Table 2.
Demographic and disease characteristics
| HLH with TMA 16/23 (70%) | HLH without TMA 7/23 (30%) | |
|---|---|---|
| Median age in years (range) | 1.5 (0.4–23) | 1 (0.1–4) |
| Female gender | 10 | 4 |
|
Genetic HLH diagnosis No genetic diagnosis/no other triggers EBV-HLH (no genetic diagnosis) |
7 6 3 |
5 1 1 |
| TMA diagnosis (requires 5 of 7 criteria to be met) | ||
| 11/16 met 7 criteria | 1/7 met 4 criteria | |
| 4/16 met 6 criteria | 2/7 met 3 criteria | |
| 1/16 met 5 criteria | 4/7 met 2 criteria | |
| Organ injury | ||
| Multi-organ dysfunction syndrome (MODS) | 15/16 (94%) | 4/7 (57%) |
| Hypertension, severe | 16/16 (100%) | 0/7 (0%) |
| Nephrotic range proteinuria | 13/16 (81%) | 1/7 (14%) |
| Elevated sC5b-9 (> 244 ng/mL) | 13/14 (93%) | 0/4 (0%) |
| Renal failure | 8/16 (50%) | 0 (0%) |
| Renal replacement therapy | 7/16 (44%) | 0/7 (0%) |
| Cystatin C GFR < 50 mL/min | 13/16 (81%) | 2/6 (33%) |
| Positive pressure ventilation | 12/16 (75%) | 4/7 (57%) |
| Pulmonary hypertension | 4/15 (27%) | 1/7 (14%) |
| Serositis requiring surgical interventions | 6/16 (38%) | 0 (0%) |
| GI symptoms (bleeding, ileus) | 7/16 (44%) | 0/7 (0%) |
| CNS symptoms (any)a | 11/16 (69%) | 5/7 (71%) |
| CNS bleeding | 6/11 (55%) | 1/7 (14%)b |
| PICU admission | 12/16 (75%) | 4/7 (57%) |
| Death | 6/16 (37.5%) | 3/7 (43%) |
Additional information is listed in Supplemental Table 1
aClinically attributed to HLH diagnosis
bPerinatal intraventricular hemorrhage (IVH)