PMID- 34907813
OWN - NLM
STAT- MEDLINE
DCOM- 20211224
LR  - 20220429
IS  - 1941-837X (Electronic)
IS  - 1369-6998 (Linking)
VI  - 25
IP  - 1
DP  - 2022 Jan-Dec
TI  - Assessing health-related quality-of-life in patients with symptomatic obstructive 
      hypertrophic cardiomyopathy: EQ-5D-based utilities in the EXPLORER-HCM trial.
PG  - 51-58
LID - 10.1080/13696998.2021.2011301 [doi]
AB  - AIMS: To assess the effects of mavacamten on health-related quality-of-life 
      (HRQoL) in symptomatic obstructive hypertrophic cardiomyopathy (HCM) and estimate 
      health utilities by New York Heart Association (NYHA) functional class. MATERIALS 
      AND METHODS: Patients with symptomatic obstructive HCM were randomized to 
      30 weeks of mavacamten or to placebo treatment, with or without beta-blocker or 
      calcium channel blocker monotherapy, in EXPLORER-HCM (ClinicalTrials.gov 
      identifier: NCT03470545). Health utility was measured using the EuroQoL 
      5-dimension 5-level (EQ-5D-5L) index score with the US value set. The 30-week 
      changes in EQ-5D-5L index score and EuroQoL visual analog scale (EQ-VAS) score 
      were compared between the two arms using linear regression, and the proportions 
      of patients with a meaningful improvement were compared using logistic 
      regression. The meaningful change thresholds were estimated using both 
      distribution- and anchor-based approaches. Mean utilities by NYHA class were 
      estimated for each arm using a generalized estimating equation. RESULTS: Compared 
      with placebo (N = 89), patients receiving mavacamten (N = 96) had significantly 
      greater 30-week improvement in EQ-5D-5L index score (mavacamten = 0.084; placebo 
      = 0.009; adjusted difference = 0.073 [95% confidence interval = 0.027-0.118]) and 
      EQ-VAS score (mavacamten = 8.5; placebo = 0.7; adjusted difference = 7.5 [95% 
      confidence interval = 1.8-13.2]), and a significantly higher proportion of these 
      patients showed meaningful improvement in EQ-5D-5L index score and EQ-VAS score. 
      Both outcomes were correlated with the Kansas City Cardiomyopathy Questionnaire 
      Clinical Summary Score (KCCQ CSS) and HCM Symptom Questionnaire 
      Shortness-of-Breath (HCMSQ SoB) subscore, two patient-reported anchor variables. 
      Additionally, mean utilities significantly decreased with higher NYHA functional 
      class (values for NYHA class I, II, and III/IV - mavacamten = 0.950, 0.866, and 
      0.708; placebo = 0.952, 0.850, and 0.704). CONCLUSIONS: Compared with placebo, 
      mavacamten significantly improved EQ-5D-5L index score and EQ-VAS score - and 
      thus HRQoL - among patients with symptomatic obstructive HCM. Patients with a 
      higher NYHA functional class had a lower health utility value.
FAU - Xie, Jipan
AU  - Xie J
AUID- ORCID: 0000-0001-5937-0936
AD  - Analysis Group, Inc., Los Angeles, CA, USA.
FAU - Wang, Yan
AU  - Wang Y
AUID- ORCID: 0000-0002-9326-0148
AD  - Analysis Group, Inc., Los Angeles, CA, USA.
FAU - Xu, Yaping
AU  - Xu Y
AD  - MyoKardia, Inc., a wholly owned subsidiary of Bristol Myers Squibb, Brisbane, CA, 
      USA.
FAU - Fine, Jennifer T
AU  - Fine JT
AD  - MyoKardia, Inc., a wholly owned subsidiary of Bristol Myers Squibb, Brisbane, CA, 
      USA.
FAU - Lam, Jenny
AU  - Lam J
AUID- ORCID: 0000-0003-3738-2973
AD  - Bristol Myers Squibb, Brisbane, CA, USA.
FAU - Garrison, Louis P
AU  - Garrison LP
AUID- ORCID: 0000-0002-9468-4989
AD  - University of Washington, Seattle, WA, USA.
LA  - eng
SI  - ClinicalTrials.gov/NCT03470545
PT  - Journal Article
PT  - Randomized Controlled Trial
PL  - England
TA  - J Med Econ
JT  - Journal of medical economics
JID - 9892255
SB  - IM
MH  - *Cardiomyopathy, Hypertrophic/drug therapy
MH  - Humans
MH  - Pain Measurement
MH  - *Quality of Life
MH  - Surveys and Questionnaires
OTO - NOTNLM
OT  - EQ-5D-5L index score
OT  - EQ-VAS score
OT  - I
OT  - I1
OT  - I15
OT  - I3
OT  - I31
OT  - Obstructive hypertrophic cardiomyopathy
OT  - health-related quality-of-life
OT  - utility
EDAT- 2021/12/16 06:00
MHDA- 2021/12/25 06:00
CRDT- 2021/12/15 08:44
PHST- 2021/12/16 06:00 [pubmed]
PHST- 2021/12/25 06:00 [medline]
PHST- 2021/12/15 08:44 [entrez]
AID - 10.1080/13696998.2021.2011301 [doi]
PST - ppublish
SO  - J Med Econ. 2022 Jan-Dec;25(1):51-58. doi: 10.1080/13696998.2021.2011301.