Table 1.

Patients with ADA SCID: treatment parameters and outcomes

UPN	Age at GT (mo)*	CD34+ cells (×106/kg)*	VCN*	Bu AUC (μM × min)*	Follow-up (y after GT)	ERT status	IgRT status
401	184	0.6	0.6	Not measured	11	Resumed ERT 6.5 mo post-GT	On
402	4	1.7	0.71	5437	11	Off ERT since GT	On
403	97	0.92	0.185	3871	11	Off ERT since GT	On
404	3	7.1	1.96	3532	10	Off ERT since GT	Off
405	8	7.62	2	5469	9	Off ERT since GT	Off
406	17	8	2.6	2427	9	Off ERT since GT	On
407	14	4, 1.6	1.0, 2.3†	3232	9	Off ERT since GT	On
408	3	6.85	2.68	5344	8	Off ERT since GT	Off
409	20	2.86	1.22	5608	8	Off ERT since GT	On
410	3	8.41	2.38	6714	8	Off ERT since GT	Off

^*Data for age at gene therapy, CD34⁺ cell dose, cell product vector copy number, and busulfan area-under-the-curve are from Shaw et al. (ADA gene mutations for these patients are listed in Table 1).¹³

^†Because of a manufacturing deviation, subject 407 received 2 separate cell products.

Bu AUC, busulfan area-under-the-curve; UPN, unique patient number.