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

*Corresponding author for this work

Research output: Contribution to journalArticleAcademicpeer-review

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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 languageEnglish
Article numbere29358
JournalPediatric Blood and Cancer
Issue number1
Early online date26 Oct 2021
Publication statusPublished - Jan 2022

Bibliographical 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 ‘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


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