Table 1:
Baseline characteristics of patients undergoing autologous hematopoietic cell transplant (aHCT) for neuroblastoma and association with development of transplant associated thrombotic microangiopathy (TA-TMA)
| Characteristics | TA-TMA (n=10) |
No TA-TMA (n=131) |
p-valuea |
|---|---|---|---|
| Age in years | 2.5 [1.8-5.2] | 4 [0.8-22.9] | 0.05 |
| Male sex | 6 (60%) | 80 (61%) | 1.0 |
| Race | 0.57 | ||
| • White | 5/10 (50%) | 79/124 (64%) | |
| • Black | 0/10 (0%) | 6/124 (5%) | |
| • Otherb | 5/10 (50%) | 39/124 (31%) | |
| Hispanic ethnicity | 2/10 (20%) | 26/126 (21%) | 1.0 |
| INSS stage 4 at diagnosis | 9 (90%) | 107 (82%) | 1.0 |
| Primary tumor site abdominal | 7 (70%) | 110 (84%) | 0.37 |
| Additional therapy beyond induction | |||
| chemotherapy and surgery prior to aHCT | |||
| Conditioningc | |||
| • None | 6 (60%) | 66 (50%) | 0.75 |
| • lrinotecan/temozolomide/chl4:18 | 0 (0%) | 5 (4%) | 1.0 |
| • 131I-MIBG therapy +/− chemotherapyd | 1 (10%) | 26 (20%) | 0.69 |
| • Local radiation | 3 (30%) | 23 (18%) | 0.39 |
| • Other cytotoxic chemotherapy | 3 (30%) | 38 (29%) | 1.0 |
| Disease status prior to transplant | 0.33 | ||
| • Complete response or very good partial response | 6 (60%) | 78 (60%) | |
| • Partial response | 3 (30%) | 30 (23%) | |
| • Stable disease | 0 (0%) | 18 (14%) | |
| • Progressive disease | 1 (10%) | 4 (3%) | |
| History of prior relapse | 1 (10%) | 7 (5%) | 0.45 |
| Nuclear GFR (ml/min/1.73m2) prior to transplant | 107.2 [76.6-128] (n=8) | 115.4 [63-234] (n=87) | 0.23 |
| GFR <100 ml/min/1.73m2 prior to transplant | 2/8 (25%) | 22/87 (25%) | 1.0 |
| Renal or renovascular involvement from neuroblastoma at diagnosis | 4/7 (57.1%) | 40/89 (45%) | 0.70 |
| Nephrectomy prior to transplant | 0/10 (0%) | 14/129 (11%) | 0.60 |
| Days from diagnosis to transplant | 187.5 [141-596] | 202 [138-1162] | 0.45 |
| Type of conditioning chemotherapy | <0.001 | ||
| • Single CEMd | 4 (40%) | 64 (49%) | |
| • Bu/Mel | 0 (0%) | 56 (43%) | |
| • Cy/Thio (intended for tandem) | 1 (10%) | 2 (2%) | |
| • Cy/Thio and CEM (tandem) | 5 (50%) | 7 (5%) | |
| • Other | 0 (0%) | 2 (2%) | |
| Transplant time period (years) | 0.35 | ||
| • 2000-2006 | 2 (20%) | 48 (37%) | |
| • 2007-2011 | 2 (20%) | 37 (28%) | |
| • 2012-2017 | 6 (60%) | 46 (35%) |
Data presented as number of patients (%) with denominator written if different than total sample size (n) for the column for categorical variables and as median [range] for continuous variables.
Continuous variables were compared using the Wilcoxon rank-sum test and categorical variables using the Fisher’s exact test.
The “other” patients were divided among Asian, Native Hawaiian or Pacific Islander, other or mixed.
Individual patients may be listed multiple times if they received multiple therapies prior to transplantation therefore we did not use a family-wise comparison.
This includes those patients (13) who received 131I-MIBG with single CEM as pre-transplant conditioning as part of a clinical trial.
Abbreviations: Bu/Mel- busulfan and melphalan, CEM-carboplatin, etoposide, melphalan, Cy/Thio- cyclophosphamide and thiotepa, GFR- glomerular filtration rate, INSS- International neuroblastoma staging system, MIBG- metaiodobenzylguanidine