Craniocervical dystonia questionnaire (CDQ-24): development and validation of a disease-specific quality of life instrument

Müller, Jörg; Wissel, Jörg; Kemmler, Georg; Voller, Bernhard; Bodner, Todd; Schneider, Alessandra; Wenning, Gregor K.; Poewe, Werner

doi:10.1136/jnnp.2003.013441

Cited by 130 publications

(123 citation statements)

References 17 publications

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“…This instrument addresses the perceptions and concerns of the patients and it can be used to assess the impact of the disease in areas not covered by the Short Form-36 Health Survey (SF-36) 8,9 . Even as a generic measure of health status widely adopted to assess QoL 8 , the SF-36 has not been used in this study, since it has undergone a considerable amount of reliability tests and validity studies, proving to be impaired in patients with craniocervical dystonia 9 . The 24 questions of the CDQ-24 are divided in five areas: stigma (6), emotional wellbeing (5), pain (3), activities of daily living (6) and family/social life (4).…”

Section: Methodsmentioning

confidence: 99%

The physical, social and emotional aspects are the most affected in the quality of life of the patients with cervical dystonia

Werle

Takeda

Zonta

et al. 2014

Arq. Neuro-Psiquiatr.

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Section: Methodsmentioning

confidence: 99%

The physical, social and emotional aspects are the most affected in the quality of life of the patients with cervical dystonia

Werle

Takeda

Zonta

et al. 2014

Arq. Neuro-Psiquiatr.

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“…Disease-specific HR-QOL measures have been developed for patients suffering from neuropsychiatric conditions bearing similarities to GTS, including cervical dystonia [50] and hemifacial spasm [51]. However, until recently, there has been no tool to measure GTS-specific HR-QOL.…”

Section: The Gts-qol: a Disease-specific Hr-qol Measurementioning

confidence: 99%

Health-Related Quality of Life in Gilles de la Tourette Syndrome: A Decade of Research

Cavanna

David

Bandera

et al. 2013

Behavioural Neurology

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Abstract. Gilles de la Tourette syndrome (GTS) is a neurodevelopmental condition characterised by multiple motor and phonic tics and associated behavioural problems, carrying a significant burden on patients' lives. Although the term health related-quality of life (HR-QOL) has only been used in recent years, several studies have long addressed the impact of GTS on physical, psychological and social aspects of wellbeing of both children and adults with GTS. We set out to answer the question "Is HR-QOL affected by GTS and, if so, in what domains?" by conducting a systematic literature review of published original studies addressing HR-QOL in both children and adult patients with GTS. This review focuses on the current evidence on the impact of GTS on patients' lives, mainly informed by studies using generic functional impairment and HR-QOL measures from the last decade, and expands on the new opportunities introduced by the recently developed GTS-specific HR-QOL scales (GTS-QOL and GTS-QOL-C&A). Analysis of the first decade of studies specifically addressing HR-QOL in GTS suggests that co-morbid conditions are key factors in determining HR-QOL in young patients, whilst the picture is more complex in adults with GTS. These findings offer some general directions for both current clinical practice and future research. Keywords: Gilles de la Tourette syndrome, health-related quality of life, functional impairment, tics, behavioural problems The functional impact of Gilles de la Tourette syndrome across the lifespanGilles de la Tourette syndrome (GTS) is a life-long neurodevelopmental disorder defined by the presence of multiple motor and phonic tics [1]. Tics are repetitive, uncontrollable movements or vocalizations that usually present in childhood but may change with re- * Corresponding author: Dr. Andrea Eugenio Cavanna, MD, PhD., Department of Neuropsychiatry, The Barberry National Centre for Mental Health, 25 Vincent Drive, Birmingham B15 2FG, UK. Tel.: +44 0121 3012317; Fax: +44 0121 3012291; E-mail: cavanna77@tin.it. gard to their location, frequency and severity over time [2]. GTS affects approximately 1% of school-age youngsters and is three to four times more common in males than females [3]. GTS is increasingly recognised as a complex disorder, with a wide spectrum of associated behavioural problems that can accompany the motor and phonic tics. These problems include ticrelated symptoms (ranging from socially inappropriate statements to actual self-harm) and co-morbid psychiatric disorders, such as obsessive-compulsive disorder (OCD) and attention-deficit hyperactivity disorder (ADHD) [4,5]. Large studies conducted both in clinical populations [2,6] and in the community [7] have consistently found that only about 10% of individuals with GTS do not present with behavioural co-morbidities.

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“…The Burke-Fahn-Marsden Scale, Unified Dystonia Rating Scale, and Global Dystonia Rating Scale were developed to evaluate generalized dystonia, but are also used for other dystonia types [44,46]. The Tsui score [47], the Cervical Dystonia Severity Scale [48], the Cervical Dystonia Impact Profile [49], the Craniocervical Dystonia Questionnaire [50], and the Toronto Western Spasmodic Torticollis Rating Scale [51] have been developed for cervical dystonia; the Jankovic Rating Scale [52] is used for blepharospasm. These scales vary in their ease of use in the clinical setting and appropriateness for use in clinical trials [45].…”

Section: Trial Design Featuresmentioning

confidence: 99%

Designing Clinical Trials for Dystonia

et al. 2014

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With advances in the understanding of the pathophysiology of dystonia, novel therapeutics are being developed. Such therapies will require clinical investigation ranging from exploratory studies to examine safety, tolerability, dosage selection, and preliminary efficacy to confirmatory studies to evaluate efficacy definitively. As dystonia is a rare and complex disorder with clinical and etiological heterogeneity, clinical trials will require careful consideration of the trial design, including enrollment criteria, concomitant medication use, and outcome measures. Given the complexities of designing and implementing efficient clinical trials, it is important for clinicians and statisticians to collaborate closely throughout the clinical development process and that each has a basic understanding of both the clinical and statistical issues that must be addressed. To facilitate designing appropriate clinical trials in this field, we review important general clinical trial and regulatory principles, and discuss the critical components of trials with an emphasis on considerations specific to dystonia. Additionally, we discuss designs used in early exploratory, late exploratory, and confirmatory phases, including adaptive designs.

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Craniocervical dystonia questionnaire (CDQ-24): development and validation of a disease-specific quality of life instrument

Cited by 130 publications

References 17 publications

The physical, social and emotional aspects are the most affected in the quality of life of the patients with cervical dystonia

The physical, social and emotional aspects are the most affected in the quality of life of the patients with cervical dystonia

Health-Related Quality of Life in Gilles de la Tourette Syndrome: A Decade of Research

Designing Clinical Trials for Dystonia

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