Nusinersen injections in adults and children with spinal muscular atrophy: a single-center experience

Abstract

PurposeNusinersen is a drug approved in December 2016 for treatment of spinal muscular atrophy (SMA). We want to share our initial experience with image-guided, non-image-guided, and port-delivered nusinersen injections in a large single-center SMA patient cohort, treating both pediatric and adult patients with focus on technical considerations and other patient concerns from a combined perspective of patient, neurologist, and radiologist.MethodsAll nusinersen injections between February 2017 and September 2018 were retrospectively reviewed. We obtained age, sex, SMA type and technical details of the injections and postprocedure complications for each procedure.ResultsA total of 52 patients (24 women [46%]; 4 patients with SMA-1 [7.6%]; 30 patients with SMA-2 [57.8%]; 18 patients with SMA-3 [34.6%]; mean age, 25.5 years [7 months to 62 years]) with a total of 265 injections were included. Of the 265 injections, 206 (77.9%) were performed with local anesthetic, 25 (9.4%) with moderate sedation, and 23 (8.6%) under general anesthesia. We performed 65 CT-guided transforaminal injections in 13 patients, 106 fluoroscopy-guided lumbar punctures in 24 patients and 83 lumbar punctures in 16 patients using conventional technique. Only 6 of 265 injections (2.2%) ended up with a post-lumbar puncture headache (PLPH) requiring medical treatment. None required an epidural blood patch. Fourteen PLPH (5.2%) occurred and resolved at the same day without any treatment. After 6 of 265 injections (2.2%), patients reported soreness at the injection site which resolved spontaneously. Three elected to have an intrathecal reservoir placement (2 lumbar, 1 intraventricular) with a total of 11 injections. One patient with lumbar catheter developed infection after surgery with subsequent meningitis and treatment delay. After the resolution of meningitis, a new intraventricular reservoir was placed without any complication in the following injections.ConclusionWith the introduction of nusinersen treatment, neurologists and radiologists play an important role in treatment of SMA patients and therefore should be familiar with different techniques and complications of drug administration. Using good technique, it is possible to have very low complication rates even in this complex patient population, and various image-guided procedures can be a safe alternative to surgical approach, even in the most difficult cases.