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

doi:10.1080/14779072.2020.1748011

PROMIS® and Neuro-QoL^TM 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

Research output: Contribution to journal › Article › peer-review

11 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 language	English (US)
Pages (from-to)	231-238
Number of pages	8
Journal	Expert review of cardiovascular therapy
Volume	18
Issue number	4
DOIs	https://doi.org/10.1080/14779072.2020.1748011
State	Published - Apr 2 2020
Externally published	Yes

Keywords

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

ASJC Scopus subject areas

Internal Medicine
Cardiology and Cardiovascular Medicine

Access to Document

10.1080/14779072.2020.1748011

Cite this

@article{0e3b10fb2d1a48c884252e6569f5d830,

title = "PROMIS{\textregistered} and Neuro-QoLTM measures are valid measures of health-related quality of life among patients with familial chylomicronemia syndrome",

abstract = "Background: FCS significantly affects health-related quality of life (HRQOL). Legacy patient-reported outcome measures are often not sensitive to FCS{\textquoteright}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.",

keywords = "Familial Chylomicronemia Syndrome, Neuro-QoL, PROMIS, health-related quality of life, symptom burden",

author = "Fox, {Rina S.} and Peipert, {John Devin} and Montserrat Vera-Llonch and Glenn Phillips and David Cella",

note = "Funding Information: Funding for this work was provided to Northwestern University by Akcea Therapeutics. Montserrat Vera Llonch and Glenn Phillips contributed to this work as part of their employment with Akcea Therapeutics. Rina S. Fox, John Devin Peipert, and David Cella contributed to this work at Northwestern University as contracted by Akcea Therapeutics. We thank Maria Corona for facilitating project management. We also thank the study participants for their time and contributions. Funding Information: This paper was funded by Akcea Therapeutics. Funding for this work was provided to Northwestern University by Akcea Therapeutics. Montserrat Vera Llonch and Glenn Phillips contributed to this work as part of their employment with Akcea Therapeutics. Rina S. Fox, John Devin Peipert, and David Cella contributed to this work at Northwestern University as contracted by Akcea Therapeutics. We thank Maria Corona for facilitating project management. We also thank the study participants for their time and contributions. Publisher Copyright: {\textcopyright} 2020, {\textcopyright} 2020 Informa UK Limited, trading as Taylor & Francis Group.",

year = "2020",

month = apr,

day = "2",

doi = "10.1080/14779072.2020.1748011",

language = "English (US)",

volume = "18",

pages = "231--238",

journal = "Expert review of cardiovascular therapy",

issn = "1477-9072",

publisher = "Taylor and Francis Ltd.",

number = "4",

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TY - JOUR

T1 - PROMIS® and Neuro-QoLTM measures are valid measures of health-related quality of life among patients with familial chylomicronemia syndrome

AU - Fox, Rina S.

AU - Peipert, John Devin

AU - Vera-Llonch, Montserrat

AU - Phillips, Glenn

AU - Cella, David

N1 - Funding Information: Funding for this work was provided to Northwestern University by Akcea Therapeutics. Montserrat Vera Llonch and Glenn Phillips contributed to this work as part of their employment with Akcea Therapeutics. Rina S. Fox, John Devin Peipert, and David Cella contributed to this work at Northwestern University as contracted by Akcea Therapeutics. We thank Maria Corona for facilitating project management. We also thank the study participants for their time and contributions. Funding Information: This paper was funded by Akcea Therapeutics. Funding for this work was provided to Northwestern University by Akcea Therapeutics. Montserrat Vera Llonch and Glenn Phillips contributed to this work as part of their employment with Akcea Therapeutics. Rina S. Fox, John Devin Peipert, and David Cella contributed to this work at Northwestern University as contracted by Akcea Therapeutics. We thank Maria Corona for facilitating project management. We also thank the study participants for their time and contributions. Publisher Copyright: © 2020, © 2020 Informa UK Limited, trading as Taylor & Francis Group.

PY - 2020/4/2

Y1 - 2020/4/2

N2 - 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.

AB - 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.

KW - Familial Chylomicronemia Syndrome

KW - Neuro-QoL

KW - PROMIS

KW - health-related quality of life

KW - symptom burden

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