Cochrane Db Syst Rev
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Spinal muscular atrophy (SMA) is caused by a homozygous deletion of the survival motor neuron 1 (SMN1) gene on chromosome 5, or a heterozygous deletion in combination with a point mutation in the second SMN1 allele. This results in degeneration of anterior horn cells, which leads to progressive muscle weakness. By definition, children with SMA type I are never able to sit without support and usually die or become ventilator dependent before the age of two years. There have until very recently been no drug treatments to influence the course of SMA. We undertook this updated review to evaluate new evidence on emerging treatments for SMA type I. The review was first published in 2009 and previously updated in 2011. ⋯ Based on the very limited evidence currently available regarding drug treatments for SMA type 1, intrathecal nusinersen probably prolongs ventilation-free and overall survival in infants with SMA type I. It is also probable that a greater proportion of infants treated with nusinersen than with a sham procedure achieve motor milestones and can be classed as responders to treatment on clinical assessments (HINE-2 and CHOP INTEND). The proportion of children experiencing adverse events and serious adverse events on nusinersen is no higher with nusinersen treatment than with a sham procedure, based on evidence of moderate certainty. It is uncertain whether riluzole has any effect in patients with SMA type I, based on the limited available evidence. Future trials could provide more high-certainty, longer-term evidence to confirm this result, or focus on comparing new treatments to nusinersen or evaluate them as an add-on therapy to nusinersen.
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Cochrane Db Syst Rev · Dec 2019
Meta AnalysisManagement of drainage for malignant ascites in gynaecological cancer.
Ascites is the accumulation of fluid within the abdominal cavity. Most women with advanced ovarian cancer and some women with advanced endometrial cancer need repeated drainage for ascites. Guidelines to advise those involved in the drainage of ascites are usually produced locally and are generally not evidence-based. Managing drains that improve the efficacy and quality of the procedure is key in making recommendations that could improve the quality of life (QoL) for women at this critical period of their lives. ⋯ Currently, there is insufficient evidence to recommend the most appropriate management of drainage for malignant ascites among women with gynaecological cancer, as there was only very low-certainty evidence from one small RCT at overall high risk of bias.
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Primary delusional infestation (DI) is a primary psychiatric disorder characterised by delusions and abnormal tactile sensations. The pathophysiology is undecided and treatment includes both pharmacological and non-pharmacological options. There is currently no Cochrane Review of the treatments used. Primary DI is a diagnosis often encountered by both dermatologists and psychiatrists, with a large associated disease burden. ⋯ Currently there is no evidence from RCTs available to compare treatment of primary DI with placebo. We cannot, therefore, make any conclusions regarding the effects of treatments (pharmacological or non-pharmacological) for primary DI. This lack of evidence for treatment of primary DI has implications for research and practice. Robust randomised trials are indicated.
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Cochrane Db Syst Rev · Dec 2019
Meta AnalysisLaparoscopic colposuspension for urinary incontinence in women.
Laparoscopic colposuspension was one of the first minimal access operations for treating stress urinary incontinence in women, with the presumed advantages of shorter hospital stays and quicker return to normal activities. This Cochrane Review was last updated in 2010. ⋯ The data indicate that, in terms of subjective cure of incontinence within 18 months, there is probably little difference between laparoscopic colposuspension and open colposuspension, or between laparoscopic colposuspension and midurethral sling procedures. Much of the evidence is low quality, meaning that a considerable degree of uncertainty remains about laparoscopic colposuspension. Future trials should recruit adequate numbers, conduct long-term follow-up and measure clinically important outcomes. A brief economic commentary identified three studies. We have not quality-assessed them and they should be interpreted in light of the findings on clinical effectiveness.
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This review is one in a series of Cochrane Reviews of interventions for shoulder disorders. ⋯ At the moment, we are uncertain whether rotator cuff repair surgery provides clinically meaningful benefits to people with symptomatic tears; it may provide little or no clinically important benefits with respect to pain, function, overall quality of life or participant-rated global assessment of treatment success when compared with non-operative treatment. Surgery may not improve shoulder pain or function compared with exercises, with or without glucocorticoid injections. The trials included have methodology concerns and none included a placebo control. They included participants with mostly small degenerative tears involving the supraspinatus tendon and the conclusions of this review may not be applicable to traumatic tears, large tears involving the subscapularis tendon or young people. Furthermore, the trials did not assess if surgery could prevent arthritic changes in long-term follow-up. Further well-designed trials in this area that include a placebo-surgery control group and long follow-up are needed to further increase certainty about the effects of surgery for rotator cuff tears.