Characteristics of XLP1 patients not receiving HSCT
| . | . | Number . |
|---|---|---|
| Age at first symptom | 8 y 8 mo (6 mo-40 y) | |
| Age at death | 7.5 y (1-31 y) | |
| Time from presentation to death | 17.3 mo (1 NK) 9 d-18 y | |
| Time from first symptom (in those patients alive) | 12 y (1 NK) 1-39 y | |
| Presenting symptom | ||
| HLH | 31.3% | 15/48 |
| FIM | 10.4% | 5/48 |
| Lymphoma | 16.7% | 8/48 |
| Dysgammaglobulinemia | 29.2% | 14/48 |
| Other | 12.5% | 6/48 |
| Features | ||
| HLH | 33.3% | 16/48 |
| FIM | 12.5% | 6/48 |
| Lymphoma | 20.1% | 10/48 |
| Dysgammaglobulinemia | 56.3% | 27/48 |
| Gut | 8.3% | 4/48 |
| Other | 14.6% | 7/48 |
| EBV status | ||
| EBV+ | 66.6% | 32/48 |
| EBV− | 14.6% | 7/48 |
| Unknown | 18.8% | 9/48 |
| Mortality | 37.5% | 18/48 (4 EBV−) |
| Associated with HLH | 81.3% | 13/16 |
| Associated with FIM | 33.3% | 2/6 |
| Associated with lymphoma | 20% | 2/10 1 had previous HLH and died during chemotherapy; 1 had recurrent lymphoma and many other problems |
| Immunoglobulin replacement | ||
| Yes | 70% | 21/30 |
| No | 23.3% | 7/30 |
| Unknown | 6.7% | 2/30 |
| . | . | Number . |
|---|---|---|
| Age at first symptom | 8 y 8 mo (6 mo-40 y) | |
| Age at death | 7.5 y (1-31 y) | |
| Time from presentation to death | 17.3 mo (1 NK) 9 d-18 y | |
| Time from first symptom (in those patients alive) | 12 y (1 NK) 1-39 y | |
| Presenting symptom | ||
| HLH | 31.3% | 15/48 |
| FIM | 10.4% | 5/48 |
| Lymphoma | 16.7% | 8/48 |
| Dysgammaglobulinemia | 29.2% | 14/48 |
| Other | 12.5% | 6/48 |
| Features | ||
| HLH | 33.3% | 16/48 |
| FIM | 12.5% | 6/48 |
| Lymphoma | 20.1% | 10/48 |
| Dysgammaglobulinemia | 56.3% | 27/48 |
| Gut | 8.3% | 4/48 |
| Other | 14.6% | 7/48 |
| EBV status | ||
| EBV+ | 66.6% | 32/48 |
| EBV− | 14.6% | 7/48 |
| Unknown | 18.8% | 9/48 |
| Mortality | 37.5% | 18/48 (4 EBV−) |
| Associated with HLH | 81.3% | 13/16 |
| Associated with FIM | 33.3% | 2/6 |
| Associated with lymphoma | 20% | 2/10 1 had previous HLH and died during chemotherapy; 1 had recurrent lymphoma and many other problems |
| Immunoglobulin replacement | ||
| Yes | 70% | 21/30 |
| No | 23.3% | 7/30 |
| Unknown | 6.7% | 2/30 |