Publicação
Deep brain stimulation in dystonia
| Resumo: | Dystonia is a painful and disabling disorder characterised by painful involuntary posturing of the affected region(s). Deep brain stimulation (DBS) is an intervention typically reserved for refractory or severe cases, although uncertainty exists regarding its efficacy, safety and tolerability. Objectives To compare the efficacy, safety, and tolerability of deep brain stimulation versus sham-stimulation, best medical therapy or placebo in people with dystonia. Search methods We identified studies for inclusion in the review using the Cochrane Movement Disorders Group Trials Register, the Cochrane Central Register of Controlled Trials (CENTRAL), MEDLINE, Embase, reference lists of included articles, conference proceedings, grey literature, and clinical trial registries. All elements of the search, with no language restrictions, were last run in October 2016. Selection criteria Double-blind, parallel, randomised, placebo-controlled trials (RCTs) comparing DBS versus sham-stimulation, best medical care or placebo in adults with dystonia. Data collection and analysis Two independent authors assessed records, selected included studies, extracted data using a paper pro forma, and evaluated the risk of bias. We resolved disagreements by consensus or by consulting a third author. We performed meta-analyses using a random-effects model, for the comparison DBS versus sham-stimulation to estimate pooled effects and corresponding 95% confidence intervals (95% CI). The primary efficacy outcome was improvement on any validated symptomatic rating scale, and the primary safety outcome was adverse events. Main results We included two RCTs, enrolling a total of 102 participants. Both trials evaluated the effect of DBS on the GPi nucleus, with outcome assessment at 3 and 6 months of stimulation. One of the studies included participants with generalised and segmental dystonia, while the other included participants with focal (cervical) dystonia. Both studies were considered at high risk for performance and for-profit bias, while the first study was at high risk of detection bias, and the second was retrospectively registered. As assessed using the GRADE methodology, DBS may improve cervical dystonia related impairment, overall subjective evaluation of clinical status, overall functional capacity and mood. DBS may have little or no difference in overall physical functioning related quality of life and overall mental health quality of life, as these outcomes had a null effect. We are uncertain whether DBS improves overall generalised or segmental dystonia related impairment, overall subjective evaluation of clinical status, overall physical functioning related quality of life, overall mental health related quality of life, overall dystonia related functional capacity, and mood. We pooled outcomes related to safety and tolerability, given that both trials used the same intervention and comparison and we considered that DBS may have little or no difference in the risk of adverse events and tolerability. Authors’ conclusions There is low quality evidence that DBS may improve overall efficacy in the treatment of cervical dystonia in adults with moderate to severe dystonia. Due to very low quality of evidence, it is unclear whether DBS may improve overall efficacy in generalised and segmental dystonia. Treatment with DBS may appear to be safe and tolerable in patients. Further research is still needed to establish clinical efficacy, safety and tolerability. |
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| Autores principais: | Prescott, David Alexandre de Azoia Ferreira |
| Assunto: | Distonia Estimulação cerebral profunda Revisão sistemática |
| Ano: | 2017 |
| País: | Portugal |
| Tipo de documento: | dissertação de mestrado |
| Tipo de acesso: | acesso restrito |
| Instituição associada: | Universidade de Lisboa |
| Idioma: | inglês |
| Origem: | Repositório da Universidade de Lisboa |
| Resumo: | Dystonia is a painful and disabling disorder characterised by painful involuntary posturing of the affected region(s). Deep brain stimulation (DBS) is an intervention typically reserved for refractory or severe cases, although uncertainty exists regarding its efficacy, safety and tolerability. Objectives To compare the efficacy, safety, and tolerability of deep brain stimulation versus sham-stimulation, best medical therapy or placebo in people with dystonia. Search methods We identified studies for inclusion in the review using the Cochrane Movement Disorders Group Trials Register, the Cochrane Central Register of Controlled Trials (CENTRAL), MEDLINE, Embase, reference lists of included articles, conference proceedings, grey literature, and clinical trial registries. All elements of the search, with no language restrictions, were last run in October 2016. Selection criteria Double-blind, parallel, randomised, placebo-controlled trials (RCTs) comparing DBS versus sham-stimulation, best medical care or placebo in adults with dystonia. Data collection and analysis Two independent authors assessed records, selected included studies, extracted data using a paper pro forma, and evaluated the risk of bias. We resolved disagreements by consensus or by consulting a third author. We performed meta-analyses using a random-effects model, for the comparison DBS versus sham-stimulation to estimate pooled effects and corresponding 95% confidence intervals (95% CI). The primary efficacy outcome was improvement on any validated symptomatic rating scale, and the primary safety outcome was adverse events. Main results We included two RCTs, enrolling a total of 102 participants. Both trials evaluated the effect of DBS on the GPi nucleus, with outcome assessment at 3 and 6 months of stimulation. One of the studies included participants with generalised and segmental dystonia, while the other included participants with focal (cervical) dystonia. Both studies were considered at high risk for performance and for-profit bias, while the first study was at high risk of detection bias, and the second was retrospectively registered. As assessed using the GRADE methodology, DBS may improve cervical dystonia related impairment, overall subjective evaluation of clinical status, overall functional capacity and mood. DBS may have little or no difference in overall physical functioning related quality of life and overall mental health quality of life, as these outcomes had a null effect. We are uncertain whether DBS improves overall generalised or segmental dystonia related impairment, overall subjective evaluation of clinical status, overall physical functioning related quality of life, overall mental health related quality of life, overall dystonia related functional capacity, and mood. We pooled outcomes related to safety and tolerability, given that both trials used the same intervention and comparison and we considered that DBS may have little or no difference in the risk of adverse events and tolerability. Authors’ conclusions There is low quality evidence that DBS may improve overall efficacy in the treatment of cervical dystonia in adults with moderate to severe dystonia. Due to very low quality of evidence, it is unclear whether DBS may improve overall efficacy in generalised and segmental dystonia. Treatment with DBS may appear to be safe and tolerable in patients. Further research is still needed to establish clinical efficacy, safety and tolerability. |
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