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. 2021 Mar;27(2):305-313.
doi: 10.1111/hae.14241. Epub 2021 Feb 16.

Shortening the paediatric Haemophilia Activities List (pedHAL) based on pooled data from international studies

Isolde A R Kuijlaars  1 , Janjaap van der Net  2 , Vanessa Bouskill  3 , Pamela Hilliard  4 , Agne Juodyte  5 , Kate Khair  6 , Sonata Saulyte Trakymiene  5 , Kathelijn Fischer  1
Affiliations

Shortening the paediatric Haemophilia Activities List (pedHAL) based on pooled data from international studies

Isolde A R Kuijlaars et al. Haemophilia. 2021 Mar.

Abstract

Introduction: The paediatric Haemophilia Activities List (pedHAL) was developed to measure activities and participation in children and youth with haemophilia. Results from international studies provide an opportunity to determine which items are universally important.

Aim: The aim of this study was to determine which items of the pedHAL are redundant to construct a shorter version of the pedHAL.

Methods: This study is a cross-sectional multicentre secondary analysis on pooled data of published studies using the pedHAL (7 domains, 53 items, optimum score: 100) in children with haemophilia A/B aged 4-18 years. To identify redundant items, the following aspects were evaluated: floor and ceiling effects, proportions of missing and 'not applicable' responses, inter-item correlations, component loadings in an exploratory factor analysis, internal consistency and item-total correlations.

Results: Data on 315 patients with haemophilia from 6 studies were evaluated. Median age was 12.2 years) (range 4.0-18.0), 87.3% had severe haemophilia and 80.3% received prophylaxis. Median (IQR) pedHAL sum score was 96.7 (88.0-100). After a stepwise procedure, 31 items were removed, resulting in a pedHALshort of 22 items, representing all original 7 domains. Most remaining items belonged to the domains 'sitting/kneeling/standing' and 'functions of the legs'. The pedHALshort sum score was similar to the original pedHAL sum score, with small differences in 5 domains.

Conclusion: This clinimetric study resulted in >50% reduction of the length of the pedHAL. The 22-item pedHALshort reduces patient burden and is expected to capture the information on activities and participation. The pedHALshort needs validation in other populations.

Keywords: activities; haemophilia; participation; patient-reported outcome.

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Conflict of interest statement

K. Fischer has received speaker's fees from Bayer, Baxter/Shire, SOBI/Biogen, CSL Behring, Octapharma, Pfizer, NovoNordisk; performed consultancy for Bayer, Baxter, Biogen, CSL‐Behring, Freeline, NovoNordisk, Pfizer, Roche and SOBI; and has received research support from Bayer, Pfizer, Baxter/Shire, and Novo Nordisk, Biogen. K Fischer is the epidemiologist for the EUHASS and PedNet registries. K. Fischer is member of the group that developed the pedHAL. IAR Kuijlaars and J. van der Net do not have any conflict of interest regarding this manuscript other than membership of the group that developed the pedHAL. The other authors have no competing interests.

Figures

FIGURE 1
FIGURE 1
Box plots of the differences in pedHAL and pedHALshort scores for patients with three different treatment regimens.
See this image and copyright information in PMC

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