Demographics and transplantation features
| . | No Ema . | Ema . | Total . | P value . |
|---|---|---|---|---|
| Age, median (range), mo | 18 (3-204) | 10 (3-214) | 17.5 (3-214) | .42 |
| Underlying cause, n (%) | .44 | |||
| Primary HLH | 23 (82) | 20 (91) | 43 (86) | |
| EBV related | 3 (11) | 2 (9) | 5 (10) | |
| VUS/no identified trigger | 2 (7) | 0 (0) | 2 (4) | |
| HLA match, n (%) | .06 | |||
| MRD | 3 (11) | 4 (19) | 7 (15) | |
| MMRD (haploidentical) | 0 (0) | 1 (5) | 1 (2) | |
| MUD | 14 (52) | 10 (48) | 23 (48) | |
| MMUD | 10 (37) | 6 (29) | 16 (33) | |
| 1 allele mismatch | 6 (22) | 3 (14) | 9 (19) | |
| 2 allele mismatches | 4 (15) | 3 (14) | 7 (15) | |
| Conditioning regimen, n (%) | .13 | |||
| Alemtuzumab/Flu/Mel | 25 (89) | 15 (68%) | 40 (80) | |
| Alemtuzumab/Flu/Mel/TT | 2 (7) | 5 (23) | 7 (14) | |
| Alemtuzumab/Flu/Bu | 1 (4) | 1 (5) | 2 (4) | |
| ATG/Flu/Mel/TT | 0 (0) | 1 (5) | 1 (2) | |
| Stem cell source, n (%) | .20 | |||
| Bone marrow | 21 (78) | 13 (62) | 34 (71) | |
| Cord blood | 2 (7) | 8 (38) | 10 (21) | |
| PBSC | 4 (15) | 0 (0) | 4 (8) | |
| Graft cell dose, median (IQR) | ||||
| TNC/kg, ×108 | 10 (5.1-12.3) | 10 (4.2-12.2) | 10 (4.5-12.2) | .82 |
| CD34+ cells per kg, ×106 | 8.7 (5.2-11.2) | 10.8 (9.9-15.3) | 9.9 (6.3-13.4) | .02 |
| GVHD prophylaxis, n (%) | ||||
| CSA/Pred | 18 (67) | 10 (48) | 28 (58) | |
| CSA/MMF | 2 (7) | 3 (11) | 5 (10) | |
| Tacro/Pred | 2 (7) | 0 (0) | 2 (4) | |
| Tacro/MMF | 0 (0) | 1 (5) | 1 (2) | |
| T-cell–depleted graft | 4 (15) | 5 (24) | 9 (19) | |
| Other | 1 (4) | 2 (10) | 3 (6) |
| . | No Ema . | Ema . | Total . | P value . |
|---|---|---|---|---|
| Age, median (range), mo | 18 (3-204) | 10 (3-214) | 17.5 (3-214) | .42 |
| Underlying cause, n (%) | .44 | |||
| Primary HLH | 23 (82) | 20 (91) | 43 (86) | |
| EBV related | 3 (11) | 2 (9) | 5 (10) | |
| VUS/no identified trigger | 2 (7) | 0 (0) | 2 (4) | |
| HLA match, n (%) | .06 | |||
| MRD | 3 (11) | 4 (19) | 7 (15) | |
| MMRD (haploidentical) | 0 (0) | 1 (5) | 1 (2) | |
| MUD | 14 (52) | 10 (48) | 23 (48) | |
| MMUD | 10 (37) | 6 (29) | 16 (33) | |
| 1 allele mismatch | 6 (22) | 3 (14) | 9 (19) | |
| 2 allele mismatches | 4 (15) | 3 (14) | 7 (15) | |
| Conditioning regimen, n (%) | .13 | |||
| Alemtuzumab/Flu/Mel | 25 (89) | 15 (68%) | 40 (80) | |
| Alemtuzumab/Flu/Mel/TT | 2 (7) | 5 (23) | 7 (14) | |
| Alemtuzumab/Flu/Bu | 1 (4) | 1 (5) | 2 (4) | |
| ATG/Flu/Mel/TT | 0 (0) | 1 (5) | 1 (2) | |
| Stem cell source, n (%) | .20 | |||
| Bone marrow | 21 (78) | 13 (62) | 34 (71) | |
| Cord blood | 2 (7) | 8 (38) | 10 (21) | |
| PBSC | 4 (15) | 0 (0) | 4 (8) | |
| Graft cell dose, median (IQR) | ||||
| TNC/kg, ×108 | 10 (5.1-12.3) | 10 (4.2-12.2) | 10 (4.5-12.2) | .82 |
| CD34+ cells per kg, ×106 | 8.7 (5.2-11.2) | 10.8 (9.9-15.3) | 9.9 (6.3-13.4) | .02 |
| GVHD prophylaxis, n (%) | ||||
| CSA/Pred | 18 (67) | 10 (48) | 28 (58) | |
| CSA/MMF | 2 (7) | 3 (11) | 5 (10) | |
| Tacro/Pred | 2 (7) | 0 (0) | 2 (4) | |
| Tacro/MMF | 0 (0) | 1 (5) | 1 (2) | |
| T-cell–depleted graft | 4 (15) | 5 (24) | 9 (19) | |
| Other | 1 (4) | 2 (10) | 3 (6) |
Continuous variables were compared using the Mann-Whitney U test. Categorical variables with 2 different parameters were compared using the Fisher exact test and variables with >2 groups with the Kruskal-Wallis test. For statistical analysis, underlying cause was categorized as primary HLH vs other, HLA match by matched vs mismatched, and conditioning regimen as 3 different groups (alemtuzumab/flu/mel vs alemtuzumab/flu/mel/TT vs other). Due to the large variability in GVHD prophylaxis, comparison between groups was not feasible. The total N for each analysis varied by parameter due to the 2 deaths during the preparative regimen.
Boldface type signifies statistical significance (P < .05).
Bu, busulfan; CSA, cyclosporine; Ema, emapalumab; Flu, fludarabine; Mel, melphalan; MMF, mycophenolate mofetil; MMRD, mismatched related donor; MMUD, mismatched unrelated donor; MRD, matched related donor; MUD, matched unrelated donor; PBSCs, peripheral blood stem cells; Pred, prednisone; TNCs, total nucleated cells; TT, thiotepa; VUS, variant of unclear significance.