Given the multiple treatments for acute attacks of HAE do we need to use more than one of the medications? What happens if I have recurrent, continued swelling after a treatment?

Jul 27, 2015

Dr C:Excellent question—it is indeed fortunate to have an array of choices for ‘on demand’ treatment of acute attacks. We now have four approved effective products: 1) Kalbitor an inhibitor of the enzyme plasma kallikrein which is responsible for generation of bradykinin which drives the vascular leak and swelling, 2) Firazyr which blocks the receptor or ‘docking site’ for bradykinin on the vascular epithelium, 3) Berinert which is a plasma derived C1 inhibitor which can arrest the swelling by inhibiting activation of the contact system and 4) Ruconest–also a C1 inhibitor which is a recombinant product. As our readers are aware the first two products are administered subcutaneously and the second two require intravenous access. The importance of having an effective product available and a treatment plan cannot be over emphasized—nor can the mantra “treat early”. Marc, do you have any thoughts for our readers regarding the need for multiple medications or the issue of re-treatment during a prolonged or recurrent attack?

Dr R: The treatment of angioedema attacks in HAE requires a highly individualized plan.  So there’s no single recipe that is going to work best for every person.  Everyone with HAE should have an acute medication available – talking through the treatment options with your personal physician is the first important step.  We know from the several large clinical studies that have been done over the years that a single dose of any of the FDA-approved acute medications is generally sufficient to stop the attack in the vast majority of cases (~90%).  In rare cases, a repeat dose of medication may be needed, but this is where it’s important to carefully consider the surrounding treatment issues.  Is there another medication that will work more effectively for a specific person?  Was the initial dose given properly and early in the attack so as to expect optimal efficacy? Was sufficient time allowed to judge drug efficacy before giving a second dose?  There are rare instances where acute medications may be combined to treat severe attacks, though in my experience this usually isn’t required if medication is given early and appropriately.  But “troubleshooting” the acute treatment plan with your personal physician is of critical importance if symptoms aren’t relieved as effectively as expected.

Dr. C: Thank you Marc. I completely agree with the importance of partnering with your physician to find the plan that works best for your individual circumstance and experience. Such factors as the route of drug delivery, ability to learn self treatment or preference for a visiting nurse (Kalbitor) may influence selection.   The emerging area of pharmacogenomics is an interesting topic—in the future it may serve to ‘marry’ the right drug to the right person. Bruce, what are your thoughts here?

Dr. Z: I couldn’t agree more with you. It’s not only an interesting area, but it’s a direction that we will certainly be paying a lot of attention to in the future. Right now, we pick the best on-demand medicine based on vague preferences and intuition. Luckily, most of the medicines work most of the time in most of the patients. But wouldn’t it be so much better to be able to say, “we know that this particular medicine is the best one for you”? The attempt to do this has been given a specific name – “precision medicine”. The information that we’ll need to make these precise recommendations will come not only from genomics, but also from many other types of information such as epigenomics, metabolomics, etc. that we hope to gather in the future. Once we can do this, we’ll expect the on-demand medicine to work with the first dose, virtually all of the time, in all of our patients. Sounds better, doesn’t it?

Dr. C:  Indeed it does, thank you both for your input. Again it is wonderful that we have these treatment choices to discuss and more on the horizon. We look forward to your input, and will be back next ‘question of the week’.

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