A new hemophilia treatment

When the FDA approved a new treatment for some hemophilia patients, including those (like me) with Factor XI deficiency, a few people asked what that would mean for me. I emailed Carolyn Francis, the hemophilia coordinator at Georgetown University Hospital, to find out, and her response was – “it’s complicated.” Here’s the story, as sI understand it.

The new treatment, called Octaplas®, is administered in much the same way as the fresh frozen plasma (FFP) I have received in the past. (See below for the back-story, if you don’t already know it.) Octaplas® is made of pooled human plasma that’s treated with solvents and detergents to scrub out viruses, including hepatitis A, herpes simplex, and parvovirus. In its current iteration, it has been used in Europe since 2006. FDA’s action last week means it will be marketed in the United States.

Along with scrubbing and cleansing, the manufacturer says the fact that its product is made of pooled plasma has an additional benefit: the potential to dilute antibodies in a donor’s plasma that, if present in large numbers, can cause allergic reactions, including swelling, hives, and anaphalaxis. The last time I had a plasma infusion, I had a severe reaction that had to be treated with steroids, so I would welcome this relief. With a pooled product, perhaps whatever made me swell up would have been diluted so that at least the reaction would not have been so sever.

But let’s not all jump for joy at the FDA’s approval of Octaplas®. The agency’s action was only one hurdle, Carolyn told me. The head of the hospital’s blood bank says the facility has not decided whether to bring the product into its supply. First, it would have to be approved by a committee of people who will consider cost, benefit, and risk before deciding. Even before that, it likely would have to be approved by Medstar, Georgetown’s parent company.

Cost is critical because the substance reportedly is expensive. The Georgetown committee probably will wait and see whether Medicare approves its use before going on to consider whether its benefits outweigh the risks of using it. If not, they are not likely to approve it either.

Even if they decide to include Octaplas® in their blood supply, my case might not warrant its use. The first group most likely to use it would be those with thrombotic thrombocytopenic purpura (TTP), a rare blood disorder that suppresses an enzyme and thereby causes clots to form in small blood vessels. TTP is generally treated by plasmapheresis, a process in which the plasma is removed from the patient’s blood and replaced with FFP. These plasma exchanges sometimes need to be repeated daily for one to eight weeks, increasing the patient’s chance of having a transfusion reaction.

People who have had reactions to past infusions might (or might not) be treated with Octaplas®, and cost might not be the only consideration. One severe side effect of the substance found during clinical trials was anaphylactic shock, which also is a complication seen in FFP treatment. If we weren’t eliminating that risk, what would make this new treatment worthwhile?

I’m only writing this to show what makes this decision so complicated. I’m not the one who will decide – that task will be left up to my hematologist and his blood supplier at Georgetown. Let’s hope we don’t have reason to find out anytime soon!

My back-story

For those who don’t know, here’s the background: I am Factor XI-deficient big-time. In past tests my Factor XI level has been measured at less than 0.5% of normal. This genetic disorder is autosomal dominant, meaning it’s carried on the X chromosome and can affect both males and females. My daughters have been tested and found to have between 50% and 60% of normal levels, which is generally thought to be “enough to get by” without pretreatment. My case is more severe (as we saw when Allison was born and I bled a lot) and I am pre-treated before surgical procedures and invasive tests.

My bleeding condition was know when I was young (I bled a lot after a tonsillectomy) but not diagnosed until the 1970s, when I happened into the care of Alan Klatsky in Hartford. Before I got pregnant he had me tested for clotting factors after hearing previous physicians’ suspicions that I had Christmas disease (Factor IX deficiency).

For a long time now I’ve been treated by Craig Kessler, head of Georgetown’s Coagulation Laboratory. I always appreciate the time he and Carolyn are willing to take to help me learn.