PROMIS® and Neuro-QoLTM measures are valid measures of health-related quality of life among patients with familial chylomicronemia syndrome

Rina S. Fox, John Devin Peipert, Montserrat Vera-Llonch, Glenn Phillips, David Cella*

*Corresponding author for this work

Research output: Contribution to journalArticlepeer-review

2 Scopus citations

Abstract

Background: FCS significantly affects health-related quality of life (HRQOL). Legacy patient-reported outcome measures are often not sensitive to FCS’s impact. NIH PROMIS and Neuro-QoL measures may accurately capture HRQOL in FCS patients. This study assessed a broad range of PROMIS and Neuro-QoL measures covering physical, mental, and social HRQOL to determine their suitability for the FCS population. Methods: Adult FCS patients in the United States (N = 25) were recruited to an online survey study and completed several PROMIS short forms and Neuro-QoL computer adaptive tests. Results: Scores were more than 0.5 standard deviations (SD) worse than the normative mean on 10 of 16 normed measures, and more than 0.75 SDs worse than the normative mean on two measures. Responses at the floor and ceiling were occasionally observed, marginal reliabilities were strong, and significant differences across performance status (ps < 0.05) provided preliminary support for construct validity. The measures correlated with each other strongly and as expected. Conclusion: Results support the ability of PROMIS and Neuro-QoL measures to detect HRQOL impairment among patients with FCS. PROMIS and Neuro-QoL measures captured the functional impact and symptom burden associated with FCS, and the broad range of symptom severity experienced by patients with FCS.

Original languageEnglish (US)
Pages (from-to)231-238
Number of pages8
JournalExpert review of cardiovascular therapy
Volume18
Issue number4
DOIs
StatePublished - Apr 2 2020

Keywords

  • Familial Chylomicronemia Syndrome
  • Neuro-QoL
  • PROMIS
  • health-related quality of life
  • symptom burden

ASJC Scopus subject areas

  • Internal Medicine
  • Cardiology and Cardiovascular Medicine

Fingerprint

Dive into the research topics of 'PROMIS® and Neuro-QoL<sup>TM</sup> measures are valid measures of health-related quality of life among patients with familial chylomicronemia syndrome'. Together they form a unique fingerprint.

Cite this