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

Background: Intrathecal delivery of 2-hydroxypropyl-beta-cyclodextrin (VTS-270, adrabetadex) by lumbar puncture (LP) has been performed on a biweekly schedule for over nine years for the treatment of Niemann-Pick type C1 (NPC1) at Rush University Medical Center.

Methods: Over this time 59 patients with NPC1 have been treated with 2935 infusions, performed with either a 22-G 3-inch Whitacre or a 22-G 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 patients treated for NPC. The NPC1 cohort ranged in age from one to 31 years at the initiation of treatment.

Results: Of the 59 patients treated 33 (55.9%) had no adverse events ever after an LP and 26 (44.1%) had adverse effects at some time. Of the 2935 LPs, adverse events occurred after 3.3% of LPs. Adverse events most observed were headache (1.2% of LPs), nausea (0.3%), vomiting (1.0%), and back pain (1.3%).

Conclusions: This study shows that frequent repeated LPs are possible for delivery of intrathecal therapy and can be accomplished with a very low rate of post-LP adverse events.