PAP Treatment
Most of Friday was spent quietly in our room, wondering where the doctors were, but the answers came late in the afternoon. She had not forgotten about us but was actually very hard at work on our case. Our “new “ Lung Specialist is going on holidays (just our luck) and wanted to get everything together before she left, as well, she is the one who knows most about PAP here and so she felt it was easier to do all the paper work, etc herself then get someone else to do it. James’ medication is VERY expensive, as well it is still on trial, especially the way that they will be giving it to him. So first they have to receive approval from Health Canada to actually go forward with the treatment. Proof that it is needed and should be effective is required, lots of paperwork etc in order to get it approved and then she had to actually order the medication, which comes from Seattle. But she got it all done, approval was made, medication order and it arrived by the evening … amazing really!!! Approval also means that we’ve been approved for coverage and can take the medication (and equipment to run it) home if it shows to be effective after a week. Originally she had said that we would need to prove after one week that it was effective to get home coverage, but now she said that it’s all covered for us to take it home … if we do indeed see positive results How long have we worked to get oxygen approval and monitors (which we still haven’t gotten a fixed price on)? And in one day such a rare and expensive medication is approved.
The doctor sat down with me today to talk in more detail. The meeting brought reality and new light to James’ disorder. Much of the information we have read on PAP is with regards to auto-immune PAP and this is more treatable with better outcome then the PAP that James is thought to have …since most of our research was based on this as it is more common, it showed a pretty good prognosis for patients with PAP, with it not being uncommon to go into remission. So far records of the LPI related PAP show that it does not go into remission and treatment effectiveness is not well known yet. I was once asked if people die from LPI and I had responded that if diagnosed and treated it is manageable and that the only deaths I have read about related to LPI have all been because of PAP … at the time I had no idea that James had lung issues to this extent. They cannot give us any idea of the prognosis for James. We are a trial and only the Lord knows what direction things will go.
As for James condition she classified it as moderately severe. I think here we can see the difference of someone who is looking at the test results versus someone who looks at James’ clinically. This is the issue we ran into at Mac, we always heard “clinically he looks well” and they seemed to focus on this more then what the tests were actually showing, always expressing surprise at what they found when they did tests. We were also advised that not treating James is not an option (technically it’s always an option), she estimated he would have 1-3 years if we went this route. So although James is a trial and we do not know the long term effects of the medication we are using … we have no other option. The doctors may be in control of the medication we administer, but we know that there is no better medicine then prayer and faith and trust in our Lord who is ultimately in control.
Surfactant produced in the lungs is normally either recycled by lung cells or destroyed (catabolized) by alveolar macrophages in the lungs. GM-CSF (Granulocyte-Macrophage Colony-Stimulating Factor) is required to stimulate surfactant catabolism and other functions in these cells. In PAP, very high levels of antibodies against GM-CSF are present in the blood and lungs and completely neutralize the activity of GM-CSF. Without GM-CSF-stimulation, alveolar macrophages are unable to breakdown surfactant. Consequently, surfactant accumulates progressively leading to impaired oxygen absorption in the lungs and symptoms.
I talked about treatments before , but more specifically the Whole Lung Lavage as this is the most common treatment. We had read about the experimental GM-CSF treament but did not really think this was something that would be used ... I guess we should have realized in a hospital like Sick Kids that's where you would use these new treatments.
All sounds simple ... but this is a trial. They have never done this in an LPI case and have only used the inhaled method they are using on James once before in a child. We have no idea if the treatment will work. GM-CSF is used in other disorders as well as in cancer, but this is done by IV. The method we are using is new, making James an even bigger guinea pig. Trials have shown inhaling the medication is more effective as it gets to the source quicker.
Originally they said he would use a mask to inhale it, but when he actually had the treatment they used a mouth piece. He had to hold the mouth piece between his lips for about 15 minutes. The doctor explain that when using a mask they get about 5% of the GM-CSF because our nose acts as a filter, when breathing in it by the mouth he gets about 20% ... much more effective. When they came with the mouth peice I thought they were nuts to expect him to keep it in his mouth, but our little James often suprises us and he is much more cooperative with this then he ever would have been with a mask (he goes beserk when he sees masks ... especially since being on BiPaP). He did not like the idea at first and it took a minute or two to get him to cooperate, then he started gagging and vomitting, likely because he was so worked up. But after throwing up he settled down and sat good with it. He has had two treatments so far - Friday night and this morning. Again this morning he got a bit worked up at first, but settled down quicker and sat there nicely. The doctor came during his treatment and was impressed with how good he was.
