Individual patient outcomes
| Dose level . | Age, y . | Sex . | Diagnosis . | Disease status at BMT . | MRD status . | Refined DRI . | Prior lines of therapy . | HCT-CI . | Evaluable for primary endpoint (event) . | aGVHD∗ . | Engraftment syndrome . | SOS . | TMA . | Persistent host T-cell chimerism . | cGVHD† . | NRM . | NRM (cause) . | Relapse (day) . | Alive (day of death) . | ||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Yes/No . | Max. grade . | Yes/No . | Max severity . | Systemic immune suppression . | |||||||||||||||||||
| DL1 PTCy 50 mg/kg per day on days +3/+4 | 23 | F | ALL | CR3 | Negative | High | 3 | 4 | Yes | No | - | No | No | No | No | No | - | - | No | - | Yes (187) | No (389) | |
| 25 | M | ALL | CR1 | Negative | Intermediate | 2 | 7 | Yes | No | - | No | No | No | No | No | - | - | No | - | No | Yes | ||
| 21 | M | ALL | CR1 | Negative | Intermediate | 2 | 4 | Yes | No | - | No | No | No | No | No | - | - | No | - | No | Yes | ||
| 27 | M | AML | CR1 | Positive | Intermediate | 3 | 6 | Yes | Yes | 4 | No | No | No | No | No | - | - | Yes | aGVHD, viral/bacterial pneumonia | No | No (165) | ||
| 38 | F | DLBCL | PD | N/A | Very high | 8 | 7 | Yes | No | - | No | Yes | No | No | Yes | Mild | No | No | - | No | Yes | ||
| DL2 PTCy 25 mg/kg per day on days +3/+4 | Phase 1 | 28 | F | ALL | CR3 | Negative | High | 3 | 3 | Yes | No | - | No | No | No | Yes | No | - | - | No | - | Yes (335) | No (417) |
| 38 | F | ALL | CR1 | Negative | Intermediate | 3 | 5 | Yes | Yes | 1 | No | Yes | No | No | Yes | Severe | Yes | No | - | No | Yes | ||
| 43 | M | AML | CR2 | Positive | High | 2 | 4 | Yes | No | - | No | No | No | No | Yes | Mild | No | No | - | Yes (312) | No (542) | ||
| 28 | F | ALL | CR1 | Negative | Intermediate | 2 | 2 | Yes | No | - | No | No | No | No | No | - | - | No | - | No | Yes | ||
| 26 | F | AML | CR1 | Negative | Intermediate | 1 | 6 | No (graft failure) | N/A‡ | - | N/A‡ | No | No | N/A | N/A‡ | - | - | No | - | No | Yes | ||
| 47 | F | ALL | CR1 | Positive | Intermediate | 1 | 1 | Yes | No | - | No | No | No | No | No | - | - | No | - | No | Yes | ||
| 27 | M | HL | PD | N/A | High | 3 | 2 | Yes | No | - | No | No | No | No | Yes | Moderate | Yes | Yes | Interstitial lung disease (chemotherapy) | No | No (237) | ||
| Phase 2 | 57 | M | DLBCL | PD | N/A | Very high | 8 | 7 | Yes | No | - | No | No | No | No | No | - | - | Yes | Bacterial pneumonia | No | No (112) | |
| 42 | M | ALL | CR5 | Negative | High | 6 | 3 | Yes | No | - | No | No | No | Yes | No | - | - | No | - | Yes (286) | No (339) | ||
| 30 | M | ALL | CR1 | Negative | Intermediate | 2 | 4 | Yes | No | - | No | No | No | No | Yes | Moderate | No | No | - | No | Yes | ||
| 31 | M | ALL | CR3 | Negative | High | 3 | 2 | Yes | No | - | No | No | No | No | No | - | - | No | - | No | Yes | ||
| 47 | F | DLBCL | PD | N/A | Very high | 10 | 2 | Yes | No | - | No | No | No | No | No | - | - | No | - | Yes (217) | No (333) | ||
| 28 | M | ALL | CR1 | Negative | Intermediate | 2 | 4 | Yes | Yes | 1 | No | No | Yes§ | No | Yes | Moderate | Yes | No | - | No | Yes | ||
