000			03678nam a22004337a 4500
008			240723s20242024 xxu||||| |||| 00| 0 eng d
022			_a2193-6544
024			_a10.1007/s40119-024-00362-9 [pii]
040			_aOvid MEDLINE(R)
099			_a38521883
245			_aReal-World Effectiveness of High-Dose Tafamidis on Neurologic Disease Progression in Mixed-Phenotype Variant Transthyretin Amyloid Cardiomyopathy.
251			_aCardiology & Therapy. 2024 Mar 23
252			_aCardiol. ther.. 2024 Mar 23
253			_aCardiology and therapy
260			_c2024
260			_p2024 Mar 23
265			_saheadofprint
265			_tPublisher
266			_d2024-07-23
520			_aCONCLUSION: 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			_aINTRODUCTION: 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			_aMETHODS: 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			_aRESULTS: 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			_aTRIAL REGISTRATION: ClinicalTrials.gov: NCT05139680. Copyright © 2024. The Author(s).
546			_aEnglish
650			_zAutomated
650			_aIN PROCESS -- NOT YET INDEXED
651			_aMedStar Health Research Institute
651			_aMedStar Heart & Vascular Institute
651			_aMedStar Washington Hospital Center
656			_aNeurology
657			_aJournal Article
700			_aMerrill, Emma _bMHRI
700			_aSheikh, Farooq H _bMHVI
700			_aStreicher, Nicholas _bMWHC
790			_aStreicher N, Amass L, Wang R, Stephens JM, LeMasters T, Raina R, Merrill E, Sheikh FH
856			_uhttps://dx.doi.org/10.1007/s40119-024-00362-9 _zhttps://dx.doi.org/10.1007/s40119-024-00362-9
942			_cART _dArticle
999			_c14204 _d14204