Anesthetic management of pediatric patients undergoing intrathecal nusinersen administration for treatment of spinal muscular atrophy: A single‐center experience

Abstract

Background Spinal muscular atrophy is a group of autosomal recessive inherited neurological disorders secondary to a genetic mutation that leads to progressive muscle weakness and atrophy. Recently approved by the Food and Drug Administration, Nusinersen is the first treatment specifically for spinal muscular atrophy. This drug must be administered intrathecally, as it does not cross the blood‐brain barrier. Aims Retrospective review of the individualized perioperative care of spinal muscular atrophy type I and II patients for intrathecal Nusinersen injection. Methods After approval from the Institutional Review Board and obtaining informed written consent from parents, we reviewed the anesthetic charts of nine patients who underwent 58 Nusinersen injections over a 23‐month period from February 2017 to December 2018. An individualized anesthetic plan was formulated based on the patient's disease severity, anxiety level, and comfort of parents as well as the provider performing the procedure. Patients underwent intrathecal Nusinersen injection under fluoroscopic guidance, with either general anesthesia, monitored anesthesia care with medications or monitored anesthesia care without medications. Patients recovered in the postanesthesia care unit for a minimum of 1 hour after injection, irrespective of the anesthetic method utilized. Analysis included patient demographics, anesthesia type relative to spinal muscular atrophy type I vs. II, and postanesthesia recovery. Results Nine patients with spinal muscular atrophy types I and II underwent 58 encounters. Five spinal muscular atrophy type I patients underwent 31 encounters, general anesthesia (9), monitored anesthesia care with medications (2), and monitored anesthesia care without medications (20). Four spinal muscular atrophy type II patients underwent 27 encounters, general anesthesia (22), monitored anesthesia care with medications (2), and monitored anesthesia care without medications (3). There were no perioperative complications. Conclusion The anesthetic plan for Nusinersen injections must be individualized to the patient's specific needs and clinical manifestations of the disease. When carefully tailored to each patient, anesthetic care is safe and successful.

Needleless inhaled anesthesia with sevoflurane: Advantages of a simplified approach for children with spinal muscular atrophy undergoing intrathecal administration of nusinersen

“…In several studies that have examined the clinical experience of nusinersen administration in SMA children, data about AEs are lacking or rough. 15,17,18,41 Bielsky et al 17 claimed there was not evidence of AEs in eight children with SMA type 2 undergoing general anesthesia using sevoflurane, nitrous oxide, and propofol for 61 LPs. However, AEs were not defined in the materials and methods section, so minor or minimal self-limiting AEs could not be excluded.…”

Procedural sedation for paediatric patients with spinal muscular atrophy undergoing intrathecal treatment

“…they mostly used non-pharmacological methods. 9 Either because the provider was inexperienced and the paediatric patient declined to be immobile or because intrathecal injection was scheduled to be done using another procedure, general anaesthesia using sevoflurane was administered to three of these five patients. The authors also reported that, after repeated nusinersen injections, two of the SMA I patients became stronger, and their anaesthetic plan was changed to using anaesthetic medication rather than local anaesthesia.…”

Predicting outcomes for spinal muscular atrophy: When the diagnosis no longer means what it used to mean

“…Sever's work adds to a growing body of literature describing the safety and efficacy of general anaesthesia and procedural sedation in these complex patients undergoing routine nusinersen administration. 3,5 The authors did not report any postoperative complications in this cohort during the first 24 h; however, the majority of postoperative complications occur in the first 1-3 days after a procedure, 6 and many studies use a 30-day benchmark when assessing postoperative mortality and rate of adverse events. Furthermore, how are these patients faring for longer, higher risk surgeries?…”