What is the role for FFP in the treatment of HAE? Do patients have a role in participating in their choice of therapy?

Jul 20, 2015

“Given the current on demand treatment options in the United States, what is the role for FFP?  Do patients have a role in participating in their choice of therapy?”

Dr. C:I would like to thank Janet Long for rescuing the stack of question cards from a previous patient summit–one of which raised this topic. As many of you recall, prior to 2008 in the United States we really had no good options for on demand treatment of attacks. It was mainly pain relief and fluids for an abdominal attack and careful monitoring and potential intubation for a laryngeal attack. Many of you had given up asking for help for swelling of the hands, feet or other areas as you knew that nothing would be done. FFP was the only option that was on the table. While effective in some cases to arrest an attack of swelling it carried the potential of actually accelerating an attack. This is explained by the fact that while FFP has the C1 inhibitor it also has the ‘fuel’, HMWK, prekallikrein and factor XII, which are used to generate bradykinin when the contact system is activated during an attack of swelling. There is also the issue that FFP is a blood-derived product. Given the current landscape of potential alternatives I believe that we should avoid FFP and advocate for one of the new effective on demand treatments such as Kalbitor, Firazyr, Berinert or Ruconest. Our HAEA Emergency 24 hour assistance line 866-841-4232 is available to assist and advocate for patients to receive optimum care. Bruce or Marc, any opinions about use of FFP?


Dr. Z:I agree with your points. In countries that don’t have access to modern on-demand therapies or during an emergency when these on-demand therapies are not available, I think that FFP still has a role in the management of HAE. Solvent detergent treated plasma (SDP) undergoes additional viral inactivation steps and so is marginally safer than FFP.   In situations where FFP or SDP is used, the physician needs to be aware that they may occasionally need to emergently treat a worsened attack. In particular, this means that the ability to quickly intubate a patient should be available when FFP or SDP is used.


Dr. C:Thank you, Bruce. What about the issue of patient participation in their management? With the help of the Medical Advisory Board there was a consensus document published in 2013. The following is an excerpt from the document emphasizing the US approach as a physician patient partnership “These expert physicians should work with their patients to assure that a defined and individualized HAE management plan is established and should also actively coordinate the patient’s care with other health care providers.”


Dr. Z: Including the individual preferences of a patient in medical decisions affecting him/her is a fundamental tenet of modern medical care. Enough said.


Dr. C: Thank you, as always we hope that this brings a measure of clarity on this topic for our readers. We look forward to your input, and will be back next ‘question of the week’.


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