Clinical and genetic features by maximum level of therapy received
| Maximum level of therapy . | Total . | Overall survivors, N . | 1-year survival estimate,* % . | 3-year survival estimate,* % . | Overall survival estimate,* % . | Associated trigger, % . | Genetic profile, % . |
|---|---|---|---|---|---|---|---|
| Observation/none | 9 | 9 | 100 | 100 | 100 | None: 22.2 | fHLH: 0.0 |
| Autoimmune: 11.1 | PIDD: 0.0 | ||||||
| Infection: 55.6 | DIAP: 0.0 | ||||||
| Malignancy: 11.1 | PIDD/DIAP: 0.0 | ||||||
| Other candidate defects: 11.1 | |||||||
| No genetic explanation: 88.9 | |||||||
| Biologics/steroids | 18 | 14 | 70 | 70.0 | 70 | None: 11.1 | fHLH: 0.0 |
| Autoimmune: 55.6 | PIDD: 0.0 | ||||||
| Infection: 33.3 | DIAP: 16.7 | ||||||
| Malignancy: 0.0† | PIDD/DIAP: 5.6 | ||||||
| Other candidate defects: 0.0 | |||||||
| No genetic explanation: 77.8 | |||||||
| Immunochemotherapy | 54 | 32 | 58 | 58 | 39 | None: 18.5 | fHLH: 9.3 |
| Autoimmune: 31.5 | PIDD: 16.7 | ||||||
| Infection: 29.6 | DIAP: 7.4 | ||||||
| Malignancy: 20.4 | PIDD/DIAP: 1.9 | ||||||
| Other candidate defects: 3.7 | |||||||
| No genetic explanation: 61.1 | |||||||
| HSCT | 38 | 21 | 75 | 57 | 40 | None: 47.4† | fHLH: 36.8† |
| Autoimmune: 10.5† | PIDD: 0.0† | ||||||
| Infection: 39.5 | DIAP: 2.6 | ||||||
| Malignancy: 2.6† | PIDD/DIAP: 2.6 | ||||||
| Other candidate defects: 2.6 | |||||||
| No genetic explanation: 55.3 |
| Maximum level of therapy . | Total . | Overall survivors, N . | 1-year survival estimate,* % . | 3-year survival estimate,* % . | Overall survival estimate,* % . | Associated trigger, % . | Genetic profile, % . |
|---|---|---|---|---|---|---|---|
| Observation/none | 9 | 9 | 100 | 100 | 100 | None: 22.2 | fHLH: 0.0 |
| Autoimmune: 11.1 | PIDD: 0.0 | ||||||
| Infection: 55.6 | DIAP: 0.0 | ||||||
| Malignancy: 11.1 | PIDD/DIAP: 0.0 | ||||||
| Other candidate defects: 11.1 | |||||||
| No genetic explanation: 88.9 | |||||||
| Biologics/steroids | 18 | 14 | 70 | 70.0 | 70 | None: 11.1 | fHLH: 0.0 |
| Autoimmune: 55.6 | PIDD: 0.0 | ||||||
| Infection: 33.3 | DIAP: 16.7 | ||||||
| Malignancy: 0.0† | PIDD/DIAP: 5.6 | ||||||
| Other candidate defects: 0.0 | |||||||
| No genetic explanation: 77.8 | |||||||
| Immunochemotherapy | 54 | 32 | 58 | 58 | 39 | None: 18.5 | fHLH: 9.3 |
| Autoimmune: 31.5 | PIDD: 16.7 | ||||||
| Infection: 29.6 | DIAP: 7.4 | ||||||
| Malignancy: 20.4 | PIDD/DIAP: 1.9 | ||||||
| Other candidate defects: 3.7 | |||||||
| No genetic explanation: 61.1 | |||||||
| HSCT | 38 | 21 | 75 | 57 | 40 | None: 47.4† | fHLH: 36.8† |
| Autoimmune: 10.5† | PIDD: 0.0† | ||||||
| Infection: 39.5 | DIAP: 2.6 | ||||||
| Malignancy: 2.6† | PIDD/DIAP: 2.6 | ||||||
| Other candidate defects: 2.6 | |||||||
| No genetic explanation: 55.3 |