دورية أكاديمية
Cardiac Amyloidosis Treatment.
| العنوان: | Cardiac Amyloidosis Treatment. |
|---|---|
| المؤلفون: | Stern LK; Smidt Heart Institute, Cedars-Sinai, Los Angeles, California, US., Patel J; Smidt Heart Institute, Cedars-Sinai, Los Angeles, California, US. |
| المصدر: | Methodist DeBakey cardiovascular journal [Methodist Debakey Cardiovasc J] 2022 Mar 14; Vol. 18 (2), pp. 59-72. Date of Electronic Publication: 2022 Mar 14 (Print Publication: 2022). |
| نوع المنشور: | Journal Article; Review |
| اللغة: | English |
| بيانات الدورية: | Publisher: Methodist DeBakey Heart & Vascular Center Country of Publication: United States NLM ID: 101508600 Publication Model: eCollection Cited Medium: Internet ISSN: 1947-6108 (Electronic) Linking ISSN: 19476108 NLM ISO Abbreviation: Methodist Debakey Cardiovasc J Subsets: MEDLINE |
| أسماء مطبوعة: | Original Publication: Houston, TX : Methodist DeBakey Heart & Vascular Center |
| مواضيع طبية MeSH: | Amyloid Neuropathies, Familial*/complications , Amyloid Neuropathies, Familial*/diagnosis , Amyloid Neuropathies, Familial*/therapy , Cardiomyopathies*/diagnosis , Cardiomyopathies*/etiology , Cardiomyopathies*/therapy , Heart Diseases*/complications , Hematopoietic Stem Cell Transplantation*/adverse effects , Immunoglobulin Light-chain Amyloidosis*/complications , Immunoglobulin Light-chain Amyloidosis*/diagnosis , Immunoglobulin Light-chain Amyloidosis*/therapy, Humans ; Prealbumin/genetics ; Prealbumin/therapeutic use ; Transplantation, Autologous/adverse effects |
| مستخلص: | Cardiac amyloidosis (CA) is a restrictive cardiomyopathy with a traditionally poor prognosis. Until recently, CA treatment options were limited and consisted predominantly of managing symptoms and disease-related complications. However, the last decade has seen significant advances in disease-modifying therapies, increased awareness of CA, and improved diagnostic methods resulting in earlier diagnoses. In this review, we provide an overview of current and experimental treatments for the predominant types of CA: transthyretin cardiac amyloidosis (ATTR-CA) and immunoglobulin light chain (AL)-mediated CA (AL-CA). The mainstay of AL-CA treatment is proteasome inhibitor-based chemotherapy with daratumumab and, when feasible, autologous stem cell transplantation. For ATTR-CA, the stabilizer tafamidis is the only US Food and Drug Administration (FDA)-approved treatment. However, promising novel therapies on the horizon target various points in the ATTR-CA amyloidogenic cascade. These include transthyretin gene ( TTR) silencing agents to prevent TTR formation, TTR tetramer stabilization and inhibition of oligomer aggregation to prevent fibril formation, anti-TTR fiber antibodies, and amyloid degradation. For end-stage CA, advanced interventions may need to be considered, including heart, heart-kidney, and, for hereditary ATTR-CA, heart-liver transplantation. Despite the evolution of treatment options, CA management remains complex due to patient frailty and therapeutic side effects or intolerance with advanced cardiac disease. This is particularly relevant for those with AL-CA, when active teamwork between the hematologist-oncologist and the cardiologist is critical for treatment success. Often, referral to an expert center is necessary for timely diagnosis, initiation of treatment, and participation in clinical trials. Competing Interests: Jignesh Patel is a consultant for Pfizer, Alnylam, Akcea, and BridgeBio. Lily Stern has no competing interests to declare. (Copyright: © 2022 The Author(s).) |
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| فهرسة مساهمة: | Keywords: amyloidosis treatment; autologous stem cell transplantation; cardiac amyloidosis; daratumumab; inotersen; light chain amyloidosis; monoclonal light chains; patisiran; tafamidis; transthyretin amyloidosis |
| المشرفين على المادة: | 0 (Prealbumin) |
| تواريخ الأحداث: | Date Created: 20220413 Date Completed: 20220414 Latest Revision: 20220414 |
| رمز التحديث: | 20240829 |
| مُعرف محوري في PubMed: | PMC8932359 |
| DOI: | 10.14797/mdcvj.1050 |
| PMID: | 35414852 |
| قاعدة البيانات: | MEDLINE |
| تدمد: | 1947-6108 |
|---|---|
| DOI: | 10.14797/mdcvj.1050 |