Table 1.
Patient characteristics
| Purged transplantation group (n=243) |
Non-purged transplantation group (n=243) |
|
|---|---|---|
| Age | ||
| <18 months | 33/243 (14%) | 31/243 (13%) |
| ≥18 months | 210/243 (86%) | 212/243 (87%) |
| INSS stage at diagnosis | ||
| Stage 1* | 2/243 (1%) | 1/243 (0·4%) |
| Stage2a/2b | 1/243 (<1%) | 5/243 (2%) |
| Stage 3 | 29/243 (12%) | 29/243 (12%) |
| Stage 4 | 208/243 (86%) | 206/243 (85%) |
| Stage 4S | 3/243 (1%) | 2/243 (1%) |
| Tumour MYCN status | ||
| Amplified | 85/194 (44%) | 87/196 (44%) |
| Non-amplified | 109/194 (56%) | 109/196 (56%) |
| Unknown | 49 | 47 |
| Tumour histology | ||
| Favourable | 7/171 (4%) | 5/173 (3%) |
| Unfavourable | 164/171 (96%) | 168/173 (97%) |
| Unknown | 72 | 70 |
| Tumour ploidy | ||
| Hyperdiploid | 84/189 (44%) | 91/196 (46%) |
| Diploid | 105/189 (56%) | 105/196 (54%) |
| Unknown | 54 | 47 |
| Bone marrow morphology | ||
| After induction cycle 2 | ||
| Negative | 107/168 (64%) | 118/177 (67%) |
| Positive | 61/168 (36%) | 59/177 (33%) |
| Unknown | 75 | 66 |
| At the end of induction | ||
| Negative | 168/208 (81%) | 181/216 (84%) |
| Positive | 40/208 (19%) | 35/216 (16%) |
| Unknown | 35 | 27 |
| PBSC day 1 immunocytology | ||
| Positive | 1/219 (<1%) | 4/220 (2%) |
| Negative | 218/219 (>99%) | 216/220 (98%) |
| No harvest | 14 | 7 |
| Unknown day 1 immunocytology† | 10 | 16 |
| TLDA analysis of PBSC from day 1 of leukapheresis‡ | ||
| Tumour detectable | 68/129 (53%) | 54/116 (47%) |
| Tumour undetectable | 61/129 (47%) | 62/116 (53%) |
| No TLDA specimen obtained or specimen of insufficient quality | 114 | 127 |
| Overall response at the end of induction | ||
| Complete response | 52/236 (22%) | 55/241 (23%) |
| Very good partial response | 62/236 (26%) | 73/241 (30%) |
| Partial response | 68/236 (29%) | 65/241 (27%) |
| Stable disease | 16/236 (7%) | 16/241 (7%) |
| Progressive disease | 38/236 (16%)§ | 32/241 (13%) |
| Unknown | 7 | 2 |
| MIBG scan at the end of induction | ||
| Complete response | 107/191 (56%) | 100/197 (51%) |
| Less than complete response | 84/191 (44%) | 97/197 (49%) |
| Unknown | 52 | 46 |
| Transplantation | ||
| Number proceeding to stem-cell transplantation | 180/243 (74%) | 192/243 (79%) |
| Number for whom any stem-cell product infused was not the product randomised | 35/180 (19%) | 5/192 (3%) |
| Number for whom back-up PBSC infusion was given | 5/180 (3%) | 6/192 (3%) |
| Number receiving post-stem-cell transplantation anti-GD2 antibody | 36/180 (20%) | 42/192 (22%) |
| Post-induction GFR | ||
| ≥100 mL/min per 1·73 m2 (normal GFR) | 163/194 (84%) | 156/201 (78%) |
| <100 mL/min per 1·73 m2 (low GFR) | 31/194 (16%) | 45/201 (22%) |
| Unknown | 49 | 42 |
Proportions have been calculated excluding patients with unknown values. INSS=international neuroblastoma staging system. PBSC=peripheral blood stem cell. TLDA=TaqMan low density array. MIBG=meta-iodobenzylguanidine. GFR=glomerular filtration rate.
Stage 1 at diagnosis and progressed to stage 4 without interval chemotherapy.
All but two patients without day 1 immunocytological data had immunocytology testing on a separate stem-cell sample before stem cell-infusion for transplantation; treating physicians chose to infuse non-immunocytology tested products in those two patients.
Percentages calculated on the basis of the number of patients who were harvested and who had a specimen of sufficient quality (129 purged, 116 non-purged, 245 overall).
One additional patient who received a transplant was retrospectively (post-transplant) determined by the treating institution to have had progressive disease at the end of induction.