PMID- 35718845
OWN - NLM
STAT- PubMed-not-MEDLINE
LR  - 20220718
IS  - 2509-4254 (Electronic)
IS  - 2509-4262 (Print)
IS  - 2509-4262 (Linking)
VI  - 6
IP  - 4
DP  - 2022 Jul
TI  - Longitudinal Psychometric Analysis of the Hypertrophic Cardiomyopathy Symptom 
      Questionnaire (HCMSQ) Using Outcomes from the Phase III EXPLORER-HCM Trial.
PG  - 575-586
LID - 10.1007/s41669-022-00340-8 [doi]
AB  - BACKGROUND: Hypertrophic cardiomyopathy (HCM) symptoms include shortness of 
      breath (SOB), fatigue, chest pain, palpitations, dizziness, and fainting. The HCM 
      Symptom Questionnaire (HCMSQ), the only patient-reported outcome instrument 
      designed to specifically measure HCM symptoms, yields four domain scores (SOB, 
      tiredness, cardiovascular symptoms, syncope) and a total score. We evaluated the 
      longitudinal psychometric properties of the HCMSQ using baseline to week 30 data 
      from the phase III EXPLORER-HCM trial (NCT03470545). METHODS: Test-retest 
      reliability was assessed via intraclass correlation of patients with stable 
      Patient Global Impression of Change (PGIC) and Patient Global Impression of 
      Severity (PGIS) responses. Sensitivity to change was assessed via Spearman 
      correlations with the Kansas City Cardiomyopathy Questionnaire (KCCQ-23) and the 
      EuroQoL visual analogue scale (EQ VAS), and via one-way ANOVA comparing change 
      groups defined on clinical (New York Heart Association [NYHA] class, left 
      ventricular outflow tract [LVOT] gradient, peak oxygen consumption [pVO(2)]) and 
      patient-reported (PGIS, PGIC) variables. Meaningful change thresholds were 
      established via PGIC/PGIS. RESULTS: All HCMSQ scores showed strong evidence of 
      test-retest reliability (intraclass correlation coefficient > 0.70). Sensitivity 
      to change was demonstrated with mostly strong/moderate correlations with KCCQ-23 
      and EQ VAS, and significant differences (p </= 0.05) in PGIS, PGIC, pVO(2), and 
      NYHA (except tiredness domain) change categories, but not LVOT gradient. 
      Clinically meaningful score reductions were >/=1 point for tiredness and 
      cardiovascular symptoms domains, >/= 2.5 points for SOB domain, and >/=2 points for 
      total score. CONCLUSIONS: Results suggest that HCMSQ is fit for purpose in 
      capturing HCM symptoms and may provide evidence of treatment benefit from the 
      patients' perspectives.
CI  - (c) 2022. The Author(s).
FAU - Reaney, Matthew
AU  - Reaney M
AUID- ORCID: 0000-0003-2918-722X
AD  - IQVIA, 3 Forbury Place, 23 Forbury Road, Reading, RG1 3JH, UK. 
      matthew.reaney@iqvia.com.
FAU - Addepalli, Prithvi
AU  - Addepalli P
AD  - IQVIA, 3 Forbury Place, 23 Forbury Road, Reading, RG1 3JH, UK.
FAU - Allen, Veleka
AU  - Allen V
AD  - IQVIA, New York, NY, USA.
FAU - Spertus, John A
AU  - Spertus JA
AUID- ORCID: 0000-0002-2839-2611
AD  - Saint Luke's Mid America Heart Institute and the University of Missouri-Kansas 
      City, Kansas City, MO, USA.
FAU - Dolan, Chantal
AU  - Dolan C
AD  - CMD Consulting, Sandy, UT, USA.
FAU - Sehnert, Amy J
AU  - Sehnert AJ
AUID- ORCID: 0000-0003-1912-1710
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.
LA  - eng
SI  - ClinicalTrials.gov/NCT03470545
PT  - Journal Article
DEP - 20220620
PL  - Switzerland
TA  - Pharmacoecon Open
JT  - PharmacoEconomics - open
JID - 101700780
PMC - PMC9283589
COIS- MR, PA, and VA are employees of IQVIA and have received funding from MyoKardia, 
      Inc., a wholly owned subsidiary of Bristol Myers Squibb, for work performed on 
      this study. JAS has served as a consultant and holds a research grant from 
      MyoKardia, Inc.; he also provides consultative services for Bayer, Janssen, 
      Merck, Novartis, Pfizer, and Terumo; JAS owns the copyright to the Kansas City 
      Cardiomyopathy Questionnaire. CD is a consultant for Bristol Myers Squibb and the 
      following companies: Alexion Pharmaceuticals, Coagulant Therapeutics, Elekta, 
      Genentech, Gilead Sciences, Global Blood Therapeutics, Lyell Immunopharma, 
      Portola Pharmaceuticals, Inc., Puma Biotechnology, and Regenxbio Inc. AJS is an 
      employee of MyoKardia, Inc., a wholly owned subsidiary of Bristol Myers Squibb, 
      and holds stocks. JTF is an employee of MyoKardia, Inc., a wholly owned 
      subsidiary of Bristol Myers Squibb, and holds stocks/options.
EDAT- 2022/06/20 06:00
MHDA- 2022/06/20 06:01
PMCR- 2022/06/20
CRDT- 2022/06/19 23:19
PHST- 2022/05/16 00:00 [accepted]
PHST- 2022/06/20 06:00 [pubmed]
PHST- 2022/06/20 06:01 [medline]
PHST- 2022/06/19 23:19 [entrez]
PHST- 2022/06/20 00:00 [pmc-release]
AID - 10.1007/s41669-022-00340-8 [pii]
AID - 340 [pii]
AID - 10.1007/s41669-022-00340-8 [doi]
PST - ppublish
SO  - Pharmacoecon Open. 2022 Jul;6(4):575-586. doi: 10.1007/s41669-022-00340-8. Epub 
      2022 Jun 20.