Cohort characteristics
| CRS . | ||||||
|---|---|---|---|---|---|---|
| ID . | Sex . | Age (y) . | Primary diagnosis . | Location of emapalumab administration . | Day 28 disease status . | Survival at 6 mo . |
| 5 | M | 16 | B-ALL | ICU | MRD negative | Y |
| 9 | F | 5 | B-ALL | ICU | MRD negative | Y |
| 10 | M | 5 | B-ALL | ICU | N/A | N |
| 12 | M | 19 | B-ALL | ICU | MRD negative | Y |
| 13 | F | 12 | B-ALL | ICU | N/A | Y |
| 22 | M | 20 | B-ALL | ICU | CRi | N/A |
| Primary hemophagocytic lymphohistiocytosis | ||||||
| ID | Sex | Age (y) | Causative mutation | Location of emapalumab administration | Subsequent HSCT? | Survival at 6 mo |
| 1 | M | 0.3 | STXBP2 | ICU | Y | Y |
| 14 | M | 0.2 | STXBP2 | ICU | Y | Y |
| 2 | F | 0.8 | PRF (homozygous) | Ward | Y | Y |
| 16 | M | 0.4 | PRF (complex heterogeneous) | Clinic | Y | Y |
| 17 | M | 1.1 | PRF (homozygous) | Clinic | Y | Y |
| Secondary hemophagocytic lymphohistiocytosis | ||||||
| ID | Sex | Age (y) | Causative disease | Location of emapalumab administration | Subsequent HSCT? | Survival at 6 mo |
| 4 | F | 15 | MAS due to unknown primary disease | ICU | N/A | Y |
| 15 | F | 11 | MAS due to sJIA | Clinic | N/A | Y |
| 6 | F | 12 | MAS due to sJIA | Ward | N/A | Y |
| 3 | M | 0.5 | SAA, iPALF | Ward | N/A | Y |
| 7 | M | 9 | SAA, monosomy 7 | ICU | N/A | N |
| 8 | F | 16 | NK lymphoma, EBV viremia | ICU | N/A | N |
| 11.1 | M | 26 | CAEBV | ICU | Y | Y |
| 11.2 | M | 27 | CAEBV, PTCL | Ward | Y | Y |
| 20 | F | 12 | EBV viremia | ICU | N/A | N |
| Other indication | ||||||
| ID | Sex | Age (y) | Primary diagnosis | Location of emapalumab administration | Indication for emapalumab | Survival at 6 mo |
| 18 | F | 21 | SAA, HSCT | ICU | Inflammatory pulmonary disease | N |
| 21 | F | 13 | Beta thalassemia major, HSCT | Ward | Crohn’s disease/GVHD | N/A |
| CRS . | ||||||
|---|---|---|---|---|---|---|
| ID . | Sex . | Age (y) . | Primary diagnosis . | Location of emapalumab administration . | Day 28 disease status . | Survival at 6 mo . |
| 5 | M | 16 | B-ALL | ICU | MRD negative | Y |
| 9 | F | 5 | B-ALL | ICU | MRD negative | Y |
| 10 | M | 5 | B-ALL | ICU | N/A | N |
| 12 | M | 19 | B-ALL | ICU | MRD negative | Y |
| 13 | F | 12 | B-ALL | ICU | N/A | Y |
| 22 | M | 20 | B-ALL | ICU | CRi | N/A |
| Primary hemophagocytic lymphohistiocytosis | ||||||
| ID | Sex | Age (y) | Causative mutation | Location of emapalumab administration | Subsequent HSCT? | Survival at 6 mo |
| 1 | M | 0.3 | STXBP2 | ICU | Y | Y |
| 14 | M | 0.2 | STXBP2 | ICU | Y | Y |
| 2 | F | 0.8 | PRF (homozygous) | Ward | Y | Y |
| 16 | M | 0.4 | PRF (complex heterogeneous) | Clinic | Y | Y |
| 17 | M | 1.1 | PRF (homozygous) | Clinic | Y | Y |
| Secondary hemophagocytic lymphohistiocytosis | ||||||
| ID | Sex | Age (y) | Causative disease | Location of emapalumab administration | Subsequent HSCT? | Survival at 6 mo |
| 4 | F | 15 | MAS due to unknown primary disease | ICU | N/A | Y |
| 15 | F | 11 | MAS due to sJIA | Clinic | N/A | Y |
| 6 | F | 12 | MAS due to sJIA | Ward | N/A | Y |
| 3 | M | 0.5 | SAA, iPALF | Ward | N/A | Y |
| 7 | M | 9 | SAA, monosomy 7 | ICU | N/A | N |
| 8 | F | 16 | NK lymphoma, EBV viremia | ICU | N/A | N |
| 11.1 | M | 26 | CAEBV | ICU | Y | Y |
| 11.2 | M | 27 | CAEBV, PTCL | Ward | Y | Y |
| 20 | F | 12 | EBV viremia | ICU | N/A | N |
| Other indication | ||||||
| ID | Sex | Age (y) | Primary diagnosis | Location of emapalumab administration | Indication for emapalumab | Survival at 6 mo |
| 18 | F | 21 | SAA, HSCT | ICU | Inflammatory pulmonary disease | N |
| 21 | F | 13 | Beta thalassemia major, HSCT | Ward | Crohn’s disease/GVHD | N/A |
Patient 11 received emapalumab at 2 separate times for different indications.
B-ALL, B-cell acute lymphoblastic leukemia; CAEBV, chronic active Epstein Barr virus; CRi, complete remission with incomplete count recovery; EBV, Epstein Barr virus; F, female; GVHD, graft versus host disease; HSTCT, hematopoietic stem cell transplant; ICU, intensive care unit; iPALF, indeterminate pediatric acute liver failure; M, male; MAS, macrophage activation syndrome; N, no; N/A. not available; NK, natural killer; PRF, perforin; PTCL, peripheral T-cell lymphoma; sJIA, systemic juvenile idiopathic arthritis; SAA, severe aplastic anemia; Y, yes.