Focal bone | Aplastic, PB 2% | CNS1 | Resolved | CR and MRD neg | None detected | Ongoing BCA and remission (4) |
| 10 | Testicular | None detected | CNS1 | Progressive | CR and MRD neg§ | Hyperdiploid and complex karyotype | EMD progression (1) |
| 11 | Spleen, bowel, pancreas, and soft tissue | 98% | CNS2 | Stable | Progression | KMT2A::AFF1 | Marrow progression (1) |
| 12 | Lung | 95% | CNS3 | NE | Progression | TSC1 truncation, IL7R, and SH2B3 (Ph-like) | Marrow progression (1) |
| 13 | Focal bone | 6.8% | CNS1 | Improved (1 mo), resolved by 2 mo | CR and MRD neg | None detected | Consolidative HCT (3) |
| 14‡ | Focal bone and focal CNS | 0.08% | CNS1 | Bone: NE; CNS: resolved | CR and MRD neg | CREBBP truncation and hyperdiploid | Ongoing BCA and remission (2) |
| 15 | Focal CNS | 5% | CNS1 | Improved (1 mo) and resolved by 2 mo | CR and MRD pos | ETV6::ETV5 and hyperdiploid | Consolidative HCT (2) |
| UPN . | Preinfusion disease evaluation . | Postinfusion response . | Notable genetics . | Clinical outcome† (mo to event/last contact) . | |||
|---|---|---|---|---|---|---|---|
| Focal EMD . | Bone marrow∗ . | CSF . | Focal EMD . | Bone marrow∗ . | |||
| 1‡ | Soft tissue | 0.06% | CNS1 | Progressive | CR and MRD neg | IKZF1 truncation and hyperdiploid | EMD progression (1) |
| 2‡ | Orbit and focal bone | NGS pos | CNS1 | Mixed (1 mo), resolved by 3 mo | CR and MRD neg | KMT2A::RELA | EMD relapse at a new site (5) |
| 3 | Pleura | 32% | CNS1 | NE | CR and NGS pos | NF1 and RASGRP4 alterations | Consolidative HCT (1) |
| 4 | Testicular and lymph nodes | 33% | CNS1 | Resolved | CR and MRD neg | KMT2A::MLLT3 | Ongoing BCA and remission (15) |
| 5 | Focal bone | 34% | CNS1 | Resolved | CR and MRD neg | None detected | Consolidative HCT (3) |
| 6 | Pancreas, kidney, and bowel | 45% | CNS1 | NE | Progression | Hypodiploid | Marrow nonresponse (1) |
| 7 | Focal bone | NGS pos | CNS1 | Stable | CR and MRD neg | None detected | Consolidative HCT (2) |
| 8‡ | Focal bone | NGS pos | CNS1 | Resolved | CR and MRD neg | KMT2A::MLLT10 | Repeat CD19 CAR infusion for the loss of BCA (4) |
| 9 | Focal bone | Aplastic, PB 2% | CNS1 | Resolved | CR and MRD neg | None detected | Ongoing BCA and remission (4) |
| 10 | Testicular | None detected | CNS1 | Progressive | CR and MRD neg§ | Hyperdiploid and complex karyotype | EMD progression (1) |
| 11 | Spleen, bowel, pancreas, and soft tissue | 98% | CNS2 | Stable | Progression | KMT2A::AFF1 | Marrow progression (1) |
| 12 | Lung | 95% | CNS3 | NE | Progression | TSC1 truncation, IL7R, and SH2B3 (Ph-like) | Marrow progression (1) |
| 13 | Focal bone | 6.8% | CNS1 | Improved (1 mo), resolved by 2 mo | CR and MRD neg | None detected | Consolidative HCT (3) |
| 14‡ | Focal bone and focal CNS | 0.08% | CNS1 | Bone: NE; CNS: resolved | CR and MRD neg | CREBBP truncation and hyperdiploid | Ongoing BCA and remission (2) |
| 15 | Focal CNS | 5% | CNS1 | Improved (1 mo) and resolved by 2 mo | CR and MRD pos | ETV6::ETV5 and hyperdiploid | Consolidative HCT (2) |
BCA, B cell aplasia; CR, complete response (<5% marrow blasts); CSF, cerebrospinal fluid; HCT, hematopoietic cell transplant; MRD, minimal residual disease by flow cytometry (0.01-5% blasts); NE, not evaluated; neg, negative result; NGS, disease detected only by next-generation sequencing (<0.01%); PB, peripheral blood; pos, positive result.
Disease burden detected by flow cytometry, unless otherwise indicated (morphology for UPN11).
First occurrence of relapse, additional systemic disease–directed therapy including consolidative HCT, or nonrelapse mortality.
Received focal RT before CAR T-cell infusion.
No detectable marrow disease before infusion.