Wild-type (senile) ATTR amyloidosis medical therapy

Wild-type (senile) ATTR amyloidosis Microchapters
Home
Patient Information
Overview
Historical Perspective
Classification
Pathophysiology
Causes
Differentiating Wild-type (senile) ATTR amyloidosis from other Diseases
Epidemiology and Demographics
Risk Factors
Screening
Natural History, Complications and Prognosis
Diagnosis
Diagnostic Study of Choice
History and Symptoms
Physical Examination
Laboratory Findings
Electrocardiogram
X-ray
Echocardiography and Ultrasound
CT scan
MRI
Other Imaging Findings
Other Diagnostic Studies
Treatment
Medical Therapy
Surgery
Primary Prevention
Secondary Prevention
Cost-Effectiveness of Therapy
Future or Investigational Therapies
Case Studies
Case #1
Wild-type (senile) ATTR amyloidosis medical therapy On the Web
Most recent articles
Most cited articles
Review articles
CME Programs
Powerpoint slides
Images
American Roentgen Ray Society Images of Wild-type (senile) ATTR amyloidosis medical therapy All Images X-rays Echo & Ultrasound CT Images MRI
Ongoing Trials at Clinical Trials.gov
US National Guidelines Clearinghouse
NICE Guidance
FDA on Wild-type (senile) ATTR amyloidosis medical therapy
CDC on Wild-type (senile) ATTR amyloidosis medical therapy
Wild-type (senile) ATTR amyloidosis medical therapy in the news
Blogs on Wild-type (senile) ATTR amyloidosis medical therapy
Directions to Hospitals Treating Psoriasis
Risk calculators and risk factors for Wild-type (senile) ATTR amyloidosis medical therapy

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	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
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.