Health-related Quality of Life in a Prospective Study of Ultrasound to Detect Cystic Fibrosis-related Liver Disease in Children

Sarah Jane Schwarzenberg; Joseph J. Palermo; Wen Ye; Suiyuan Huang; John C. Magee; Adina Alazraki; A. Jay Freeman; Roger Harned; Boaz Karmazyn; Wikrom Karnsakul; Daniel H. Leung; Simon C. Ling; Prakash Masand; Jean P. Molleston; Karen F. Murray; Oscar M. Navarro; Jennifer Lynn Nicholas; Randolph K. Otto; Shruti M. Paranjape; Marilyn J. Siegel; Janis Stoll; Alexander J. Towbin; Michael R. Narkewicz; Estella M. Alonso

doi:10.1097/MPG.0000000000003605

Health-related Quality of Life in a Prospective Study of Ultrasound to Detect Cystic Fibrosis-related Liver Disease in Children

Sarah Jane Schwarzenberg^*, Joseph J. Palermo, Wen Ye, Suiyuan Huang, John C. Magee, Adina Alazraki, A. Jay Freeman, Roger Harned, Boaz Karmazyn, Wikrom Karnsakul, Daniel H. Leung, Simon C. Ling, Prakash Masand, Jean P. Molleston, Karen F. Murray, Oscar M. Navarro, Jennifer Lynn Nicholas, Randolph K. Otto, Shruti M. Paranjape, Marilyn J. SiegelJanis Stoll, Alexander J. Towbin, Michael R. Narkewicz, Estella M. Alonso

^*Corresponding author for this work

Pediatrics

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

2 Scopus citations

Abstract

Objectives: Cystic fibrosis liver disease (CFLD) begins early in life. Symptoms may be vague, mild, or nonexistent. Progressive liver injury may be associated with decrements in patient health before liver disease is clinically apparent. We examined Health-Related Quality of Life (HRQOL) in children enrolled in a multi-center study of CFLD to determine the impact of early CFLD on general and disease-specific QOL. Methods: Ultrasound (US) patterns of normal (NL), heterogeneous (HTG), homogeneous (HMG), or nodular (NOD) were assigned in a prospective manner to predict those at risk for advanced CFLD. Parents were informed of results. We assessed parent/child-reported (age ≥5 years) HRQOL by PedsQL 4.0 Generic Core and CF Questionnaire-revised (CFQ-R) prior to US and annually. HRQOL scores were compared by US pattern at baseline (prior to US), between baseline and 1 year and at 5 years. Multivariate analysis of variance (MANOVA) with Hotelling-Lawley trace tested for differences among US groups. Results: Prior to US, among 515 participants and their parents there was no evidence that HTG or NOD US was associated with reduced PedsQL/CFQ-R at baseline. Parents of NOD reported no change in PedsQL/CFQ-R over the next year. Child-report PedsQL/CFQ-R (95 NL, 20 NOD) showed improvement between baseline and year 5 for many scales, including Physical Function. Parents of HMG children reported improved CFQ-R scores related to weight. Conclusions: Early undiagnosed or pre-symptomatic liver disease had no impact on generic or disease-specific HRQoL, and HRQoL was remarkably stable in children with CF regardless of liver involvement.

Original language	English (US)
Pages (from-to)	635-642
Number of pages	8
Journal	Journal of pediatric gastroenterology and nutrition
Volume	75
Issue number	5
DOIs	https://doi.org/10.1097/MPG.0000000000003605
State	Published - Nov 1 2022

Funding

Sources of Funding: Funded by grants from the Cystic Fibrosis Foundation (NARKEW17AB0 [to M.R.N.]) and NIDDK (U01 DK062453 [to M.R.N.] and U01 DK 062456 [to J.P.M.]). Trial identification number: Clinical Trials.gov : ; funding from National Institute of Health. S.J.S. serves as a consultant for Nestle, UpToDate, and AbbVie. A.J.F. has grant/research support through Travere Therapeutics and Allergan; serves as an advisor for Abbvie and Takeda. W.K. has received research grants from Gilead Sciences and Albireo Pharma; serves as an advisor for Mirum and Travere Therapeutics. D.H.L. has grant/research support from Abbvie, Gilead, and Mirum; serves as a consultant for Gilead, Vertex, and Merck. S.C.L. receives research funding from Abbvie and Gilead and serves as a consultant for Abbvie JPM has research funding from Gillead, Abbvie, Albireo, Mirum. K.F.M. is a consultant for Albireo and Gilead. M.R.N. serves as a consultant for Vertex, has received research grants from Gilead, AbbVie, and has a family member with stock in Merck. The remaining authors report no conflicts of interest.

