Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
Multicenter Study
. 2014 Aug;29 Suppl 3(Suppl 3):S760-6.
doi: 10.1007/s11606-014-2893-y.

Longitudinal changes in health-related quality of life for chronic diseases: an example in hemophilia A

Jiat-Ling Poon  1 , Jason N Doctor, Michael B Nichol
Affiliations
Multicenter Study

Longitudinal changes in health-related quality of life for chronic diseases: an example in hemophilia A

Jiat-Ling Poon et al. J Gen Intern Med. 2014 Aug.

Abstract

Background: Patients with well-managed rare chronic diseases such as hemophilia maintain a stable health state and health-related quality of life (HrQoL) that may be affected by acute events. Longitudinal HrQoL assessments analyzed using multivariate multilevel (MVML) modelling can determine the impact of such events on individuals (within-person effect) and identify factors influencing within-population differences (between-person effect).

Objectives: To demonstrate the application of MVML modelling in a longitudinal study of HrQoL in hemophilia A.

Methods/design/participants: Using data on 136 adults and 125 children from a two-year observational cohort study of burden of illness in US hemophilia A patients, MVML modelling determined the effect of time-invariant (sociodemographic and clinical characteristics) and time-varying factors (bleeding frequency, emergency room visits, and missed work/school days) on within-person and between-person HrQoL changes. HrQoL was assessed using the SF-12 health survey (adults) and PedsQL inventory (children) at baseline, then every 6 months.

Results: In children, within-person (p < 0.0001) and between-person (p < 0.0001) psychosocial functioning was reduced by each additional bleed and missed day (within-person: p = 0.0089; between-person: p = 0.0060). Within-person physical functioning was reduced by each additional bleed (p < 0.0001), emergency room (ER) visit (p = 0.0284), and missed day (p = 0.0473). Between-persons, additional missed days (p < 0.0001) significantly decreased physical functioning. In adults, each additional missed day reduced SF-12 Health Survey mental (p = 0.0025) and physical (p = 0.0093) component summary scores. Each additional bleed also decreased physical component summary (PCS) significantly (p = 0.0093).

Conclusions: This study demonstrated the applicability of MVML modelling in identifying time-invariant and time-varying factors influencing HrQoL in a rare chronic disease population. Small but significant within-person and between-person changes in HrQoL with each additional acute event experienced were identified, which if frequent, could have a large cumulative impact. The results suggest that MVML modelling may be applied to future studies of longitudinal change in HrQoL in other rare chronic disease populations.

PubMed Disclaimer

Figures

Figure 1
Figure 1
Adults—Study population mean and 95 % confidence interval* for follow-up HrQoL scores (N = 136). *Arrows denote 95 % confidence interval.
Figure 2
Figure 2
Children—Study population mean and 95 % confidence interval* for follow-up HrQoL scores (N = 125). *Arrows denote 95 % confidence interval.
See this image and copyright information in PMC

References

    1. World Federation of Hemophilia. Guidelines for the Management of Hemophilia: World Federation of Hemophilia; 2005.
    1. Choiniere M, Melzack R. Acute and chronic pain in hemophilia. Pain. 1987;31(3):317–331. doi: 10.1016/0304-3959(87)90161-8. - DOI - PubMed
    1. Gringeri A, Mantovani LG, Scalone L, Mannucci PM. Cost of care and quality of life for patients with hemophilia complicated by inhibitors: the COCIS Study Group. Blood. 2003;102(7):2358–2363. doi: 10.1182/blood-2003-03-0941. - DOI - PubMed
    1. Lindvall K, Von Mackensen S, Berntorp E. Quality of life in adult patients with haemophilia—a single centre experience from Sweden. Haemophilia. 2012;18(4):527–531. doi: 10.1111/j.1365-2516.2012.02765.x. - DOI - PubMed
    1. Solovieva S. Clinical severity of disease, functional disability and health-related quality of life. Three-year follow-up study of 150 Finnish patients with coagulation disorders. Haemophilia. 2001;7(1):53–63. doi: 10.1046/j.1365-2516.2001.00476.x. - DOI - PubMed

Publication types

LinkOut - more resources