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Botulinumtoxin Improves both Generic and Disease-Specific Quality of Life in Cervical Dystonia.

ABSTRACT: Botulinumtoxin injection (BoNT) into affected muscles is effective to improve motor symptoms of cervical dystonia (CD) by reducing muscle contraction and involuntary dystonic movement and posturing. However, the understanding of the effect on health-related quality of life (HR-QoL) and patient referral under HR-QoL aspects is incomplete. In this open-label clinical prospective observational study, we characterized the outcomes in CD (n = 159) from botulinumtoxin on both generic HR-QoL (EuroQol; EQ-5D-5L) and disease-specific HR-QoL [craniocervical dystonia questionnaire (CDQ-24)]. Additionally, we characterized motor and non-motor signs of dystonia including motor symptom improvement, depressive symptoms, pain, and sleep quality. We assessed patients at the end of a regular 3-month period from last injection (Timepoint1) and 4 weeks after the re-injection of BoNT (Timepoint2). We aimed to define outcomes on both generic and disease-specific HR-QoL and to evaluate predictors of therapeutic outcome in terms of stepwise multiple regression models. Patients with CD showed a robust improvement of both generic and disease-specific HR-QoL. Furthermore, motor and non-motor signs improved. Multiple regression analyses revealed that EQ-5D-5L and "satisfaction with health" (Fragen zur Lebenszufriedenheit-G) at Timepoint1 predicted treatment response on generic HR-QoL outcome (R2 = 0.284; P = 0.019). Similarly, CDQ-24 and Beck's Depression inventory at Timepoint1 predicted the treatment response on disease-specific HR-QoL (R2 = 0.253; P = 0.026). Our study underscores both generic and disease-specific HR-QoL improvements in CD, and provides useful predictors on HR-QoL outcomes.

SUBMITTER: Weiss D

PROVIDER: S-EPMC5660718 | biostudies-other | 2017

REPOSITORIES: biostudies-other

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Botulinumtoxin Improves both Generic and Disease-Specific Quality of Life in Cervical Dystonia.

Weiss Daniel D Hieber Leonhard L Sturm Justine J Börtlein Axel A Mayr Ingo I Appy Matthias M Kühnler Benedicta B Buchthal Joachim J Dippon Christian C Arnold Guy G Wächter Tobias T

Frontiers in neurology 20171024

Botulinumtoxin injection (BoNT) into affected muscles is effective to improve motor symptoms of cervical dystonia (CD) by reducing muscle contraction and involuntary dystonic movement and posturing. However, the understanding of the effect on health-related quality of life (HR-QoL) and patient referral under HR-QoL aspects is incomplete. In this open-label clinical prospective observational study, we characterized the outcomes in CD (<i>n</i> = 159) from botulinumtoxin on both generic HR-QoL (Eu ...[more]

PMID: 29114239

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Project description:BACKGROUND:This study was undertaken to explore the longitudinal patterns of health-related quality of life (HRQoL) among youth and young adults with Hemophilia A (HA) over a 3-year period. This report presents the baseline characteristics of the study cohort. METHODS:Males, 14 to 29 years of age, with predominantly severe HA were recruited from six treatment centres in Canada. Subjects completed a comprehensive survey. HRQoL was measured using: the CHO-KLAT2.0 (youth), Haemo-QoL-A (young adults) and the SF-36v2 (all). RESULTS:13 youth (mean age?=?15.7, range?=?12.9-17.9 years) and 33 young adults (mean age?=?23.6; range?=?18.4 -28.7 years) with moderate (7 %) and severe (93 %) HA were enrolled. All were on a prophylactic regimen with antihemophilic factor (Helixate FS®) during the study. The youth had minimal joint damage (mean HJHS?=?5.2) compared to young adults (mean HJHS?=?13.3). The mean HRQoL scores for youth were: 79.2 (SD?=?11.9) for the CHO-KLAT, and 53.0 (5.5) and 52.3 (6.8) for the SF-36 Physical Component Summary (PCS) and Mental Component Summary (MCS) scores respectively. The mean HRQoL scores for young adults were: 85.8 (9.5) for the Haemo-Qol-A, and 50.8 (6.4) and 50.9 (8.8) for PCS and MCS respectively. PCS and MCS scores were comparable to published Canadian norms, however significant differences were found for the domains of Physical Functioning and Bodily Pain. The disease-specific HRQoL scores were weakly correlated with the PCS for youth (CHO-KLAT vs. PCS r?=?0.28, p?=?0.35); and moderately correlated for the MCS (r?=?0.39, p?=?0.19). Haemo-QoL-A scores for young adults were strongly correlated with the PCS (r?=?0.53, p?=?0.001); and weakly correlated with the MCS (r?=?0.26, p?=?0.13). Joint status as assessed by HJHS was correlated with PCS scores. A history of lifelong prophylaxis resulted in better PCS but worse MCS scores. CONCLUSION:Despite having hemophilia, the youth in this cohort have minimal joint disease and good HRQoL. The young adults demonstrated more joint disease and slightly worse HRQoL in the domains of physical functioning and pain. The data presented here provide new information to inform the selection of Health Related Quality of Life (HRQoL) instruments for use in future clinical trials involving persons with hemophilia. TRIAL REGISTRATION:ClinicalTrials.gov : NCT01034904. Study funded by CSL Behring Canada.

Project description:BackgroundNonmotor symptoms (NMS) are highly prevalent in cervical dystonia (CD). In general, fatigue and sleep are important NMS that determine a decreased health-related quality of life (HR-QoL), but their influence in CD is unknown. The authors systematically investigated fatigue, excessive daytime sleepiness (EDS), and sleep quality in patients with CD and controls and assessed the influence of psychiatric comorbidity, pain, and dystonia motor severity. They also examined the predictors of HR-QoL.MethodsThe study included 44 patients with CD and 43 matched controls. Fatigue, EDS, and sleep quality were assessed with quantitative questionnaires and corrected for depression and anxiety using analysis of covariance. The Toronto Western Spasmodic Torticollis Rating Scale and the Clinical Global Impression Scale-jerks/tremor subscale were used to score motor severity and to assess whether motor characteristics could explain an additional part of the variation in fatigue and sleep-related measures. HR-QoL was determined with the RAND-36 item Health Survey, and predictors of HR-QoL were assessed using multiple regression.ResultsFatigue scores were increased independently from psychiatric comorbidity (4.0 vs. 2.7; P < 0.01), whereas EDS (7.3 vs. 7.4; P = 0.95) and sleep quality (6.5 vs. 6.1; P = 0.73) were highly associated with depression and anxiety. In patients with CD, motor severity did not explain the variations in fatigue (change in the correlation coefficient [ΔR2] = 0.06; P = 0.15), EDS (ΔR2 = 0.00; P = 0.96), or sleep quality (ΔR2 = 0.04; P = 0.38) scores. Fatigue, EDS, psychiatric comorbidity, and pain predicted a decreased QoL.ConclusionIndependent from psychiatric comorbidity and motor severity, fatigue appeared to be a primary NMS. Sleep-related measures were highly associated with psychiatric comorbidity, but not with motor severity. Only NMS predicted HR-QoL, which emphasizes the importance of attention to NMS in patients with CD.

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Botulinumtoxin Improves both Generic and Disease-Specific Quality of Life in Cervical Dystonia.

Publications

Botulinumtoxin Improves both Generic and Disease-Specific Quality of Life in Cervical Dystonia.

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