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

Özet

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, andport-delivered nusinersen injections in a large single-center SMA patient cohort, treating bothpediatric and adult patients with focus on technical considerations and other patient concernsfrom 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 totalof 265 injections were included. Of the 265 injections, 206 (77.9%) were performed with localanesthetic, 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 lumbarpunctures 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%) occurredand resolved at the same day without any treatment. After 6 of 265 injections (2.2%), patientsreported soreness at the injection site which resolved spontaneously. Three elected to have anintrathecal reservoir placement (2 lumbar, 1 intraventricular) with a total of 11 injections. Onepatient with lumbar catheter developed infection after surgery with subsequent meningitis andtreatment delay. After the resolution of meningitis, a new intraventricular reservoir was placedwithout any complication in the following injections.CONCLUSIONWith the introduction of nusinersen treatment, neurologists and radiologists play an importantrole in treatment of SMA patients and therefore should be familiar with different techniques andcomplications 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 procedurescan be a safe alternative to surgical approach, even in the most difficult cases.