PMID- 37711563
OWN - NLM
STAT- PubMed-not-MEDLINE
LR  - 20230917
IS  - 2297-055X (Print)
IS  - 2297-055X (Electronic)
IS  - 2297-055X (Linking)
VI  - 10
DP  - 2023
TI  - Burden of untreated transthyretin amyloid cardiomyopathy on patients and their 
      caregivers by disease severity: results from a multicenter, non-interventional, 
      real-world study.
PG  - 1238843
LID - 10.3389/fcvm.2023.1238843 [doi]
LID - 1238843
AB  - BACKGROUND: The humanistic burden of transthyretin amyloid cardiomyopathy 
      (ATTR-CM) is poorly defined. METHODS: An international study to comprehensively 
      characterize the burden of ATTR-CM on patients naive to disease-modifying therapy 
      and their unpaid primary caregivers using study-specific and established surveys 
      (patients: Kansas City Cardiomyopathy Questionnaire Overall Summary [KCCQ-OS], 
      12-Item Short Form Health Survey [SF-12], Hospital Anxiety and Depression Scale 
      [HADS], Patient-Reported Outcomes Measurement Information System [PROMIS] Fatigue 
      and Dyspnea; caregivers: SF-12, HADS, PROMIS Fatigue, Zarit Burden Interview 
      [ZBI]). All data were summarized descriptively. RESULTS: 208 patient and 
      caregiver pairs were included. 86% of patients were male, median age was 81 
      years, and 91% (141/155 with genetic testing) had wild-type ATTR-CM. Patient 
      responses characterized the mental and physical burden of ATTR-CM, which was 
      numerically higher among those who were New York Heart Association (NYHA) class 
      III (n = 43) vs. class I/II (n = 156). NYHA class III patients had particularly 
      low KCCQ-OS (36) and SF-12 physical component (27) scores, and 67% had a HADS 
      depression score >/=8. Caregivers (median age 68 years; 85% female; 59% spouse of 
      the patient; median duration of caregiving 1.5 years) reported that NYHA III 
      patients more frequently required help with a range of physical activities than 
      NYHA class I/II patients. 51% of caregivers to NYHA class III patients reported 
      at least a mild-to-moderate burden in the ZBI. A plain language summary of this 
      paper can be found as a supplemental material. CONCLUSIONS: Untreated ATTR-CM is 
      a burden to both patients and their caregivers.
CI  - (c) 2023 Ponti, Hsu, Damy, Villacorta, Verheyen, Keohane, Wang, Ines, Kumar, 
      Munteanu and Cappelli.
FAU - Ponti, Lucia
AU  - Ponti L
AD  - University of Urbino Carlo Bo, Urbino, Italy.
FAU - Hsu, Kristen
AU  - Hsu K
AD  - Amyloidosis Research Consortium, Newton, MA, United States.
FAU - Damy, Thibaud
AU  - Damy T
AD  - Referral Center for Cardiac Amyloidosis, CHU Henri Mondor, Creteil, France.
FAU - Villacorta, Eduardo
AU  - Villacorta E
AD  - Complejo Asistencial Universitario de Salamanca, Salamanca, Spain.
FAU - Verheyen, Nicolas
AU  - Verheyen N
AD  - Medical University of Graz, Graz, Austria.
FAU - Keohane, Denis
AU  - Keohane D
AD  - Pfizer Inc., New York, NY, United States.
FAU - Wang, Ronnie
AU  - Wang R
AD  - Pfizer Inc., Groton, CT, United States.
FAU - Ines, Monica
AU  - Ines M
AD  - Pfizer Inc., Porto Salvo, Portugal.
FAU - Kumar, Nisith
AU  - Kumar N
AD  - Pfizer Inc., New York, NY, United States.
FAU - Munteanu, Carmen
AU  - Munteanu C
AD  - Pfizer Inc., New York, NY, United States.
FAU - Cappelli, Francesco
AU  - Cappelli F
AD  - Tuscan Regional Amyloidosis Referral Centre, Careggi University Hospital, 
      Florence, Italy.
LA  - eng
PT  - Journal Article
DEP - 20230829
PL  - Switzerland
TA  - Front Cardiovasc Med
JT  - Frontiers in cardiovascular medicine
JID - 101653388
PMC - PMC10497948
OTO - NOTNLM
OT  - caregiver burden
OT  - chronic heart failure
OT  - healthcare surveys
OT  - heart failure
OT  - patient-reported outcome measures
COIS- DK, RW, MI, NK, and CM are employees of Pfizer and hold stock/stock options. TD 
      has received consulting fees from Alnylam, GlaxoSmithKline, Pfizer, and Prothena; 
      honoraria from Alnylam, Pfizer, and Prothena; research grants from 
      GlaxoSmithKline and Pfizer; and clinical trial support from Alnylam, Ionis, and 
      Pfizer. The institution that EV is employed by has received an unconditional 
      research grant from Pfizer. NV has received research grant, honoraria for 
      advisory board participation, and speaker's fees from Pfizer. FC reports 
      honoraria for advisory board participation from Pfizer, Alnylam, Novo Nordisk, 
      and Akcea; his institution has received an unconditional research grant from 
      Pfizer. The remaining authors declare that the research was conducted in the 
      absence of any commercial or financial relationships that could be construed as a 
      potential conflict of interest. This study was supported by Pfizer. Pfizer 
      contributed to the study design, management, and collection of data. In their 
      role as authors, employees of Pfizer were involved in the design or conduct of 
      the study, interpretation of data, preparation, review, and approval of the 
      manuscript and the decision to submit for publication, along with their 
      co-authors. The study sponsor approved the manuscript from an intellectual 
      property perspective, but had no right to veto the publication.
EDAT- 2023/09/15 06:42
MHDA- 2023/09/15 06:43
PMCR- 2023/01/01
CRDT- 2023/09/15 03:53
PHST- 2023/06/12 00:00 [received]
PHST- 2023/07/27 00:00 [accepted]
PHST- 2023/09/15 06:43 [medline]
PHST- 2023/09/15 06:42 [pubmed]
PHST- 2023/09/15 03:53 [entrez]
PHST- 2023/01/01 00:00 [pmc-release]
AID - 10.3389/fcvm.2023.1238843 [doi]
PST - epublish
SO  - Front Cardiovasc Med. 2023 Aug 29;10:1238843. doi: 10.3389/fcvm.2023.1238843. 
      eCollection 2023.