With less than a week remaining before Ryan’s scheduled
second arrival at Seattle Cancer Care Alliance (SCCA) to resume the
pre-transplant work-up, I received a call yesterday from one of the doctors we
have been working with there, Dr. Laurie Burroughs. The national non-malignant
board that she chairs met to discuss Ryan’s case and all of the doctors said
his case is very challenging.
The board felt that he doesn’t currently meet the diagnosis
for Aplastic Anemia due the increase in cellularity of his bone marrow
(although his biopsy a few weeks ago shows that it has gone back down some).
However, he still does not have any test results that confirm MDS, so he
doesn’t technically meet that criteria either. There is still one outstanding
test, the big “UW Oncoplex” to look at over 100 individual genes. If they don’t
find any mutations from that test, and nothing else changes with the next bone
marrow biopsy, then his official new diagnosis will likely be “underlying bone
marrow failure” (I believe this implies that it is probably something genetic,
but they don’t know what).
All of the doctors on the board agreed with our prior
decision to hold off on the transplant in February, but that it is very
reasonable to proceed with transplant at this point. However, there apparently
was quite a bit of debate between the doctors as to which pre-conditioning
regimen (chemo and/or radiation before transplant) to use; the majority of the
board favored a more aggressive approach.
Dr. Burroughs presented the option for Ryan to participate
in a phase 2 clinical trial she is the Principal Investigator of, that has had
some very promising results. It involves using three different chemotherapy
agents prior to transplant, Treosulfan, Fludarabine & Anti-thymosite
Globulin (ATG), without radiation. (clinicaltrials.gov, protocol 2256 –
Identifier 00919503) So far, there have been no patients with liver toxicity,
which is often a problem with one of the standard chemo agents (Cytoxin).
Everyone on the non-malignant board indicated that this would be a good
approach for Ryan.
The caveat… Either the drug company or FDA (I am not clear)
recently asked them to lower the dose of Treosulfan used. Since then, they have
had problems with engraftment failure (meaning the donor bone marrow fails to
set up shop in the patient’s body or fails after engrafting). The same study is
being conducted in Europe ; they never
decreased the dosage of Treosulfan in their trial and have not had problems
with engraftment failure. Therefore, Dr. Burroughs just sent a letter to the
FDA requesting permission to reinstate the higher dose. The FDA has 30 days to
respond. If they do not deny the request within 30 days then it is
automatically approved.
In the meantime, Ryan has been burning through platelets at
a faster pace and his ANC (white blood cells that fight infection) keep
dipping. He just got platelets in the ER last Thursday night and today, a week
later, his platelet count was back down to 4,000 and his ANC is 400. Now that
his ANC is back under 500, we have to be even more hyper-vigilant about germs,
the types of foods he can eat, etc. I suspect the lower counts are due to the
discontinuation of his Tacrolimus (immunosuppressant medication). However, the
doctors took him off of it because they said, in theory, it shouldn’t have been
helping him because they don’t have any signs that indicate his bone marrow
failure is autoimmune.
On a lighter note, I picked Ryan up from Grandma N’s house
after work yesterday and he had taken a bath and spiked his hair into a mohawk.
He very matter of factly said “Yeah, I wanted to do a mohawk before I get bald.”
