I’m back from a fantastic time at the Florida Hemophilia Association’s annual family meeting. About 330 people attended, and we presented Pulse on the Road, which I’ll write about in a few days, once I have the photos back! There was a motivational speaker, our insurance symposium, a men’s rap session, a woman’s session, and a thorough presentation by Dr. Glenn Pierce of Biogen Idec on long acting factor and gene therapy, and an

energetic kids program.

 This morning I attended a very detailed and comprehensive review of prophylaxis. You know, many families do prophy, and I have read about it, and researched it, and written about it for years. But today reminded me, you just can’t get enough education on the subject, and we all need to be reminded of the basics. Dr. Joanna Davis, Medical
Director, Div. of Pediatric Hematology/Oncology, University of Miami Hemophilia Treatment Center, and local “celebrity” as she was introduced, gave a five star review of hemophilia basics and how to do prophy the right way.

I liked how she described prophy as an attempt to mimic “nature” by boosting factor levels up in the normal range by planned and frequent infusions, whether a child needs them or not. She reviewed the difference between different prophy regimens:

Primary prophylaxis: begins with the first bleed, and is often an indefinite course of therapy. It prevents joint damage.

Secondary prophylaxis: aimed at treating a target joint or providing coverage during a high-risk situation such as team sports, or after a surgical procedure. It stabilizes or improves joint function.

Then she covered some basic definitions that we all need to know:

Peak level: the level of factor activity reached immediately after an infusion.

Trough: the lowest level of factor activity left over before infusing again.

Half-life: the time it takes for the factor levels to drop to half of what was infused. For FVIII: about 8-12 hours, for FIX, about 24-48 hours.

She explained in practical terms what this meant for prophy: let’s say you infuse on a Monday and you peak at 100%, for example. By Monday evening, a factor VIII patient may be then at 50% factor activity (which means still in the normal range). By Tuesday, the factor activity drops to 25%, and by Tuesday evening, maybe down to 12%. Just before infusing on Wednesday morning, the patient’s levels may be down to 2%–the trough. So prophy is scheduled according to a series of the peaks and troughs of factor activity.

Dr. Davis reminded the audience that on Friday, you may do your third prophy shot of the week. By Monday you’ll be depleted. She reported that
some families do a double dose of factor on Friday, to boost levels high, so the half-life does not bring the factor activity down to 2% by Monday morning.

Some of the barriers to prophy? Venous access—many children’s veins can’t take all the infusions and needle sticks, so a central venous access
device might be in order. This presents its own risks, such as blood clots. And sometimes, children may balk at such frequent infusions, and teens can be in denial and refuse to do their infusions (oh yeah, I can vouch for that one!). And the big issue is cost. Prophy ain’t cheap.

The best time for prophy is the morning, even though our ridiculously over-scheduled lives often make us want to wait till evening. Think about it: infuse at night, when things are quiet, and by morning you’ll only have half the factor working! Best to infuse in the morning, when your child is most active and when the factor is working strong, at 100%.

This was a great overview of prophy and the audience responded
with excellent questions.

After Dr. Davis’s talk, Alan Shier, of ATHN, spoke about the importance
of keeping logs about bleeds and presented the ATHNAdvoy, and wow! What a great
presentation too and what a great device. This is a web-based tool that will
help you track your bleeds and product usage, is accessible by your treatment
center, and can produce reports in a heartbeat. This is also available as a
mobile phone app, so you can use your Smartphone to quickly scan in your product,
report your bleed, and then you will always have a constant record, which you
can even print out in colorful reports. THIS COULD COME IN HANDY IN THE NEW
INSURANCE REFORM CLIMATE! Have I said that clearly enough? We should all be
recording every vial of factor we use. I would highly recommend using the
ATHNAdvoy.

About 11,032 hemophilia patients have already registered for this, which is astounding. All the data is kept in aggregate and no personal information,
including your name is kept. A bit hard to explain all this in a blog; I recommend
visiting www.ATHN.org to lean more!

Thanks to Debbi Adamkin and team for a fabulous time and meeting! (Debbi, aka Wonder Woman, I owe you a dance off)

Great Book I am Reading

In Cold Blood by Truman Capote

After watching the movie “Capote,” starring Philip Seymour Hoffman (fantastic), I felt compelled to read this, especially as the movie ended with the fateful words that Capote never wrote another book, and died of alcoholism. If the story of the writing of the book is compelling, the book is sheer brilliance. Capote does more in this book than document the shocking 1959 murders of Herbert Clutter, a wealthy farmer from Holcomb, Kansas, his wife, and two teens. Capote interviewed everyone and anyone connected to the case and living in Holcomb, including the murderers themselves, taking six years, and created a whole new genre in literature—the non-fiction novel. It certainly became the first true crime novel and was a raging success. Skillfully, almost lyrically written, well paced although it seems to weave its way through the minds of the killers at leisure, this is a masterpiece and not to be missed.