Health-related quality of life in infants, toddlers and young children with sickle cell disease

Maite E. Houwing; Madieke J. Muntendam; Maud M. van Muilekom; Lorynn Teela; Karin Fijnvandraat; Anne P. J. de Pagter; Henriëtte Heijboer; Hedy A. van Oers; Marjon H. Cnossen; Lotte Haverman

doi:https://doi.org/10.1002/pbc.29358

Health-related quality of life in infants, toddlers and young children with sickle cell disease

Maite E. Houwing, Madieke J. Muntendam, Maud M. van Muilekom, Lorynn Teela, Karin Fijnvandraat, Anne P. J. de Pagter, Henriëtte Heijboer, Hedy A. van Oers, Marjon H. Cnossen, Lotte Haverman

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

6 Citations (Scopus)

Abstract

Background: Little is known about health-related quality of life (HRQoL) in young children with sickle cell disease living in a European country. Methods: A retrospective cross-sectional evaluation of TNO-AZL Preschool Children Quality of Life questionnaire (TAPQOL, 0–1 year) and Pediatric Quality of Life Inventory (PedsQL, 2–7 years) data was conducted. Study participants included caregivers of children with sickle cell disease aged 0–7 years attending the sickle cell centre at the Erasmus Medical Center or the Amsterdam University Medical Centers between April 2012 and October 2020. Comparisons were made with normative data on HRQoL in the general paediatric population. Results: The study enrolled 136 caregivers of 136 children. In children aged 0–5 years, no significant differences emerged between children with sickle cell disease and the general population. However, in children aged 5–7 years, children with sickle cell disease scored significantly lower on all subscales except for emotional functioning. Multiple regression models showed a negative association between age and HRQoL. No association was found between HRQoL and disease severity or sociodemographic characteristics. Conclusions: This study demonstrates that HRQoL is negatively correlated with age in young children with sickle cell disease with a significantly lower HRQoL in 5- to 7-year-olds when compared to the general population. Our study underlines the importance of measuring HRQoL in young children to identify patients with impaired HRQoL early in life in order to be able to intervene accordingly. Future research should focus on deepening the knowledge of factors influencing HRQoL in children with sickle cell disease.

Original language	English
Article number	e29358
Journal	Pediatric Blood and Cancer
Volume	69
Issue number	1
Early online date	2021
DOIs	https://doi.org/10.1002/pbc.29358
Publication status	Published - Jan 2022

Keywords

child
infant
patient-reported outcomes
quality of life
sickle cell disease

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@article{b34cff318fc2480a8bf0580eaf5fdb58,

title = "Health-related quality of life in infants, toddlers and young children with sickle cell disease",

abstract = "Background: Little is known about health-related quality of life (HRQoL) in young children with sickle cell disease living in a European country. Methods: A retrospective cross-sectional evaluation of TNO-AZL Preschool Children Quality of Life questionnaire (TAPQOL, 0–1 year) and Pediatric Quality of Life Inventory (PedsQL, 2–7 years) data was conducted. Study participants included caregivers of children with sickle cell disease aged 0–7 years attending the sickle cell centre at the Erasmus Medical Center or the Amsterdam University Medical Centers between April 2012 and October 2020. Comparisons were made with normative data on HRQoL in the general paediatric population. Results: The study enrolled 136 caregivers of 136 children. In children aged 0–5 years, no significant differences emerged between children with sickle cell disease and the general population. However, in children aged 5–7 years, children with sickle cell disease scored significantly lower on all subscales except for emotional functioning. Multiple regression models showed a negative association between age and HRQoL. No association was found between HRQoL and disease severity or sociodemographic characteristics. Conclusions: This study demonstrates that HRQoL is negatively correlated with age in young children with sickle cell disease with a significantly lower HRQoL in 5- to 7-year-olds when compared to the general population. Our study underlines the importance of measuring HRQoL in young children to identify patients with impaired HRQoL early in life in order to be able to intervene accordingly. Future research should focus on deepening the knowledge of factors influencing HRQoL in children with sickle cell disease.",

keywords = "child, infant, patient-reported outcomes, quality of life, sickle cell disease",

author = "Houwing, {Maite E.} and Muntendam, {Madieke J.} and {van Muilekom}, {Maud M.} and Lorynn Teela and Karin Fijnvandraat and {de Pagter}, {Anne P. J.} and Henri{\"e}tte Heijboer and {van Oers}, {Hedy A.} and Cnossen, {Marjon H.} and Lotte Haverman",

note = "Funding Information: This investigator‐initiated research project was partially funded by Novartis and by the Rotary Foundation. Funding Information: Dr. Marjon H. Cnossen's institution has received investigator‐initiated research and travel grants as well as speaker fees over the years from The Netherlands Organisation for Scientific Research (NWO), the Netherlands Organisation for Health Research and Development (ZonMw), the Dutch {\textquoteleft}Innovatiefonds Zorgverzekeraars{\textquoteright}, Pfizer, Baxter/Baxalta/Shire, Bayer Schering Pharma, CSL Behring, Sobi and Biogen Idec, Novo Nordisk, Novartis and Nordic Pharma, and for serving as a steering board member for f Roche, Bayer and Novartis for which fees go to the Erasmus MC as an institution. Dr. Anne P. J. de Pagter has received a grant from Rotary Foundation for the institution. All other authors declare no conflict of interest relevant to the contents of this manuscript. Publisher Copyright: {\textcopyright} 2021 The Authors. Pediatric Blood & Cancer published by Wiley Periodicals LLC",

year = "2022",

month = jan,

doi = "https://doi.org/10.1002/pbc.29358",

language = "English",

volume = "69",

journal = "Pediatric Blood and Cancer",

issn = "1545-5009",

publisher = "Wiley-Liss Inc.",

number = "1",

}

TY - JOUR

T1 - Health-related quality of life in infants, toddlers and young children with sickle cell disease

AU - Houwing, Maite E.

