Feasibility analysis of intrathecal administration strategy of nusinersen based on Cobb angle in children with spinal muscular atrophy.

This retro-prospective observational study described the experience in lumbar puncture procedures in children with spinal muscular atrophy (SMA) with and without neuromuscular scoliosis in a single center. The technical feasibility of intrathecal nusinersen administration was the main limiting factor. A total of 457 technically successful intrathecal injections based on a hierarchical strategy in Cobb angle were reviewed in 81 SMA children aged 0.75–13.5 years who were referred for nusinersen injections in our hospital from October 2019 to December 2022. Under local anesthesia, conventional lumbar puncture was performed on 47 patients without spinal deformity (Cobb angle of 0–10°) and 20 patients with moderate scoliosis (Cobb angle of 10–50°). Ultrasound-assisted lumbar puncture was performed on 12 patients with moderate scoliosis but lordosis. A combination of ultrasound imaging and three-dimensional CT under sedation was performed in the remaining 14 patients with severe scoliosis (Cobb angle >50°). No severe complications were found. Cobb angle is an important basis for intrathecal administration of nusinersen. It is feasible and suitable to carry out intrathecal nusinersen injection under ultrasound combined with three-dimensional CT imaging for children with severe scoliosis. • Many children with SMA have scoliosis causing challenging intrathecal access. • Personalized therapies are required to fit patients with different Cobb angle. • Ultrasound positioning was helpful for intrathecal injections within scoliosis. • Preoperative sedation helped to reduce patient's anxiety, thus got good cooperation. [ABSTRACT FROM AUTHOR]