Low Risk Profile of Long-Term Repeated Lumbar Puncture for Intrathecal Delivery of 2-Hydroxypropyl-beta-Cyclodextrin in Patients with Niemann-Pick Type C

Intrathecal delivery of 2-Hydroxypropyl-beta-cyclodextrin (VTS-270, adrabetadex) by lumbar puncture (LP) has been performed on a 2-weekly schedule for over 9 years for the treatment of Niemann-Pick type C1 (NPC1) at Rush University Medical Center (RUMC). Over this time 59 patients with NPC1 have been treated with 2935 infusions, performed with either a 22 gauge 3 inch Whitacre or a 22 gauge 2 inch Gertie-Marx atraumatic needles, with or without general anesthesia. Adverse events potentially related to the LP infusion were collected from records for all treated NPC patients.
Source: Pediatric Neurology - Category: Neurology Authors: Tags: Research Paper Source Type: research