Table 1.

Demographics, clinical characteristics, and details of intrathecal injections

Clinical characteristics	Case 1	Case 2	Case 3	Case 4	Case 5	Case 6	Case 7
Age at first dose (y)	12	12	12	19	16	13	15
Sex	Male	Female	Male	Male	Female	Female	Female
Copies of SMN2 gene	3	3	NA	3	3	2	NA
SMA subtype	2b	1c	3a	2b	3a	1c	2a
Motor function	Sit independently	Sit on the back	Sit independently	Sit independently	Sit independently	Sit on the back	Sit on the back
Spine features	Spinal fusion	Spinal fusion	Spinal fusion	Spinal fusion	Spinal fusion	Severe scoliosis	Severe scoliosis
Lumbar laminotomy	No	L3–L4	No	L3–L4	L3–L4	–	–
Time-interval between spine surgery and first dose nusinersen	5 months	1 year and 11 months	1 month	3 years and 4 months	1 month	–	–
Cobb’s angle	13°	45°	10°	87°	18°	77°	75°
Rotation of spinea	0	IV	0	IV	I	III	IV
No. of procedures	4	4	4	1	1	4	1
Injection level	L4–L5	L3–L4	L3–L4	L3–L4	L3–L4	L4–L5	L5–S1
No. of interspace level adjustments per procedure	1/1/1/1	1/1/1/1	1/1/1/1	1	1	3/1/1/1	1
No. of insertions per procedure	2/2/1/1	3/2/1/1	2/1/1/1	1	1	8/2/1/1	2
Operation time (min)b	35/34/18/19	45/28/17/15	20/18/14/13	17	19	56/37/17/15	37
Puncture time (min)c	12/8/5/3	20/10/3/2	7/3/2/2	3	3	30/15/3/3	5
Adverse events	Transient leg numbness, postpuncture headache	Post-puncture headache	No	No	No	Lumbar pain	No
CHOP INTEND score before first and last dose	29/36	19/30	29/37	NA	NA	21/26	NA

SMA spinal muscular atrophy, SMN survival motor neuron, NA not available

^aRotation of the spine was referred to as the maximum degree of lumbar spine rotation on X-ray, according to the Nash and Moe criteria

^bOperation time was defined as the time from the preprocedure scanning to the completion of the intrathecal injection

^cPuncture time was defined as the time from the needle puncturing the skin to the outflow of cerebrospinal fluid