Dataset Information

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

ABSTRACT:

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.

SUBMITTER: Usuba K

PROVIDER: S-EPMC6611476 | biostudies-literature | 2019 Jul

REPOSITORIES: biostudies-literature

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Publications

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

Usuba Koyo K Price Victoria E VE Blanchette Victor V Abad Audrey A Altisent Carmen C Buchner-Daley Loretta L Carneiro Jorge D A JDA Feldman Brian M BM Fischer Kathelijn K Grainger John J Holzhauer Susanne S Luke Koon-Hung KH Meunier Sandrine S Ozelo Margareth M Tang Ling L Antunes Sandra V SV Villaça Paula P Wakefield Cindy C Wharfe Gilian G Wu Runhui R Young Nancy L NL

Research and practice in thrombosis and haemostasis 20190423 3

<h4>Background</h4>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.<h4>Methods</h4>The aim of this study was to assess the impact of hemophilia severity and treatment regimen on ...[more]

PMID: 31294327

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Project description:STUDY QUESTION:How has the timing of women's reproductive events (including ages at menarche, first birth, and natural menopause, and the number of children) changed across birth years, racial/ethnic groups and educational levels? SUMMARY ANSWER:Women who were born in recent generations (1970-84 vs before 1930) or those who with higher education levels had menarche a year earlier, experienced a higher prevalence of nulliparity and had their first child at a later age. WHAT IS KNOWN ALREADY:The timing of key reproductive events, such as menarche and menopause, is not only indicative of current health status but is linked to the risk of adverse hormone-related health outcomes in later life. Variations of reproductive indices across different birth years, race/ethnicity and socioeconomic positions have not been described comprehensively. STUDY DESIGN, SIZE, DURATION:Individual-level data from 23 observational studies that contributed to the International Collaboration for a Life Course Approach to Reproductive Health and Chronic Disease Events (InterLACE) consortium were included. PARTICIPANTS/MATERIALS, SETTING, METHODS:Altogether 505 147 women were included. Overall estimates for reproductive indices were obtained using a two-stage process: individual-level data from each study were analysed separately using generalised linear models. These estimates were then combined using random-effects meta-analyses. MAIN RESULTS AND THE ROLE OF CHANCE:Mean ages were 12.9 years at menarche, 25.7 years at first birth, and 50.5 years at natural menopause, with significant between-study heterogeneity (I2 > 99%). A linear trend was observed across birth year for mean age at menarche, with women born from 1970 to 1984 having menarche one year earlier (12.6 years) than women born before 1930 (13.5 years) (P for trend = 0.0014). The prevalence of nulliparity rose progressively from 14% of women born from 1940-49 to 22% of women born 1970-84 (P = 0.003); similarly, the mean age at first birth rose from 24.8 to 27.3 years (P = 0.0016). Women with higher education levels had fewer children, later first birth, and later menopause than women with lower education levels. After adjusting for birth year and education level, substantial variation was present for all reproductive events across racial/ethnic/regional groups (all P values < 0.005). LIMITATIONS, REASONS FOR CAUTION:Variations of study design, data collection methods, and sample selection across studies, as well as retrospectively reported age at menarche, age at first birth may cause some bias. WIDER IMPLICATIONS OF THE FINDINGS:This global consortium study found robust evidence on variations in reproductive indices for women born in the 20th century that appear to have both biological and social origins. STUDY FUNDING/COMPETING INTEREST(S):InterLACE project is funded by the Australian National Health and Medical Research Council project grant (APP1027196). GDM is supported by the Australian National Health and Medical Research Council Principal Research Fellowship (APP1121844).

Dataset Information

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

Background

Methods

Results

Conclusions

Publications

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

Similar Datasets

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