Knowledge gaps in health-related quality of life research performed in children with bleeding disorders - A scoping review
© 2024 The Authors. Haemophilia published by John Wiley & Sons Ltd..
INTRODUCTION: Bleeding disorders (BDs) may influence health-related quality of life (HRQoL) in children and caregivers. Measuring HRQoL gives insight into domains requiring support and provides an opportunity to evaluate the effects of novel therapies.
AIM: To gain insight in the current body of literature on HRQoL in children with BDs in order to identify knowledge gaps for research and further development of this field.
METHODS: Scoping review.
RESULTS: We included 53 articles, describing studies mainly performed in Europe and North-America (60.4%) and mostly within the last ten years. Only 32% studies included children <4 years. Almost all studies (47/53, 88.7%) were performed in boys with haemophilia, pooling haemophilia A and B (n = 21) and different disease severities (n = 20). Thirteen different generic and five disease-specific HRQoL-questionnaires were applied; all questionnaires were validated for haemophilia specifically. Six (11,3%) combined generic and disease-specific questionnaires. Self-reports were most frequently applied (40/53, 75.5%), sometimes combined with proxy and/or parent-reports (17/53, 32.1%). Eleven studies used a reference group (20.8%). Statistical analyses mostly consisted of mean and SD (77.4%).
CONCLUSION: HRQoL-research is mainly performed in school-aged boys with haemophilia, treated in developed countries. Pitfalls encountered are the pooling of various BDs, subtypes and severities, as well as the application of multiple generic questionnaires prohibiting comparison of results. More attention is needed for broader study populations including other BDs, young children, feminine bleeding issues and platelet disorders, as well as the use of HRQoL as an effect-measurement tool for medical interventions, and more thorough statistical analysis.
| Medienart: |
E-Artikel |
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| Erscheinungsjahr: |
2024 |
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| Erschienen: |
2024 |
| Enthalten in: |
Zur Gesamtaufnahme - volume:30 |
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| Enthalten in: |
Haemophilia : the official journal of the World Federation of Hemophilia - 30(2024), 2 vom: 15. März, Seite 295-305 |
| Sprache: |
Englisch |
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| Beteiligte Personen: |
Huisman, Elise J [VerfasserIn] |
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| Links: |
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| Themen: |
Blood coagulation disorders [MESH] |
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| Anmerkungen: |
Date Completed 14.03.2024 Date Revised 14.03.2024 published: Print-Electronic Citation Status MEDLINE |
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| doi: |
10.1111/hae.14941 |
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| funding: |
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| Förderinstitution / Projekttitel: |
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| PPN (Katalog-ID): |
NLM368066983 |
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| 520 | |a © 2024 The Authors. Haemophilia published by John Wiley & Sons Ltd. | ||
| 520 | |a INTRODUCTION: Bleeding disorders (BDs) may influence health-related quality of life (HRQoL) in children and caregivers. Measuring HRQoL gives insight into domains requiring support and provides an opportunity to evaluate the effects of novel therapies | ||
| 520 | |a AIM: To gain insight in the current body of literature on HRQoL in children with BDs in order to identify knowledge gaps for research and further development of this field | ||
| 520 | |a METHODS: Scoping review | ||
| 520 | |a RESULTS: We included 53 articles, describing studies mainly performed in Europe and North-America (60.4%) and mostly within the last ten years. Only 32% studies included children <4 years. Almost all studies (47/53, 88.7%) were performed in boys with haemophilia, pooling haemophilia A and B (n = 21) and different disease severities (n = 20). Thirteen different generic and five disease-specific HRQoL-questionnaires were applied; all questionnaires were validated for haemophilia specifically. Six (11,3%) combined generic and disease-specific questionnaires. Self-reports were most frequently applied (40/53, 75.5%), sometimes combined with proxy and/or parent-reports (17/53, 32.1%). Eleven studies used a reference group (20.8%). Statistical analyses mostly consisted of mean and SD (77.4%) | ||
| 520 | |a CONCLUSION: HRQoL-research is mainly performed in school-aged boys with haemophilia, treated in developed countries. Pitfalls encountered are the pooling of various BDs, subtypes and severities, as well as the application of multiple generic questionnaires prohibiting comparison of results. More attention is needed for broader study populations including other BDs, young children, feminine bleeding issues and platelet disorders, as well as the use of HRQoL as an effect-measurement tool for medical interventions, and more thorough statistical analysis | ||
| 650 | 4 | |a Journal Article | |
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| 700 | 1 | |a Cnossen, Marjon H |e verfasserin |4 aut | |
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