Keywords

cirrhosis
cystic fibrosis
functional outcomes
health-related quality of life
nodular liver
patient reported outcomes
wellness

ASJC Scopus subject areas

Pediatrics, Perinatology, and Child Health
Gastroenterology

UN SDGs

This output contributes to the following UN Sustainable Development Goals (SDGs)

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10.1097/MPG.0000000000003605

Cite this

Schwarzenberg, S. J., Palermo, J. J., Ye, W., Huang, S., Magee, J. C., Alazraki, A., Freeman, A. J., Harned, R., Karmazyn, B., Karnsakul, W., Leung, D. H., Ling, S. C., Masand, P., Molleston, J. P., Murray, K. F., Navarro, O. M., Nicholas, J. L., Otto, R. K., Paranjape, S. M., ... Alonso, E. M. (2022). Health-related Quality of Life in a Prospective Study of Ultrasound to Detect Cystic Fibrosis-related Liver Disease in Children. Journal of pediatric gastroenterology and nutrition, 75(5), 635-642. https://doi.org/10.1097/MPG.0000000000003605

@article{da1150b2cabc498cac6fdec502380e68,

title = "Health-related Quality of Life in a Prospective Study of Ultrasound to Detect Cystic Fibrosis-related Liver Disease in Children",

abstract = "Objectives: Cystic fibrosis liver disease (CFLD) begins early in life. Symptoms may be vague, mild, or nonexistent. Progressive liver injury may be associated with decrements in patient health before liver disease is clinically apparent. We examined Health-Related Quality of Life (HRQOL) in children enrolled in a multi-center study of CFLD to determine the impact of early CFLD on general and disease-specific QOL. Methods: Ultrasound (US) patterns of normal (NL), heterogeneous (HTG), homogeneous (HMG), or nodular (NOD) were assigned in a prospective manner to predict those at risk for advanced CFLD. Parents were informed of results. We assessed parent/child-reported (age ≥5 years) HRQOL by PedsQL 4.0 Generic Core and CF Questionnaire-revised (CFQ-R) prior to US and annually. HRQOL scores were compared by US pattern at baseline (prior to US), between baseline and 1 year and at 5 years. Multivariate analysis of variance (MANOVA) with Hotelling-Lawley trace tested for differences among US groups. Results: Prior to US, among 515 participants and their parents there was no evidence that HTG or NOD US was associated with reduced PedsQL/CFQ-R at baseline. Parents of NOD reported no change in PedsQL/CFQ-R over the next year. Child-report PedsQL/CFQ-R (95 NL, 20 NOD) showed improvement between baseline and year 5 for many scales, including Physical Function. Parents of HMG children reported improved CFQ-R scores related to weight. Conclusions: Early undiagnosed or pre-symptomatic liver disease had no impact on generic or disease-specific HRQoL, and HRQoL was remarkably stable in children with CF regardless of liver involvement.",

keywords = "cirrhosis, cystic fibrosis, functional outcomes, health-related quality of life, nodular liver, patient reported outcomes, wellness",

author = "Schwarzenberg, {Sarah Jane} and Palermo, {Joseph J.} and Wen Ye and Suiyuan Huang and Magee, {John C.} and Adina Alazraki and Freeman, {A. Jay} and Roger Harned and Boaz Karmazyn and Wikrom Karnsakul and Leung, {Daniel H.} and Ling, {Simon C.} and Prakash Masand and Molleston, {Jean P.} and Murray, {Karen F.} and Navarro, {Oscar M.} and Nicholas, {Jennifer Lynn} and Otto, {Randolph K.} and Paranjape, {Shruti M.} and Siegel, {Marilyn J.} and Janis Stoll and Towbin, {Alexander J.} and Narkewicz, {Michael R.} and Alonso, {Estella M.}",

year = "2022",

month = nov,

day = "1",

doi = "10.1097/MPG.0000000000003605",

language = "English (US)",

volume = "75",

pages = "635--642",

journal = "Journal of pediatric gastroenterology and nutrition",

issn = "0277-2116",

publisher = "John Wiley and Sons Inc.",

number = "5",

}

Schwarzenberg, SJ, Palermo, JJ, Ye, W, Huang, S, Magee, JC, Alazraki, A, Freeman, AJ, Harned, R, Karmazyn, B, Karnsakul, W, Leung, DH, Ling, SC, Masand, P, Molleston, JP, Murray, KF, Navarro, OM, Nicholas, JL, Otto, RK, Paranjape, SM, Siegel, MJ, Stoll, J, Towbin, AJ, Narkewicz, MR & Alonso, EM 2022, 'Health-related Quality of Life in a Prospective Study of Ultrasound to Detect Cystic Fibrosis-related Liver Disease in Children', Journal of pediatric gastroenterology and nutrition, vol. 75, no. 5, pp. 635-642. https://doi.org/10.1097/MPG.0000000000003605

TY - JOUR

T1 - Health-related Quality of Life in a Prospective Study of Ultrasound to Detect Cystic Fibrosis-related Liver Disease in Children

AU - Schwarzenberg, Sarah Jane

AU - Palermo, Joseph J.

