Our Xyrem Journey
Back in the summer of 2011, we moved from England to the USA for two reasons. One was to seek expert care for Mathilda at the Centre for Narcolepsy Research at Stanford. The other was to get her treatment.
That treatment was a drug called Xyrem. Otherwise known as Sodium Oxybate.
Until then, Mathilda had spent 18 months battling the symptoms of Narcolepsy and Cataplexy.
She was three years old.
She hallucinated at night and when she wasn't sleeping in the day (EDS) she was dealing with Cataplexy. Worse than that, she had almost completely withdrawn, succumbing to the psychological damage of hypnogogic and hypnopompic hallucinations and even self- harming. I felt like I had lost her.
The medication she was prescribed in England was Modafinil which did help her stay awake in the afternoons but did nothing for her night-time symptoms or Cataplexy. And it came at a cost. Modafinil made Mathilda aggressive and angry which added another layer to her behavioral problems.
The decision to sell our home and move all three of our kids to a place they had never been to before, far far away from our family was not taken lightly. The Professor left his job at the University of Bristol where he had been working as a Reader in Theology for the previous 5 years and took up at position here in LA in an entirely different context. Our middle child Elliot (10) was all right with the move and now at the age of 15 is very settled here. Our eldest daughter Liberty on the other hand was not. At 12-years old she didn't want to leave her friends and grandparents. Right now she is back in London for Christmas having saved up enough money for a plane ticket.
But we were desperate. I could not envisage a future for Mathilda without effective treatment. I wasn't sure how we would all go on coping being virtually housebound as a family and caring for her around the clock. Fundamentally I thought that if she didn't get treatment soon, she would lose her childhood.
Within 3 days of landing in LA we had Xyrem. I never expected to come away from our first appointment with Dr. Mignot with two bottles of Sodium Oxybate. But at the end of our afternoon with him the Professor carried Mathilda to our car whilst I trailed behind holding the medication as if was liquid gold.
I should add that the Professor and I were ignorant of a very important fact, which was just as well because if we had any idea that Xyrem is only effective in 80% of PWN we might not be here.
He was sure she was in the 20% (being a glass empty kind of guy). But having risked everything to get here I had to believe Mathilda was in the majority. The odds were surely in her favor!
However, it took a very long time before that became apparent. Certainly over a year, but probably closer to 18 months before Mathilda's dose became optimal.
Initially her dose was very low so it was difficult to see the benefits. Every time we tried to titrate up she was hit hard with nausea, vomiting, headaches and loss of appetite. I remember Mathilda throwing up some mornings in our apartment or in the car on the way to school. Once I pulled in to a garage and she threw up there. Another time it was outside the school on the grass and countless times in the classroom. Within 7 months she lost so much weight I had to pull her out of school and follow the advice of a dietician to help her gain enough weight to stay on her dose.
With every increase in Xyrem, there was a new round of side effects--and so it went on.
But, and this is a BIG BUT--during that time we saw some major improvements. Here are the main ones:
- Her nightly hallucinations stopped once she dad taken each dose.
- Her Excessive Daytime Sleepiness condensed into 3-4 regular naps a day rather than her dropping off anytime and anywhere. This meant we could to some extent plan things like going out!
- She was well enough to attend school for the first time. The benefits for this are obvious but I am going to be honest here and admit to the fact that those hours gave me respite care and time with Liberty and Elliot who were struggling living in a new country. It also gave me time to look for a more permanent home, though that took another year.
- Her Cataplexy improved by about 80% (the other 20% is dealt with in the form of Straterra). This meant she no longer fell down and injured herself and was willing to risk laughing. Her strong sense of fun slowly returned and she managed to do things she had wanted to try like ride a bike and roller skate .
- She stopped self harming and was less depressed.
- We began to see elements of Mathilda’s personality return like chattiness, imaginative play, and a desire to be with others.
- She re-engaged with learning and hobbies such as reading and writing and playing. Her creative side emerged enough to provide a form of art therapy during which time she drew incredible pictures. I am sure a psychologist would have a field day with those.
It transpired then that Xyrem was a wonder drug of sorts for Mathilda but some days I almost gave up hope and there were times when the Professor and I questioned our decision to move.
If I could with hindsight speak to my then 41 years old self, I might say something like this:
- Buckle up. The Xyrem journey is long and rough because it seems to have a sweet spot which can be difficult to find. There’s no magic dose. Rarely are all the benefits seen in a matter of weeks.
- Be patient with the side effects. Try probiotics at night and anti-emetics in the morning. Prepare for weight loss, then once stabilized, weight gain.
- Get a timed safe to keep the second dose of Xyrem out of reach before the three hours are up. Then there’s no risk of an overdose.
- Find respite care sooner and more frequently–before you become depressed yourself. In other words, don’t underestimate the toll of being a caregiver has on you and your whole family.
- Don’t hang in there with a school that isn’t working. Life is too short; childhood is even shorter.
With grateful for thanks to Dr. Mignot and the staff at Stanford for taking care of Mathilda for the past 5 and a half years.