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Beyond Hermansky-Pudlak Syndrome

In my fantasy life, I secretly hope that one day some curious or enterprising young doctor or researcher develops an interest in Hermansky-Pudlak Syndrome, and then says to themselves, “Gee, I wonder what’s it’s like to live with HPS?”

In this little fantasy, this doctor or researcher is enterprising enough to hop onto some blog search engine, like Technorati or Google blog search, and stumble upon either my blog, or that of one of the other HPS’ers. Far fetched, I know.

Yet we in the HPS community strive to put a face to our medical maladies. So often the bench researchers doing the truly life saving and ground breaking work never get to meet someone with HPS (except for the mice.) We hope that by knowing us, they better understand just how important and valuable their work is to us.

Donna Appell, President of the HPS Network, and her family even plan family vacations around which labs they’d like to visit – just so the researchers can meet Ashley, her daughter.

So, it’s in that vein, and in the spirit of the fantasy, that I’d like to mention one other group of HPS’ers – the mystery group.

Scientists have to come up with definitions for things. It’s just the nature of the beast. So, they’ve created a list of common attributes those of us with HPS share and doctors use this list to make a diagnosis. People with HPS, by definition, have a lack of dense bodies, tiny sacs of chemicals on the edges of blood platelet cells. Those chemicals are evidently responsible for a lot – including part of the blood clotting process. People with HPS also have albinism, and may develop other symptoms such as colitis or pulmonary fibrosis.

But, what about the increasing number of people that don’t fit so neatly into that definition?

If there is some young, enterprising researcher type lurking out there in cyberland, you might be interested to know there are an increasing number of people who test negative for HPS under the current definition – yet they share many of the same symptoms. There’s clearly something else going on out there. Soon science is either going to have to modify the definition of HPS, or name a separate, yet likely related, syndrome.

Some of these “mystery” types have albinism, and another HPS symptom like bleeding, or colitis, or pulmonary fibrosis – but they’ve got dense bodies on their platelets. Some have two or more of these symptoms, but have fewer than the normal number of dense bodies on their platelets. A handful don’t have albinism, but lack dense bodies altogether – just like the HPS’ers.

I sometimes worry that we’re losing some of these “mystery HPS’ers” because they receive a negative diagnosis and assume their problems therefore couldn’t be HPS related. Mind you, people with albinism don’t have some immunity to the many other maladies of life. It’s certainly possible, even probable, that many of these folks simply have albinism and their other issues really are a completely separate disease process. But, still, I hope that those testing for HPS who do have symptoms stay in touch. I suspect the research still has many secrets to uncover.

We have a number of these “mystery HPS’ers” involved with the HPS Network. They find us the closest fit to what they’re experiencing and seem to appreciate the support. I have a sneaking suspicion that one day one of these “mystery” cases is going to provide a clue that could help us begin to put the complete puzzle together.

Comments

Care said…
As the Mom of one of these Mystery kids - I appreciate so much the support I've found in the HPS Network. I have no idea what Ethan's diagnosis will end up being, but regardless I've gotten so much valuable information and support. I hope our trip to NIH in August brings answers (more likely more questions) and I hope more and more of those "mystery HPS-ers" find their way to the network, and to the researchers.
Whitey said…
I have a question, Are you wanting a cure?
Oh yes, most certianly. And, I think it will happen one day. Perhaps it won't happen for me, but the progress that has been made in the past four years is really exciting.
Anonymous said…
Hey There

This is Sheena, one of the "mysteries" I guess you could say. I"ve been living with this "condition" my entire life and I am 32 now. I have learned to deal with each aspect serarately but would one day love to know what the overall picture makes up. I am optimistic if we all work together and stick together that one day they'll find a cute or at the very least find out what it is we have. Until then I am honored to be a part of such a wonderful group.

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