March 10, 2012 mcfadyena

Fifth Intrathecal Dose at UNC

Trey received his 5th intrathecal dose of 30mg of Elaprase Tuesday, March 6 by lumbar puncture. His white count was again less than one, which means no reactions. Yahoo! By Wednesday afternoon I felt like we’d been in the hospital for days and days and days.

On the sixth month (fifth dose because he missed his November dose due to intrathecal port infection and removal at the end of October), like the first month, Trey has an IV in for 4 days and 3 nights for PK’s (pharmacokinetic studies). Blood draws constantly (15 on Thursday alone). They actually added more blood draws to the protocol because the enzyme is being released from the brain into the bloodstream for so long (at least 2 days) that they want more information! To give you a comparison, the enzyme from Trey’s weekly IV Elaprase infusions is out of his bloodstream within hours of his infusion. So, while this is GREAT news, it makes for a long week.
PK’s are done on monthes 1, 6, 19 & 31, so we won’t have to do the IV thing for another year. In the mean time, our long hospital stays are DONE! Instead of staying for three nights in hospital, for the next 3 doses, we only have to stay over night! And then, from month 10 onwards (aside from PK monthes) we only have to stay for a few hours after dosing! We’re in the home stretch!! For now. I say ‘for now’ because trial life has taught me not to assume or get too accustomed to what ‘was’ going to or ‘what I was told’ is going to happen. Things (ie. protocol, criteria) change so quickly, the moment you assume something, it changes.
Another significant decision made this week was that Trey will NOT have an intrathecal port placed next week. Back in October, Trey’s intrathecal port was removed due to infection. The neurosurgeons would not put in a new device until they were confident there was no lingering infection. To make a long story short, somewhere between then and now, after a lot of reading and questions and debate and conversation, I wanted to explore not putting in a new device. The current device in use is less than ideal and I have many reservations. However, I didn’t know how Dr. Muenzer would react to my request/hope.
Dr. Muenzer is AMAZING. He listens (like REALLY listens), contemplates, tells me if he doesn’t know or that he’ll need to look into or think about something more. It’s one thing when you have limited interactions with someone. It’s easy (or easier) for someone to be amazing when you don’t see them often. But for him to continue to listen and respond with thought and to respect and support decisions that may ‘make waves’ when you see parents daily for close to half a month, EACH month, is nothing short of unbelievable. After pondering this a lot, I think it’s because through all the med school and lab work, he’s held onto his humanity. He still sees Trey and our family as people, not a diagnosis or a number. This past week alone, he must have spent 3 or more hours just talking with Trey, my mom and I. We are SO fortunate to have him running this trial.
So, long story long, for the time being, we will be dosing by lumbar puncture month by month, assessing how things are going and making decisions based on new information that becomes available.
We have this coming week and the following week off. March 27-30, Trey will have end of study tests (the actual trial is only six monthes, now Trey rolls into the extension study), same as what was tested for him to qualify for the trial back in August, which will culminate on Friday with the LONG procedures under general anesthetic: MRI, ABR, bronch, LP. April 3rd Trey has his sixth IT dose and April 5th we get to make our way home!!
Thank you for all your support. It’s been quite a year.


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