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