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With many new medications available and on the horizon to treat #sicklecelldisease, there is still decreased awareness and confusion as to where they may fit in.
Here’s a brief thread summarizing these medications and treatments. #sicklecellawareness #sicklecellmatters
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Here’s a brief thread summarizing these medications and treatments. #sicklecellawareness #sicklecellmatters
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I talk a lot about #hydroxyurea but can't leave it out due to the long (30+years) track record showing it works.
HU should be the foundation for all HbSS and some HbSC patients and other meds should be added to and not in place of it.
Primary mechanism is to increase HbF
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HU should be the foundation for all HbSS and some HbSC patients and other meds should be added to and not in place of it.
Primary mechanism is to increase HbF
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L-glutamine (Endari) is an oral powder taken 2x/day.
The proposed mechanism, though not clear, is that it reduces oxidant stress.
Published data showed slight reduction in pain compared to placebo (median 2.0 vs. 3.0).
Could be considered for patients with significant pain.
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The proposed mechanism, though not clear, is that it reduces oxidant stress.
Published data showed slight reduction in pain compared to placebo (median 2.0 vs. 3.0).
Could be considered for patients with significant pain.
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Voxelotor (Oxbryta) is an oral medication (3 tabs) taken 1x/ day that increases Hb binding to oxygen and thus stabilizes Hb and reduces sickling.
HOPE showed increased Hb but no data yet to clearly show clinical benefits.Could be considered for pts with low Hb despite HU use.
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HOPE showed increased Hb but no data yet to clearly show clinical benefits.Could be considered for pts with low Hb despite HU use.
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Crizanlizumab (Adakveo) is an IV medication (monthly) that binds to and inhibits P-selectin, a molecule that is important in causing vasoocclusion and pain.
SUSTAIN study showed reduction (but not prevention) in pain.
Consider for patients with frequent painful crises.
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SUSTAIN study showed reduction (but not prevention) in pain.
Consider for patients with frequent painful crises.
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#GeneTherapy is exciting and getting closer to becoming a reality.There are several methods that are being developed to #CureSCD.
To date, # of patients treated is small and follow-up is short, but we're getting closer. More data needed to show benefits and confirm safety.
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To date, # of patients treated is small and follow-up is short, but we're getting closer. More data needed to show benefits and confirm safety.
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There are many other drugs in development to reduce sickling, improve Hb, increase HbF, among other mechanisms.
These include PKR activators, PDE9 inhibitors, sGC stimulators, and BCL11A inhibition.
This is an exciting time and I hope we can continue to improve outcomes.
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These include PKR activators, PDE9 inhibitors, sGC stimulators, and BCL11A inhibition.
This is an exciting time and I hope we can continue to improve outcomes.
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