Table 3.

Survival of modern-era relapsed/refractory neuroblastoma patients, overall and according to standard risk factors determined at the time of diagnosis (n=383 patients)

	Number of patients n (%)	PFS from start of early-phase trial enrollment			OS from start of early-phase trial enrollment
		1-yr ±SE (%)	4-yr ±SE (%)	p-value	1-yr ±SE (%)	4-yr ±SE (%)	p-value
Overall	383	21±2	6±1	NA	57±3	20±2	NA
Risk Group
Low	12 (5)	28±14	9±9		55±15	28±14
Intermediate	7 (3)	29±17	0	0.9	57±19	29±22	0.8
High	214 (92)	20±3	6±2		58±3	15±3
unknown	150
Era* of first early-phase enrollment
8/02 – 4/09 (pre-dinutuximab)	282 (74)	19±2	5±1	0.1	53±3	17±2	0.008
5/09 – 1/14 (post-dinutuximab)	101 (26)	28±5	10±4		69±5	26±6
INSS stage#
1,2,3,4S	29 (12)	18±7	5±4	0.6	46±10	15±8	0.5
4	205 (88)	22±3	6±2		59±4	17±3
unknown	149
Age at diagnosis#
<18 months	17 (7)	35±12	28±11	0.1	46±12	32±12	0.4
≥18 months	218 (93)	20±3	5±2		59±3	16±2
unknown	148
MYCN status#
Not amplified	163 (84)	23±3	7±2	0.003	62±4	21±3	<0.0001
Amplified	32 (16)	13±6	0		30±8	0
unknown	188
Ploidy#
Hyperdiploid	91 (50)	20±4	3±2	0.5	49±5	21±5	0.8
Diploid	91 (50)	24±5	10±3		59±5	15±4
unknown	201
Histology#
Favorable	12 (7)	25±13	17±11	0.5	57±15	29±14	0.4
Unfavorable	165 (93)	19±3	5±2		55±4	16±3
unknown	206
MKI#
Low/Intermediate	95 (77)	16±4	6±3	0.2	54±5	15±4	0.3
High	29 (23)	30±9	5±5		40±9	9±6
unknown	259
Grade of differentiation#
Differentiating	14 (10)	14±10	0	1.0	57±13	7±7	0.5
Undifferentiated	126 (90)	20±4	7±2		54±5	15±3
unknown	243
11q#
No LOH	17 (55)	30±13	0	0.02	67±12	10±9	0.03
LOH	14 (45)	0	0		31±13	0
unknown	352
1p#
No LOH	24 (75)	19±9	0	0.4	55±11	6±5	0.3
LOH	8 (25)	0	0		50±18	0
unknown	351
Prior transplant
No	101 (36)	26±5	8±3	0.2	58±5	27±5	0.5
Yes	180 (64)	18±3	4±2		61±4	16±3
unknown	102
TTFR#
<30 months	146 (83)	15±3	4±2	0.3	48±4	11±3	0.055
≥30 months	30 (17)	21±8	0		72±8	17±8
unknown	207

^#At the time of diagnosis

NA – Not applicable

^*Efficacy of dinutuximab identified 4/2009