JAMA : the journal of the American Medical Association
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Randomized Controlled Trial Multicenter Study
Repurposing diflunisal for familial amyloid polyneuropathy: a randomized clinical trial.
Familial amyloid polyneuropathy, a lethal genetic disease caused by aggregation of variant transthyretin, induces progressive peripheral nerve deficits and disability. Diflunisal, a nonsteroidal anti-inflammatory agent, stabilizes transthyretin tetramers and prevents amyloid fibril formation in vitro. ⋯ Among patients with familial amyloid polyneuropathy, the use of diflunisal compared with placebo for 2 years reduced the rate of progression of neurological impairment and preserved quality of life. Although longer-term follow-up studies are needed, these findings suggest benefit of this treatment for familial amyloid polyneuropathy.
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Randomized Controlled Trial Multicenter Study
Effect of progenitor cell mobilization with granulocyte-macrophage colony-stimulating factor in patients with peripheral artery disease: a randomized clinical trial.
Many patients with peripheral artery disease (PAD) have walking impairment despite therapy. Experimental studies in animals demonstrate improved perfusion in ischemic hind limb after mobilization of bone marrow progenitor cells (PCs), but whether this is effective in patients with PAD is unknown. ⋯ Therapy with GM-CSF 3 times a week did not improve treadmill walking performance at the 3-month follow-up. The improvements in some secondary outcomes with GM-CSF suggest that it may warrant further study in patients with claudication.
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Randomized Controlled Trial Multicenter Study
Effect of nortriptyline on symptoms of idiopathic gastroparesis: the NORIG randomized clinical trial.
Gastroparesis remains a challenging syndrome to manage, with few effective treatments and a lack of rigorously controlled trials. Tricyclic antidepressants are often used to treat refractory symptoms of nausea, vomiting, and abdominal pain. Evidence from well-designed studies for this use is lacking. ⋯ Among patients with idiopathic gastroparesis, the use of nortriptyline compared with placebo for 15 weeks did not result in improvement in overall symptoms. These findings do not support the use of nortriptyline for idiopathic gastroparesis.
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Randomized Controlled Trial Comparative Study
Prolonged exposure vs supportive counseling for sexual abuse-related PTSD in adolescent girls: a randomized clinical trial.
Evidence-based treatments for posttraumatic stress disorder (PTSD) have not been established for adolescents despite high prevalence of PTSD in this population. ⋯ Adolescents girls with sexual abuse-related PTSD experienced greater benefit from prolonged exposure therapy than from supportive counseling even when delivered by counselors who typically provide supportive counseling.
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Randomized Controlled Trial
Cognitive behavioral therapy plus amitriptyline for chronic migraine in children and adolescents: a randomized clinical trial.
Early, safe, effective, and durable evidence-based interventions for children and adolescents with chronic migraine do not exist. ⋯ Among young persons with chronic migraine, the use of CBT plus amitriptyline resulted in greater reductions in days with headache and migraine-related disability compared with use of headache education plus amitriptyline. These findings support the efficacy of CBT in the treatment of chronic migraine in children and adolescents.