Anesthetic management of pediatric patients with Niemann-Pick disease type C for intrathecal 2-hydroxypropyl-ß-cyclodextrin injection.

BACKGROUND: Niemann-Pick disease type C is an autosomal-recessive, lysosomal storage disorder with variable age of onset and a heterogeneous clinical presentation that includes neurological, psychiatric, and visceral findings. 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 anesthesia at two tertiary care centers was conducted from December 2015 through April 2019. Data were extracted for analysis included preoperative 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-cyclodextrin administration 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%-3.1%). Minor adverse events (emesis, delirium, hypotension, seizure, and airway obstruction) were observed in 19/394 encounters (4.8%, 95% CI 3.0%-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 and effective option for pediatric patients with Niemann-Pick disease type C undergoing serial intrathecal injections of 2-hydroxypropyl-beta-cyclodextrin, supporting this technique as a viable option for anesthetic care in these patients.

Pneumothorax after tracheostomy closure with successful nonsurgical management.

A 3-year-old girl presented for routine closure of her tracheostomy site. She was intubated easily for the procedure, and the wound was closed with a drain in place. In recovery, the mother noticed fullness in the patient's submandibular region, and on examination, the girl had subcutaneous emphysema in the neck bilaterally. She returned to the operating room for exploration, and air was released from the surgical site. The wound was again closed with a drain in place, and the patient was extubated uneventfully. After arriving to the pediatric intensive care unit for monitoring, the patient acutely developed respiratory distress and was found to have pneumomediastinum and pneumothorax and was emergently intubated. She was observed closely, and the following day, the pneumothorax improved, and she successfully extubated without further complication.