AU - Muntendam, Madieke J.

AU - van Muilekom, Maud M.

AU - Teela, Lorynn

AU - Fijnvandraat, Karin

AU - de Pagter, Anne P. J.

AU - Heijboer, Henriëtte

AU - van Oers, Hedy A.

AU - Cnossen, Marjon H.

AU - Haverman, Lotte

N1 - Funding Information: This investigator‐initiated research project was partially funded by Novartis and by the Rotary Foundation. Funding Information: Dr. Marjon H. Cnossen's institution has received investigator‐initiated research and travel grants as well as speaker fees over the years from The Netherlands Organisation for Scientific Research (NWO), the Netherlands Organisation for Health Research and Development (ZonMw), the Dutch ‘Innovatiefonds Zorgverzekeraars’, Pfizer, Baxter/Baxalta/Shire, Bayer Schering Pharma, CSL Behring, Sobi and Biogen Idec, Novo Nordisk, Novartis and Nordic Pharma, and for serving as a steering board member for f Roche, Bayer and Novartis for which fees go to the Erasmus MC as an institution. Dr. Anne P. J. de Pagter has received a grant from Rotary Foundation for the institution. All other authors declare no conflict of interest relevant to the contents of this manuscript. Publisher Copyright: © 2021 The Authors. Pediatric Blood & Cancer published by Wiley Periodicals LLC

PY - 2022/1

Y1 - 2022/1

N2 - Background: Little is known about health-related quality of life (HRQoL) in young children with sickle cell disease living in a European country. Methods: A retrospective cross-sectional evaluation of TNO-AZL Preschool Children Quality of Life questionnaire (TAPQOL, 0–1 year) and Pediatric Quality of Life Inventory (PedsQL, 2–7 years) data was conducted. Study participants included caregivers of children with sickle cell disease aged 0–7 years attending the sickle cell centre at the Erasmus Medical Center or the Amsterdam University Medical Centers between April 2012 and October 2020. Comparisons were made with normative data on HRQoL in the general paediatric population. Results: The study enrolled 136 caregivers of 136 children. In children aged 0–5 years, no significant differences emerged between children with sickle cell disease and the general population. However, in children aged 5–7 years, children with sickle cell disease scored significantly lower on all subscales except for emotional functioning. Multiple regression models showed a negative association between age and HRQoL. No association was found between HRQoL and disease severity or sociodemographic characteristics. Conclusions: This study demonstrates that HRQoL is negatively correlated with age in young children with sickle cell disease with a significantly lower HRQoL in 5- to 7-year-olds when compared to the general population. Our study underlines the importance of measuring HRQoL in young children to identify patients with impaired HRQoL early in life in order to be able to intervene accordingly. Future research should focus on deepening the knowledge of factors influencing HRQoL in children with sickle cell disease.

AB - Background: Little is known about health-related quality of life (HRQoL) in young children with sickle cell disease living in a European country. Methods: A retrospective cross-sectional evaluation of TNO-AZL Preschool Children Quality of Life questionnaire (TAPQOL, 0–1 year) and Pediatric Quality of Life Inventory (PedsQL, 2–7 years) data was conducted. Study participants included caregivers of children with sickle cell disease aged 0–7 years attending the sickle cell centre at the Erasmus Medical Center or the Amsterdam University Medical Centers between April 2012 and October 2020. Comparisons were made with normative data on HRQoL in the general paediatric population. Results: The study enrolled 136 caregivers of 136 children. In children aged 0–5 years, no significant differences emerged between children with sickle cell disease and the general population. However, in children aged 5–7 years, children with sickle cell disease scored significantly lower on all subscales except for emotional functioning. Multiple regression models showed a negative association between age and HRQoL. No association was found between HRQoL and disease severity or sociodemographic characteristics. Conclusions: This study demonstrates that HRQoL is negatively correlated with age in young children with sickle cell disease with a significantly lower HRQoL in 5- to 7-year-olds when compared to the general population. Our study underlines the importance of measuring HRQoL in young children to identify patients with impaired HRQoL early in life in order to be able to intervene accordingly. Future research should focus on deepening the knowledge of factors influencing HRQoL in children with sickle cell disease.

KW - child

KW - infant

KW - patient-reported outcomes

KW - quality of life

KW - sickle cell disease

UR - http://www.scopus.com/inward/record.url?scp=85118168265&partnerID=8YFLogxK

U2 - https://doi.org/10.1002/pbc.29358

DO - https://doi.org/10.1002/pbc.29358

M3 - Article

C2 - 34699122

SN - 1545-5009

VL - 69

JO - Pediatric Blood and Cancer

JF - Pediatric Blood and Cancer

IS - 1

M1 - e29358

ER -

Health-related quality of life in infants, toddlers and young children with sickle cell disease

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Keywords

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