Wild-type (senile) ATTR amyloidosis medical therapy: Difference between revisions
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==Overview== | ==Overview== | ||
Pharmacologic medical therapies for wild-type ATTR amyloidosis related polyneuropathy include [[patisiran]] and indotersen. Several other medications are under investigation, but liver transplantation remains the gold standard for therapy. | |||
==Medical Therapy== | ==Medical Therapy== | ||
Pharmacologic medical therapies for wild-type ATTR amyloidosis related polyneuropathy include [[patisiran]] and indotersen. Several other medications are under investigation, but liver transplantation remains the gold standard for therapy. | |||
{| class="wikitable" | |||
|+ | |||
! | |||
!MOA | |||
!Dosaeg | |||
|- | |||
|[[Patisiran]] | |||
| | |||
* RNA interference, a cellular process in which small interfering RNAs (siRNAs) control gene expression by mediating the cleavage of specific messenger RNAs (mRNAs). | |||
* Patisiran comprises siRNAs that are specific for TTR mRNA, formulated in lipid nanoparticles. | |||
| | |||
* Administration is via intravenous infusion every 3 weeks. | |||
|- | |||
|Inotersen | |||
| | |||
* Antisense oligonucleotide that causes degradation of mutant and wild-type transthyretin mRNA by binding TTR mRNA. | |||
* Results in reduced TTR protein levels in serum and tissue. | |||
| | |||
* Administer once-weekly subcutaneous injection | |||
|- | |||
| colspan="3" |Drugs under clinical trials | |||
|- | |||
|[[Tafamidis]] | |||
| | |||
* Binds to TTR with negative cooperativity and kinetically stabilizes wild-type native TTR and mutant TTR. | |||
* Halts the amyloidogenic cascade. | |||
| | |||
|- | |||
|[[Tolcapone]] | |||
| | |||
| | |||
|} | |||
=== Treatment of cardiac involvement === | |||
* Prophylactic placement of an implantable cardioverter defibrillator (ICD) is recommended to prevent sudden cardiac death. | |||
* Use of commonly employed drugs such as diuretics, beta-blockers, angiotensin-converting enzyme (ACE) inhibitors, and angiotensin receptor blockers (ARBs) in amyloidosis is complicated. | |||
* Due to the restrictive effect of the disease, ventricular compliance is poor and end-diastolic volumes are low. | |||
* Patients often require a higher filling pressure to distend the stiffened heart, and diuretic therapy reduces preload, which can further reduce stroke volume and systolic blood press | |||
* Digoxin binds to amyloid fibrils and can lead to locally high levels; it also must be used with caution. | |||
==References== | ==References== |
Revision as of 15:17, 2 December 2019
Wild-type (senile) ATTR amyloidosis Microchapters |
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Editor-In-Chief: C. Michael Gibson, M.S., M.D. [1]
Overview
Pharmacologic medical therapies for wild-type ATTR amyloidosis related polyneuropathy include patisiran and indotersen. Several other medications are under investigation, but liver transplantation remains the gold standard for therapy.
Medical Therapy
Pharmacologic medical therapies for wild-type ATTR amyloidosis related polyneuropathy include patisiran and indotersen. Several other medications are under investigation, but liver transplantation remains the gold standard for therapy.
MOA | Dosaeg | |
---|---|---|
Patisiran |
|
|
Inotersen |
|
|
Drugs under clinical trials | ||
Tafamidis |
|
|
Tolcapone |
Treatment of cardiac involvement
- Prophylactic placement of an implantable cardioverter defibrillator (ICD) is recommended to prevent sudden cardiac death.
- Use of commonly employed drugs such as diuretics, beta-blockers, angiotensin-converting enzyme (ACE) inhibitors, and angiotensin receptor blockers (ARBs) in amyloidosis is complicated.
- Due to the restrictive effect of the disease, ventricular compliance is poor and end-diastolic volumes are low.
- Patients often require a higher filling pressure to distend the stiffened heart, and diuretic therapy reduces preload, which can further reduce stroke volume and systolic blood press
- Digoxin binds to amyloid fibrils and can lead to locally high levels; it also must be used with caution.