Table 1.
Characteristics of children with DAH post HCT by NRM versus Alive or Relapsed.
| Variable | Alive/Relapsed (n=13, 34%), N (%) |
NRM (n=27, 68%), N (%) |
p-value |
|---|---|---|---|
| Age years (median, range)+ | 3 (0-24) | 11 (0.28- 20.5) | 0.02 |
| Sex^
Female Male |
10 (40) 3 (20) |
15 (60) 12 (80) |
0.29 |
| Race^
White Black/African American Asian Other/Declined |
4 (17) 4 (40) 2 (67) 3 (75) |
19 (83) 6 (60) 1 (33) 1 (25) |
0.02 |
| Ethnicity^
Hispanic Non-Hispanic |
2 (33) 11 (32) |
4 (67) 23 (68) |
1.0 |
| HCT Indication^
Heme Malignancy Immune Def/Dys Non-malignant Heme Solid Tumor Neuro/Metabolic |
6 (32) 1 (13) 1 (20) 4 (80) 1 (33) |
13 (68) 7 (88) 4 (80) 1 (20) 2 (67) |
0.16 |
| HCT Type^
Allogeneic Autologous |
9 (28) 4 (80) |
26 (74) 1 (20) |
0.03 |
| Cell Source*^
Bone Marrow Peripheral Blood Umbilical Cord |
3 (14) 3 (30) 3 (75) |
18 (86) 7 (70) 1 (25) |
0.02 |
| Donor*^
Related Unrelated |
3 (21) 6 (29) |
11 (79) 15 (71) |
0.71 |
| HLAMismatch*@^
8/8 7/8 ≤6/8 |
3 (15) 1 (17) 2 (40) |
17 (85) 5 (83) 3 (60) |
0.51 |
| Preparative Intensity^
Myeloablative RIC Non-myeloablative |
12 (44) 1 (8) 0 (0) |
15 (56) 11 (92) 1 (100) |
0.04 |
| Acute GVHD Prophylaxis*^ CNI Methotrexate MMF Abatacept T-cell depletion |
6 (21) 3 (20) 3 (21) 1 (33) 2 (40) |
23 (79) 12 (80) 11 (79) 2 (67) 3 (60) |
0.88 |
| TA-TMA^ | 9 (30) | 21 (70) | 0.70 |
| Eculizumab^ | 9 (35) | 17 (65) | 1.0 |
| SOS^ | 4 (21) | 15 (79) | 0.19 |
| Defibrotide^ | 4 (22) | 14 (78) | 0.31 |
| Maximum aGVHD*^
Grade 0-2 Grade 3-4 |
7 (28) 2 (20) |
18 (72) 8 (80) |
0.69 |
| Concurrent Systemic Infection#%^ | 8 (35) | 15 (65) | 1.0 |
| Concurrent Pulmonary Infection#%^ | 7 (35) | 13 (65) | 1.0 |
| Day first pulmonary bleed (median, range) | 59 (52) | 36 (163) | 0.74 |
| Number of pulmonary bleeds (median, range)+ | 1 (1-4) | 1 (1-4) | 0.53 |
*Allogeneic HCT recipients only (n=35), #within 4 weeks of first pulmonary bleed, %diagnosed via bacterial, viral, or fungal culture or next generation sequencing of BAL fluid. @excluding cord blood, ^Fishers Exact Test, +Wilcoxon-Rank Sum Test, Abbreviations: human leukocyte antigen (HLA), reduced intensity conditioning (RIC), graft versus host disease (GVHD), calcineurin inhibitor (CNI), mycophenolate mofetil (MMF), transplant associated thrombotic microangiopathy (TA-TMA), sinusoidal obstructive syndrome (SOS), graft versus host disease (GVHD), hematologic malignancy (heme malignancy), immune deficiency/dysregulation (immune def/dys), non-malignant blood disorder (non-malignant heme). Bold indicates significant differences.