AU - Ye, Wen

AU - Huang, Suiyuan

AU - Magee, John C.

AU - Alazraki, Adina

AU - Freeman, A. Jay

AU - Harned, Roger

AU - Karmazyn, Boaz

AU - Karnsakul, Wikrom

AU - Leung, Daniel H.

AU - Ling, Simon C.

AU - Masand, Prakash

AU - Molleston, Jean P.

AU - Murray, Karen F.

AU - Navarro, Oscar M.

AU - Nicholas, Jennifer Lynn

AU - Otto, Randolph K.

AU - Paranjape, Shruti M.

AU - Siegel, Marilyn J.

AU - Stoll, Janis

AU - Towbin, Alexander J.

AU - Narkewicz, Michael R.

AU - Alonso, Estella M.

PY - 2022/11/1

Y1 - 2022/11/1

N2 - Objectives: Cystic fibrosis liver disease (CFLD) begins early in life. Symptoms may be vague, mild, or nonexistent. Progressive liver injury may be associated with decrements in patient health before liver disease is clinically apparent. We examined Health-Related Quality of Life (HRQOL) in children enrolled in a multi-center study of CFLD to determine the impact of early CFLD on general and disease-specific QOL. Methods: Ultrasound (US) patterns of normal (NL), heterogeneous (HTG), homogeneous (HMG), or nodular (NOD) were assigned in a prospective manner to predict those at risk for advanced CFLD. Parents were informed of results. We assessed parent/child-reported (age ≥5 years) HRQOL by PedsQL 4.0 Generic Core and CF Questionnaire-revised (CFQ-R) prior to US and annually. HRQOL scores were compared by US pattern at baseline (prior to US), between baseline and 1 year and at 5 years. Multivariate analysis of variance (MANOVA) with Hotelling-Lawley trace tested for differences among US groups. Results: Prior to US, among 515 participants and their parents there was no evidence that HTG or NOD US was associated with reduced PedsQL/CFQ-R at baseline. Parents of NOD reported no change in PedsQL/CFQ-R over the next year. Child-report PedsQL/CFQ-R (95 NL, 20 NOD) showed improvement between baseline and year 5 for many scales, including Physical Function. Parents of HMG children reported improved CFQ-R scores related to weight. Conclusions: Early undiagnosed or pre-symptomatic liver disease had no impact on generic or disease-specific HRQoL, and HRQoL was remarkably stable in children with CF regardless of liver involvement.

AB - Objectives: Cystic fibrosis liver disease (CFLD) begins early in life. Symptoms may be vague, mild, or nonexistent. Progressive liver injury may be associated with decrements in patient health before liver disease is clinically apparent. We examined Health-Related Quality of Life (HRQOL) in children enrolled in a multi-center study of CFLD to determine the impact of early CFLD on general and disease-specific QOL. Methods: Ultrasound (US) patterns of normal (NL), heterogeneous (HTG), homogeneous (HMG), or nodular (NOD) were assigned in a prospective manner to predict those at risk for advanced CFLD. Parents were informed of results. We assessed parent/child-reported (age ≥5 years) HRQOL by PedsQL 4.0 Generic Core and CF Questionnaire-revised (CFQ-R) prior to US and annually. HRQOL scores were compared by US pattern at baseline (prior to US), between baseline and 1 year and at 5 years. Multivariate analysis of variance (MANOVA) with Hotelling-Lawley trace tested for differences among US groups. Results: Prior to US, among 515 participants and their parents there was no evidence that HTG or NOD US was associated with reduced PedsQL/CFQ-R at baseline. Parents of NOD reported no change in PedsQL/CFQ-R over the next year. Child-report PedsQL/CFQ-R (95 NL, 20 NOD) showed improvement between baseline and year 5 for many scales, including Physical Function. Parents of HMG children reported improved CFQ-R scores related to weight. Conclusions: Early undiagnosed or pre-symptomatic liver disease had no impact on generic or disease-specific HRQoL, and HRQoL was remarkably stable in children with CF regardless of liver involvement.

KW - cirrhosis

KW - cystic fibrosis

KW - functional outcomes

KW - health-related quality of life

KW - nodular liver

KW - patient reported outcomes

KW - wellness

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U2 - 10.1097/MPG.0000000000003605

DO - 10.1097/MPG.0000000000003605

M3 - Article

C2 - 36070552

AN - SCOPUS:85141003651

SN - 0277-2116

VL - 75

SP - 635

EP - 642

JO - Journal of pediatric gastroenterology and nutrition

JF - Journal of pediatric gastroenterology and nutrition

IS - 5

ER -

Health-related Quality of Life in a Prospective Study of Ultrasound to Detect Cystic Fibrosis-related Liver Disease in Children

Abstract

Funding

Keywords

ASJC Scopus subject areas

UN SDGs

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