The intrathecal administration of nusinersen (Spinraza) can prove problematic in patients with SMN1 gene-related proximal spinal muscular atrophy (SMA) due to spinal deformation that is either pre-existing or acquired during treatment.
American clinicians have put in place, experimentally, a catheter linking the spinal canal to an implanted subcutaneous chamber, thus facilitating iterative injections of this product.
The results obtained in 17 patients, both children and adults, who received nusinersen in this way, with between nine and twelve repeated administrations each, showed that:
- the complication rate is not negligible, but is acceptable: mechanical problems, CSF leaks or sepsis.
- the functional efficacy of the treatment does not seem to be affected.
The authors recommend this method for patients with complex anatomies.
