Anesthetic Management of Pediatric Patients with Niemann-Pick Disease Type C for Intrathecal 2-hydroxypropyl-beta-cyclodextrin Injection.

BACKGROUND: Niemann-Pick disease type C is an autosomal-recessive, lysosomal storage disorder with variable age of onset and a heterogeneous clinical presentationthat includes neurological, psychiatric, and visceralfindings.Serial intrathecal injections of 2-hydroxypropyl-beta-cyclodextrin are being evaluated as a treatment modality for Niemann-Pick disease type C with a subset of patients requiring anesthesia for this procedure. AIMS: The aim of this study was to evaluate the safety of anesthesia provided for patients undergoing intrathecal injection of 2-hydroxypropyl-beta-cyclodextrin. METHODS: A retrospective review of pediatric patients who received serial intrathecal injections of 2-hydroxypropyl-beta-cyclodextrin with anesthesiawith anesthesia at two tertiary care centers was conducted from December 2015 through April 2019.Data was extracted for analysis included pre-operative comorbidities, demographics, vital signs,intraoperative anesthesia course, airway management technique, venous access, postoperative course, and perioperative complications. In total, 19 patients were identified and a total of 394 anesthetic encounters were included in this study. RESULTS: All 394 2-hydroxypropyl-beta-cyclodextrinadministration procedures were successfully performed and there were no changes made in the anesthetic plan during the anesthesia encounters. Three hundred forty-nine anesthetics were performed utilizing inhalation induction and mask maintenance and 45 anesthetics were performed with placement of a supraglottic airway device due to patient body habitus and provider preference. The incidence of a major adverse event (aspirations, arterial desaturation) was 5/394 (1.3%, 95% CI 0.05% to 3.1%). Minor adverse events (emesis, delirium, hypotension, seizure, airway obstruction) were observed in19/394encounters(4.8%, 95% CI 3.0% to 7.5%). CONCLUSIONS: Our findings suggest that general anesthesia induced via inhalation induction and maintained with volatile anesthetic via mask or supraglottic airway is a safe andeffective option for pediatric patients with Niemann-Pick disease type C undergoing serial intrathecal injections of 2-hydroxypropyl-beta-cyclodextrin, supporting thistechnique as a viable option for anesthetic care in these patients.