| 50 | F | AML | CR1 | Negative | High | 3 | 1 | Yes | No | - | No | No | No | No | No | - | - | Yes | Interstitial lung disease (chemo vs infection) | No | No (357) | ||
| 53 | M | PMF | SD | N/A | Intermediate | 1 | 3 | Yes | No | - | No | No | No | No | Yes | Mild | No | No | - | No | Yes | ||
| 39 | M | HL | CR4 | Negative‖ | Low | 7 | 0 | Yes | No | - | Yes | No | No | No | Yes | Moderate | No | No | - | No | Yes | ||
| 40 | M | CML | CP | Positive | Intermediate | 3 | 4 | No (early relapse) | N/A¶ | - | No | No | No | N/A | N/A¶ | - | - | No | - | Yes (41) | No (161) | ||
| 30 | F | ALL | CR2 | Negative | High | 2 | 5 | Yes | No | - | No | No | No | Yes | No | - | - | No | - | No | Yes | ||
| 28 | M | DLBCL | CR3 | Negative‖ | Intermediate | 4 | 2 | Yes | No | - | No | No | No | No | No | - | - | No | - | No | Yes | ||
| 34 | M | ALL | CR5 | Negative | High | 8# | 1 | Yes | No | - | No | No | No | No | No | - | - | No | - | No | Yes | ||
| 21 | M | AML | CR2 | Negative | Low | 2 | 3 | Yes | No | - | No | No | No | Yes | No | - | - | Yes | ARDS/infection (toxo, AdV) after second HCT | No | No (161) | ||
| 52 | M | PMF | SD | N/A | Intermediate | 4 | 5 | No (graft failure) | N/A‡ | - | N/A‡ | No | No | N/A | N/A‡ | - | - | No | - | No | Yes | ||
| 18 | F | AML | CR1 | Negative | Intermediate | 3 | 0 | Yes | No | - | No | No | No | No | Yes | Mild | No | No | - | No | Yes | ||
| DL3 PTCy 25 mg/kg on day +4 | 28 | M | ALL | CR3 | Negative | High | 6 | 8 | No (NRM) | No | - | No | Yes | No | N/A | N/A∗∗ | - | - | Yes | SOS | No | No (33) | |
| 23 | F | HL | CR1 | Negative‖ | Low | 4 | 0 | Yes | Yes | 2 | No | No | No | No | No | - | - | No | - | No | Yes | ||
| 30 | M | ALL | CR2 | Negative | High | 2 | 6 | Yes | Yes | 1 | No | No | No | No | No | - | - | Yes | CMV pneumonitis/enteritis | No | No (102) | ||
| 20 | M | AML | CR1 | Negative | Intermediate | 3 | 3 | Yes | Yes | 1 | Yes | No | No | No | Yes | Moderate | Yes | Yes | Unknown†† | No | No (545) | ||
| 22 | F | AML | CR1 | Negative | Intermediate | 1 | 0 | Yes | No | - | No | No | No | Yes | No | - | - | Yes | Bacterial and fungal pneumonia | No | No (502) | ||
| 26 | F | AML | CR1 | Negative | High | 1‡‡ | 6 | No (Early relapse) | N/A§§ | - | N/A§§ | No | No | N/A | N/A§§ | - | - | No | - | Yes (24) | No (300) | ||
| 21 | F | MDS | SD | N/A | High | 4 | 2 | Yes | No | - | Yes | No | No | No | Yes | Severe | Yes | No | - | Yes (369) | No (394) | ||
| Dose level . | Age, y . | Sex . | Diagnosis . | Disease status at BMT . | MRD status . | Refined DRI . | Prior lines of therapy . | HCT-CI . | Evaluable for primary endpoint (event) . | aGVHD∗ . | Engraftment syndrome . | SOS . | TMA . | Persistent host T-cell chimerism . | cGVHD† . | NRM . | NRM (cause) . | Relapse (day) . | Alive (day of death) . | ||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Yes/No . | Max. grade . | Yes/No . | Max severity . | Systemic immune suppression . | |||||||||||||||||||
| DL1 PTCy 50 mg/kg per day on days +3/+4 | 23 | F | ALL | CR3 | Negative | High | 3 | 4 | Yes | No | - | No | No | No | No | No | - | - | No | - | Yes (187) | No (389) | |
