New and Noteworthy Information—October 2014
Regular blood transfusion therapy significantly reduced the recurrence of cerebral infarct in children with sickle cell anemia, according to a study published in the August 21 issue of the New England Journal of Medicine. During the three-year study, 196 children ages 5 through 15 with sickle cell anemia who had previously had a silent stroke were followed. Children who underwent regular transfusions were 58% less likely to have another silent stroke or an overt stroke, while those who had no transfusions were more than twice as likely to experience repeat strokes. In addition, children who had monthly transfusions were less likely to have a range of other sickle cell anemia–related problems, such as episodes of extreme pain. Overall, 295 pain episodes occurred among children who did not receive transfusions, compared with 126 episodes among those receiving treatment.
Stroke incidence and mortality rates decreased from 1987 to 2011, according to a study published in the July 16 issue of JAMA. The findings were based on data from the Atherosclerosis Risk in Communities cohort of 15,792 US residents between the ages of 45 and 64 who were monitored during the 1980s. The new study followed the progress of 14,357 participants who were free of stroke in 1987 and monitored hospitalizations from stroke and deaths from 1987 to 2011. Stroke incidence decreased over time in Caucasians and African Americans, with an age-adjusted incidence rate ratio of 0.76. The absolute decrease was 0.93 per 1,000 person-years overall. The overall mortality rate after stroke decreased over time (hazard ratio, 0.80), with an absolute decrease of 8.09 per 100 strokes after 10 years.
The FDA has approved Vimpat (lacosamide) C-V as monotherapy in the treatment of partial-onset seizures in patients with epilepsy ages 17 and older. The monotherapy approval for Vimpat is based on a phase III historical-control conversion to lacosamide monotherapy study in adult patients with epilepsy with partial-onset seizures. This study met its primary end point, demonstrating that the exit percentage for patients converting to lacosamide (400 mg/day) was lower than the historical control exit percentage used as a comparator. Lacosamide (300 mg/day) also met the prespecified criteria for efficacy. Based on individual patients’ needs, physicians can choose between Vimpat formulations—tablets, oral solution, or injection. Vimpat (UCB; Brussels) is already approved in the US as adjunctive treatment for partial-onset seizures in patients in this age group.
Disruption of intestinal homeostasis is an early and immune-mediated event in experimental autoimmune encephalomyelitis, according to a study published September 3 in PLoS ONE. Investigators observed structural changes in the mucous membrane of the small intestine and an increase in inflammatory T cells, as well as a reduction in immunosuppressive cells. “Our findings provide support for the idea that a damaged intestinal barrier can prevent the body ending an autoimmune reaction in the normal manner, leading to a chronic disease such as multiple sclerosis,” stated the study authors. “In particular, an increased understanding of the regulation of tight junctions at the blood–brain barrier and in the intestinal wall may be crucial for design of future innovative therapies.”
Children and adolescents with autism have a surplus of synapses in the brain due to reduced developmental spine pruning, investigators reported in the September 3 issue of Neuron. Researchers examined brains from children with autism who had died from other causes. Thirteen brains were from children ages 2 to 9, 13 brains were from children ages 13 to 20, and 22 brains were from children without autism. The investigators measured synapse density in a small section of tissue in each brain by counting the number of tiny spines that branch from the cortical neurons. During late childhood, spine density had decreased by about half in the control brains, compared with 16% in the brains from patients with autism. “Hundreds of genes have been linked to autism, but almost all of our human subjects had overactive mTOR and decreased autophagy, and all appear to have a lack of normal synaptic pruning,” stated study authors.
Macromolecular proton fraction (MPF) mapping enables quantitative assessment of demyelination in normal-appearing brain tissues and shows primary clinical relevance of gray matter damage in multiple sclerosis (MS), according to a study published online ahead of print September 10 in Radiology. Researchers examined 30 patients with MS, 18 with relapsing-remitting MS (RRMS) and 12 with secondary progressive MS. Fourteen healthy controls also were included. Each participant underwent MRI on a 3-T imager, and the investigators reconstructed 3-D whole-brain MPF maps to examine normal-appearing white matter, gray matter, and MS lesions. MPF was significantly lower in both white and gray matter in patients with RRMS, compared with healthy controls, and it was significantly reduced in normal-appearing brain tissues and lesions of patients with secondary progressive MS, compared with patients with RRMS with the largest relative decrease in gray matter.
