Demographics and outcomes for ex vivo expansion patients
. | . | . | . | . | . | Acute . | . | . | . | . | |
|---|---|---|---|---|---|---|---|---|---|---|---|
| Patient no. . | Diagnosis . | Age, y . | Weight, kg . | HLA match . | Conditioning regimen . | GVHD grade . | GVHD locations . | Days of EFS . | Event . | Outcome . | |
| 1 | CID | 1.27 | 6.0 | 4/6 | Bu/Cy/ATG | 0 | NA | 80 | Death | Pulmonary failure | |
| 2 | Lesch-Nyhan syndrome | 5.00 | 13.7 | 4/6 | Bu/Mel/ATG | IV | Skin, gut | 158 | Death | Sepsis after dental procedure | |
| 3 | Infant ALL, CR2 | 3.92 | 17.1 | 5/6 | Bu/Mel/ATG | 1 | Skin, gut | > 1587 | NA | Alive and well | |
| 4 | ALL, CR3 | 5.67 | 19.4 | 4/6 | TBI/Mel*45/ATG | 0 | NA | 153 | Relapse | Died of relapse | |
| 5 | ANLL, relapse | 11.50 | 34.2 | 5/6 | TBI/Mel*45/ATG | 0 | NA | > 1566 | NA | Alive and well | |
| 6 | CD7+ acute leukemia | 15.25 | 43.4 | 3/6 | TBI/Mel*45/ATG | II | Skin, gut | 196 | Relapse | Died of relapse | |
| 7 | WAS | 4.00 | 15.9 | 4/6 | Bu/Cy/ATG | 0 | NA | > 1509 | NA | Alive and well | |
| 8 | WAS, MDS | 1.00 | 8.8 | 4/6 | Bu/Cy/ATG | 0 | NA | > 1501 | NA | Alive and well | |
| 9 | MDS/ANLL | 1.00 | 8.6 | 5/6 | Bu/Mel/ATG | 0 | NA | > 1482 | NA | Alive and well | |
| 10 | ALL, relapse | 7.75 | 24.1 | 4/6 | Bu/Mel/ATG | IV | Liver | 94 | Graft failure | Died of GVHD | |
| 11 | HD, relapse | 23.58 | 77.8 | 4/6 | Bu/Mel/ATG | 0 | NA | 41 | Death | Hemorrhagic stroke | |
| 12 | WAS | 3.92 | 17.1 | 4/6 | Bu/Cy/ATG | 1 | Skin | > 1453 | NA | Alive and well | |
| 13 | WAS | 0.58 | 6.0 | 4/6 | Bu/Cy/ATG | IV | Skin, gut | 99 | Death | GVHD | |
| 14 | T-ALL, relapse | 14.25 | 64.7 | 4/6 | TBI/Mel*45/ATG | 0 | NA | 17 | Death | Sepsis, respiratory failure | |
| 15 | WAS | 2.00 | 11.1 | 5/6 | Bu/Cy/ATG | 0 | NA | 42 | Graft failure | Alive and well after 2nd UCB | |
| 16 | MDS | 11.75 | 52.2 | 4/6 | TBI/Mel*45/ATG | 0 | NA | 43 | Death | Toxoplasmosis | |
| 17 | CML, accelerating | 8.00 | 31.6 | 4/6 | TBI/Mel*45/ATG | 0 | NA | 121 | Autoreconstitution | Died undergoing 2nd UCB | |
| 18 | MDS | 9.83 | 32.8 | 4/6 | TBI/Mel*45/ATG | III | Gut | > 1419 | NA | Alive and well | |
| 19 | ALL, Ph+, relapse | 11.92 | 30.7 | 4/6 | TBI/Mel*45/ATG | I | Skin | > 1405 | NA | Alive and well | |
| 20 | JCML | 0.58 | 8.1 | 4/6 | Bu/Mel/ATG | 0 | NA | 220 | Relapse | Alive with relapse JCML | |
| 21 | Osteopetrosis | 0.66 | 7.3 | 4/6 | Bu/Cy/ATG | I | NA | 123 | Death | Stroke | |
| 22 | MDS, monosomy 7 | 3.25 | 16.0 | 5/6 | Bu/Mel/ATG | 0 | NA | > 1383 | NA | Alive and well | |
| 23 | ANLL, relapse | 3.75 | 13.1 | 5/6 | TBI/Mel*45/ATG | 0 | NA | 42 | Graft failure | Died undergoing 2nd UCB | |
| 24 | ANLL | 15.83 | 47.4 | 3/6 | TBI/Mel*45/ATG | 0 | NA | 191 | Death | Relapsed ALL | |
| 25 | WAS | 2.00 | 12.0 | 4/6 | Bu/Cy/ATG | IV | Skin, gut, liver | 42 | Death | Adenovirus | |
| 26 | WAS | 2.33 | 9.9 | 4/6 | Bu/Cy/ATG | IV | Liver | 287 | Death | Sepsis, EBV lymphoma | |
| 27 | ALL, CR2 | 6.00 | 20.5 | 5/6 | TBI/Mel*45/ATG | I | Skin | > 1299 | NA | Alive and well | |
| 28 | AML, relapse | 36.20 | 69.3 | 4/6 | TBI/Mel*45/ATG | II | Skin | 462 | Death | Died of relapse | |
| Median | 4.50 | 17.10 | |||||||||
| Mean | 7.60 | 25.67 | |||||||||
| SD | 8.05 | 20.34 | |||||||||
. | . | . | . | . | . | Acute . | . | . | . | . | |
|---|---|---|---|---|---|---|---|---|---|---|---|
| Patient no. . | Diagnosis . | Age, y . | Weight, kg . | HLA match . | Conditioning regimen . | GVHD grade . | GVHD locations . | Days of EFS . | Event . | Outcome . | |
