Spoiler Alert – If you haven’t watched Season 4 yet, you might want to skip this post.
Whether you hate to love or love to hate Frank Underwood, there is no question that this season – with the attempted assassination, wild hallucinations, and the emergency liver transplant – was intense and even sometimes difficult to watch. Perhaps especially for me. I don’t even know if I would have paid much attention to the doctors description of high ammonia levels before Jacob, but now that I’ve received an unwelcome education, I watched rapt, terrified of what can happen when a liver fails to do its job.
I haven’t discussed liver transplants much (at all?) on this blog because that course of treatment is not currently part of the conversation with Jacob’s doctor. A liver transplant would “cure” Jacob’s citrullinemia. We could serve him a prime rib the next day. (Actually, there would probably be some restrictions on what you could eat the day after major surgery, but you get the picture.)
There is some disagreement in the UCD community over whether it is wiser to transplant early and often, if you will, or manage the disorder with diet and medication, as we have done for the last 13 months. In short, the arguments, as best I understand them, go like this:
Pro-Transplant: This disorder is a ticking time-bomb. A single crisis of hyperammonemia, and the child could suffer permanent brain damage. Besides, we don’t know what effect the permanently high citrulline levels (in children with citrullinemia, specifically) have on the brain. Some people contend that high citrulline can cause headaches and autism-like symptoms in otherwise well-managed children. Also, we don’t really know the long-term impact of ammonia scavenger medications like Buphenyl. In short, it is safer to transplant, because issues related to transplants are more predictable. There is simply more research. In fact, a 2013 study determined that “[liver transplant] was associated with the eradication of hyperammonemia, removal of dietary restrictions, and potentially improved neurocognitive development.”
Sounds pretty good, except…
Anti-Transplant: An organ transplant is a major surgery, and irreversible. Why would you take the risk of transplanting an otherwise healthy child, when the treatment options and management protocols for UCDs are getting better every day? Either way, the child will be on some kind of medication permanently, whether it’s an ammonia scavenger drug or anti-rejection immunosuppressants. Besides, considerable research is being done on liver therapies that could eliminate the need for transplant well within our lifetimes. If the UCD is well managed, it’s better to wait and see what kinds of options you will have in the upcoming years.
So, yes, we are somewhere between the devil and the deep blue sea here.
Last year, in coordination with the National Urea Cycle Disorders Foundation, the Patient-Centered Outcomes Research Institute (PCORI) approved a $2 million study to determine which treatment approach, if any, has the best outcomes. The project is scheduled to take 37 months. Is it possible that we will be able to put this debate to rest by the time that Jacob turns 4?