Shellye Horowitz
Forum Replies Created
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There are a few drugs in trials for Hemophilia A – they have had some really great success with Hemophilia B – the drugs are harder to develop for Hem A.
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As far as I understand, the mechanism for Hemlibra is different and it does not work on demand, but must build up in your system. People who are on Hemlibra still need to take factor if they get a bleed – but many bleeds are prevented by taking it.
Hemlibra is not the actual factor, but similar enough that it is able to help bind FIX to FX to continue the clotting cascade (as I understand it, but someone else may be able to explain it better). When having an actual bleed, factor is more effective to stop the bleed – Hemlibra is great to prevent bleeds – it is for prophylaxis.
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Thank you for sharing the link to your book. What aspects of the hemophilia struggle are shared in your book?
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You wrote that you think it is good to not be able to trace hemophilia and I would love to hear more from you about that. What do you think are the benefits of not being able to trace it?
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I totally get putting off treating when you are too busy, but do take care of you!!! I love that your 7 year old lets you have it if you do not treat like you should!
This past week was crazy at work and on Thursday morning my target joints were hurting for the first time in over a month…. I couldn’t figure out why and then I realized I had totally missed a prophylaxis dose. It DOES make a difference for sure and my joints were mad at me!
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I feel that I am more tired after I take factor. When on prophy I feel like I have mild fatigue at all times, but I am also so happy to have less pain and not be bleeding, so it is a tradeoff for me for sure.
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That is not the way it was developed – it was meant to be a subcutaneous injection. I think the point was to create something that did not require IV administration, as that is easier for most people!
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It is interesting. I hope one day they will find a cure through gene therapy.
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Hemlibra works differently than factor so it is a bit hard to compare. It is not meant to treat bleeds in real time, but to prevent them. It apparently keeps a person at the equivalent of a FVIII level @ 30% (look it up, don’t quote me) so that it is good to prevent spontaneous and mild bleeds.
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That must be hard – I cannot imagine having prophylactic care and then losing it. I am very active, so without it I get bleeds a lot… or would have to sit at home and do nothing. You are correct, in some countries it must be so difficult – some people cannot get factor at all.
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Very true – so many “victims” of hemophilia – those who got HIV and Hepatitis, like my dad; women who they thought could not have it, and many in countries that lack access to diagnosis and treatment. It is not an easy disease for sure. Do you have access to prophylactic treatment for bleed prevention where you live?
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It is my understanding that there are clinical trials right now for Hemlibra in those with mild hemophilia – again, taken regularly to prevent a bleed.
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I am sure that was hard too – it is not easy to miss school due to injuries/ bleeds.
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Oh my – yes, when one needs factor, immobilization is not that helpful – better to have factor. I had an experience once where I had an ankle injury for a year and they had me in casts too – it only healed when 9 months after injury I finally got factor…
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That must have been so hard – to not have access to treatment that you needed.
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That is great – there are so many things that people with hemophilia struggle with. What have been some of your biggest challenges? For me, it was getting a diagnosis and treatment.