The Jamaican Haemophilia Registry: Describing the burden of disease

Wharfe, Gilian; Buchner-Daley, L M; Gibson, TN; Hilliard, Pamela; Usuba, Koyo; Abad, Audrey; Boma-Fischer, Laurence; Bouskill, Vanessa; Lillicrap, David; Lowe, Y; Lowe, Dwight; Palmer-Mitchell, N; Rand, Margaret L.; Teitel, Jerome; Tuttle, Angie; Watson, Amanda; White, Rebecca; Young, Nancy L.; Blanchette, Victor S.

doi:10.1111/hae.13517

Cited by 7 publications

(10 citation statements)

References 26 publications

(47 reference statements)

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“…Therefore, the data may not be representative for the entire US population. The HAL scores in the current data (median HAL sum: 65.0) were comparable to HAL scores in PWH from Jamaica (median: 66.1) and Brazil (weighted mean: 66.4), but lower than HAL scores in PWH from the United Kingdom (median: 80) and the Netherlands (median: 96) 18–21 . In Sweden, PWH with a later onset of treatment showed a median HAL sum score of 56, compared to a median of 98 in PWH with early treatment 22 .…”

Section: Discussionsupporting

confidence: 51%

Shortening the Haemophilia Activities List (HAL) from 42 items to 18 items

et al. 2021

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Introduction:The Haemophilia Activities List (HAL) was developed to measure activities and participation in persons with haemophilia (PWH). Shortening the questionnaire may facilitate use of the HAL. Aim:The aim of this study was to determine which items of the HAL are redundant, to construct a shorter version of the HAL, and to determine the construct validity of the HAL short .Methods: A secondary analysis was performed on pooled data of two published studies using the HAL (seven domains, 42 items, optimum score: 100) in adults with haemophilia A/B. Data were divided into a derivation (62%) and a validation set (38%).Redundant items were identified by evaluation of: 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. Correlations with the SF-36 and EQ-5D-5L were used to determine construct validity of the HAL short .Results: Data on 680 PWH were evaluated. In the derivation dataset (n = 420), median age was 30 years (range 18-80), 43% had severe haemophilia and 61% received prophylaxis. Median (IQR) HAL sum score was 65.0 (55.7-88.8). The stepwise procedure resulted in a HAL short of 18 items with a median sum score of 63.3 (54.4-86.7). Construct validity was similar for the HAL and HAL short in the validation dataset (n = 260). Conclusion:This clinimetric study resulted in a >50% shortening of the HAL. The 18item HAL short reduces patient burden and is expected to capture the information on activities and participation. The HAL short needs further validation.

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Section: Discussionsupporting

confidence: 51%

Shortening the Haemophilia Activities List (HAL) from 42 items to 18 items

et al. 2021

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“…This is a secondary analysis of pooled data, using comparable variables, from 7 studies in 9 countries that measured HRQoL using the CHO‐KLAT . The countries, representing cultural and economic diversity, included Brazil, Canada, China, France, Germany, Jamaica, the Netherlands, Spain, and the United Kingdom.…”

Section: Methodsmentioning

confidence: 99%

“…The Canadian Hemophilia Outcomes–Kids Life Assessment Tool (CHO‐KLAT) is a disease‐specific HRQoL instrument validated for use in boys 4‐18 years of age. It has been used in a number of clinical studies and was found to be sensitive to clinically important changes in the setting of the use of factor (F)VIII/FIX concentrates for the management of boys with hemophilia . Pooled CHO‐KLAT data from clinical studies in 9 countries provided a sample size large enough to allow evaluation of the impact of treatment on HRQoL while controlling for confounding relationships.…”

Section: Introductionmentioning

confidence: 99%

Impact of prophylaxis on health‐related quality of life of boys with hemophilia: An analysis of pooled data from 9 countries

Usuba

Price

Blanchette

et al. 2019

Research and Practice in Thrombosis and Haemostasis

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Background Prophylaxis reduces the frequency of bleeds in boys with severe hemophilia and is the standard care for their management in resource‐abundant countries. The effect of prophylaxis on Health‐Related Quality of Life (HRQoL) has not been established, because the sample sizes of most studies are too small to explore the relationship of multiple factors that influence HRQoL. Methods The aim of this study was to assess the impact of hemophilia severity and treatment regimen on HRQoL and to establish the minimum important difference (MID) using the international level of score distributions. HRQoL data were pooled from 7 studies across 9 countries. HRQoL was measured using the Canadian Hemophilia Outcomes–Kids’ Life Assessment Tool (CHO‐KLAT). A mixed‐effect linear regression analysis was employed to assess the impact of prophylaxis on the CHO‐KLAT score. Results Data from 401 boys with hemophilia were analyzed (57.6% severe hemophilia and 57.6% receiving prophylaxis). The model revealed that receiving prophylaxis was significantly associated with higher HRQoL (regression coefficient 8.5, 95% confidence interval [CI] 3.9‐13.1). Boys with severe hemophilia had a significantly lower HRQoL as compared to boys with moderate and mild hemophilia whose CHO‐KLAT scores were 7.0 and 6.6 points higher, respectively. There was a significant interaction between treatment and disease severity (P = 0.023), indicating prophylaxis has the most significant impact in boys with severe hemophilia. Based on these pooled data, the MID of the CHO‐KLAT was established at 6.5. Conclusions This study confirms the positive effect of prophylaxis on HRQoL in boys with hemophilia in a real‐world setting and provides initial benchmarks for interpreting HRQoL scores based on use of the CHO‐KLAT instrument.

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“…Several items in the Hemophilia Activities List (HAL), for example, are not applicable in India and Jamaica, while the Functional Independence Score in Hemophilia (FISH), an instrument developed in India, performs well in these countries. 38,39 Similarly, the FISH shows ceiling effects and fails to detect early changes in joint health in high-income countries with early prophylaxis. 40 Health care organizations may choose the tool that is most appropriate and feasible in their situation.…”

Section: Health Outcomementioning

confidence: 99%