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 |