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

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.