LLM 2021 | Treatment options for rare bleeding disorders
Suchitra Acharya, MD, MBBS, Northwell Health, New Hyde Park, New York, NY, provides an overview of the current treatment landscape for bleeding disorders. Single factor concentrates remain the current standard of care for various ultra-rare bleeding disorders with the exception of factor V and XI deficiencies. However, the role of factor concentrates as prophylaxis remain unclear, as use of prophylactic factor concentrate for factor XIII deficiency may result in intercranial bleeds, whereas prophylactic factor concentrate is beneficial for patients with fibrinogen deficiency. This interview took place at the Lymphoma, Leukemia & Myeloma Congress 2021.
Transcript (edited for clarity)
So the treatment of these ultra-rare disorders has actually come a very long way. Back in the 50s and 60s, we used plasma cryoprecipitate, but now I think we should be really proud of the fact that we have single factor concentrates, which are highly purified that are available for most of these ultra-rare disorders, except for factor V deficiency and factor XI deficiency in the United States. And so, I think it’s really an exciting time because we really have the availability of appropriate treatments...
So the treatment of these ultra-rare disorders has actually come a very long way. Back in the 50s and 60s, we used plasma cryoprecipitate, but now I think we should be really proud of the fact that we have single factor concentrates, which are highly purified that are available for most of these ultra-rare disorders, except for factor V deficiency and factor XI deficiency in the United States. And so, I think it’s really an exciting time because we really have the availability of appropriate treatments.
The difficulty is I think of providers availing themselves of the knowledge about these factor concentrates, especially since they’re so rare and making themselves familiar with these factor products and how to use them and in what context. The other issue is I think it’s not currently clear to us is the role of prophylaxis, that this preventative use of these factor concentrates in order to prevent bleeding.
I would say there’s at least a couple of disorders, for example, factor XIII deficiency, where I think we automatically do recommend prophylaxis because factor XIII deficiency, severe factor XIII deficiency actually can increase your risk for intracranial bleeds, which could be devastating in any individual. And so for XIII, I think that’s what we do. For severe, for example, fibrinogen deficiencies, especially in a pregnant woman, replacement factor concentrate is also recommended, but with the other factor deficiencies, I think the jury’s out there as to where you would really institute prophylaxis.
Now, for example, if an individual has had repeated joint bleeds, say, then it’s probably one of the strategies that you would use if you’ve had two or more joint bleeds. Again, extrapolating from the hemophilia world that you would start prophylaxis in these ultra-rare patients. But for others, I think we are still trying to figure out what the best way to implement these treatment strategies is.
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