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The C word and the Clinical Trial
This post is a hard one to write. It's filled with so much fear and hope. I'm going to address my last two videos first, answering questions about the visit to UVA/Pittsburgh and the BSEP protein test, then getting into the C word and the Clinical Trial.
BSEP and Biliary Diversion
When we went to UVA there was a lot of optimism around Levi's condition. He LOOKS GREAT and I think this deceives many. We came away from our visit thinking the BSEP (Bile Salt Export Pump) protein dye test would confirm PFIC2 or another PFIC, and determine whether Levi was a candidate for Biliary Diversion surgery.
Here is what we've come to understand about this.
The BSEP protein dye test CAN confirm PFIC2 and the absence of protein in Levi's liver cells as we were advised by UVA/Pittsburgh.
The presence of the BSEP protein in Levi's liver cells DOES NOT mean he is not PFIC2. The protein could be present but non-functioning which still means he is PFIC2.
Our medical team at Georgetown confirmed Levi's diagnosis of PFIC2 the following ways:
Clinical presentation.
Biopsy results of liver cells.
DNA - although inconclusive he does have one known genetic mutation for PFIC2.
Levi presents like many PFIC2 children our doctors see from Saudi Arabia (so strange).
Because Levi is PFIC2 our Georgetown team feels very strongly that Biliary Diversion surgery is not an option for him. Which brings me to the C Word.
The C Word
The largest reason why Levi is not a candidate for Biliary Diversion surgery is because the Georgetown team believes he is at a HIGH risk for the C word. Many kids who have PFIC2 clear their bilirubin as the disease waxes and wanes. You may notice from my Facebook posts that we go through periods of intense itching and then it tapers however, when Levi's bilirubin tapers it never gets to a normal number (less than 1). His bilirubin has never gone below 4. This means that Levi's liver is replacing cells at a rapid rate without any rest time. At some point there could be C cells and they could grow. THIS is why our Georgetown team has him listed, with exception points, at 25. Dr. Yazigi is not willing to risk the C word. Our visit today was not the first time she has mentioned the C word but it was the first time she spoke with such fear. She knows how much of a worrier I am and I think she was protecting me from the fear, from the children she has lost to C as a result of this disease. I don't envy her and I certainly feel more fearful today than I have ever before.
The Clinical Trial
But there is a silver lining. At the same time that she showed us her fear, she gave us hope. Last Friday, she was presented with an opportunity to begin a clinical trial at Georgetown (because GT is an international center, they take many kids from overseas who do not have living donor options). The medication presented by the Pharmaceutical company (just saying Pharmaceutical company makes me shudder a little) has been found to help other proteins carry the bile out of the liver (I'm going off memory here and am not good with all this chemistry/biology, so more to come).
The primary goal of the clinical trial is to decrease the pruritis (itching). The byproduct, is that the bile clears from the liver eliminating the overproduction of cells, eliminating the C risk, reducing the strain on the liver and the need for TRANSPLANT. It's hard to even believe. This could be an ANSWERED PRAYER! While not a cure, it could be 1 pill a day for the rest of his life to keep his liver functioning normally. There could be no transplant, no immune suppression, no hospital visits because of a normal cold. It could be what we've been asking God for.
But (you knew there was going to be a but) the trial has to pass Dr. Yazigi's scrutiny (she just got some preliminary information), it has to pass the board and attorney's review. This could take months especially because the board may be on recess for the summer (really?!).
Additionally, we need 1 more test to confirm his eligibility: the BSEP protein dye test (what the what - we see you paving the way God)!
So what now
We push forward with the clinical trial: It turns out, there is another center doing a similar clinical trial - Pittsburgh. We've already asked if Levi could be a part of that trial while we wait at GT. The team at UVA/Pittsburgh is who initiated the BSEP test, so there is a chance they already have us on their radar for their clinical trial (we have a call into their surgeon to ask). Being in the trial does not preclude us from transplant. If something happens, we go in, he and I or his number goes up and he gets the next liver.
We watch Levi like a hawk: He goes in for quarterly MRI's (which means sedation) to confirm there is no C. We watch his labs monthly for C markers. We forge ahead with him on the transplant list while also doing the clinical trial.
We PRAY like CRAZY!
Pray
Here are some things you can pray for:
No C - not now, not ever
The pharmaceutical team - that they are presenting a good drug with minimal side effects that does what they say it does
Dr. Yazigi, the board and attorneys - that through diligent research they will find this to be a benefit to Georgetown and their patients as long as it is healthy
Qualification - that Levi qualifies to be a part of the trial
Time - time to do the clinical trial
Results - that Levi's body would respond well and that this will truely be a magic pill (blessed pill?)
Thanksgiving - pray prayers of thanksgiving to God who has provided a way when there seemed to be no way (isn't he sort of famous for that :))
Our fear - that God would take away all our fear while we watch Him do what only He can.
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