| 25 | M | ALL | CR1 | Negative | Intermediate | 2 | 7 | Yes | No | - | No | No | No | No | No | - | - | No | - | No | Yes | ||
| 21 | M | ALL | CR1 | Negative | Intermediate | 2 | 4 | Yes | No | - | No | No | No | No | No | - | - | No | - | No | Yes | ||
| 27 | M | AML | CR1 | Positive | Intermediate | 3 | 6 | Yes | Yes | 4 | No | No | No | No | No | - | - | Yes | aGVHD, viral/bacterial pneumonia | No | No (165) | ||
| 38 | F | DLBCL | PD | N/A | Very high | 8 | 7 | Yes | No | - | No | Yes | No | No | Yes | Mild | No | No | - | No | Yes | ||
| DL2 PTCy 25 mg/kg per day on days +3/+4 | Phase 1 | 28 | F | ALL | CR3 | Negative | High | 3 | 3 | Yes | No | - | No | No | No | Yes | No | - | - | No | - | Yes (335) | No (417) |
| 38 | F | ALL | CR1 | Negative | Intermediate | 3 | 5 | Yes | Yes | 1 | No | Yes | No | No | Yes | Severe | Yes | No | - | No | Yes | ||
| 43 | M | AML | CR2 | Positive | High | 2 | 4 | Yes | No | - | No | No | No | No | Yes | Mild | No | No | - | Yes (312) | No (542) | ||
| 28 | F | ALL | CR1 | Negative | Intermediate | 2 | 2 | Yes | No | - | No | No | No | No | No | - | - | No | - | No | Yes | ||
| 26 | F | AML | CR1 | Negative | Intermediate | 1 | 6 | No (graft failure) | N/A‡ | - | N/A‡ | No | No | N/A | N/A‡ | - | - | No | - | No | Yes | ||
| 47 | F | ALL | CR1 | Positive | Intermediate | 1 | 1 | Yes | No | - | No | No | No | No | No | - | - | No | - | No | Yes | ||
| 27 | M | HL | PD | N/A | High | 3 | 2 | Yes | No | - | No | No | No | No | Yes | Moderate | Yes | Yes | Interstitial lung disease (chemotherapy) | No | No (237) | ||
| Phase 2 | 57 | M | DLBCL | PD | N/A | Very high | 8 | 7 | Yes | No | - | No | No | No | No | No | - | - | Yes | Bacterial pneumonia | No | No (112) | |
| 42 | M | ALL | CR5 | Negative | High | 6 | 3 | Yes | No | - | No | No | No | Yes | No | - | - | No | - | Yes (286) | No (339) | ||
| 30 | M | ALL | CR1 | Negative | Intermediate | 2 | 4 | Yes | No | - | No | No | No | No | Yes | Moderate | No | No | - | No | Yes | ||
| 31 | M | ALL | CR3 | Negative | High | 3 | 2 | Yes | No | - | No | No | No | No | No | - | - | No | - | No | Yes | ||
| 47 | F | DLBCL | PD | N/A | Very high | 10 | 2 | Yes | No | - | No | No | No | No | No | - | - | No | - | Yes (217) | No (333) | ||
| 28 | M | ALL | CR1 | Negative | Intermediate | 2 | 4 | Yes | Yes | 1 | No | No | Yes§ | No | Yes | Moderate | Yes | No | - | No | Yes | ||
| 50 | F | AML | CR1 | Negative | High | 3 | 1 | Yes | No | - | No | No | No | No | No | - | - | Yes | Interstitial lung disease (chemo vs infection) | No | No (357) | ||
| 53 | M | PMF | SD | N/A | Intermediate | 1 | 3 | Yes | No | - | No | No | No | No | Yes | Mild | No | No | - | No | Yes | ||
| 39 | M | HL | CR4 | Negative‖ | Low | 7 | 0 | Yes | No | - | Yes | No | No | No | Yes | Moderate | No | No | - | No | Yes | ||
| 40 | M | CML | CP | Positive | Intermediate | 3 | 4 | No (early relapse) | N/A¶ | - | No | No | No | N/A | N/A¶ | - | - | No | - | Yes (41) | No (161) | ||
| 30 | F | ALL | CR2 | Negative | High | 2 | 5 | Yes | No | - | No | No | No | Yes | No | - | - | No | - | No | Yes | ||