| 1 | CID | 1.27 | 6.0 | 4/6 | Bu/Cy/ATG | 0 | NA | 80 | Death | Pulmonary failure | |
| 2 | Lesch-Nyhan syndrome | 5.00 | 13.7 | 4/6 | Bu/Mel/ATG | IV | Skin, gut | 158 | Death | Sepsis after dental procedure | |
| 3 | Infant ALL, CR2 | 3.92 | 17.1 | 5/6 | Bu/Mel/ATG | 1 | Skin, gut | > 1587 | NA | Alive and well | |
| 4 | ALL, CR3 | 5.67 | 19.4 | 4/6 | TBI/Mel*45/ATG | 0 | NA | 153 | Relapse | Died of relapse | |
| 5 | ANLL, relapse | 11.50 | 34.2 | 5/6 | TBI/Mel*45/ATG | 0 | NA | > 1566 | NA | Alive and well | |
| 6 | CD7+ acute leukemia | 15.25 | 43.4 | 3/6 | TBI/Mel*45/ATG | II | Skin, gut | 196 | Relapse | Died of relapse | |
| 7 | WAS | 4.00 | 15.9 | 4/6 | Bu/Cy/ATG | 0 | NA | > 1509 | NA | Alive and well | |
| 8 | WAS, MDS | 1.00 | 8.8 | 4/6 | Bu/Cy/ATG | 0 | NA | > 1501 | NA | Alive and well | |
| 9 | MDS/ANLL | 1.00 | 8.6 | 5/6 | Bu/Mel/ATG | 0 | NA | > 1482 | NA | Alive and well | |
| 10 | ALL, relapse | 7.75 | 24.1 | 4/6 | Bu/Mel/ATG | IV | Liver | 94 | Graft failure | Died of GVHD | |
| 11 | HD, relapse | 23.58 | 77.8 | 4/6 | Bu/Mel/ATG | 0 | NA | 41 | Death | Hemorrhagic stroke | |
| 12 | WAS | 3.92 | 17.1 | 4/6 | Bu/Cy/ATG | 1 | Skin | > 1453 | NA | Alive and well | |
| 13 | WAS | 0.58 | 6.0 | 4/6 | Bu/Cy/ATG | IV | Skin, gut | 99 | Death | GVHD | |
| 14 | T-ALL, relapse | 14.25 | 64.7 | 4/6 | TBI/Mel*45/ATG | 0 | NA | 17 | Death | Sepsis, respiratory failure | |
| 15 | WAS | 2.00 | 11.1 | 5/6 | Bu/Cy/ATG | 0 | NA | 42 | Graft failure | Alive and well after 2nd UCB | |
| 16 | MDS | 11.75 | 52.2 | 4/6 | TBI/Mel*45/ATG | 0 | NA | 43 | Death | Toxoplasmosis | |
| 17 | CML, accelerating | 8.00 | 31.6 | 4/6 | TBI/Mel*45/ATG | 0 | NA | 121 | Autoreconstitution | Died undergoing 2nd UCB | |
| 18 | MDS | 9.83 | 32.8 | 4/6 | TBI/Mel*45/ATG | III | Gut | > 1419 | NA | Alive and well | |
| 19 | ALL, Ph+, relapse | 11.92 | 30.7 | 4/6 | TBI/Mel*45/ATG | I | Skin | > 1405 | NA | Alive and well | |
| 20 | JCML | 0.58 | 8.1 | 4/6 | Bu/Mel/ATG | 0 | NA | 220 | Relapse | Alive with relapse JCML | |
| 21 | Osteopetrosis | 0.66 | 7.3 | 4/6 | Bu/Cy/ATG | I | NA | 123 | Death | Stroke | |
| 22 | MDS, monosomy 7 | 3.25 | 16.0 | 5/6 | Bu/Mel/ATG | 0 | NA | > 1383 | NA | Alive and well | |
| 23 | ANLL, relapse | 3.75 | 13.1 | 5/6 | TBI/Mel*45/ATG | 0 | NA | 42 | Graft failure | Died undergoing 2nd UCB | |
| 24 | ANLL | 15.83 | 47.4 | 3/6 | TBI/Mel*45/ATG | 0 | NA | 191 | Death | Relapsed ALL | |
| 25 | WAS | 2.00 | 12.0 | 4/6 | Bu/Cy/ATG | IV | Skin, gut, liver | 42 | Death | Adenovirus | |
| 26 | WAS | 2.33 | 9.9 | 4/6 | Bu/Cy/ATG | IV | Liver | 287 | Death | Sepsis, EBV lymphoma | |
| 27 | ALL, CR2 | 6.00 | 20.5 | 5/6 | TBI/Mel*45/ATG | I | Skin | > 1299 | NA | Alive and well | |
| 28 | AML, relapse | 36.20 | 69.3 | 4/6 | TBI/Mel*45/ATG | II | Skin | 462 | Death | Died of relapse | |
| Median | 4.50 | 17.10 | |||||||||
| Mean | 7.60 | 25.67 | |||||||||
| SD | 8.05 | 20.34 | |||||||||
CID indicates combined innumodefieciency; Bu, busulfan; Cy, cyclophosphamide; NA, not applicable; Mel, melphalan; ALL, acute lymphoblastic leukemia; CR2, second complete remission; CR3, third CR; ANLL, acute nonlymphocytic leukemia; WAS, Wiskott-Aldrich; WAS, MDS, Wiskott-Aldrich with evidence of myelodyspalsia; HD, Hodgkin disease; T-ALL, T-cell phenotype of acute lymphoblastic leukemia; CML, chronic myelogenous leukemia; Ph+, Philadelphia chromosome-positive; and JCML, juvenile CML.