What is GM-CSF doesn't work?? Then we move to the more common treatment of Whole Lung Lavage (WLL). This is where they literally wash out the lungs. When they do this they keep one lung going while they wash the other one out ... with up to 50L (compared to 100ml in a bronchoscopy) of saline water. They allow you to recuperate for several days and then do the other lung. When reading on the internet it all sounds so simple but we are told there are great risks to doing this treatment as you wash the good with the bad and put the lungs under great strain for the process. Even anesthetics are difficult and require a anesthesis who is specifically trained to use double tubes to keep the lungs going seperately. For a normal adult this is a critical procedure and for James ... well it's very critical. They have never done this on someone so young and his lungs are not large enough for the equipment to even go down as they normally would. All she said is that if it came to that they would find a way to do it ... but this would be no small procedure. There is also a possibility that even if the GM-CSF treatment is working they will still need to do the WLL in order to get out the build-up that he has accumulated in the past year.
The long term prognisis? Absolutely no idea. She explained that James is very sick and wants us to know ahead of time that there is just as much chance they cannot make him better as there is that they can make him better ... they really don't know what to tell us, but only that we can be thankful there are treatments available. She said if we had come to them about 6 years ago they probably wouldn't have known what to do with him ...we have options now and all we can do is pray that they will work.
A few other factors which remind us of our "wasted" time at Mac. The Lung Specialist says that James does not have aspiration pneumonia (which our old specialist insisted he did) and that the lipids they seen to give this diagnosis are PAP related. She explained that this is the difference on having a adult specialist (as ours was) and a child specialist. She said children who are mentally and physically fine, as James is, do not get aspiration pneumonia - she has never seen it - because they are able to swallow and function properly ... as we know James can ... and therefore know how to use their coughing reflux etc to control any reflux. James likely does have reflux but it is probably mild. This is not uncommon in children, especially those with lung problems. She explained that when the lungs are under stress they also put extra stress on the esophagis and food track therefore causing some reflux. So if we get the lungs relaxed and under control his reflux should also settle. But ... the reflux that he has is likely mild and not really a factor. So ... in other words ... all we went through to put a J-tube in was not necessary, and we are extremely thankful that they were not able to do the fundoplication surgery right away. There is no reason to remove the J-tube since it's in now and won't hurt, but should it come out we could go back to a g-tube and likely just using reflux medicine (Losec) will keep the amount of reflux he probably has under control. Those are things to worry about in the future, one thing at a time. First we try get the lungs under control then we worry about how extensive his reflux really is.
Although I say "wasted" time at Mac, it wasn't all wasted and I do realize they were doing the best they could. Our frustration remains mostly with the Lung Specialist who never showed up and never took the time to really listen and for some reason with all the proof in front of him refused to see that this was PAP and James needed further help. He had said we would take a wait and see approach ... we are now told that waiting could mean James' life. Our time was not completely wasted either as no further tests were needed to be done at Sick Kids, we could move straight to treatment. We still love Mac, we just don't care for the Lung Specialist (no matter how positive the nurses talk about him) ... we will gladly drive the distance to Toronto each time again to have a doctor who listens and talks to us at our level!!
As for James himself ... the last couple days he has not been happy. There has not been the shine in his eyes that there usually is. He badly wants to go home, more so then I've ever seen him ask at Mac. I have to remind myself that just because I think of it as just another hospital room, to him it is a new surround and usually it takes him a few days to adapt with each admission to Mac, so he needs to adapt here too. Today he started to perk up again and we were able to wean him down a bit more on his oxygen ... he is now okay at just under .5L and they were attempting .25L this afternoon.
It's been an emotional few days, especially Friday when we realized that just because we got the diagnosis didn't mean we were on the road to recovery ...our reading of PAP did not prepare us for this prognosis. We pray that recovery is the case, but know that ultimately the Lord will decide ... and so we pray for strength to accept whatever is put before us in the days, weeks, months or years ahead. I look at James in a new manner now ... our happy, cheerful, energetic little guy who is such a little charmer ... oh how much he has gone through and still has to go through ... and all the what-if's that automatically come as a parent. I hope that he continues to improve the next couple days so that I too may feel more relax and happy ... happiness is contagious, James has a way of bringing that out in people.
"Do not be anxious about anything, but in everything, by prayer and petition, with thanksgiving, present your requests to God. And the peace of God, which transcends all understanding, will guard your hearts and your minds in Christ Jesus." Phillipians 4:6,7
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