Table 1.

Patient-related characteristics and outcomes for 83 patients with SCID

	N	Deaths	5-Year OS, % (95% CI)	P	Neurologic events	5-Year cumulative incidence of neurologic events, % (95% CI)	P	Neurologic event–free survivors	5-Year NEFS, % (95% CI)	P
Overall	83	21	78 (69-87)		8	11 (4-18)		54	68 (58-78)
Sex				.06
Male	49	16	69 (56-82)		6	15 (7-31)	.28	27	57 (43-71)	.03
Female	34	5	91 (82-100)		2	7 (2-24)		27	85 (73-97)
Trigger for diagnosis
Infection/autoimmunity	49	18	67 (54-80)	.02	8	20 (7-23)	.03	23	51 (37-65)	<.001
FH	13	1	100 (73-100)		0	0 (0-27)		12	100 (72-100)
NBS	21	2	90 (77-100)		0	0 (0-18)		19	90 (77-100)
Pre-HCT infection				.004
No	34	3	97 (91-100)		0	0 (0-12)	.006	31	97 (91-100)	<.001
Yes	49	18	63 (49-76)		8	21 (8-24)		23	49 (32-60)
Age at HCT				.002
<3.5 mo	39	4	92 (84-100)		1	3 (1-7)	.02	34	90 (80-99)	<.001
>3.5 mo	44	17	66 (52-70)		7	20 (7-34)		20	50 (35-65)
Genotype				.007
IL2RG/JAK3	20	2	90 (85-100)		3	16 (6-46)	.73	15	75 (56-94)	.03
RAG1/2	13	2	85 (65-100)		1	9 (1-59)		10	77 (54-99)
DCLRE1C	23	7	83 (67-98)		1	4 (1-30)		15	78 (61-95)
IL7R/CD3D	12	1	92 (76-100)		2	17 (5-59)		9	75 (51-99)
Rare/unknown*	15	9	40 (15-65)		1	11 (2-71)		5	33 (10-57)
SCID classification				.05
Typical	71	16	82 (72-91)		8	13 (4-21)	.26	47	70 (59-81)	.36
Leaky/Omenn	12	5	53 (22-85)		0	0 (0-28)		7	53 (22-85)

^*Other genotypes included: LIG4 (n = 1) and RMRP (n=1). Thirteen were unknown.