Monday, March 20, 2017

Week 187: Live long and prosper

Had you asked me 3 months ago whether we would make it from one regularly scheduled clinic visit to the next without a sick visit or a hospitalization in between (given that the months in question were January, February, and March), I would have laughed at you. But, thanks to the power of Cayston, we did it. Our appointment went quite well, another minor miracle. Somehow, without really telling me about it, Lemon managed to pack on a pound and a half between when I weighed him at home last and when we got to the clinic. So, although if you average out from December until now, he isn't quite meeting his weight gain goal, he isn't doing half badly, either. We talked with our dietitian, and decided not to go up on the tube feed amount for now--we'd like to keep Lemon a little hungry, just on the off-chance that he decides to eat any solid food ever. On that note, I've got him scheduled for an evaluation at a feeding clinic later this week, so more on that soon I imagine.

After the clinic visit part of the appointment was over, we stayed on to help out with a research study. I would say participate, but it wasn't exactly that. As many of you know, a key measure of disease severity in CF is pulmonary function, which can be measured by two tests. Our center is going to participate in a new study that requires the use of these tests in kids aged 3-6, so they need their operators to prove that they can get accurate results in kids that young. When we got called to participate, I was a little skeptical, since Lemon is not exactly at the most cooperative stage of life, but I'm vehemently pro-research so I figured we should try to do whatever we could to help.

So, after the clinic appointment, we went to the pulmonary function lab to see what Lemon would do. He actually did much better than I expected at the first test, aided by a computer screen that displayed a birthday cake with candles that went out when he blew in the tube correctly. I think we got at least one trial on that was good enough to help certify our clinic staff. The other test was not as successful--that one required having a mask form an air-tight seal on his face for several consecutive minutes. He wasn't afraid of having a mask over his face, but he had to sit really, really still in order for the mask to maintain its seal. And sitting really, really still is something he is really, really not good at right now. So, for that test, we weren't able to get a run that was good enough to count towards certifying the operator. Honestly, watching both tests made me wonder how accurate they can be in kids as young as Lemon--it seems like we really need some other ways to measure lung function in kids this age.

In other news (literally--this story has been picked up a lot of places including the New York Times and the Washington Post), a study came out last week that found that patients with CF in Canada have a median survival that is 10 years longer than in the US. Both countries had similar survival up until around 2005, when the length of survival in Canada started to pull away from the US. There are a number of factors that may have contributed to this difference. The Canadians figured out the importance of a high-fat, high-calorie diet for CF patients earlier on (in the 1970's) and it took some time for that finding to be broadly applied throughout the US. So, adults with CF in Canada may be reflecting a benefit of that early childhood nutrition that the US patients missed. A higher percentage of Canadian CF patients get life-extending lung transplants than is seen in the US (although even in Canada, the percentage of CF patients who get a lung transplant is pretty low, just over 10%). So, what is behind this big difference? A key clue is that patients in the US who have private health insurance live just as long as Canadian ones (who all are covered under Canada's single-payer healthcare system). In other words, access to healthcare matters tremendously, and the importance of healthcare access is made starkly and unequivocally clear by a challenging medical condition like CF.

This study makes another important point, though, which is that everyone benefits from the study of CF and other rare diseases. Rare diseases like CF are the ones that have detailed registries to enable research like the study we just talked about--research that drew on almost 3 decades of meticulous record-keeping and follow-up. Rare diseases are at the cutting edge of personalized medicine and gene therapy, leading the way for strategies to tackle more common ailments like cancer and diabetes. Rare diseases are at the forefront of health economics, testing how the tremendous cost of these personalized medications can be borne in the face of finite resources.

Of course, to me, research on rare diseases is more than a societal good, and more than a scientific interest. To me, it is a very personal passion. The progress that has extend the lifespan of CF patients from 5 years in 1950 to 40.6 years (in the US) or 50.9 years (in Canada) today is astonishing, but obviously there is more work to be done. And the registries, the research on genetics, the development and testing of drugs, the design of gene therapies? That stuff is expensive. In this day and age, when federal support for medical research may be wavering, research supported by private foundations like the Cystic Fibrosis Foundation is more important than ever. So, please click here and donating to our Great Strides team to sustain these life-saving efforts. It's incredibly important to our family, and--in ways that we can't necessarily anticipate yet--it will be important to yours.

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