Our daughter, Phoebe, 7 years old, was diagnosed with aHUS on Thanksgiving weekend 2012. On Thanksgiving, our very healthy daughter woke up vomiting. We assumed she had a stomach virus. By Sunday, we knew she wasn't o.k. She was pale and had slightly yellow eyes. She was extremely lethargic and was still vomiting.
Her original numbers from day 1 at the hospital were:
hgb = 5.5
platelets = 12
creatinine = 2.39
BUN = 154
LDH = 909
Our doctor informed us that it was aHUS and told us that there were options for treatment. They ran a bunch of blood tests. Her C3 was low, but her C4 was normal. They sent her blood to the lab in Cincinnati. There they found that her Factor H autoantibody was at 3,500. From what we understand, the level should have been between 0 and 22.
From this finding, our doctor informed us it is genetic. We sent Phoebe's blood to Iowa earlier this week for the genetic test. The doctor wanted to wait until her blood levels were better before sending out for the test.
Phoebe was released from the hospital a week after going in. Her body responded to the plasma infusions very well.
When she was released from the hospital, her blood levels were:
hgb = 7.5
plt = 149
creatinine = .99
BUN = 57
LDH = ??
In the hospital she received 1 platelet transfusion, about 4 blood transfusions, and 3 plasma transfusions.
They gave her two vaccination shots (pneumonia & meningococcal). We were told that Soliris might be a possibility.
Several days later, Phoebe's platelet and hgb levels started going backwards.
Our doctor put Phoebe on CellCept (mycophenolate) and prednisone (steroid). The doctor then had Phoebe go into the hospital for 2-3 plasma infusions a week.
We were told that this should be the first method of trying to get this episode of aHus into remission.
Little by little the numbers have gotten better.
Her numbers on 1/4/13:
hgb = 12.1
Platelets = 255
creatinine = .44
BUN = 17
LDH = 242
From what I understand, the plasma helps block the negative effects of the autoantibodies. Suppressing the immune system will theoretically shut her immune system down and have it stop creating these antibodies.
We were originally told that we would be able to pull Phoebe off the plasma and see what happens, but I don't know when that will happen.
Our doctor has always left Soliris as a last option. Our doctor made it clear that she was discussing Phoebe's case with many different doctors around the world. We have since talked to one of those doctors in Cincinnati. This doctor made a lot clear and we felt that the way things were going were good. We have been frustrated with the time the infusions take since the hospital doesn't have an infusion room. Phoebe has to be admitted and the whole ordeal takes 8-12 hours. We feel terrible about having a seemingly healthy girl spend that much time in a hospital bed. But we felt the plan was good, even though my wife and I are somewhat uncertain about the end game.
We initiated a second opinion in early December from a hospital in Chicago that we are sure has had cases of aHus. We weren't able to meet with this doctor until 1/3/13. By this date, Phoebe's numbers were very good. This doctor said that we should stop the CellCept, taper the steroid, and go straight to Soliris. He said Phoebe would get 1 infusion a week for 4 weeks and then 1 every other week. He said that the plasma should stop because we are allowing Phoebe to develop antibodies against a blood product, which could complicate a future kidney transplant if one is needed, although he stressed that is quite unlikely. He said that he would keep Phoebe on Soliris for 3 - 6 months. Then we would take her off it and see if her factor H antibody levels stay in the right range. We understand that we have a lifetime of monitoring and that she could always have a relapse.
This sounded like a good method of attack. But we talked to a third doctor who didn't completely disagree with the idea of Soliris, but said that he would definitely keep Phoebe on the immune suppression because we need to make sure we stop her body's ability to produce the antibodies. Also, he said that the plasma seems to work for now and doesn't see any pressing reason to jump to Soliris at this time.
Now we are a little confused because her numbers are so good, it doesn't seem right to stop the current treatment, and we have been given hope that we will stop the plasma very soon. We are nervous about starting another round of infusions if they aren't necessary. However, the doctor who recommends stopping treatment makes a lot of sense. But so do the other doctors.
I know many people who belong to the foundation have far more experience with this than we do. We also know that each case has variations. But we are curious to hear from anyone willing to share with us thoughts on these three ways to treat:
1. Plasma (once a week to once every other week) + immune suppressant (cellcept and prednisone). We have gotten great results so far, but in the midst of the worse whooping cough and flu season the country has seen in decade(s), we are terrified about her immune system being shut down. We seem to be tapering off the plasma, but the infusions seem to take forever.
2. Soliris + immune suppressant (CellCept and Prednisone) This doctor's argument is that if we don't suppress the immune system, then when we take her off Soliris, we run the risk of the antibodies being created again and having a relapse.
3. Soliris (We are told the Soliris infusion is 30 minutes and the whole experience can be done in about 2 hours because it would be done in an infusion outpatient. We are also told that the Soliris won't shut her immune system down like the cellcept so she will be less at risk of getting sick. Plus this doctor says this the more sure way to treat the illness).
We will continue to post more information as we progress. Any info you want to share with us is great. Otherwise, thank you for having a foundation like this. We have learned a lot and with an illness that makes one feel very alone, it is nice to have a community.