Assessing the true burden of early-onset dystonia: A pilot study to evaluate the health related quality of life and the impact of dystonia severity and pain.

Eggink, H. (Wieke) (2013) Assessing the true burden of early-onset dystonia: A pilot study to evaluate the health related quality of life and the impact of dystonia severity and pain. thesis, Medicine.

Text EgginkW.pdf Restricted to Registered users only Download (1MB)

Abstract

Background: Dystonia is a hyperkinetic movement disorder that may manifest before the age of 26, also referred to as early-onset dystonia. The disease course varies greatly and may lead to severe motor impairment interfering with multiple domains of development. Current treatment strategies are mainly directed to motor functioning, despite the evidence suggests dystonia severity does not adequately reflect the perceived quality of life. Moreover, non-motor features such as psychiatric disorders and pain are recognised in adults with dystonia accounting for a significant lower health related quality of life (HRQOL). Experts believe treatment of dystonia should be focussed on the aspects that are important to the patient and caregiver. Despite this opinion, little is known about HRQOL in early onset dystonia, as well as about factors that influence the perceived burden. Objective: To analyse the HRQOL in early-onset dystonia patients and their caregivers and in addition, to identify the impact of dystonia severity and extent of pain on HRQOL. Methods: A pilot study was conducted in dystonia patients aged 8-25years between January and April 2013. Patients were recruited from the Neurology and Rehabilitation outpatient clinics of the University Medical Center Groningen. The primary HRQOL measure was the parent-proxy version of the generic Pediatric Quality of Life Inventory (PedsQL). In addition, self-report PedsQL and a disease-specific Caregiver Priorities & Child Health Index of Life with Disabilities (CPCHILD) was used. Dystonia severity was measured by the Burke-Fahn-Marsden dystonia rating scale (BFMDRS) and pain with the Wong-Baker FACES (≤8years) or the Numeric Rating Scale (>8years). Pearson correlation or Spearman’s rho were used to assess the association between the parent-proxy and self-report versions of the PedsQL, the two HRQOL measures (Parent-proxy PedsQL vs CPCHILD) and concordance of dystonia severity and pain with HRQOL. Linear (multiple) regressions were performed to identify the impact of motor severity and pain on HRQOL. Results: A total of 15 patients were included (mean age 13.9 years, 11 male). PedsQL scores were impaired in total HRQOL as well as motor and psychosocial sub domains. Correlation between parent-proxy and self-report versions were moderate to very strong (p<0.01). Dystonia severity was solely related to the physical functioning (r=0.725, p=0.002) and did not significantly total HRQOL (14%, p=0.092). An increasing extent of pain was moderately to very strongly associated with decreased scores on all sub domains and total HRQOL (p<0.05) and resulted in a significant predictor of total HRQOL (65%, p<0.001). Total parent-proxy PedsQL and CPCHILD scores did correlate well for ambulant patients ( =0.787, p=0.01), but there was no correlation in non-ambulant patients ( =-0.464; p=0.177). The dystonia severity and pain together accounted for a significant amount of the variance in total CPCHILD score (85%, p<0.001) Conclusion: Early-onset dystonia causes marked impairments in both motor and psychosocial domains, as well as total HRQOL. This perceived burden is significantly influenced by the extent of pain, whereas the role of dystonia severity tends to depend on the HRQOL measure used. The contribution of both predictors did not explain the entire variance of HRQOL. Next to pain and dystonia severity, additional non-motor features may play an important role in the perception of the impact of dystonia on the life of patients and their caregivers.

Item Type:	Thesis (Thesis)
Supervisor name:	Koning-Tijssen, Professor doctor M.A.J. de
Faculty:	Medical Sciences
Date Deposited:	25 Jun 2020 11:06
Last Modified:	25 Jun 2020 11:06
URI:	https://umcg.studenttheses.ub.rug.nl/id/eprint/2631

Actions (login required)

View Item