| 28 | M | DLBCL | CR3 | Negative‖ | Intermediate | 4 | 2 | Yes | No | - | No | No | No | No | No | - | - | No | - | No | Yes | ||
| 34 | M | ALL | CR5 | Negative | High | 8# | 1 | Yes | No | - | No | No | No | No | No | - | - | No | - | No | Yes | ||
| 21 | M | AML | CR2 | Negative | Low | 2 | 3 | Yes | No | - | No | No | No | Yes | No | - | - | Yes | ARDS/infection (toxo, AdV) after second HCT | No | No (161) | ||
| 52 | M | PMF | SD | N/A | Intermediate | 4 | 5 | No (graft failure) | N/A‡ | - | N/A‡ | No | No | N/A | N/A‡ | - | - | No | - | No | Yes | ||
| 18 | F | AML | CR1 | Negative | Intermediate | 3 | 0 | Yes | No | - | No | No | No | No | Yes | Mild | No | No | - | No | Yes | ||
| DL3 PTCy 25 mg/kg on day +4 | 28 | M | ALL | CR3 | Negative | High | 6 | 8 | No (NRM) | No | - | No | Yes | No | N/A | N/A∗∗ | - | - | Yes | SOS | No | No (33) | |
| 23 | F | HL | CR1 | Negative‖ | Low | 4 | 0 | Yes | Yes | 2 | No | No | No | No | No | - | - | No | - | No | Yes | ||
| 30 | M | ALL | CR2 | Negative | High | 2 | 6 | Yes | Yes | 1 | No | No | No | No | No | - | - | Yes | CMV pneumonitis/enteritis | No | No (102) | ||
| 20 | M | AML | CR1 | Negative | Intermediate | 3 | 3 | Yes | Yes | 1 | Yes | No | No | No | Yes | Moderate | Yes | Yes | Unknown†† | No | No (545) | ||
| 22 | F | AML | CR1 | Negative | Intermediate | 1 | 0 | Yes | No | - | No | No | No | Yes | No | - | - | Yes | Bacterial and fungal pneumonia | No | No (502) | ||
| 26 | F | AML | CR1 | Negative | High | 1‡‡ | 6 | No (Early relapse) | N/A§§ | - | N/A§§ | No | No | N/A | N/A§§ | - | - | No | - | Yes (24) | No (300) | ||
| 21 | F | MDS | SD | N/A | High | 4 | 2 | Yes | No | - | Yes | No | No | No | Yes | Severe | Yes | No | - | Yes (369) | No (394) | ||
AdV, adenovirus; ALL, acute lymphoblastic leukemia; AML, acute myeloid leukemia; ARDS, acute respiratory distress syndrome; BMT, bone marrow transplantation; CML, chronic myelogenous leukemia; CP, chronic phase; CR1/2/3/4/5, first/second/third/fourth/fifth complete remission; DLBCL, diffuse large B-cell lymphoma; F, female; HCT-CI, HCT-specific comorbidity index; HL, Hodgkin lymphoma; M, male; MDS, myelodysplastic syndrome; MRD, measurable residual disease; N/A, not applicable; PD, progressive disease; PMF, primary myelofibrosis; SD, stable disease; SOS, sinusoidal obstructive syndrome; TMA, thrombotic microangiopathy; Toxo, toxoplasmosis.
Diagnosed and graded per Keystone criteria.
Diagnosed and staged per National Institutes of Health (2014) consensus criteria.
Primary graft failure requiring second transplant.
No clinical sequelae other than anemia and thrombocytopenia that resolved with discontinuation of sirolimus.
Based on negative positron emission tomography imaging (HL and DLBCL), negative bone marrow (HL and DLBCL), and cerebrospinal fluid analysis by flow cytometry (DLBCL).
Loss of graft because of disease relapse.
Includes prior allogeneic HCT for primary disease.
Follow-up <100 days because of NRM.
International patient with NRM due to meningitis per verbal family report only; no confirmatory documentation available.
Also had prior allogeneic HCT for sickle cell disease.
Disease relapse resulting in engraftment failure.