Another Delay while We Wait on the FDA

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.

Hopefully, the third time is a charm – our new SCCA arrival date is May 6th. Ryan will likely go in-patient around May 20th, to start chemo, and they are targeting May 28th for the transplant.

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.”