MARC details
| 000 -LEADER |
|---|
| fixed length control field |
03678nam a22004337a 4500 |
| 008 - FIXED-LENGTH DATA ELEMENTS--GENERAL INFORMATION |
|---|
| fixed length control field |
240723s20242024 xxu||||| |||| 00| 0 eng d |
| 022 ## - INTERNATIONAL STANDARD SERIAL NUMBER |
|---|
| International Standard Serial Number |
2193-6544 |
| 024 ## - OTHER STANDARD IDENTIFIER |
|---|
| Standard number or code |
10.1007/s40119-024-00362-9 [pii] |
| 040 ## - CATALOGING SOURCE |
|---|
| Original cataloging agency |
Ovid MEDLINE(R) |
| 099 ## - LOCAL FREE-TEXT CALL NUMBER (OCLC) |
|---|
| PMID |
38521883 |
| 245 ## - TITLE STATEMENT |
|---|
| Title |
Real-World Effectiveness of High-Dose Tafamidis on Neurologic Disease Progression in Mixed-Phenotype Variant Transthyretin Amyloid Cardiomyopathy. |
| 251 ## - Source |
|---|
| Source |
Cardiology & Therapy. 2024 Mar 23 |
| 252 ## - Abbreviated Source |
|---|
| Abbreviated source |
Cardiol. ther.. 2024 Mar 23 |
| 253 ## - Journal Name |
|---|
| Journal name |
Cardiology and therapy |
| 260 ## - PUBLICATION, DISTRIBUTION, ETC. |
|---|
| Year |
2024 |
| 260 ## - PUBLICATION, DISTRIBUTION, ETC. |
|---|
| Publication date |
2024 Mar 23 |
| 265 ## - SOURCE FOR ACQUISITION/SUBSCRIPTION ADDRESS [OBSOLETE] |
|---|
| Publication status |
aheadofprint |
| 265 ## - SOURCE FOR ACQUISITION/SUBSCRIPTION ADDRESS [OBSOLETE] |
|---|
| Medline status |
Publisher |
| 266 ## - Date added to catalog |
|---|
| Date added to catalog |
2024-07-23 |
| 520 ## - SUMMARY, ETC. |
|---|
| Abstract |
CONCLUSION: This is the first real-world study to demonstrate the potential value of high-dose tafamidis for delaying neurologic disease progression in patients with mixed-phenotype ATTRv-CM. The findings underscore the importance of multidisciplinary assessment for patients with ATTR amyloidosis. |
| 520 ## - SUMMARY, ETC. |
|---|
| Abstract |
INTRODUCTION: Transthyretin amyloidosis (ATTR) is a progressive, heterogeneous rare disease manifesting as ATTR polyneuropathy (ATTR-PN), ATTR cardiomyopathy (ATTR-CM), or a mixed phenotype. Tafamidis meglumine (20 mg po qd) is approved in some markets to delay neurologic progression in ATTR-PN, while high-dose tafamidis (80/61 mg po qd) is approved worldwide to reduce cardiovascular mortality and cardiovascular-related hospitalization in ATTR-CM. The objective of this study was to assess the real-world benefit of high-dose tafamidis for delaying neurologic progression in patients with mixed-phenotype variant ATTR-CM (ATTRv-CM). |
| 520 ## - SUMMARY, ETC. |
|---|
| Abstract |
METHODS: This exploratory, retrospective, observational cohort study evaluated anonymized electronic medical records and included adult patients with mixed-phenotype ATTRv-CM treated with high-dose tafamidis for at least 6 months. Neurologic assessments included the Medical Research Council (MRC) Scale for Muscle Strength, Neuropathy Impairment Score (NIS) muscle weakness subscale, and Polyneuropathy Disability (PND) instrument. Modified body mass index (mBMI) was also assessed. |
| 520 ## - SUMMARY, ETC. |
|---|
| Abstract |
RESULTS: Patients (N = 10) started tafamidis treatment an average of 3.8 months after diagnosis, with an average treatment duration of 20.8 months. Seven of 10 patients demonstrated normal muscle strength on the MRC scale throughout the study, and 9 of 10 patients had no decline in muscle strength during the post-treatment period. The NIS muscle weakness subscale score was <= 60 for all patients in the study at all time points, suggesting normal function to mild impairment. Six of 10 patients had no change in walking capacity as measured by the PND instrument at pre- and post-assessments, while one-third of patients had a decrease in PND stage (signaling improvement) from pre- to post-assessment. mBMI remained relatively stable throughout the study. |
| 520 ## - SUMMARY, ETC. |
|---|
| Abstract |
TRIAL REGISTRATION: ClinicalTrials.gov: NCT05139680. Copyright © 2024. The Author(s). |
| 546 ## - LANGUAGE NOTE |
|---|
| Language note |
English |
| 650 ## - SUBJECT ADDED ENTRY--TOPICAL TERM |
|---|
| Indexing |
Automated |
| 650 ## - SUBJECT ADDED ENTRY--TOPICAL TERM |
|---|
| Topical term or geographic name entry element |
IN PROCESS -- NOT YET INDEXED |
| 651 ## - SUBJECT ADDED ENTRY--GEOGRAPHIC NAME |
|---|
| Institution |
MedStar Health Research Institute |
| 651 ## - SUBJECT ADDED ENTRY--GEOGRAPHIC NAME |
|---|
| Institution |
MedStar Heart & Vascular Institute |
| 651 ## - SUBJECT ADDED ENTRY--GEOGRAPHIC NAME |
|---|
| Institution |
MedStar Washington Hospital Center |
| 656 ## - INDEX TERM--OCCUPATION |
|---|
| Department |
Neurology |
| 657 ## - INDEX TERM--FUNCTION |
|---|
| Medline publication type |
Journal Article |
| 700 ## - ADDED ENTRY--PERSONAL NAME |
|---|
| Local Authors |
Merrill, Emma |
| Institution Code |
MHRI |
| 700 ## - ADDED ENTRY--PERSONAL NAME |
|---|
| Local Authors |
Sheikh, Farooq H |
| Institution Code |
MHVI |
| 700 ## - ADDED ENTRY--PERSONAL NAME |
|---|
| Local Authors |
Streicher, Nicholas |
| Institution Code |
MWHC |
| 790 ## - Authors |
|---|
| All authors |
Streicher N, Amass L, Wang R, Stephens JM, LeMasters T, Raina R, Merrill E, Sheikh FH |
| 856 ## - ELECTRONIC LOCATION AND ACCESS |
|---|
| DOI |
<a href="https://dx.doi.org/10.1007/s40119-024-00362-9">https://dx.doi.org/10.1007/s40119-024-00362-9</a> |
| Public note |
https://dx.doi.org/10.1007/s40119-024-00362-9 |
| 942 ## - ADDED ENTRY ELEMENTS (KOHA) |
|---|
| Koha item type |
Journal Article |
| Item type description |
Article |
