Choices

Nathan's bone marrow is officially severely aplastic, having only 5-10% cellularity, meaning his bone marrow cannot even come close to making the blood cells that his body needs. His liver is improving but his bone marrow clearly is not, in fact it is failing. And he now has a very low neutrophil count; neutrophils are a type of white blood cell that fights off infections, and a low count is a huge indicator of his poor bone marrow health. We kinda knew is was coming, as much as we had hoped it wouldn't, watching his blood counts trickle down each day was pretty telling but seeing the bone marrow results seals the deal so to speak. It appears that now is the time, or soon at least, that he will need to begin some sort of real treatment to try to reverse this before it's too late. Let's assume he has non-viral hepititis associated aplastic anemia, rather than HLH which is also still a possibility. Generally a child with severe aplastic anemia who has a sibling match for their bone marrow would go directly to a bone marrow transplant. While the process is difficult and long, it is also incredibly effective, giving a child like Nathan around a 90-95% chance of a complete long term reversal. That's incredible, truly, bone marrow failure sounds pretty ominous so knowing such an effective cure exists is beyond my comprehension. The alternative, for children without a matched sibling bone marrow donor, would be to try a combination of medications alone, and only attempting a bone marrow transplant if that is ineffective or if there is a subsequent relapse. These medications have their side effects but the therapy is not even close to as intense as that which would occur with a bone marrow transplant. Medication alone will reverse around 80% of severe aplastic anemia patients initially, taking around two to three months for this reversal to even begin occurring. Approximately 30% of those children relapse within a reasonably short period of time (usually 2 years), effectively giving it a 56% cure rate. Having our two children as bone marrow matches for one another essentially gives us the choice...it is a gift and a curse, and I am feeling paralyzed. On one hand, I have a medication regime that is reasonably likely to cure him but it will take a long time to know for sure and taking that time will reduce the possible effectiveness of the bone marrow transplant by about ten percent if he does end up going down that road, and it would also be precious time wasted knowing we went against the standard evidence based recommendations for his condition. On the other hand, I have an almost certain cure which many other families wish they had, but one which will essentially will make him extremely sick for a few good weeks, severely weakening his immune system in order to replace it with his sister's. Our doctor, who is among the absolute best for this diagnosis, feels like either choice is valid and appropriate for his situation. While the sibling transplant is the usual preferred first course of action, we can try the medication first, especially during this time when we feel there is still a chance that he may have HLH, as this same medication is also sometimes used to treat that syndrome. So the bottom line decision... (a) try the meds now and risk having to do a slightly less effective bone marrow transplant in two months time, or (b) wait a week or two while we watch his daily labs and rule out HLH completely then plan on a bone marrow transplant right away. Pros and cons of all sides. My head is spinning. I will have many many questions for the doctors tomorrow. The good news is that the liver biopsy has yet again been postponed, simply because it may not really help us in the immediate future as we make these very big decisions. For what it's worth, Nathan said he wants a bone marrow tansplant because then he knows he will get better for sure, clearly he too is tired of all the waiting as well and wants the more sure thing even knowing how sick it will make him and how long he will be hospitalized as a result. I have to take that preference into account as I have been so comforted by his ability to think logically and rationally despite all that he is going through.