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In a randomized trial that enrolled 200 patients within 6 hours of their having sustained severe closed head traumatic brain injury, Robertson and colleagues assessed the effect of erythropoietin administration and 2 transfusion thresholds (7 and 10 g/dL) on neurological recovery. The authors report that neither administration of erythropoietin nor maintaining the hemoglobin concentration greater than 10 g/dL resulted in improved neurological outcome at 6 months.
Author Audio Interview
Myeloablative allogeneic hematopoietic stem cell transplantation (HSCT) is curative for children with sickle cell disease, but toxicity may be prohibitive in adults. In a study that involved 30 patients aged 16 to 65 years with severe sickle cell disease or thalassemia, Hsieh and colleagues assessed the efficacy and safety of a low-intensity, nonmyeloablative regimen and HSCT from human leukocyte antigen–matched siblings. The authors report that at a median follow-up of 3.4 years, 29 patients were alive and 26 patients had long-term stable donor engraftment and normalized hemoglobin. In an Editorial, King and DiPersio discuss the potential to cure sickle cell disease in adults.
Editorial and Related Article
Author Video Interview
Sorafenib is the only systemic therapy shown to improve overall survival in advanced hepatocellular carcinoma and the benefit is largely transient and modest. Zhu and colleagues assessed the efficacy of everolimus vs placebo—both combined with best supportive care—in a randomized study of 546 patients with advanced hepatocellular carcinoma for whom sorafenib treatment had failed and found no beneficial effect of everolimus on overall survival.
Mitochondrial respiratory chain disorders are difficult to diagnose at the molecular level because many different genes are potentially involved. In a study involving 53 unrelated patients who had biochemical evidence of multiple respiratory chain complex defects, Taylor and colleagues assessed whether whole-exome sequencing could be used to define the molecular basis of disease. The authors report that exome sequencing identified potential nuclear gene mutations—involving 18 different genes—in 32 of the 53 patients studied.
Generalized anxiety disorder (GAD) and panic disorder are common but often unrecognized in primary care settings. In a review of 76 prospective studies that assessed the diagnostic accuracy of self-report screening instruments for GAD or panic disorder, Herr and colleagues found that 2 screening instruments, the GAD-7 for generalized anxiety disorder and the Patient Health Questionnaire for panic disorder, had good performance characteristics, and are feasible for use in primary care.
Continuing Medical Education
In this JAMA Guide to Statistics and Methods article, Detry and Lewis discuss the application of the intention-to-treat principle in the analysis, interpretation, and clinical use of data from randomized clinical trials. In an Editorial, Livingston discusses this new JAMA feature.
Related Article and Editorial
A recent article in JAMA Dermatology reported that data from the National Ambulatory Medical Care Survey suggest that few physicians advise patients about sunscreen use—even when patients have a history of skin cancer. In this From the JAMA Network article, Federman and colleagues discuss guidelines for counseling about sun-protective behaviors.
Highlights. JAMA. 2014;312(1):1-3. doi:10.1001/jama.2013.279585