A mini review of Patisiran's efficacy in the management of transthyretin cardiac amyloidosis

被引：0

作者：

Gbolahan Olatunji ^{[1
]}

Emmanuel Kokori ^{[1
]}

Ikponmwosa Jude Ogieuhi ^{[2
]}

Chimezirim Ezeano ^{[3
]}

Oluwatobi Omoworare ^{[4
]}

Doyin Olatunji ^{[5
]}

Sai Gautham Kanagala ^{[6
]}

Ayilola Ayotomiwa Elisha ^{[7
]}

Deborah Aboyeji ^{[1
]}

Awoyinfa Michael Oluwatobiloba ^{[8
]}

Komolafe Babajide Ayodeji ^{[9
]}

Owusu Yaa Asieduwaa ^{[10
]}

Emmanuel Obokhai Uduigwome ^{[11
]}

Ismaila Ajayi Yusuf ^{[12
]}

Olawale Olanisa ^{[13
]}

Nicholas Aderinto ^{[14
]}

Aarushi Venkatraman ^{[15
]}

Yewande Abigail Adebayo ^{[16
]}

机构：

[1] University of Ilorin,Department of Medicine and Surgery

[2] Siberian State Medical University,Health Science Centre

[3] University of North Texas,Department of Health Sciences

[4] Lagos State University College of Medicine,Department of Internal Medicine

[5] Western Illinois University,College of Medicine

[6] Metropolitan Hospital Center,Department of Medicine and Surgery

[7] Evercare Hospital,Department of Medicine and Surgery

[8] University of Lagos,undefined

[9] Pearls Specialist Hospital,undefined

[10] Kwame Nkrumah University of Science and Technology,undefined

[11] Lagos University Teaching Hospital,undefined

[12] Obafemi Awolowo University Teaching Hospital,undefined

[13] Trinity Health Centre,undefined

[14] Ladoke Akintola University of Technology,undefined

[15] Madras Medical College,undefined

[16] Glangwili General Hospital,undefined

来源：

Discover Medicine | / 1卷 / 1期

关键词：

Transthyretin cardiac amyloidosis; Patisiran; RNA interference; Therapeutic intervention; Disease modification;

D O I：

10.1007/s44337-024-00066-y

中图分类号：

学科分类号：

摘要：

This mini-review provides a narrative analysis of the emerging therapeutic potential of Patisiran in managing Transthyretin Cardiac Amyloidosis (ATTR-CA). ATTR-CA, characterized by the deposition of misfolded transthyretin proteins in cardiac tissue, leads to progressive heart failure, significantly impacting affected individuals' quality of life and prognosis. Current treatment options for ATTR-CA are limited, necessitating the exploration of novel interventions like Patisiran. Patisiran, an RNA interference therapeutic, targets hepatic transthyretin protein production, thereby reducing amyloid deposits. While proven effective and safe in treating ATTR amyloidosis with polyneuropathy, its impact on cardiac manifestations is less studied. Positive outcomes include improved health status, enhanced quality of life, and preservation of functional capacity. Studies demonstrate sustained benefits, highlighting the potential for long-term positive effects. Reductions in cardiac amyloid burden and regression and prevention of deterioration in various cardiac parameters suggest a potential disease-modifying effect. Despite promising results, infusion-related reactions and adverse events necessitate careful consideration of long-term tolerability. Overall, Patisiran emerges as a promising intervention, offering hope for improved patient outcomes in the complex landscape of ATTR-CA management.

引用

共 91 条

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