Abstract

GA5-98

Using Perioperative Surgical Home Goals to Facilitate Access to a Novel Therapy: Spinraza® (nusinersen) Intrathecal Treatment for Children with Spinal Muscular Atrophy

Berkelhamer M, Bass N, Miller C
University Hospitals Cleveland Medical Center, Rainbow Babies and Children's Hospital, Cleveland, Ohio, USA

The Perioperative Surgical Home (PSH) model was conceived to improve quality by coordinating care before, during, and after surgical procedures. The PSH provides an entry point for patients to receive care, and ensure coordination and continuity of care. The goals of the PSH: to enhance value while achieving a better experience for patients, better health outcomes, and lower costs were envisioned by the Institute of Health Care Improvement.

The paradigm of the Perioperative Surgical Home has applications across the entire spectrum of procedures requiring anesthesia. Pediatric patients require anesthesia for a variety of painful therapeutic and diagnostic procedures. Children receive anesthesia to prevent pain, anxiety, and movement during invasive procedures such as lumbar puncture.

At Rainbow Babies and Children’s Hospital, we applied the concept of PSH to deliver a novel treatment to patients with Spinal Muscular Atrophy (SMA). Using the PSH paradigms, we were able to quickly provide a portal of entry for SMA patients to receive a new, complicated therapy regimen, under general anesthesia.

SMA, previously untreatable, is a potentially fatal disease, with little hope for preventing the deterioration of motor milestones and respiratory function or improvement of muscle strength until Spinraza® (nusinersen) was approved by the Federal Drug Administration on December 23, 2016. Spinraza® is administered intrathecally via lumbar puncture in a recommended series of doses.

The Spinraza® intrathecal injection regimen requires predictable scheduling of repeated care episodes under general anesthesia. Creating consistent “block time” for the Neurologists to initiate the course of Spinraza® therapy to their SMA patients, ensured access to the growing, eligible patient population.

Spinraza® treatment is initiated with 4 loading doses. Three loading doses are administered at 14-day intervals. The 4th loading dose is given 30 days after the 3rd dose. Maintenance dosing occurs once every 4 months thereafter, and continued indefinitely or until side effects preclude further treatment.

Coordination of perioperative care for infants and children receiving general anesthesia for Spinraza® therapy is facilitated by established outpatient surgery care pathways, and experienced medical staff in the pediatric operating suite. These patients did not meet selection criteria for sedation by non-anesthesia providers. Infants and children with SMA have a variety of complex respiratory and airway pathologies. Their respiratory parameters evolve over the course of treatment, The anesthetic technique is tailored to the SMA type and degree of severity.
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Continuity of care is ensured by a team of pediatric care providers. Pulmonologists optimize respiratory parameters preoperatively, Radiologists facilitate imaging to guide lumbar puncture when necessary,
Patient data is documented by accepted motor scales for SMA. Increased mobility, maintenance or improvement of respiratory function may improve health outcomes for pediatric SMA patients.

No anesthetic related complications have occurred in our series of SMA patients receiving Spinraza® intrathecal injections.

References:
ASA: Perioperative surgical home. http://asahq.org/psh